Tara Haelle

ORLANDO, FLORIDA — The rationale for using obesity medications in pediatric patients is that it’s using “a biological intervention to treat a biologically based disease,” according to Claudia Fox, MD, MPH, an associate professor of pediatrics and codirector of the Center for Pediatric Obesity Medicine at the University of Minnesota, Minneapolis. At the annual meeting of the American Academy of Pediatrics (AAP), Fox provided an overview of what obesity medications are approved for in youth and how to determine which medications may be best for different patients.

“This field is changing so rapidly that even over the course of the last 3 or 4 months, the verbiage around what we should be calling these interventions has changed,” Fox noted. Instead of “anti-obesity” medications, “most of us are now using the term obesity medications to highlight or to reduce chances of stigma and bias that can come along with this topic.”

Jessica Ivers, MD, a pediatrician at Swedish Pediatrics in Seattle, Washington, said she found the session very informative, particularly because she doesn’t think many pediatricians currently feel very comfortable prescribing obesity medications.

“It answered questions that any general pediatrician would have, and it’s kind of a new field that people are learning about,” Ivers said. “I think we just need more education. It’s just too new, and people haven’t had the education and the support from colleagues to [use the medications].”

Fox first reminded attendees of precisely what obesity is: A chronic, relapsing, multifactorial, neurobehavioral disease that involves the accumulation and/or distribution of excess body fat that results in impaired health. AAP clinical practice guidelines currently advise that youth aged 12 years or older who have obesity be offered weight loss pharmacotherapy as an adjunct to lifestyle treatment, taking into consideration the indications, risks, and benefits of each medication.

That doesn’t necessarily mean every child aged 12 years or older with a body mass index (BMI) of at least the 95th percentile should be prescribed one of these medications, Fox said. But pediatricians should start becoming familiar with the options and recognize that part of reducing the stigma of this disease is emphasizing that these medications are prescribed not for “weight loss” but to treat the disease of obesity, Fox said. The guidelines advise “early, intensive care” and focusing on the whole child, “using a family-centered and nonstigmatizing approach that acknowledges obesity’s biologic, social, and structural drivers.”
 

Offer the Full Spectrum of Care Early On

Early intervention means starting obesity treatment at diagnosis, without watchful waiting or the previously recommended staged approach. Instead of trying lifestyle therapy for 3-6 months, then considering the addition of medication, and then considering bariatric surgery, “we should be offering the full spectrum of obesity care as appropriate for that individual patient,” Fox said.

Some children with severe obesity may need the combination of lifestyle therapy and pharmacotherapy right up front, whereas another might be able to try lifestyle therapy alone for a while first. “What we know is that, for most interventions, whether it is lifestyle therapy, a medication, or bariatric surgery, early response typically predicts longer-term response,” Fox said. A study conducted by her group, for example, found that a 3% BMI reduction after 1 month with lifestyle therapy was very predictive of clinically meaningful BMI reduction at 1 year.

As with any medical treatment, physicians need to weigh the risks of the medication — short-term side effects and unknown long-term risks (or benefits) — against the risks of not treating. Because obesity is a progressive disease, “if we don’t treat it, most will develop comorbid conditions, or worsening of their already present comorbid conditions, and this does indeed lead to shortened life expectancy,” Fox said. Those who should be treated with medication are obviously those in whom the benefits outweigh the risks, Fox said, which depends on their age, their comorbidities, the severity of obesity, and the safety and efficacy of medication options.

“If I have a patient who has maybe class 2 obesity but no other comorbid conditions, I may be less inclined to start an obesity medication than a kid who has class 1 obesity and obstructive sleep apnea, for instance,” Fox said. “Some of the medications are very, very potent and effective. If you have a kid who maybe has less severe forms of obesity, perhaps they don’t need something that’s so potent.”

BMI trajectory is also a factor to consider. She said she may not be too concerned about a 16-year-old who has always been at the 95th percentile and is otherwise healthy, but the situation is different for a 16-year-old who used to be in the 25th percentile and has rapidly progressed to the 50th and then 75th percentiles in a trajectory heading straight up.

Another factor that may come into play is the patient and family preferences, though Fox noted that weight bias and stigma often interfere here. If obesity medications are brought up, the family may bring up the need for more exercise and better meal prep at home.

“They have this sense that they just need to try harder, that if they did that, the obesity would somehow get better on its own,” Fox said. “That’s an internalized bias that it’s somehow their fault, rather than realizing that this is indeed a biological disorder.”

Finally, clinicians may want to consider the child’s response to lifestyle therapy and whether they have already had bariatric surgery because these medications can be prescribed in people who did not have an adequate response to surgery.
 

Overview of the Medications

There are currently six obesity medications approved by the Food and Drug Administration (FDA) for use in youth: Phentermine, orlistat, liraglutide, phentermine/topiramate, semaglutide, and setmelanotide.

Of these, orlistat is rarely used now because it results in the least amount of change in BMI (about a 3% loss change in BMI), has a lot of gastrointestinal side effects, often is not covered by insurance, and is expensive out of pocket. Setmelanotide is indicated only in those aged 6 years or older who have obesity because of Bardet-Biedl syndrome or one of three other rare genetic conditions: a POMC, LEPR, or PCSK1 deficiency. Fox therefore focused on the other medications besides these two.

While nearly all the currently available obesity medications are only approved in those aged 12 years or older, Fox noted that studies are ongoing at younger ages, so some of these medications may receive approval in younger populations in the future. The only one currently available for a younger age is liraglutide, which is approved down to 6 years old in children with type 2 diabetes.

“Very young kids who have very severe forms of obesity need intervention, and unfortunately, at this point, we really don’t have much to offer them,” Fox said.

Fox highlighted six key factors to consider in selecting a medication for those aged 12 years or older, though one of these, in the US healthcare system, can tend to trump all the others. Those factors are mechanism of action, side effect profile, effects on other diagnoses, patient/family preferences, provider comfort, and finally — the potentially overruling one — insurance coverage and access.

“These days, insurance coverage and access are really the No. 1 driver when I’m seeing a patient,” Fox said. “The first thing I do is look at their insurance and then also look at what kind of updates our pharmacist has given us about which medication is currently in stock.”

Each medication has different properties that should be considered with the child’s health profile. For example, topiramate is a carbonic anhydrase inhibitor so likely shouldn’t be prescribed in a child who is taking any other carbonic anhydrase inhibitor. Fox said she probably wouldn’t prescribe phentermine in a child with severe anxiety because it might enhance the anxiety effect. But if a child has migraines, she may be more inclined to try phentermine/topiramate first because the topiramate may help with the migraines. Similarly, if a child has type 2 diabetes or prediabetes, she may lean toward one of the glucagon-like peptide 1 (GLP-1) agonist drugs.
 

Liraglutide and Semaglutide

Liraglutide and semaglutide are both GLP-1 receptor agonists administered subcutaneously to reduce appetite, increase satiety, slow gastric emptying, and reduce the food reward response in the brain. Liraglutide can result in up to 4.5%-5% change in BMI, and semaglutide, the most potent of all the medications, can result in up to a 17% change in BMI.

Liraglutide and semaglutide are both approved for patients aged 12 years or older who weigh at least 60 kg and have a BMI of at least the 95th percentile. Liraglutide is also approved for those aged 10 years or older with type 2 diabetes. Both are contraindicated in those with a family history of medullary thyroid cancer or multiple endocrine neoplasia II. The risks to watch for include pancreatitis and gallbladder disease. Also keep in mind if you have a patient with type 1 diabetes and insulin resistance; prescribing a GLP-1 agonist is appropriate, but their insulin needs will decrease, necessitating close monitoring of their blood glucose, Fox noted.

These GLP-1 medications can be considered for those who have insurance coverage for them, who have diabetes or prediabetes, who are comfortable with daily (liraglutide) or weekly (semaglutide) injections, who have food cravings, and who have poor satiety or satiation. Without insurance, these medications are very expensive.

The most common side effects include injection site reactions and nausea, vomiting, and diarrhea, though all these usually fade and can be minimized with small portions and slower eating if needed. Less common possible side effects can include abdominal pain, constipation, headache, dizziness, fatigue, and hypoglycemia. If patients develop severe belly pain that radiates to their back, they should be assessed for pancreatitis.

It’s also important to demonstrate for patients how to do the injections, Fox said. Liraglutide dosing begins at 0.6 mg daily for a week, followed by a week at 1.2 mg, a week at 1.8 mg, a week at 2.4 mg, and then 3 mg daily. Semaglutide dosing starts at 0.25 mg weekly for 4 weeks, then going up each subsequent month as needed to 0.5 mg, then 1 mg, then 1.7 mg, and finally 2.4 mg. Though there’s no standard follow-up schedule for these medications, Fox suggested considering monthly visits for the first 3 months and then every 2-3 months to assess heart rate and blood pressure, the injection site, adherence, side effects, and the effect on BMI and eating.

“Are they getting appetite suppression, but not too much appetite suppression?” Fox said. “Just like in eating disorder treatment, we want our patients to eat regularly spaced meals. If their appetite is so suppressed that they are hardly eating anything, that’s a problem.”

Fox also offered the following additional pearls about these medications:

• Though manufacturers have struggled to keep up with demand, the shortages of these medications are improving. However, beware the compounding pharmacies filling the gap because compounded medications are not FDA approved, and quality control issues are a concern.
• Prior authorizations are usually needed, and common reasons for denial to anticipate include lack of documentation on not having contraindications, the patient not following a low-calorie diet or engaging in physical activity, and the patient not having seen a registered dietitian.
• Patients should expect gastrointestinal side effects, but ondansetron can be prescribed to lessen the intensity.

Phentermine/Topiramate

Phentermine/topiramate extended-release is a once-daily oral tablet, with the phentermine acting to reduce appetite (by simulating the release of norepinephrine) and the topiramate reducing caloric intake and food reward response (by increasing gamma-aminobutyric acid activity). It’s approved for those aged 12 years or older with a BMI of at least the 95th percentile and should be considered in those with strong hunger, low energy, binge eating disorder, or migraines, as well as those who have insurance coverage for it. It can result in up to a 10% change in BMI.

Contraindications include pregnancy, substance use, cardiovascular disease (though it’s okay in patients with controlled hypertension), hyperthyroidism, glaucoma, and monoamine oxidase inhibitor (MAOI) use. Fox emphasized the teratogenic effects, so patients capable of pregnancy need to be on reliable birth control. The most common side effects include paresthesia, dizziness, dysgeusia, insomnia, and constipation.

A risk of topiramate is kidney stones, so patients should drink a lot of water, especially in hot weather, Fox said. Other risks can include metabolic acidosis, suicidality, poor cognitive function, high blood pressure, and renal impairment.

“If your patient is struggling academically, I might use this medication a bit more cautiously, particularly when the dose gets above 100 mg a day,” Fox said. “That’s when the cognitive effects tend to emerge more strongly.”

Patients with congenital heart disease should meet with their cardiologist before starting this medication, and although patients taking selective serotonin reuptake inhibitors (SSRIs) can take this, there is a potential increased risk for serotonin syndrome because phentermine has a little bit of serotonergic activity, she said.

Before prescribing, do an exam to ensure the patient doesn’t have a heart murmur, isn’t hypertensive, isn’t pregnant, has normal kidney function, and has bicarbonate in a reasonable range. Dosing begins with a daily 3.75/23-mg capsule for 2 weeks, followed by 2 weeks at 7.5 mg/46 mg. As with the GLP-1 drugs, Fox advises considering monthly follow-ups for the first 3 months and then visits every 2-3 months. Each visit should include the assessment of cardiovascular health, heart rate, blood pressure, side effects, pregnancy risk, and the medication’s effect on BMI and eating. If the patient is tolerating a dose of 7.5 mg/46 mg, it can be increased to 11.25 mg/69 mg for 2 weeks and then to 15 mg/92 mg. Bicarbonate and creatinine should be checked every 6-12 months; if bicarbonate < 18 mEq/L, the dose should be reduced and then bicarbonate should be checked again a month later.

Fox noted that this drug is expected to go off patent in late 2024 or in 2025, which will substantially reduce the cost. It’s also possible to prescribe phentermine and topiramate separately, which may reduce costs or help with insurance coverage and can allow for evening dosing of topiramate.
 

Phentermine

Phentermine alone is only approved for those older than 16 years who have a BMI of at least 30, or at least 27 with weight-related comorbidities, and it’s not approved for use longer than 12 weeks. It results in a BMI change of up to 5%. It should be considered in those with strong hunger and low energy and in those who don’t have adequate insurance coverage because out-of-pocket costs can be as little as $5/mo.

Contraindications are the same as those for the combined pill above: Substance use, cardiovascular disease, hyperthyroidism, glaucoma, MAOI use, and agitation. Again, take caution with patients who have hypertension, have congenital heart disease, or take SSRIs or insulin.

Side effects can include palpitations, tachycardia, dry mouth, headache, insomnia, and anxiety. The dose starts at 15 mg daily, and Fox advises following a similar follow-up as with the other medications, at which clinicians should assess BMI, the medication’s effect on eating, cardiovascular health, and side effects and have a discussion about off-label use. Off-label use refers to prescriptions lasting longer than 12 weeks, but it’s arguably safer than attention-deficit/hyperactivity disorder stimulants because of the lower addiction potential, Fox said.
 

What Else to Know

Because obesity is a chronic disease, treatment will be ongoing, Fox noted. A lot of people will ask when or where the “off-ramp” for these medications is, but many people will need these medications long term just as someone with other chronic diseases requires lifetime pharmacotherapy. The treatment intensity will vary based on disease severity and individual characteristics, Fox said.

For those feeling overwhelmed by the options, Fox advises clinicians to start by picking one medication to learn and then spending the time to read the FDA package insert in full. Get samples and then closely follow patients to learn that medication well before moving on to learn another. She also noted the opportunity for pediatricians to see a pediatric obesity medicine fellowship.

No external funding was used for the presentation. Fox is a site principal investigator for clinical trials sponsored by Novo Nordisk and Eli Lilly. Ivers had no disclosures.

A version of this article first appeared on Medscape.com.

ORLANDO, FLORIDA — The rationale for using obesity medications in pediatric patients is that it’s using “a biological intervention to treat a biologically based disease,” according to Claudia Fox, MD, MPH, an associate professor of pediatrics and codirector of the Center for Pediatric Obesity Medicine at the University of Minnesota, Minneapolis. At the annual meeting of the American Academy of Pediatrics (AAP), Fox provided an overview of what obesity medications are approved for in youth and how to determine which medications may be best for different patients.

“This field is changing so rapidly that even over the course of the last 3 or 4 months, the verbiage around what we should be calling these interventions has changed,” Fox noted. Instead of “anti-obesity” medications, “most of us are now using the term obesity medications to highlight or to reduce chances of stigma and bias that can come along with this topic.”

Jessica Ivers, MD, a pediatrician at Swedish Pediatrics in Seattle, Washington, said she found the session very informative, particularly because she doesn’t think many pediatricians currently feel very comfortable prescribing obesity medications.

“It answered questions that any general pediatrician would have, and it’s kind of a new field that people are learning about,” Ivers said. “I think we just need more education. It’s just too new, and people haven’t had the education and the support from colleagues to [use the medications].”

Fox first reminded attendees of precisely what obesity is: A chronic, relapsing, multifactorial, neurobehavioral disease that involves the accumulation and/or distribution of excess body fat that results in impaired health. AAP clinical practice guidelines currently advise that youth aged 12 years or older who have obesity be offered weight loss pharmacotherapy as an adjunct to lifestyle treatment, taking into consideration the indications, risks, and benefits of each medication.

That doesn’t necessarily mean every child aged 12 years or older with a body mass index (BMI) of at least the 95th percentile should be prescribed one of these medications, Fox said. But pediatricians should start becoming familiar with the options and recognize that part of reducing the stigma of this disease is emphasizing that these medications are prescribed not for “weight loss” but to treat the disease of obesity, Fox said. The guidelines advise “early, intensive care” and focusing on the whole child, “using a family-centered and nonstigmatizing approach that acknowledges obesity’s biologic, social, and structural drivers.”
 

Offer the Full Spectrum of Care Early On

Early intervention means starting obesity treatment at diagnosis, without watchful waiting or the previously recommended staged approach. Instead of trying lifestyle therapy for 3-6 months, then considering the addition of medication, and then considering bariatric surgery, “we should be offering the full spectrum of obesity care as appropriate for that individual patient,” Fox said.

Some children with severe obesity may need the combination of lifestyle therapy and pharmacotherapy right up front, whereas another might be able to try lifestyle therapy alone for a while first. “What we know is that, for most interventions, whether it is lifestyle therapy, a medication, or bariatric surgery, early response typically predicts longer-term response,” Fox said. A study conducted by her group, for example, found that a 3% BMI reduction after 1 month with lifestyle therapy was very predictive of clinically meaningful BMI reduction at 1 year.

As with any medical treatment, physicians need to weigh the risks of the medication — short-term side effects and unknown long-term risks (or benefits) — against the risks of not treating. Because obesity is a progressive disease, “if we don’t treat it, most will develop comorbid conditions, or worsening of their already present comorbid conditions, and this does indeed lead to shortened life expectancy,” Fox said. Those who should be treated with medication are obviously those in whom the benefits outweigh the risks, Fox said, which depends on their age, their comorbidities, the severity of obesity, and the safety and efficacy of medication options.

“If I have a patient who has maybe class 2 obesity but no other comorbid conditions, I may be less inclined to start an obesity medication than a kid who has class 1 obesity and obstructive sleep apnea, for instance,” Fox said. “Some of the medications are very, very potent and effective. If you have a kid who maybe has less severe forms of obesity, perhaps they don’t need something that’s so potent.”

BMI trajectory is also a factor to consider. She said she may not be too concerned about a 16-year-old who has always been at the 95th percentile and is otherwise healthy, but the situation is different for a 16-year-old who used to be in the 25th percentile and has rapidly progressed to the 50th and then 75th percentiles in a trajectory heading straight up.

Another factor that may come into play is the patient and family preferences, though Fox noted that weight bias and stigma often interfere here. If obesity medications are brought up, the family may bring up the need for more exercise and better meal prep at home.

“They have this sense that they just need to try harder, that if they did that, the obesity would somehow get better on its own,” Fox said. “That’s an internalized bias that it’s somehow their fault, rather than realizing that this is indeed a biological disorder.”

Finally, clinicians may want to consider the child’s response to lifestyle therapy and whether they have already had bariatric surgery because these medications can be prescribed in people who did not have an adequate response to surgery.
 

Overview of the Medications

There are currently six obesity medications approved by the Food and Drug Administration (FDA) for use in youth: Phentermine, orlistat, liraglutide, phentermine/topiramate, semaglutide, and setmelanotide.

Of these, orlistat is rarely used now because it results in the least amount of change in BMI (about a 3% loss change in BMI), has a lot of gastrointestinal side effects, often is not covered by insurance, and is expensive out of pocket. Setmelanotide is indicated only in those aged 6 years or older who have obesity because of Bardet-Biedl syndrome or one of three other rare genetic conditions: a POMC, LEPR, or PCSK1 deficiency. Fox therefore focused on the other medications besides these two.

While nearly all the currently available obesity medications are only approved in those aged 12 years or older, Fox noted that studies are ongoing at younger ages, so some of these medications may receive approval in younger populations in the future. The only one currently available for a younger age is liraglutide, which is approved down to 6 years old in children with type 2 diabetes.

“Very young kids who have very severe forms of obesity need intervention, and unfortunately, at this point, we really don’t have much to offer them,” Fox said.

Fox highlighted six key factors to consider in selecting a medication for those aged 12 years or older, though one of these, in the US healthcare system, can tend to trump all the others. Those factors are mechanism of action, side effect profile, effects on other diagnoses, patient/family preferences, provider comfort, and finally — the potentially overruling one — insurance coverage and access.

“These days, insurance coverage and access are really the No. 1 driver when I’m seeing a patient,” Fox said. “The first thing I do is look at their insurance and then also look at what kind of updates our pharmacist has given us about which medication is currently in stock.”

Each medication has different properties that should be considered with the child’s health profile. For example, topiramate is a carbonic anhydrase inhibitor so likely shouldn’t be prescribed in a child who is taking any other carbonic anhydrase inhibitor. Fox said she probably wouldn’t prescribe phentermine in a child with severe anxiety because it might enhance the anxiety effect. But if a child has migraines, she may be more inclined to try phentermine/topiramate first because the topiramate may help with the migraines. Similarly, if a child has type 2 diabetes or prediabetes, she may lean toward one of the glucagon-like peptide 1 (GLP-1) agonist drugs.
 

Liraglutide and Semaglutide

Liraglutide and semaglutide are both GLP-1 receptor agonists administered subcutaneously to reduce appetite, increase satiety, slow gastric emptying, and reduce the food reward response in the brain. Liraglutide can result in up to 4.5%-5% change in BMI, and semaglutide, the most potent of all the medications, can result in up to a 17% change in BMI.

Liraglutide and semaglutide are both approved for patients aged 12 years or older who weigh at least 60 kg and have a BMI of at least the 95th percentile. Liraglutide is also approved for those aged 10 years or older with type 2 diabetes. Both are contraindicated in those with a family history of medullary thyroid cancer or multiple endocrine neoplasia II. The risks to watch for include pancreatitis and gallbladder disease. Also keep in mind if you have a patient with type 1 diabetes and insulin resistance; prescribing a GLP-1 agonist is appropriate, but their insulin needs will decrease, necessitating close monitoring of their blood glucose, Fox noted.

These GLP-1 medications can be considered for those who have insurance coverage for them, who have diabetes or prediabetes, who are comfortable with daily (liraglutide) or weekly (semaglutide) injections, who have food cravings, and who have poor satiety or satiation. Without insurance, these medications are very expensive.

The most common side effects include injection site reactions and nausea, vomiting, and diarrhea, though all these usually fade and can be minimized with small portions and slower eating if needed. Less common possible side effects can include abdominal pain, constipation, headache, dizziness, fatigue, and hypoglycemia. If patients develop severe belly pain that radiates to their back, they should be assessed for pancreatitis.

It’s also important to demonstrate for patients how to do the injections, Fox said. Liraglutide dosing begins at 0.6 mg daily for a week, followed by a week at 1.2 mg, a week at 1.8 mg, a week at 2.4 mg, and then 3 mg daily. Semaglutide dosing starts at 0.25 mg weekly for 4 weeks, then going up each subsequent month as needed to 0.5 mg, then 1 mg, then 1.7 mg, and finally 2.4 mg. Though there’s no standard follow-up schedule for these medications, Fox suggested considering monthly visits for the first 3 months and then every 2-3 months to assess heart rate and blood pressure, the injection site, adherence, side effects, and the effect on BMI and eating.

“Are they getting appetite suppression, but not too much appetite suppression?” Fox said. “Just like in eating disorder treatment, we want our patients to eat regularly spaced meals. If their appetite is so suppressed that they are hardly eating anything, that’s a problem.”

Fox also offered the following additional pearls about these medications:

• Though manufacturers have struggled to keep up with demand, the shortages of these medications are improving. However, beware the compounding pharmacies filling the gap because compounded medications are not FDA approved, and quality control issues are a concern.
• Prior authorizations are usually needed, and common reasons for denial to anticipate include lack of documentation on not having contraindications, the patient not following a low-calorie diet or engaging in physical activity, and the patient not having seen a registered dietitian.
• Patients should expect gastrointestinal side effects, but ondansetron can be prescribed to lessen the intensity.

Phentermine/Topiramate

Phentermine/topiramate extended-release is a once-daily oral tablet, with the phentermine acting to reduce appetite (by simulating the release of norepinephrine) and the topiramate reducing caloric intake and food reward response (by increasing gamma-aminobutyric acid activity). It’s approved for those aged 12 years or older with a BMI of at least the 95th percentile and should be considered in those with strong hunger, low energy, binge eating disorder, or migraines, as well as those who have insurance coverage for it. It can result in up to a 10% change in BMI.

Contraindications include pregnancy, substance use, cardiovascular disease (though it’s okay in patients with controlled hypertension), hyperthyroidism, glaucoma, and monoamine oxidase inhibitor (MAOI) use. Fox emphasized the teratogenic effects, so patients capable of pregnancy need to be on reliable birth control. The most common side effects include paresthesia, dizziness, dysgeusia, insomnia, and constipation.

A risk of topiramate is kidney stones, so patients should drink a lot of water, especially in hot weather, Fox said. Other risks can include metabolic acidosis, suicidality, poor cognitive function, high blood pressure, and renal impairment.

“If your patient is struggling academically, I might use this medication a bit more cautiously, particularly when the dose gets above 100 mg a day,” Fox said. “That’s when the cognitive effects tend to emerge more strongly.”

Patients with congenital heart disease should meet with their cardiologist before starting this medication, and although patients taking selective serotonin reuptake inhibitors (SSRIs) can take this, there is a potential increased risk for serotonin syndrome because phentermine has a little bit of serotonergic activity, she said.

Before prescribing, do an exam to ensure the patient doesn’t have a heart murmur, isn’t hypertensive, isn’t pregnant, has normal kidney function, and has bicarbonate in a reasonable range. Dosing begins with a daily 3.75/23-mg capsule for 2 weeks, followed by 2 weeks at 7.5 mg/46 mg. As with the GLP-1 drugs, Fox advises considering monthly follow-ups for the first 3 months and then visits every 2-3 months. Each visit should include the assessment of cardiovascular health, heart rate, blood pressure, side effects, pregnancy risk, and the medication’s effect on BMI and eating. If the patient is tolerating a dose of 7.5 mg/46 mg, it can be increased to 11.25 mg/69 mg for 2 weeks and then to 15 mg/92 mg. Bicarbonate and creatinine should be checked every 6-12 months; if bicarbonate < 18 mEq/L, the dose should be reduced and then bicarbonate should be checked again a month later.

Fox noted that this drug is expected to go off patent in late 2024 or in 2025, which will substantially reduce the cost. It’s also possible to prescribe phentermine and topiramate separately, which may reduce costs or help with insurance coverage and can allow for evening dosing of topiramate.
 

Phentermine

Phentermine alone is only approved for those older than 16 years who have a BMI of at least 30, or at least 27 with weight-related comorbidities, and it’s not approved for use longer than 12 weeks. It results in a BMI change of up to 5%. It should be considered in those with strong hunger and low energy and in those who don’t have adequate insurance coverage because out-of-pocket costs can be as little as $5/mo.

Contraindications are the same as those for the combined pill above: Substance use, cardiovascular disease, hyperthyroidism, glaucoma, MAOI use, and agitation. Again, take caution with patients who have hypertension, have congenital heart disease, or take SSRIs or insulin.

Side effects can include palpitations, tachycardia, dry mouth, headache, insomnia, and anxiety. The dose starts at 15 mg daily, and Fox advises following a similar follow-up as with the other medications, at which clinicians should assess BMI, the medication’s effect on eating, cardiovascular health, and side effects and have a discussion about off-label use. Off-label use refers to prescriptions lasting longer than 12 weeks, but it’s arguably safer than attention-deficit/hyperactivity disorder stimulants because of the lower addiction potential, Fox said.
 

What Else to Know

Because obesity is a chronic disease, treatment will be ongoing, Fox noted. A lot of people will ask when or where the “off-ramp” for these medications is, but many people will need these medications long term just as someone with other chronic diseases requires lifetime pharmacotherapy. The treatment intensity will vary based on disease severity and individual characteristics, Fox said.

For those feeling overwhelmed by the options, Fox advises clinicians to start by picking one medication to learn and then spending the time to read the FDA package insert in full. Get samples and then closely follow patients to learn that medication well before moving on to learn another. She also noted the opportunity for pediatricians to see a pediatric obesity medicine fellowship.

No external funding was used for the presentation. Fox is a site principal investigator for clinical trials sponsored by Novo Nordisk and Eli Lilly. Ivers had no disclosures.

A version of this article first appeared on Medscape.com.

ORLANDO, FLORIDA — The rationale for using obesity medications in pediatric patients is that it’s using “a biological intervention to treat a biologically based disease,” according to Claudia Fox, MD, MPH, an associate professor of pediatrics and codirector of the Center for Pediatric Obesity Medicine at the University of Minnesota, Minneapolis. At the annual meeting of the American Academy of Pediatrics (AAP), Fox provided an overview of what obesity medications are approved for in youth and how to determine which medications may be best for different patients.

“This field is changing so rapidly that even over the course of the last 3 or 4 months, the verbiage around what we should be calling these interventions has changed,” Fox noted. Instead of “anti-obesity” medications, “most of us are now using the term obesity medications to highlight or to reduce chances of stigma and bias that can come along with this topic.”

Jessica Ivers, MD, a pediatrician at Swedish Pediatrics in Seattle, Washington, said she found the session very informative, particularly because she doesn’t think many pediatricians currently feel very comfortable prescribing obesity medications.

“It answered questions that any general pediatrician would have, and it’s kind of a new field that people are learning about,” Ivers said. “I think we just need more education. It’s just too new, and people haven’t had the education and the support from colleagues to [use the medications].”

Fox first reminded attendees of precisely what obesity is: A chronic, relapsing, multifactorial, neurobehavioral disease that involves the accumulation and/or distribution of excess body fat that results in impaired health. AAP clinical practice guidelines currently advise that youth aged 12 years or older who have obesity be offered weight loss pharmacotherapy as an adjunct to lifestyle treatment, taking into consideration the indications, risks, and benefits of each medication.

That doesn’t necessarily mean every child aged 12 years or older with a body mass index (BMI) of at least the 95th percentile should be prescribed one of these medications, Fox said. But pediatricians should start becoming familiar with the options and recognize that part of reducing the stigma of this disease is emphasizing that these medications are prescribed not for “weight loss” but to treat the disease of obesity, Fox said. The guidelines advise “early, intensive care” and focusing on the whole child, “using a family-centered and nonstigmatizing approach that acknowledges obesity’s biologic, social, and structural drivers.”
 

Offer the Full Spectrum of Care Early On

Early intervention means starting obesity treatment at diagnosis, without watchful waiting or the previously recommended staged approach. Instead of trying lifestyle therapy for 3-6 months, then considering the addition of medication, and then considering bariatric surgery, “we should be offering the full spectrum of obesity care as appropriate for that individual patient,” Fox said.

Some children with severe obesity may need the combination of lifestyle therapy and pharmacotherapy right up front, whereas another might be able to try lifestyle therapy alone for a while first. “What we know is that, for most interventions, whether it is lifestyle therapy, a medication, or bariatric surgery, early response typically predicts longer-term response,” Fox said. A study conducted by her group, for example, found that a 3% BMI reduction after 1 month with lifestyle therapy was very predictive of clinically meaningful BMI reduction at 1 year.

As with any medical treatment, physicians need to weigh the risks of the medication — short-term side effects and unknown long-term risks (or benefits) — against the risks of not treating. Because obesity is a progressive disease, “if we don’t treat it, most will develop comorbid conditions, or worsening of their already present comorbid conditions, and this does indeed lead to shortened life expectancy,” Fox said. Those who should be treated with medication are obviously those in whom the benefits outweigh the risks, Fox said, which depends on their age, their comorbidities, the severity of obesity, and the safety and efficacy of medication options.

“If I have a patient who has maybe class 2 obesity but no other comorbid conditions, I may be less inclined to start an obesity medication than a kid who has class 1 obesity and obstructive sleep apnea, for instance,” Fox said. “Some of the medications are very, very potent and effective. If you have a kid who maybe has less severe forms of obesity, perhaps they don’t need something that’s so potent.”

BMI trajectory is also a factor to consider. She said she may not be too concerned about a 16-year-old who has always been at the 95th percentile and is otherwise healthy, but the situation is different for a 16-year-old who used to be in the 25th percentile and has rapidly progressed to the 50th and then 75th percentiles in a trajectory heading straight up.

Another factor that may come into play is the patient and family preferences, though Fox noted that weight bias and stigma often interfere here. If obesity medications are brought up, the family may bring up the need for more exercise and better meal prep at home.

“They have this sense that they just need to try harder, that if they did that, the obesity would somehow get better on its own,” Fox said. “That’s an internalized bias that it’s somehow their fault, rather than realizing that this is indeed a biological disorder.”

Finally, clinicians may want to consider the child’s response to lifestyle therapy and whether they have already had bariatric surgery because these medications can be prescribed in people who did not have an adequate response to surgery.
 

Overview of the Medications

There are currently six obesity medications approved by the Food and Drug Administration (FDA) for use in youth: Phentermine, orlistat, liraglutide, phentermine/topiramate, semaglutide, and setmelanotide.

Of these, orlistat is rarely used now because it results in the least amount of change in BMI (about a 3% loss change in BMI), has a lot of gastrointestinal side effects, often is not covered by insurance, and is expensive out of pocket. Setmelanotide is indicated only in those aged 6 years or older who have obesity because of Bardet-Biedl syndrome or one of three other rare genetic conditions: a POMC, LEPR, or PCSK1 deficiency. Fox therefore focused on the other medications besides these two.

While nearly all the currently available obesity medications are only approved in those aged 12 years or older, Fox noted that studies are ongoing at younger ages, so some of these medications may receive approval in younger populations in the future. The only one currently available for a younger age is liraglutide, which is approved down to 6 years old in children with type 2 diabetes.

“Very young kids who have very severe forms of obesity need intervention, and unfortunately, at this point, we really don’t have much to offer them,” Fox said.

Fox highlighted six key factors to consider in selecting a medication for those aged 12 years or older, though one of these, in the US healthcare system, can tend to trump all the others. Those factors are mechanism of action, side effect profile, effects on other diagnoses, patient/family preferences, provider comfort, and finally — the potentially overruling one — insurance coverage and access.

“These days, insurance coverage and access are really the No. 1 driver when I’m seeing a patient,” Fox said. “The first thing I do is look at their insurance and then also look at what kind of updates our pharmacist has given us about which medication is currently in stock.”

Each medication has different properties that should be considered with the child’s health profile. For example, topiramate is a carbonic anhydrase inhibitor so likely shouldn’t be prescribed in a child who is taking any other carbonic anhydrase inhibitor. Fox said she probably wouldn’t prescribe phentermine in a child with severe anxiety because it might enhance the anxiety effect. But if a child has migraines, she may be more inclined to try phentermine/topiramate first because the topiramate may help with the migraines. Similarly, if a child has type 2 diabetes or prediabetes, she may lean toward one of the glucagon-like peptide 1 (GLP-1) agonist drugs.
 

Liraglutide and Semaglutide

Liraglutide and semaglutide are both GLP-1 receptor agonists administered subcutaneously to reduce appetite, increase satiety, slow gastric emptying, and reduce the food reward response in the brain. Liraglutide can result in up to 4.5%-5% change in BMI, and semaglutide, the most potent of all the medications, can result in up to a 17% change in BMI.

Liraglutide and semaglutide are both approved for patients aged 12 years or older who weigh at least 60 kg and have a BMI of at least the 95th percentile. Liraglutide is also approved for those aged 10 years or older with type 2 diabetes. Both are contraindicated in those with a family history of medullary thyroid cancer or multiple endocrine neoplasia II. The risks to watch for include pancreatitis and gallbladder disease. Also keep in mind if you have a patient with type 1 diabetes and insulin resistance; prescribing a GLP-1 agonist is appropriate, but their insulin needs will decrease, necessitating close monitoring of their blood glucose, Fox noted.

These GLP-1 medications can be considered for those who have insurance coverage for them, who have diabetes or prediabetes, who are comfortable with daily (liraglutide) or weekly (semaglutide) injections, who have food cravings, and who have poor satiety or satiation. Without insurance, these medications are very expensive.

The most common side effects include injection site reactions and nausea, vomiting, and diarrhea, though all these usually fade and can be minimized with small portions and slower eating if needed. Less common possible side effects can include abdominal pain, constipation, headache, dizziness, fatigue, and hypoglycemia. If patients develop severe belly pain that radiates to their back, they should be assessed for pancreatitis.

It’s also important to demonstrate for patients how to do the injections, Fox said. Liraglutide dosing begins at 0.6 mg daily for a week, followed by a week at 1.2 mg, a week at 1.8 mg, a week at 2.4 mg, and then 3 mg daily. Semaglutide dosing starts at 0.25 mg weekly for 4 weeks, then going up each subsequent month as needed to 0.5 mg, then 1 mg, then 1.7 mg, and finally 2.4 mg. Though there’s no standard follow-up schedule for these medications, Fox suggested considering monthly visits for the first 3 months and then every 2-3 months to assess heart rate and blood pressure, the injection site, adherence, side effects, and the effect on BMI and eating.

“Are they getting appetite suppression, but not too much appetite suppression?” Fox said. “Just like in eating disorder treatment, we want our patients to eat regularly spaced meals. If their appetite is so suppressed that they are hardly eating anything, that’s a problem.”

Fox also offered the following additional pearls about these medications:

• Though manufacturers have struggled to keep up with demand, the shortages of these medications are improving. However, beware the compounding pharmacies filling the gap because compounded medications are not FDA approved, and quality control issues are a concern.
• Prior authorizations are usually needed, and common reasons for denial to anticipate include lack of documentation on not having contraindications, the patient not following a low-calorie diet or engaging in physical activity, and the patient not having seen a registered dietitian.
• Patients should expect gastrointestinal side effects, but ondansetron can be prescribed to lessen the intensity.

Phentermine/Topiramate

Phentermine/topiramate extended-release is a once-daily oral tablet, with the phentermine acting to reduce appetite (by simulating the release of norepinephrine) and the topiramate reducing caloric intake and food reward response (by increasing gamma-aminobutyric acid activity). It’s approved for those aged 12 years or older with a BMI of at least the 95th percentile and should be considered in those with strong hunger, low energy, binge eating disorder, or migraines, as well as those who have insurance coverage for it. It can result in up to a 10% change in BMI.

Contraindications include pregnancy, substance use, cardiovascular disease (though it’s okay in patients with controlled hypertension), hyperthyroidism, glaucoma, and monoamine oxidase inhibitor (MAOI) use. Fox emphasized the teratogenic effects, so patients capable of pregnancy need to be on reliable birth control. The most common side effects include paresthesia, dizziness, dysgeusia, insomnia, and constipation.

A risk of topiramate is kidney stones, so patients should drink a lot of water, especially in hot weather, Fox said. Other risks can include metabolic acidosis, suicidality, poor cognitive function, high blood pressure, and renal impairment.

“If your patient is struggling academically, I might use this medication a bit more cautiously, particularly when the dose gets above 100 mg a day,” Fox said. “That’s when the cognitive effects tend to emerge more strongly.”

Patients with congenital heart disease should meet with their cardiologist before starting this medication, and although patients taking selective serotonin reuptake inhibitors (SSRIs) can take this, there is a potential increased risk for serotonin syndrome because phentermine has a little bit of serotonergic activity, she said.

Before prescribing, do an exam to ensure the patient doesn’t have a heart murmur, isn’t hypertensive, isn’t pregnant, has normal kidney function, and has bicarbonate in a reasonable range. Dosing begins with a daily 3.75/23-mg capsule for 2 weeks, followed by 2 weeks at 7.5 mg/46 mg. As with the GLP-1 drugs, Fox advises considering monthly follow-ups for the first 3 months and then visits every 2-3 months. Each visit should include the assessment of cardiovascular health, heart rate, blood pressure, side effects, pregnancy risk, and the medication’s effect on BMI and eating. If the patient is tolerating a dose of 7.5 mg/46 mg, it can be increased to 11.25 mg/69 mg for 2 weeks and then to 15 mg/92 mg. Bicarbonate and creatinine should be checked every 6-12 months; if bicarbonate < 18 mEq/L, the dose should be reduced and then bicarbonate should be checked again a month later.

Fox noted that this drug is expected to go off patent in late 2024 or in 2025, which will substantially reduce the cost. It’s also possible to prescribe phentermine and topiramate separately, which may reduce costs or help with insurance coverage and can allow for evening dosing of topiramate.
 

Phentermine

Phentermine alone is only approved for those older than 16 years who have a BMI of at least 30, or at least 27 with weight-related comorbidities, and it’s not approved for use longer than 12 weeks. It results in a BMI change of up to 5%. It should be considered in those with strong hunger and low energy and in those who don’t have adequate insurance coverage because out-of-pocket costs can be as little as $5/mo.

Contraindications are the same as those for the combined pill above: Substance use, cardiovascular disease, hyperthyroidism, glaucoma, MAOI use, and agitation. Again, take caution with patients who have hypertension, have congenital heart disease, or take SSRIs or insulin.

Side effects can include palpitations, tachycardia, dry mouth, headache, insomnia, and anxiety. The dose starts at 15 mg daily, and Fox advises following a similar follow-up as with the other medications, at which clinicians should assess BMI, the medication’s effect on eating, cardiovascular health, and side effects and have a discussion about off-label use. Off-label use refers to prescriptions lasting longer than 12 weeks, but it’s arguably safer than attention-deficit/hyperactivity disorder stimulants because of the lower addiction potential, Fox said.
 

What Else to Know

Because obesity is a chronic disease, treatment will be ongoing, Fox noted. A lot of people will ask when or where the “off-ramp” for these medications is, but many people will need these medications long term just as someone with other chronic diseases requires lifetime pharmacotherapy. The treatment intensity will vary based on disease severity and individual characteristics, Fox said.

For those feeling overwhelmed by the options, Fox advises clinicians to start by picking one medication to learn and then spending the time to read the FDA package insert in full. Get samples and then closely follow patients to learn that medication well before moving on to learn another. She also noted the opportunity for pediatricians to see a pediatric obesity medicine fellowship.

No external funding was used for the presentation. Fox is a site principal investigator for clinical trials sponsored by Novo Nordisk and Eli Lilly. Ivers had no disclosures.

A version of this article first appeared on Medscape.com.

ORLANDO, FLORIDA — Asthma is the most common cause of chronic cough in children, but it’s important to be aware of other differential diagnoses for those patients who have less common concerns, according to Rajeev Bhatia, MD, division chief of pulmonology at Phoenix Children’s in Phoenix, Arizona. Bhatia reviewed both the major causes of chronic cough as well as the rare zebras to watch out for in a presentation at the American Academy of Pediatrics (AAP) 2024 National Conference.

“When you see a patient for cough, the most important thing is, history, history, history,” Bhatia said. “There are many, many, many clues in the history, age of onset, and duration.” That includes starting at birth to ensure you don’t miss key details such as a preterm birth. It’s also important to discuss what the cough sounds like, how frequent it is, what makes it better, what makes it worse, and how it’s affecting the child and others around them — all of which can help narrow the diagnosis.

Jose Quijada, DO, a pediatrician with CommuniCare Pediatrics in San Antonio, found the session “incredibly useful” not only because of the specific pointers about each condition but also because of the realistic case studies he included throughout.

“Sometimes when you’re practicing, you focus on what’s most common,” Quijada said, so it was helpful to get a review of some of the key features and red flags that point to less common causes that may need to be considered. He particularly appreciated the discussion of habitual cough and potential treatments because those can be challenging patients and it can be tough to find a middle ground with how much workup to do.
 

Common Causes of Chronic Cough

The coughing from inadequately controlled asthma tends to be nonproductive and worse at night or in the early morning, Bhatia explained, and it’s often accompanied by wheezing, shortness of breath, and chest tightness. While fractional exhaled nitric oxide can be useful, “studies show that it is more useful for to monitor the symptoms rather than to diagnose the asthma,” Bhatia told attendees, but he also added that spirometry can be normal in patients with asthma. In young patients, the Asthma Predictive Index can be an invaluable tool, he also said.

Another common cause of chronic cough is a postinfectious cough, which lasts more than 3 weeks after resolution of acute upper respiratory infection. It occurs in about one in 10 children after a viral infection such as a rhinovirus or respiratory syncytial virus infection and results from extensive inflammation and disruption of the airway epithelial integrity. Chest x-rays are usually normal in these patients, and the cough will usually resolve on its own. Albuterol has not been shown to be any more effective than placebo for a postinfectious cough, and antibiotics similarly have no clinically useful role.

A wet cough that lasts for at least 4 weeks and appears to have no other specific cause may be protracted bacterial bronchitis (PBB). While the cause of PBB isn’t known, it could be due to frequent illnesses that cause airway inflammation and injury over time, thereby making it easier for bacteria to grow and cause infection. Risk factors include tracheomalacia/bronchomalacia and childcare attendance, and typical pathogens include Haemophilus influenzae, Streptococcus pneumonia, and Moraxella catarrhalis.

While an x-ray can be done, radiography is often normal in patients with PBB. Bronchoscopy will reveal purulent secretions. PBB should resolve with at least 2 weeks of antibiotics, including amoxicillin-clavulanate, but the course can be extended to 4 weeks if the cough persists. However, about 40% of patients will have recurrence, and those with recurrent PBB or with a chronic cough unresponsive to 4 weeks of antibiotics should be referred to a pediatric pulmonologist.

Upper airway cough syndrome is the updated name for postnasal drip, which can occur with both allergic and nonallergic rhinitis and chronic rhinosinusitis. This is often a dry, throat-clearing cough that can be accompanied by headaches, nasal congestion, and sinus or facial tenderness. An examination will reveal posterior pharynx cobblestoning, Bhatia said, and empirical use of intranasal steroids can be both diagnostic and therapeutic for upper airway cough syndrome. He also emphasized the importance of taking an environmental history and avoiding exposure of environmental triggers.
 

Uncommon Pulmonary Causes of Chronic Cough

After discussing frequent reasons patients may present with a chronic cough, Bhatia went on to discuss the less common things to consider if the provider has eliminated the other possibilities. These include both pulmonary causes as well as congenital malformations, gastrointestinal causes, and habit cough.

A child who presents with a sudden onset of cough or wheeze in the absence of an upper respiratory infection may have a retained foreign body. This cough can be mistaken for bronchiolitis, asthma, croup, and other infectious conditions, especially because a partial obstruction can make diagnosis confusing or challenging. Adding to the challenge is that most foreign bodies will be radiolucent. A decubitus chest x-ray could be useful, but bronchoscopy is necessary for diagnosis. Bhatia stressed that it’s easy to miss a foreign body in younger children and that the wheezing can be more prominent on one side or another.

Cystic fibrosis, another uncommon cause of chronic cough, is ideally diagnosed via newborn screening, but screening is imperfect and can involve missed diagnoses or false negatives. Over 75% of cystic fibrosis cases are diagnosed by age 2, but that means a substantial number of cases still are not diagnosed until older childhood or later. This cough will be a chronic productive/wet cough.

A family history of cystic fibrosis may be present but doesn’t have to be, so signs to look for include poor weight gain, sinusitis, nasal polyps, clubbing, and isolation of suspicious organisms from a respiratory culture, such as Pseudomonas aeruginosa or Burkholderia cepacia complex. Clubbing in the fingers is a particularly telltale symptom of undiagnosed cystic fibrosis, and bronchiectasis of unknown etiology will be present. Suspicion of cystic fibrosis should lead to a referral to a cystic fibrosis center for a sweat test.

Even rarer than cystic fibrosis is primary ciliary dyskinesia (PCD), an inherited autosomal recessive disease that occurs in about one in 20,000 live births and involves a structural or functional defect in the cilia. About half of all patients with PCD will have situs inversus — an arrangement of chest and abdominal organs that is a mirror image of typical human anatomical presentation — but most people with situs inversus do not have PCD. One type of PCD is Kartagener syndrome, identified by the triad of situs inversus totalis, chronic sinusitis, and bronchiectasis.

Children with PCD present with a chronic productive cough and recurrent pneumonias, and nearly all patients will have rhinosinusitis. About 60% of patients will develop respiratory symptoms such as mild distress or cough in their first month, and recurrent otitis media is common in these patients. PCD diagnosis is based on a combination of genetic testing, nasal nitric oxide, and evaluation of ciliary motion and structure. Clinical suspicion of PCD should lead to a specialist referral.

Nearly all people with PCD will eventually develop bronchiectasis, where the priority should be airway clearance using antibiotics for acute exacerbations and chronic azithromycin therapy for recurrent exacerbations. Patients with chronic rhinosinusitis, chronic otitis media, and nasal polyposis should be referred to an ENT specialist.
 

Other Uncommon Causes of Chronic Cough

A non-pulmonary, uncommon cause of chronic cough is a vascular ring, a congenital anomaly in which blood vessels encircle and potentially constrict the esophagus and/or trachea. The most common type is a double aortic arch, but a right aortic arch or pulmonary artery sling is also possible. These coughs sound harsh and are usually accompanied by stridor, dyspnea and feeding problems. Workup includes an echocardiogram, a CT angiogram, and possibly a bronchoscopy to determine the extent of the airway narrowing. In symptomatic patients, surgery is indicated for correction.

Another congenital malformation that can cause chronic cough is a tracheoesophageal fistula, which occurs in about one in 3500 live births, commonly linked to trisomy disorders and VACTERL. Several types of tracheoesophageal fistula exist, and H-type fistula is associated with late onset symptoms. The cough can be wet or dry and sometimes sounds like barking because of the associated airway collapse. Patients often have recurrent pneumonia, bronchitis, and cough or cyanosis with feeding. Workup should include an upper gastrointestinal series but not with barium, Bhatia said, because that can cause pneumonitis. Instead, the series should be done with a thickened water-soluble contrast material, and a bronchoscopy may be indicated as well.

Though common as a condition in adults, gastroesophageal reflux disease (GERD) is a rare, but possible, cause of chronic cough in children. More often, the reflux is the result of the cough rather than the cause. The most sensitive tool for assessing GERD is esophageal 24-hour pH/impedance reflux monitoring. However, treatment of the reflux for cough is not recommended unless the patient has clinical features of GERD, including dystonic neck posturing in infants, heartburn, regurgitation, or other symptoms. If the patient has clinical symptoms, then treatment is acid suppressive therapy for 4-8 weeks, followed by a clinical reassessment.

An uncommon cause of chronic cough with no biological mechanism is habit cough. Habit cough is most easily distinguished from other coughs by its sounds, a “large, loud, honking noise,” Bhatia said. It also lacks a clear trigger and is usually absent during sleep, but it can be continuous during the day. Frustratingly, the patients themselves often don’t seem bothered by the cough, but “it’s very disruptive in the school and everywhere else,” Bhatia said. Families and/providers will often have tried multiple treatments and seen no improvement with habit cough.

The first thing to do with habit cough is reassure the family that there’s nothing serious going on because they are often worried by this point. Several non-pharmaceutical treatments can be effective, such as suggestion therapy or the “warm water technique,” in which the patient takes a sip of warm water every time they feel the urge to cough. “If they’re able to break the cycle, most of the time, they are fine,” Bhatia said. In rarer cases, more involved behavioral interventions may be indicated, such as a psychology referral if an underlying anxiety or other behavioral disorder is contributing.
 

Newer Causes of Cough

Two more recent causes of cough to watch for are long COVID and e-cigarette or vaping product–associated lung injury (EVALI), Bhatia said. The clearest sign of EVALI is a history of e-cigarette/vaping exposure, but clinical symptoms include a dry cough that occurs with dyspnea and chest pain. A chest x-ray may show diffuse, hazy, or consolidative opacities. Sometimes antibiotics or steroids can be helpful, but the evidence isn’t strong, and the most effective treatment is stopping e-cigarette use. Less commonly, passive exposure to vaping can also be associated with EVALI.

The most recent research on long COVID suggests that about 10-20% of children with acute COVID develop long COVID, and about a quarter of these patients develop a chronic dry cough. It’s often associated with fatigue and shortness of breath, which can be assessed with cardiopulmonary exercise testing. Sometimes a short trial of inhaled steroids can help.

Bhatia also mentioned a handful of other uncommon causes of chronic cough that most American pediatricians are unlikely to see: Childhood interstitial lung disease, tuberculosis, use of Angiotensin-Converting Enzyme inhibitors, and a build-up of ear wax via the Arnold’s nerve reflex.
 

Evaluation and Workup

Bhatia also discussed what to cover while taking a history and questions to ask. The history should include the type of cough, the onset timing (sudden vs gradual), associated symptoms, the cough trajectory, medications the patient is taking, and the patient’s past medical history and environmental exposures. Those attributes are included in this more comprehensive list of questions to consider during evaluation, adapted from a list provided in a 2019 article in Paediatric Respiratory Reviews:

• Age of onset and duration?
• Was the onset sudden or associate with an illness?
• Is the cough wet or dry?
• What does the cough sound like?
• How often does the cough occur?
• Is it progressive?
• Is it present during sleep?
• Are there any other associated symptoms, such as wheeze, dyspnea, vomiting, chest pain, etc?
• Are there any exacerbating factors or known triggers?
• Are there any relieving factors, including a trial of bronchodilators?
• Has there been exposure to auto-irritants, such as secondhand smoke?
• What is the cough’s effect on the child and on others around the child?
• Does the child have any other underlying conditions such as neuromuscular disease or asthma?
• What medications is the child taking or has recently taken?
• Is there a family history of atopy and/or respiratory disease?

Bhatia also recommended paying special attention to the following red flags or key features that may help more quickly narrow the diagnosis and often require a specialist referral:

• Digital clubbing, failure to thrive, or low tone
• An abnormal cardiac exam
• Tachypnea, hypoxemia, chest retractions, or hemoptysis
• Abnormal breath sounds such as unilateral wheezing or coarse crackles
• Abnormal spirometry in those aged 5 and older showing reversible obstruction, which often indicates asthma
• An abnormal chest x-ray with, for example, bilateral infiltrates, hyperinflation, right middle lobe syndrome, situs inversus, unilateral hyperlucency, a right aortic arch, etc.

No external funding was used for the presentation. Bhatia and Quijada had no disclosures.
 

A version of this article appeared on Medscape.com.

ORLANDO, FLORIDA — Asthma is the most common cause of chronic cough in children, but it’s important to be aware of other differential diagnoses for those patients who have less common concerns, according to Rajeev Bhatia, MD, division chief of pulmonology at Phoenix Children’s in Phoenix, Arizona. Bhatia reviewed both the major causes of chronic cough as well as the rare zebras to watch out for in a presentation at the American Academy of Pediatrics (AAP) 2024 National Conference.

“When you see a patient for cough, the most important thing is, history, history, history,” Bhatia said. “There are many, many, many clues in the history, age of onset, and duration.” That includes starting at birth to ensure you don’t miss key details such as a preterm birth. It’s also important to discuss what the cough sounds like, how frequent it is, what makes it better, what makes it worse, and how it’s affecting the child and others around them — all of which can help narrow the diagnosis.

Jose Quijada, DO, a pediatrician with CommuniCare Pediatrics in San Antonio, found the session “incredibly useful” not only because of the specific pointers about each condition but also because of the realistic case studies he included throughout.

“Sometimes when you’re practicing, you focus on what’s most common,” Quijada said, so it was helpful to get a review of some of the key features and red flags that point to less common causes that may need to be considered. He particularly appreciated the discussion of habitual cough and potential treatments because those can be challenging patients and it can be tough to find a middle ground with how much workup to do.
 

Common Causes of Chronic Cough

The coughing from inadequately controlled asthma tends to be nonproductive and worse at night or in the early morning, Bhatia explained, and it’s often accompanied by wheezing, shortness of breath, and chest tightness. While fractional exhaled nitric oxide can be useful, “studies show that it is more useful for to monitor the symptoms rather than to diagnose the asthma,” Bhatia told attendees, but he also added that spirometry can be normal in patients with asthma. In young patients, the Asthma Predictive Index can be an invaluable tool, he also said.

Another common cause of chronic cough is a postinfectious cough, which lasts more than 3 weeks after resolution of acute upper respiratory infection. It occurs in about one in 10 children after a viral infection such as a rhinovirus or respiratory syncytial virus infection and results from extensive inflammation and disruption of the airway epithelial integrity. Chest x-rays are usually normal in these patients, and the cough will usually resolve on its own. Albuterol has not been shown to be any more effective than placebo for a postinfectious cough, and antibiotics similarly have no clinically useful role.

A wet cough that lasts for at least 4 weeks and appears to have no other specific cause may be protracted bacterial bronchitis (PBB). While the cause of PBB isn’t known, it could be due to frequent illnesses that cause airway inflammation and injury over time, thereby making it easier for bacteria to grow and cause infection. Risk factors include tracheomalacia/bronchomalacia and childcare attendance, and typical pathogens include Haemophilus influenzae, Streptococcus pneumonia, and Moraxella catarrhalis.

While an x-ray can be done, radiography is often normal in patients with PBB. Bronchoscopy will reveal purulent secretions. PBB should resolve with at least 2 weeks of antibiotics, including amoxicillin-clavulanate, but the course can be extended to 4 weeks if the cough persists. However, about 40% of patients will have recurrence, and those with recurrent PBB or with a chronic cough unresponsive to 4 weeks of antibiotics should be referred to a pediatric pulmonologist.

Upper airway cough syndrome is the updated name for postnasal drip, which can occur with both allergic and nonallergic rhinitis and chronic rhinosinusitis. This is often a dry, throat-clearing cough that can be accompanied by headaches, nasal congestion, and sinus or facial tenderness. An examination will reveal posterior pharynx cobblestoning, Bhatia said, and empirical use of intranasal steroids can be both diagnostic and therapeutic for upper airway cough syndrome. He also emphasized the importance of taking an environmental history and avoiding exposure of environmental triggers.
 

Uncommon Pulmonary Causes of Chronic Cough

After discussing frequent reasons patients may present with a chronic cough, Bhatia went on to discuss the less common things to consider if the provider has eliminated the other possibilities. These include both pulmonary causes as well as congenital malformations, gastrointestinal causes, and habit cough.

A child who presents with a sudden onset of cough or wheeze in the absence of an upper respiratory infection may have a retained foreign body. This cough can be mistaken for bronchiolitis, asthma, croup, and other infectious conditions, especially because a partial obstruction can make diagnosis confusing or challenging. Adding to the challenge is that most foreign bodies will be radiolucent. A decubitus chest x-ray could be useful, but bronchoscopy is necessary for diagnosis. Bhatia stressed that it’s easy to miss a foreign body in younger children and that the wheezing can be more prominent on one side or another.

Cystic fibrosis, another uncommon cause of chronic cough, is ideally diagnosed via newborn screening, but screening is imperfect and can involve missed diagnoses or false negatives. Over 75% of cystic fibrosis cases are diagnosed by age 2, but that means a substantial number of cases still are not diagnosed until older childhood or later. This cough will be a chronic productive/wet cough.

A family history of cystic fibrosis may be present but doesn’t have to be, so signs to look for include poor weight gain, sinusitis, nasal polyps, clubbing, and isolation of suspicious organisms from a respiratory culture, such as Pseudomonas aeruginosa or Burkholderia cepacia complex. Clubbing in the fingers is a particularly telltale symptom of undiagnosed cystic fibrosis, and bronchiectasis of unknown etiology will be present. Suspicion of cystic fibrosis should lead to a referral to a cystic fibrosis center for a sweat test.

Even rarer than cystic fibrosis is primary ciliary dyskinesia (PCD), an inherited autosomal recessive disease that occurs in about one in 20,000 live births and involves a structural or functional defect in the cilia. About half of all patients with PCD will have situs inversus — an arrangement of chest and abdominal organs that is a mirror image of typical human anatomical presentation — but most people with situs inversus do not have PCD. One type of PCD is Kartagener syndrome, identified by the triad of situs inversus totalis, chronic sinusitis, and bronchiectasis.

Children with PCD present with a chronic productive cough and recurrent pneumonias, and nearly all patients will have rhinosinusitis. About 60% of patients will develop respiratory symptoms such as mild distress or cough in their first month, and recurrent otitis media is common in these patients. PCD diagnosis is based on a combination of genetic testing, nasal nitric oxide, and evaluation of ciliary motion and structure. Clinical suspicion of PCD should lead to a specialist referral.

Nearly all people with PCD will eventually develop bronchiectasis, where the priority should be airway clearance using antibiotics for acute exacerbations and chronic azithromycin therapy for recurrent exacerbations. Patients with chronic rhinosinusitis, chronic otitis media, and nasal polyposis should be referred to an ENT specialist.
 

Other Uncommon Causes of Chronic Cough

A non-pulmonary, uncommon cause of chronic cough is a vascular ring, a congenital anomaly in which blood vessels encircle and potentially constrict the esophagus and/or trachea. The most common type is a double aortic arch, but a right aortic arch or pulmonary artery sling is also possible. These coughs sound harsh and are usually accompanied by stridor, dyspnea and feeding problems. Workup includes an echocardiogram, a CT angiogram, and possibly a bronchoscopy to determine the extent of the airway narrowing. In symptomatic patients, surgery is indicated for correction.

Another congenital malformation that can cause chronic cough is a tracheoesophageal fistula, which occurs in about one in 3500 live births, commonly linked to trisomy disorders and VACTERL. Several types of tracheoesophageal fistula exist, and H-type fistula is associated with late onset symptoms. The cough can be wet or dry and sometimes sounds like barking because of the associated airway collapse. Patients often have recurrent pneumonia, bronchitis, and cough or cyanosis with feeding. Workup should include an upper gastrointestinal series but not with barium, Bhatia said, because that can cause pneumonitis. Instead, the series should be done with a thickened water-soluble contrast material, and a bronchoscopy may be indicated as well.

Though common as a condition in adults, gastroesophageal reflux disease (GERD) is a rare, but possible, cause of chronic cough in children. More often, the reflux is the result of the cough rather than the cause. The most sensitive tool for assessing GERD is esophageal 24-hour pH/impedance reflux monitoring. However, treatment of the reflux for cough is not recommended unless the patient has clinical features of GERD, including dystonic neck posturing in infants, heartburn, regurgitation, or other symptoms. If the patient has clinical symptoms, then treatment is acid suppressive therapy for 4-8 weeks, followed by a clinical reassessment.

An uncommon cause of chronic cough with no biological mechanism is habit cough. Habit cough is most easily distinguished from other coughs by its sounds, a “large, loud, honking noise,” Bhatia said. It also lacks a clear trigger and is usually absent during sleep, but it can be continuous during the day. Frustratingly, the patients themselves often don’t seem bothered by the cough, but “it’s very disruptive in the school and everywhere else,” Bhatia said. Families and/providers will often have tried multiple treatments and seen no improvement with habit cough.

The first thing to do with habit cough is reassure the family that there’s nothing serious going on because they are often worried by this point. Several non-pharmaceutical treatments can be effective, such as suggestion therapy or the “warm water technique,” in which the patient takes a sip of warm water every time they feel the urge to cough. “If they’re able to break the cycle, most of the time, they are fine,” Bhatia said. In rarer cases, more involved behavioral interventions may be indicated, such as a psychology referral if an underlying anxiety or other behavioral disorder is contributing.
 

Newer Causes of Cough

Two more recent causes of cough to watch for are long COVID and e-cigarette or vaping product–associated lung injury (EVALI), Bhatia said. The clearest sign of EVALI is a history of e-cigarette/vaping exposure, but clinical symptoms include a dry cough that occurs with dyspnea and chest pain. A chest x-ray may show diffuse, hazy, or consolidative opacities. Sometimes antibiotics or steroids can be helpful, but the evidence isn’t strong, and the most effective treatment is stopping e-cigarette use. Less commonly, passive exposure to vaping can also be associated with EVALI.

The most recent research on long COVID suggests that about 10-20% of children with acute COVID develop long COVID, and about a quarter of these patients develop a chronic dry cough. It’s often associated with fatigue and shortness of breath, which can be assessed with cardiopulmonary exercise testing. Sometimes a short trial of inhaled steroids can help.

Bhatia also mentioned a handful of other uncommon causes of chronic cough that most American pediatricians are unlikely to see: Childhood interstitial lung disease, tuberculosis, use of Angiotensin-Converting Enzyme inhibitors, and a build-up of ear wax via the Arnold’s nerve reflex.
 

Evaluation and Workup

Bhatia also discussed what to cover while taking a history and questions to ask. The history should include the type of cough, the onset timing (sudden vs gradual), associated symptoms, the cough trajectory, medications the patient is taking, and the patient’s past medical history and environmental exposures. Those attributes are included in this more comprehensive list of questions to consider during evaluation, adapted from a list provided in a 2019 article in Paediatric Respiratory Reviews:

• Age of onset and duration?
• Was the onset sudden or associate with an illness?
• Is the cough wet or dry?
• What does the cough sound like?
• How often does the cough occur?
• Is it progressive?
• Is it present during sleep?
• Are there any other associated symptoms, such as wheeze, dyspnea, vomiting, chest pain, etc?
• Are there any exacerbating factors or known triggers?
• Are there any relieving factors, including a trial of bronchodilators?
• Has there been exposure to auto-irritants, such as secondhand smoke?
• What is the cough’s effect on the child and on others around the child?
• Does the child have any other underlying conditions such as neuromuscular disease or asthma?
• What medications is the child taking or has recently taken?
• Is there a family history of atopy and/or respiratory disease?

Bhatia also recommended paying special attention to the following red flags or key features that may help more quickly narrow the diagnosis and often require a specialist referral:

• Digital clubbing, failure to thrive, or low tone
• An abnormal cardiac exam
• Tachypnea, hypoxemia, chest retractions, or hemoptysis
• Abnormal breath sounds such as unilateral wheezing or coarse crackles
• Abnormal spirometry in those aged 5 and older showing reversible obstruction, which often indicates asthma
• An abnormal chest x-ray with, for example, bilateral infiltrates, hyperinflation, right middle lobe syndrome, situs inversus, unilateral hyperlucency, a right aortic arch, etc.

No external funding was used for the presentation. Bhatia and Quijada had no disclosures.
 

A version of this article appeared on Medscape.com.

ORLANDO, FLORIDA — Asthma is the most common cause of chronic cough in children, but it’s important to be aware of other differential diagnoses for those patients who have less common concerns, according to Rajeev Bhatia, MD, division chief of pulmonology at Phoenix Children’s in Phoenix, Arizona. Bhatia reviewed both the major causes of chronic cough as well as the rare zebras to watch out for in a presentation at the American Academy of Pediatrics (AAP) 2024 National Conference.

“When you see a patient for cough, the most important thing is, history, history, history,” Bhatia said. “There are many, many, many clues in the history, age of onset, and duration.” That includes starting at birth to ensure you don’t miss key details such as a preterm birth. It’s also important to discuss what the cough sounds like, how frequent it is, what makes it better, what makes it worse, and how it’s affecting the child and others around them — all of which can help narrow the diagnosis.

Jose Quijada, DO, a pediatrician with CommuniCare Pediatrics in San Antonio, found the session “incredibly useful” not only because of the specific pointers about each condition but also because of the realistic case studies he included throughout.

“Sometimes when you’re practicing, you focus on what’s most common,” Quijada said, so it was helpful to get a review of some of the key features and red flags that point to less common causes that may need to be considered. He particularly appreciated the discussion of habitual cough and potential treatments because those can be challenging patients and it can be tough to find a middle ground with how much workup to do.
 

Common Causes of Chronic Cough

The coughing from inadequately controlled asthma tends to be nonproductive and worse at night or in the early morning, Bhatia explained, and it’s often accompanied by wheezing, shortness of breath, and chest tightness. While fractional exhaled nitric oxide can be useful, “studies show that it is more useful for to monitor the symptoms rather than to diagnose the asthma,” Bhatia told attendees, but he also added that spirometry can be normal in patients with asthma. In young patients, the Asthma Predictive Index can be an invaluable tool, he also said.

Another common cause of chronic cough is a postinfectious cough, which lasts more than 3 weeks after resolution of acute upper respiratory infection. It occurs in about one in 10 children after a viral infection such as a rhinovirus or respiratory syncytial virus infection and results from extensive inflammation and disruption of the airway epithelial integrity. Chest x-rays are usually normal in these patients, and the cough will usually resolve on its own. Albuterol has not been shown to be any more effective than placebo for a postinfectious cough, and antibiotics similarly have no clinically useful role.

A wet cough that lasts for at least 4 weeks and appears to have no other specific cause may be protracted bacterial bronchitis (PBB). While the cause of PBB isn’t known, it could be due to frequent illnesses that cause airway inflammation and injury over time, thereby making it easier for bacteria to grow and cause infection. Risk factors include tracheomalacia/bronchomalacia and childcare attendance, and typical pathogens include Haemophilus influenzae, Streptococcus pneumonia, and Moraxella catarrhalis.

While an x-ray can be done, radiography is often normal in patients with PBB. Bronchoscopy will reveal purulent secretions. PBB should resolve with at least 2 weeks of antibiotics, including amoxicillin-clavulanate, but the course can be extended to 4 weeks if the cough persists. However, about 40% of patients will have recurrence, and those with recurrent PBB or with a chronic cough unresponsive to 4 weeks of antibiotics should be referred to a pediatric pulmonologist.

Upper airway cough syndrome is the updated name for postnasal drip, which can occur with both allergic and nonallergic rhinitis and chronic rhinosinusitis. This is often a dry, throat-clearing cough that can be accompanied by headaches, nasal congestion, and sinus or facial tenderness. An examination will reveal posterior pharynx cobblestoning, Bhatia said, and empirical use of intranasal steroids can be both diagnostic and therapeutic for upper airway cough syndrome. He also emphasized the importance of taking an environmental history and avoiding exposure of environmental triggers.
 

Uncommon Pulmonary Causes of Chronic Cough

After discussing frequent reasons patients may present with a chronic cough, Bhatia went on to discuss the less common things to consider if the provider has eliminated the other possibilities. These include both pulmonary causes as well as congenital malformations, gastrointestinal causes, and habit cough.

A child who presents with a sudden onset of cough or wheeze in the absence of an upper respiratory infection may have a retained foreign body. This cough can be mistaken for bronchiolitis, asthma, croup, and other infectious conditions, especially because a partial obstruction can make diagnosis confusing or challenging. Adding to the challenge is that most foreign bodies will be radiolucent. A decubitus chest x-ray could be useful, but bronchoscopy is necessary for diagnosis. Bhatia stressed that it’s easy to miss a foreign body in younger children and that the wheezing can be more prominent on one side or another.

Cystic fibrosis, another uncommon cause of chronic cough, is ideally diagnosed via newborn screening, but screening is imperfect and can involve missed diagnoses or false negatives. Over 75% of cystic fibrosis cases are diagnosed by age 2, but that means a substantial number of cases still are not diagnosed until older childhood or later. This cough will be a chronic productive/wet cough.

A family history of cystic fibrosis may be present but doesn’t have to be, so signs to look for include poor weight gain, sinusitis, nasal polyps, clubbing, and isolation of suspicious organisms from a respiratory culture, such as Pseudomonas aeruginosa or Burkholderia cepacia complex. Clubbing in the fingers is a particularly telltale symptom of undiagnosed cystic fibrosis, and bronchiectasis of unknown etiology will be present. Suspicion of cystic fibrosis should lead to a referral to a cystic fibrosis center for a sweat test.

Even rarer than cystic fibrosis is primary ciliary dyskinesia (PCD), an inherited autosomal recessive disease that occurs in about one in 20,000 live births and involves a structural or functional defect in the cilia. About half of all patients with PCD will have situs inversus — an arrangement of chest and abdominal organs that is a mirror image of typical human anatomical presentation — but most people with situs inversus do not have PCD. One type of PCD is Kartagener syndrome, identified by the triad of situs inversus totalis, chronic sinusitis, and bronchiectasis.

Children with PCD present with a chronic productive cough and recurrent pneumonias, and nearly all patients will have rhinosinusitis. About 60% of patients will develop respiratory symptoms such as mild distress or cough in their first month, and recurrent otitis media is common in these patients. PCD diagnosis is based on a combination of genetic testing, nasal nitric oxide, and evaluation of ciliary motion and structure. Clinical suspicion of PCD should lead to a specialist referral.

Nearly all people with PCD will eventually develop bronchiectasis, where the priority should be airway clearance using antibiotics for acute exacerbations and chronic azithromycin therapy for recurrent exacerbations. Patients with chronic rhinosinusitis, chronic otitis media, and nasal polyposis should be referred to an ENT specialist.
 

Other Uncommon Causes of Chronic Cough

A non-pulmonary, uncommon cause of chronic cough is a vascular ring, a congenital anomaly in which blood vessels encircle and potentially constrict the esophagus and/or trachea. The most common type is a double aortic arch, but a right aortic arch or pulmonary artery sling is also possible. These coughs sound harsh and are usually accompanied by stridor, dyspnea and feeding problems. Workup includes an echocardiogram, a CT angiogram, and possibly a bronchoscopy to determine the extent of the airway narrowing. In symptomatic patients, surgery is indicated for correction.

Another congenital malformation that can cause chronic cough is a tracheoesophageal fistula, which occurs in about one in 3500 live births, commonly linked to trisomy disorders and VACTERL. Several types of tracheoesophageal fistula exist, and H-type fistula is associated with late onset symptoms. The cough can be wet or dry and sometimes sounds like barking because of the associated airway collapse. Patients often have recurrent pneumonia, bronchitis, and cough or cyanosis with feeding. Workup should include an upper gastrointestinal series but not with barium, Bhatia said, because that can cause pneumonitis. Instead, the series should be done with a thickened water-soluble contrast material, and a bronchoscopy may be indicated as well.

Though common as a condition in adults, gastroesophageal reflux disease (GERD) is a rare, but possible, cause of chronic cough in children. More often, the reflux is the result of the cough rather than the cause. The most sensitive tool for assessing GERD is esophageal 24-hour pH/impedance reflux monitoring. However, treatment of the reflux for cough is not recommended unless the patient has clinical features of GERD, including dystonic neck posturing in infants, heartburn, regurgitation, or other symptoms. If the patient has clinical symptoms, then treatment is acid suppressive therapy for 4-8 weeks, followed by a clinical reassessment.

An uncommon cause of chronic cough with no biological mechanism is habit cough. Habit cough is most easily distinguished from other coughs by its sounds, a “large, loud, honking noise,” Bhatia said. It also lacks a clear trigger and is usually absent during sleep, but it can be continuous during the day. Frustratingly, the patients themselves often don’t seem bothered by the cough, but “it’s very disruptive in the school and everywhere else,” Bhatia said. Families and/providers will often have tried multiple treatments and seen no improvement with habit cough.

The first thing to do with habit cough is reassure the family that there’s nothing serious going on because they are often worried by this point. Several non-pharmaceutical treatments can be effective, such as suggestion therapy or the “warm water technique,” in which the patient takes a sip of warm water every time they feel the urge to cough. “If they’re able to break the cycle, most of the time, they are fine,” Bhatia said. In rarer cases, more involved behavioral interventions may be indicated, such as a psychology referral if an underlying anxiety or other behavioral disorder is contributing.
 

Newer Causes of Cough

Two more recent causes of cough to watch for are long COVID and e-cigarette or vaping product–associated lung injury (EVALI), Bhatia said. The clearest sign of EVALI is a history of e-cigarette/vaping exposure, but clinical symptoms include a dry cough that occurs with dyspnea and chest pain. A chest x-ray may show diffuse, hazy, or consolidative opacities. Sometimes antibiotics or steroids can be helpful, but the evidence isn’t strong, and the most effective treatment is stopping e-cigarette use. Less commonly, passive exposure to vaping can also be associated with EVALI.

The most recent research on long COVID suggests that about 10-20% of children with acute COVID develop long COVID, and about a quarter of these patients develop a chronic dry cough. It’s often associated with fatigue and shortness of breath, which can be assessed with cardiopulmonary exercise testing. Sometimes a short trial of inhaled steroids can help.

Bhatia also mentioned a handful of other uncommon causes of chronic cough that most American pediatricians are unlikely to see: Childhood interstitial lung disease, tuberculosis, use of Angiotensin-Converting Enzyme inhibitors, and a build-up of ear wax via the Arnold’s nerve reflex.
 

Evaluation and Workup

Bhatia also discussed what to cover while taking a history and questions to ask. The history should include the type of cough, the onset timing (sudden vs gradual), associated symptoms, the cough trajectory, medications the patient is taking, and the patient’s past medical history and environmental exposures. Those attributes are included in this more comprehensive list of questions to consider during evaluation, adapted from a list provided in a 2019 article in Paediatric Respiratory Reviews:

• Age of onset and duration?
• Was the onset sudden or associate with an illness?
• Is the cough wet or dry?
• What does the cough sound like?
• How often does the cough occur?
• Is it progressive?
• Is it present during sleep?
• Are there any other associated symptoms, such as wheeze, dyspnea, vomiting, chest pain, etc?
• Are there any exacerbating factors or known triggers?
• Are there any relieving factors, including a trial of bronchodilators?
• Has there been exposure to auto-irritants, such as secondhand smoke?
• What is the cough’s effect on the child and on others around the child?
• Does the child have any other underlying conditions such as neuromuscular disease or asthma?
• What medications is the child taking or has recently taken?
• Is there a family history of atopy and/or respiratory disease?

Bhatia also recommended paying special attention to the following red flags or key features that may help more quickly narrow the diagnosis and often require a specialist referral:

• Digital clubbing, failure to thrive, or low tone
• An abnormal cardiac exam
• Tachypnea, hypoxemia, chest retractions, or hemoptysis
• Abnormal breath sounds such as unilateral wheezing or coarse crackles
• Abnormal spirometry in those aged 5 and older showing reversible obstruction, which often indicates asthma
• An abnormal chest x-ray with, for example, bilateral infiltrates, hyperinflation, right middle lobe syndrome, situs inversus, unilateral hyperlucency, a right aortic arch, etc.

No external funding was used for the presentation. Bhatia and Quijada had no disclosures.
 

A version of this article appeared on Medscape.com.

ORLANDO — It’s important for pediatricians not only to understand the causes and effects of climate change but also to know how to discuss this issue with families and make risk-based adjustments to their clinical practice based on the individual health and circumstances of each patient. That’s one of the key messages delivered at the annual meeting of the American Academy of Pediatrics (AAP) by Elizabeth C. Matsui, MD, MHS, professor of population health and pediatrics and director of the Center for Health and Environment Education and Research at the University of Texas at Austin Dell Medical School. 

“Even though climate change has been here and has been affecting health already for a while, it’s just really impossible to ignore right now,” she told attendees in a session focused on climate change impacts on allergies and asthma. “The challenge is connecting the dots between something that is much larger, or feels much larger, than the patient and the family that’s in front of you.” 

The reality, however, is that climate change is now impacting patients’ health on an individual level, and pediatricians have a responsibility to understand how that’s happening and to help their families prepare for it. 

“From the perspective of someone who went into medicine to practice and take care of the individual patient, I think it has been more difficult to connect those dots, and for the people in this room, it’s our job to connect those dots,” Matsui said. She also acknowledged that many of the solutions are frustratingly limited to the policy level and challenging to implement, “but it doesn’t mean that we can’t make a difference for the patients who are in front of us.” 

Charles Moon, MD, a pediatrician and Pediatric Environmental Health Fellow at the Children’s Environmental Health Center, Icahn School of Medicine at Mount Sinai, New York City, found the talk particularly helpful in providing information about both the broader issue and what it means on a local practice level. 

“The biggest takeaway is that more people and more pediatricians are tuning in to this issue and realizing the dangers,” Moon said. “It’s clear that a larger community is forming around this, and I think we are at the cusp where more and more people will be coming in. We are really focusing on taking all the data and trying to figure out solutions. I think the solutions orientation is the most important part.” 
 

Understanding the Big Picture

Matsui opened with a general discussion of the human causes of climate change and the effects on a global scale presently and in the future. For example, over the past 800,000 years, carbon dioxide levels have never been above 300 ppm, but they surpassed that threshold in 1911 and have reached 420 ppm today. The trapping of heat in Earth’s atmosphere caused by the increase in carbon dioxide and other greenhouse gases is leading to multiple phenomena that impact health, such as longer growing seasons; increased droughts, heat waves, and wildfire seasons; and higher temperatures. These changes, in turn, affect allergens and asthma.

Children are particularly vulnerable to climate change impacts because they have a higher risk for developing asthma, allergic disease, and infections, Matsui said. Childhood is a critical period for lung and immune development, and the Environmental Protection Agency’s 2023 Climate Change and Children’s Health and Well-Being report projects that an increase of 2° C in global warming will result in an additional 34,500 pediatric asthma cases and 228,000 allergic rhinitis cases per year, driven largely by predicted increases in ozone and 2.5-µm particulate matter. The report also forecasts an increase in 6240 asthma emergency department visits and 332 additional respiratory hospitalizations per year. 

“We know that these associations that we see between climate change exposures and poor respiratory health outcomes in kids are biologically plausible,” Matsui said. “They’re not just correlation without causation. A lot of the mechanisms for how air pollution, allergies, and other factors directly affect the lungs of the airway epithelium have been worked out.” 
 

An Increase in Allergens and Viral Infections

Pediatricians should prepare for anticipated growth in allergens and viral infections. The longer growing seasons mean that pollen seasons will also lengthen. Meanwhile, higher concentrations of carbon dioxide cause individual plants to produce more pollen. 

“As the winters get warmer, mice that might not be able to survive during the winter are surviving, and mice reproduce at a very rapid rate,” she said. “The increase in moisture means that dust mites, which absorb their water — they drink by absorbing humidity that’s in the air — will be present in higher concentrations, and their range will expand.”

Fungal and mold exposures are also increasing, not just outdoors but also indoors, “and there are all sorts of allergic and respiratory health consequences of fungal exposure,” Matsui said. As hurricanes and flooding increase, storm damage can also make indoor environments more conducive to fungal and mold growth. 

Extreme weather from climate change also affects infrastructure. “When there’s healthcare infrastructure disruption and other infrastructure disruption, it adds to the challenge,” she said. “It compounds all the other threat to health from climate change, so this overall problem of climate change and health is multidimensional and very complicated.”

Then there’s the impact of climate change on respiratory viruses, which are a major driver of asthma exacerbations, Matsui said. The greater variability in daytime temperatures affects environmental reservoirs, transmission patterns, geographical ranges, and seasonality of various respiratory pathogens. The prevalence of respiratory syncytial virus infections, for example, increases during humid periods. 

“This is coupled with the fact that the projected increases in air pollution increase susceptibility to respiratory virus infections,” Matsui said. “In fact, climate change and air pollution are inextricably linked.” 
 

Climate Change and Air Pollution

Climate disruption creates extreme weather patterns that then lead to worsening air quality due to high temperatures; heavier precipitation; and more forest fires, droughts, dust storms, thunderstorms, hurricanes, stagnation events, and other extreme weather. Matsui shared a map showing the substantial increase in days with stagnant air since 1973. During stagnation events, air pollution builds up in the atmosphere because of a stable air mass that remains over a region for several days, with low-level winds and no precipitation. 

The pollutants can then contribute to rising temperatures. Black carbon particulate matter released from the burning of forests and other biomass absorbs more solar radiation, further contributing to temperature increases. Data from the National Bureau of Economic Research has shown that the US made big strides in reducing air pollution from 2009 through 2016, but it began to reverse in 2016 as severe weather events picked up. 

Pediatricians need to be cognizant of the synergistic effect of these different impacts as well. “We oftentimes talk about these problems in a silo, so we may talk about air pollution and health effects, or allergens and health effects, or heat and health effects, but all of these interact with each other and further compound the health effects,” compared to just one of them in isolation, Matsui said.

For example, air pollution increases sensitivity to allergen exposure and increases reaction severity, which disrupts the immune tolerance to allergens. “Heat and air pollution also interact, and the combination of the two is more deadly than either one alone,” she said. 

Air pollution from wildfire smoke is also more toxic to the lungs than air pollution from other sources, so if there’s wildfire-based air pollution, the impact on respiratory hospitalizations is significantly greater. Even in places that would not otherwise be at risk for wildfires, the threat remains of air pollution from more distant fires, as New York City experienced from Canadian wildfires last year. 

“This is a problem that is not just isolated to the parts of the world where the wildfires are located,” Matsui said.

Moon, who practices in New York City, said he really appreciated Matsui’s perspectives and nuanced advice as a subspecialist “because it’s obvious that the way we deliver healthcare is going to have to change based on climate change.” He hopes to see more subspecialists from other pediatric areas getting involved in looking at climate impacts and providing nuanced advice about changing clinical care similar to the examples Matsui provided. 

Air pollution can also be deadly, as a landmark case in the United Kingdom revealed a few years ago when the court ruled that a child’s death from an asthma attack was directly due to air pollution. In addition to causing worse asthma symptoms and exacerbations, air pollution also adds to the risk of developing asthma and impedes lung growth, all of which disproportionately affects disadvantaged and minoritized communities, she said. 
 

Greater Impact on Disadvantaged Populations

Matsui called attention to the equity implications of climate change impacts on health. 

“If you have a community that does not have the infrastructure and access to resources, and that same community has a prevalence of asthma that is double that of their more advantaged and white counterparts, then the impacts of climate change are going to be amplified even more,” she said.

For example, a 2019 study found that the biggest predictor of the location of ragweed plants has to do with vacant lots and demolition of housing. Ragweed plants being more common in neighborhoods with vacant lots will disproportionately affect disadvantaged neighborhoods, she said. Another study found in Baltimore that mouse allergens — specifically urine — were a bigger cause of asthma in low-income children than were cockroach allergens. 

“It’s important to consider context,” including age, gender and social and behavioral context, she said. “We as pediatricians know that children are particularly vulnerable, and what happens to them has an effect across the lifespan.” 

Furthermore, pediatricians are aware that disadvantaged and minoritized communities lack infrastructure; often live in areas with greater air pollution; often have heat islands in their communities without protection, such as tree canopy; and may be at greater flooding risk. “Poverty is also associated with increased vulnerability” because of poorer housing and infrastructure, less education, less access to care, more preexisting health conditions and greater discrimination, she said.
 

Three Cornerstone Interventions

Interventions fall into three main buckets, Matsui said: mitigation, adaption, and resilience. 

“Mitigation means reducing greenhouse gas and air pollution production and trying to enhance sinks for greenhouse gases,” she said. Mitigation strategies primarily occur at the policy level, with improved regulation, treaties, and market-based approaches, such as carbon tax and cap and trade. 

Adaptation includes actions that lessen the impact on health and environment, such as infrastructure changes and implementation of air conditioning. Examples of climate change adaptation strategies also mostly come from policy but largely at state and local levels, where individual pediatricians have a greater voice and influence. These can include changes in urban planning to address heat islands, flooding risk, and public transportation’s contribution to air pollution and climate change. It can also include changes in housing regulation and policy and investments in healthcare, such as expanded Medicaid and health insurance and investing in disaster planning and readiness. 

“Resilience is a more holistic concept,” Matsui said, “which advocates for system-wide, multilevel changes and involves a range of strategies to enhance social, human, natural, physical, and financial capacities.”
 

What Pediatricians Can Do

Pediatricians have an important role to play when it comes to climate change and health impacts. 

“The first step is sort of understanding the complexity of climate change in terms of its potential health effects, but also being prepared to talk with our patients and their families about it,” Matsui said. “The second step is advocacy.” She drew attention to the February policy statement in Pediatrics that discusses precisely the ways in which pediatricians can leverage their expertise and credibility. 

“Pediatricians are ideal advocates with whom to partner and uplift youth and community voices working to advance zero-carbon energy policy and climate justice,” she said. “There are many opportunities to advocate for climate solution policies at the local, state, national, and even international level.” 

These roles can include educating elected officials and health insurance entities about the risks that climate change poses to allergies, asthma, and child health more broadly, as well as the benefits of local solutions, including improved air quality, tree canopy, and green space. “There are lots of opportunities to engage with the community, including speaking at public hearings, serving as an expert testimony, and writing letters to the editor,” she said. 

The impact of these efforts can be further maximized by working with other healthcare professionals. Lori Byron, MD, a pediatrician from Red Lodge, Montana, who heads the AAP Chapter Climate Advocates program, noted during Q&A that every AAP chapter in the country has climate advocates. She added that the AAP is the first medical board to have climate modules in their maintenance of certification specifically designed to incorporate climate change education into well visits.
 

Adjusting Clinical Care

Meanwhile, in patient care, Matsui acknowledged it can be frustrating to think about what a massive impact climate has and simultaneously challenging to engage families in discussions about it. However, a wide range of resources are available that can be provided to patients. 

“For a patient in front of you, being informed and prepared to talk about it is the first step to being able to assess their climate change risk and provide tailored guidance,” she said. Tailored guidance takes into account the child’s specific health situation and the risks they’re most likely to encounter, such as wildfire smoke, air pollution, longer pollen seasons, environmental allergens, or disruption of infrastructure. 

“If I am seeing a patient with asthma who is allergic to a particular pollen, I can anticipate that pollen may be present in higher levels of the future, and that the season for that pollen may be longer,” Matsui said. “So if I’m thinking about allergen immunotherapy for that patient, future risk may be something that would push the conversation and the shared decision-making” from possible consideration to more serious consideration, depending on the child’s age. 

“Another example is a patient with asthma, thinking about wildfire risk and having them prepared, because we know from data that wildfire air pollution is going to be worse for that child than pollution from other sources, and there are ways for them to be prepared,” Matsui said. For instance, having an HVAC system with a high-grade air filter (at least a MERV 13) will filter the air better if a wildfire causes smoke to descend over an area. Portable, less expensive HEPA filters are also an option if a family cannot upgrade their system, and wearing an N95 or N95-equivalent mask can also reduce the impact of high air pollution levels. 

An example of thinking about the impact of potential infrastructure disruption could be ensuring patients have enough of all their medications if they’re close to running out. “It’s important for them to always have think about their medications and get those refills ahead of a storm,” she said.
 

Additional Resources 

Understanding that pediatricians may not have time to discuss all these issues or have broader conversations about climate change during visits, Matsui highlighted the AAP website of resources on climate change. In addition to resources for pediatricians, such as a basic fact sheet about climate change impacts on children’s health and the technical report that informed the policy statement, the site has multiple resources for families:

• Climate Change Impact: Safeguarding Your Family’s Health and Well-being (video), How to Talk With Children About Climate Change, Climate Change & Children’s Health: AAP Policy Explained, Climate Checkup for Children’s Health: Little Changes With Big Impact, How Climate Change Can Make Children Sick: What Parents Need to Know, Climate Change & Wildfires: Why Kids Are Most at Risk, Climate Change, Extreme Weather & Children: What Families Need to Know, Extreme Heat & Air Pollution: Health Effects on Babies & Pregnant People, and

The following resources can also be helpful to pediatricians and/or families:

• Ready.gov, AirNow, Patient Exposure and the Air Quality Index, Protecting Vulnerable Patient Populations from Climate Hazards: A Referral Guide for Health Professionals from the US Department of Health and Human Services, Low Income Home Energy Assistance Program (LIHEAP), Weatherization Assistance Program, and the Disaster Supplemental Nutrition Assistance Program (D-SNAP)

In some states, Medicaid will provide or cover the cost of air conditioning and/or air filters.

The presentation did not involve external funding. Drs. Matsui and Moon had no disclosures. 
 

A version of this article first appeared on Medscape.com.

ORLANDO — It’s important for pediatricians not only to understand the causes and effects of climate change but also to know how to discuss this issue with families and make risk-based adjustments to their clinical practice based on the individual health and circumstances of each patient. That’s one of the key messages delivered at the annual meeting of the American Academy of Pediatrics (AAP) by Elizabeth C. Matsui, MD, MHS, professor of population health and pediatrics and director of the Center for Health and Environment Education and Research at the University of Texas at Austin Dell Medical School. 

“Even though climate change has been here and has been affecting health already for a while, it’s just really impossible to ignore right now,” she told attendees in a session focused on climate change impacts on allergies and asthma. “The challenge is connecting the dots between something that is much larger, or feels much larger, than the patient and the family that’s in front of you.” 

The reality, however, is that climate change is now impacting patients’ health on an individual level, and pediatricians have a responsibility to understand how that’s happening and to help their families prepare for it. 

“From the perspective of someone who went into medicine to practice and take care of the individual patient, I think it has been more difficult to connect those dots, and for the people in this room, it’s our job to connect those dots,” Matsui said. She also acknowledged that many of the solutions are frustratingly limited to the policy level and challenging to implement, “but it doesn’t mean that we can’t make a difference for the patients who are in front of us.” 

Charles Moon, MD, a pediatrician and Pediatric Environmental Health Fellow at the Children’s Environmental Health Center, Icahn School of Medicine at Mount Sinai, New York City, found the talk particularly helpful in providing information about both the broader issue and what it means on a local practice level. 

“The biggest takeaway is that more people and more pediatricians are tuning in to this issue and realizing the dangers,” Moon said. “It’s clear that a larger community is forming around this, and I think we are at the cusp where more and more people will be coming in. We are really focusing on taking all the data and trying to figure out solutions. I think the solutions orientation is the most important part.” 
 

Understanding the Big Picture

Matsui opened with a general discussion of the human causes of climate change and the effects on a global scale presently and in the future. For example, over the past 800,000 years, carbon dioxide levels have never been above 300 ppm, but they surpassed that threshold in 1911 and have reached 420 ppm today. The trapping of heat in Earth’s atmosphere caused by the increase in carbon dioxide and other greenhouse gases is leading to multiple phenomena that impact health, such as longer growing seasons; increased droughts, heat waves, and wildfire seasons; and higher temperatures. These changes, in turn, affect allergens and asthma.

Children are particularly vulnerable to climate change impacts because they have a higher risk for developing asthma, allergic disease, and infections, Matsui said. Childhood is a critical period for lung and immune development, and the Environmental Protection Agency’s 2023 Climate Change and Children’s Health and Well-Being report projects that an increase of 2° C in global warming will result in an additional 34,500 pediatric asthma cases and 228,000 allergic rhinitis cases per year, driven largely by predicted increases in ozone and 2.5-µm particulate matter. The report also forecasts an increase in 6240 asthma emergency department visits and 332 additional respiratory hospitalizations per year. 

“We know that these associations that we see between climate change exposures and poor respiratory health outcomes in kids are biologically plausible,” Matsui said. “They’re not just correlation without causation. A lot of the mechanisms for how air pollution, allergies, and other factors directly affect the lungs of the airway epithelium have been worked out.” 
 

An Increase in Allergens and Viral Infections

Pediatricians should prepare for anticipated growth in allergens and viral infections. The longer growing seasons mean that pollen seasons will also lengthen. Meanwhile, higher concentrations of carbon dioxide cause individual plants to produce more pollen. 

“As the winters get warmer, mice that might not be able to survive during the winter are surviving, and mice reproduce at a very rapid rate,” she said. “The increase in moisture means that dust mites, which absorb their water — they drink by absorbing humidity that’s in the air — will be present in higher concentrations, and their range will expand.”

Fungal and mold exposures are also increasing, not just outdoors but also indoors, “and there are all sorts of allergic and respiratory health consequences of fungal exposure,” Matsui said. As hurricanes and flooding increase, storm damage can also make indoor environments more conducive to fungal and mold growth. 

Extreme weather from climate change also affects infrastructure. “When there’s healthcare infrastructure disruption and other infrastructure disruption, it adds to the challenge,” she said. “It compounds all the other threat to health from climate change, so this overall problem of climate change and health is multidimensional and very complicated.”

Then there’s the impact of climate change on respiratory viruses, which are a major driver of asthma exacerbations, Matsui said. The greater variability in daytime temperatures affects environmental reservoirs, transmission patterns, geographical ranges, and seasonality of various respiratory pathogens. The prevalence of respiratory syncytial virus infections, for example, increases during humid periods. 

“This is coupled with the fact that the projected increases in air pollution increase susceptibility to respiratory virus infections,” Matsui said. “In fact, climate change and air pollution are inextricably linked.” 
 

Climate Change and Air Pollution

Climate disruption creates extreme weather patterns that then lead to worsening air quality due to high temperatures; heavier precipitation; and more forest fires, droughts, dust storms, thunderstorms, hurricanes, stagnation events, and other extreme weather. Matsui shared a map showing the substantial increase in days with stagnant air since 1973. During stagnation events, air pollution builds up in the atmosphere because of a stable air mass that remains over a region for several days, with low-level winds and no precipitation. 

The pollutants can then contribute to rising temperatures. Black carbon particulate matter released from the burning of forests and other biomass absorbs more solar radiation, further contributing to temperature increases. Data from the National Bureau of Economic Research has shown that the US made big strides in reducing air pollution from 2009 through 2016, but it began to reverse in 2016 as severe weather events picked up. 

Pediatricians need to be cognizant of the synergistic effect of these different impacts as well. “We oftentimes talk about these problems in a silo, so we may talk about air pollution and health effects, or allergens and health effects, or heat and health effects, but all of these interact with each other and further compound the health effects,” compared to just one of them in isolation, Matsui said.

For example, air pollution increases sensitivity to allergen exposure and increases reaction severity, which disrupts the immune tolerance to allergens. “Heat and air pollution also interact, and the combination of the two is more deadly than either one alone,” she said. 

Air pollution from wildfire smoke is also more toxic to the lungs than air pollution from other sources, so if there’s wildfire-based air pollution, the impact on respiratory hospitalizations is significantly greater. Even in places that would not otherwise be at risk for wildfires, the threat remains of air pollution from more distant fires, as New York City experienced from Canadian wildfires last year. 

“This is a problem that is not just isolated to the parts of the world where the wildfires are located,” Matsui said.

Moon, who practices in New York City, said he really appreciated Matsui’s perspectives and nuanced advice as a subspecialist “because it’s obvious that the way we deliver healthcare is going to have to change based on climate change.” He hopes to see more subspecialists from other pediatric areas getting involved in looking at climate impacts and providing nuanced advice about changing clinical care similar to the examples Matsui provided. 

Air pollution can also be deadly, as a landmark case in the United Kingdom revealed a few years ago when the court ruled that a child’s death from an asthma attack was directly due to air pollution. In addition to causing worse asthma symptoms and exacerbations, air pollution also adds to the risk of developing asthma and impedes lung growth, all of which disproportionately affects disadvantaged and minoritized communities, she said. 
 

Greater Impact on Disadvantaged Populations

Matsui called attention to the equity implications of climate change impacts on health. 

“If you have a community that does not have the infrastructure and access to resources, and that same community has a prevalence of asthma that is double that of their more advantaged and white counterparts, then the impacts of climate change are going to be amplified even more,” she said.

For example, a 2019 study found that the biggest predictor of the location of ragweed plants has to do with vacant lots and demolition of housing. Ragweed plants being more common in neighborhoods with vacant lots will disproportionately affect disadvantaged neighborhoods, she said. Another study found in Baltimore that mouse allergens — specifically urine — were a bigger cause of asthma in low-income children than were cockroach allergens. 

“It’s important to consider context,” including age, gender and social and behavioral context, she said. “We as pediatricians know that children are particularly vulnerable, and what happens to them has an effect across the lifespan.” 

Furthermore, pediatricians are aware that disadvantaged and minoritized communities lack infrastructure; often live in areas with greater air pollution; often have heat islands in their communities without protection, such as tree canopy; and may be at greater flooding risk. “Poverty is also associated with increased vulnerability” because of poorer housing and infrastructure, less education, less access to care, more preexisting health conditions and greater discrimination, she said.
 

Three Cornerstone Interventions

Interventions fall into three main buckets, Matsui said: mitigation, adaption, and resilience. 

“Mitigation means reducing greenhouse gas and air pollution production and trying to enhance sinks for greenhouse gases,” she said. Mitigation strategies primarily occur at the policy level, with improved regulation, treaties, and market-based approaches, such as carbon tax and cap and trade. 

Adaptation includes actions that lessen the impact on health and environment, such as infrastructure changes and implementation of air conditioning. Examples of climate change adaptation strategies also mostly come from policy but largely at state and local levels, where individual pediatricians have a greater voice and influence. These can include changes in urban planning to address heat islands, flooding risk, and public transportation’s contribution to air pollution and climate change. It can also include changes in housing regulation and policy and investments in healthcare, such as expanded Medicaid and health insurance and investing in disaster planning and readiness. 

“Resilience is a more holistic concept,” Matsui said, “which advocates for system-wide, multilevel changes and involves a range of strategies to enhance social, human, natural, physical, and financial capacities.”
 

What Pediatricians Can Do

Pediatricians have an important role to play when it comes to climate change and health impacts. 

“The first step is sort of understanding the complexity of climate change in terms of its potential health effects, but also being prepared to talk with our patients and their families about it,” Matsui said. “The second step is advocacy.” She drew attention to the February policy statement in Pediatrics that discusses precisely the ways in which pediatricians can leverage their expertise and credibility. 

“Pediatricians are ideal advocates with whom to partner and uplift youth and community voices working to advance zero-carbon energy policy and climate justice,” she said. “There are many opportunities to advocate for climate solution policies at the local, state, national, and even international level.” 

These roles can include educating elected officials and health insurance entities about the risks that climate change poses to allergies, asthma, and child health more broadly, as well as the benefits of local solutions, including improved air quality, tree canopy, and green space. “There are lots of opportunities to engage with the community, including speaking at public hearings, serving as an expert testimony, and writing letters to the editor,” she said. 

The impact of these efforts can be further maximized by working with other healthcare professionals. Lori Byron, MD, a pediatrician from Red Lodge, Montana, who heads the AAP Chapter Climate Advocates program, noted during Q&A that every AAP chapter in the country has climate advocates. She added that the AAP is the first medical board to have climate modules in their maintenance of certification specifically designed to incorporate climate change education into well visits.
 

Adjusting Clinical Care

Meanwhile, in patient care, Matsui acknowledged it can be frustrating to think about what a massive impact climate has and simultaneously challenging to engage families in discussions about it. However, a wide range of resources are available that can be provided to patients. 

“For a patient in front of you, being informed and prepared to talk about it is the first step to being able to assess their climate change risk and provide tailored guidance,” she said. Tailored guidance takes into account the child’s specific health situation and the risks they’re most likely to encounter, such as wildfire smoke, air pollution, longer pollen seasons, environmental allergens, or disruption of infrastructure. 

“If I am seeing a patient with asthma who is allergic to a particular pollen, I can anticipate that pollen may be present in higher levels of the future, and that the season for that pollen may be longer,” Matsui said. “So if I’m thinking about allergen immunotherapy for that patient, future risk may be something that would push the conversation and the shared decision-making” from possible consideration to more serious consideration, depending on the child’s age. 

“Another example is a patient with asthma, thinking about wildfire risk and having them prepared, because we know from data that wildfire air pollution is going to be worse for that child than pollution from other sources, and there are ways for them to be prepared,” Matsui said. For instance, having an HVAC system with a high-grade air filter (at least a MERV 13) will filter the air better if a wildfire causes smoke to descend over an area. Portable, less expensive HEPA filters are also an option if a family cannot upgrade their system, and wearing an N95 or N95-equivalent mask can also reduce the impact of high air pollution levels. 

An example of thinking about the impact of potential infrastructure disruption could be ensuring patients have enough of all their medications if they’re close to running out. “It’s important for them to always have think about their medications and get those refills ahead of a storm,” she said.
 

Additional Resources 

Understanding that pediatricians may not have time to discuss all these issues or have broader conversations about climate change during visits, Matsui highlighted the AAP website of resources on climate change. In addition to resources for pediatricians, such as a basic fact sheet about climate change impacts on children’s health and the technical report that informed the policy statement, the site has multiple resources for families:

• Climate Change Impact: Safeguarding Your Family’s Health and Well-being (video), How to Talk With Children About Climate Change, Climate Change & Children’s Health: AAP Policy Explained, Climate Checkup for Children’s Health: Little Changes With Big Impact, How Climate Change Can Make Children Sick: What Parents Need to Know, Climate Change & Wildfires: Why Kids Are Most at Risk, Climate Change, Extreme Weather & Children: What Families Need to Know, Extreme Heat & Air Pollution: Health Effects on Babies & Pregnant People, and

The following resources can also be helpful to pediatricians and/or families:

• Ready.gov, AirNow, Patient Exposure and the Air Quality Index, Protecting Vulnerable Patient Populations from Climate Hazards: A Referral Guide for Health Professionals from the US Department of Health and Human Services, Low Income Home Energy Assistance Program (LIHEAP), Weatherization Assistance Program, and the Disaster Supplemental Nutrition Assistance Program (D-SNAP)

In some states, Medicaid will provide or cover the cost of air conditioning and/or air filters.

The presentation did not involve external funding. Drs. Matsui and Moon had no disclosures. 
 

A version of this article first appeared on Medscape.com.

ORLANDO — It’s important for pediatricians not only to understand the causes and effects of climate change but also to know how to discuss this issue with families and make risk-based adjustments to their clinical practice based on the individual health and circumstances of each patient. That’s one of the key messages delivered at the annual meeting of the American Academy of Pediatrics (AAP) by Elizabeth C. Matsui, MD, MHS, professor of population health and pediatrics and director of the Center for Health and Environment Education and Research at the University of Texas at Austin Dell Medical School. 

“Even though climate change has been here and has been affecting health already for a while, it’s just really impossible to ignore right now,” she told attendees in a session focused on climate change impacts on allergies and asthma. “The challenge is connecting the dots between something that is much larger, or feels much larger, than the patient and the family that’s in front of you.” 

The reality, however, is that climate change is now impacting patients’ health on an individual level, and pediatricians have a responsibility to understand how that’s happening and to help their families prepare for it. 

“From the perspective of someone who went into medicine to practice and take care of the individual patient, I think it has been more difficult to connect those dots, and for the people in this room, it’s our job to connect those dots,” Matsui said. She also acknowledged that many of the solutions are frustratingly limited to the policy level and challenging to implement, “but it doesn’t mean that we can’t make a difference for the patients who are in front of us.” 

Charles Moon, MD, a pediatrician and Pediatric Environmental Health Fellow at the Children’s Environmental Health Center, Icahn School of Medicine at Mount Sinai, New York City, found the talk particularly helpful in providing information about both the broader issue and what it means on a local practice level. 

“The biggest takeaway is that more people and more pediatricians are tuning in to this issue and realizing the dangers,” Moon said. “It’s clear that a larger community is forming around this, and I think we are at the cusp where more and more people will be coming in. We are really focusing on taking all the data and trying to figure out solutions. I think the solutions orientation is the most important part.” 
 

Understanding the Big Picture

Matsui opened with a general discussion of the human causes of climate change and the effects on a global scale presently and in the future. For example, over the past 800,000 years, carbon dioxide levels have never been above 300 ppm, but they surpassed that threshold in 1911 and have reached 420 ppm today. The trapping of heat in Earth’s atmosphere caused by the increase in carbon dioxide and other greenhouse gases is leading to multiple phenomena that impact health, such as longer growing seasons; increased droughts, heat waves, and wildfire seasons; and higher temperatures. These changes, in turn, affect allergens and asthma.

Children are particularly vulnerable to climate change impacts because they have a higher risk for developing asthma, allergic disease, and infections, Matsui said. Childhood is a critical period for lung and immune development, and the Environmental Protection Agency’s 2023 Climate Change and Children’s Health and Well-Being report projects that an increase of 2° C in global warming will result in an additional 34,500 pediatric asthma cases and 228,000 allergic rhinitis cases per year, driven largely by predicted increases in ozone and 2.5-µm particulate matter. The report also forecasts an increase in 6240 asthma emergency department visits and 332 additional respiratory hospitalizations per year. 

“We know that these associations that we see between climate change exposures and poor respiratory health outcomes in kids are biologically plausible,” Matsui said. “They’re not just correlation without causation. A lot of the mechanisms for how air pollution, allergies, and other factors directly affect the lungs of the airway epithelium have been worked out.” 
 

An Increase in Allergens and Viral Infections

Pediatricians should prepare for anticipated growth in allergens and viral infections. The longer growing seasons mean that pollen seasons will also lengthen. Meanwhile, higher concentrations of carbon dioxide cause individual plants to produce more pollen. 

“As the winters get warmer, mice that might not be able to survive during the winter are surviving, and mice reproduce at a very rapid rate,” she said. “The increase in moisture means that dust mites, which absorb their water — they drink by absorbing humidity that’s in the air — will be present in higher concentrations, and their range will expand.”

Fungal and mold exposures are also increasing, not just outdoors but also indoors, “and there are all sorts of allergic and respiratory health consequences of fungal exposure,” Matsui said. As hurricanes and flooding increase, storm damage can also make indoor environments more conducive to fungal and mold growth. 

Extreme weather from climate change also affects infrastructure. “When there’s healthcare infrastructure disruption and other infrastructure disruption, it adds to the challenge,” she said. “It compounds all the other threat to health from climate change, so this overall problem of climate change and health is multidimensional and very complicated.”

Then there’s the impact of climate change on respiratory viruses, which are a major driver of asthma exacerbations, Matsui said. The greater variability in daytime temperatures affects environmental reservoirs, transmission patterns, geographical ranges, and seasonality of various respiratory pathogens. The prevalence of respiratory syncytial virus infections, for example, increases during humid periods. 

“This is coupled with the fact that the projected increases in air pollution increase susceptibility to respiratory virus infections,” Matsui said. “In fact, climate change and air pollution are inextricably linked.” 
 

Climate Change and Air Pollution

Climate disruption creates extreme weather patterns that then lead to worsening air quality due to high temperatures; heavier precipitation; and more forest fires, droughts, dust storms, thunderstorms, hurricanes, stagnation events, and other extreme weather. Matsui shared a map showing the substantial increase in days with stagnant air since 1973. During stagnation events, air pollution builds up in the atmosphere because of a stable air mass that remains over a region for several days, with low-level winds and no precipitation. 

The pollutants can then contribute to rising temperatures. Black carbon particulate matter released from the burning of forests and other biomass absorbs more solar radiation, further contributing to temperature increases. Data from the National Bureau of Economic Research has shown that the US made big strides in reducing air pollution from 2009 through 2016, but it began to reverse in 2016 as severe weather events picked up. 

Pediatricians need to be cognizant of the synergistic effect of these different impacts as well. “We oftentimes talk about these problems in a silo, so we may talk about air pollution and health effects, or allergens and health effects, or heat and health effects, but all of these interact with each other and further compound the health effects,” compared to just one of them in isolation, Matsui said.

For example, air pollution increases sensitivity to allergen exposure and increases reaction severity, which disrupts the immune tolerance to allergens. “Heat and air pollution also interact, and the combination of the two is more deadly than either one alone,” she said. 

Air pollution from wildfire smoke is also more toxic to the lungs than air pollution from other sources, so if there’s wildfire-based air pollution, the impact on respiratory hospitalizations is significantly greater. Even in places that would not otherwise be at risk for wildfires, the threat remains of air pollution from more distant fires, as New York City experienced from Canadian wildfires last year. 

“This is a problem that is not just isolated to the parts of the world where the wildfires are located,” Matsui said.

Moon, who practices in New York City, said he really appreciated Matsui’s perspectives and nuanced advice as a subspecialist “because it’s obvious that the way we deliver healthcare is going to have to change based on climate change.” He hopes to see more subspecialists from other pediatric areas getting involved in looking at climate impacts and providing nuanced advice about changing clinical care similar to the examples Matsui provided. 

Air pollution can also be deadly, as a landmark case in the United Kingdom revealed a few years ago when the court ruled that a child’s death from an asthma attack was directly due to air pollution. In addition to causing worse asthma symptoms and exacerbations, air pollution also adds to the risk of developing asthma and impedes lung growth, all of which disproportionately affects disadvantaged and minoritized communities, she said. 
 

Greater Impact on Disadvantaged Populations

Matsui called attention to the equity implications of climate change impacts on health. 

“If you have a community that does not have the infrastructure and access to resources, and that same community has a prevalence of asthma that is double that of their more advantaged and white counterparts, then the impacts of climate change are going to be amplified even more,” she said.

For example, a 2019 study found that the biggest predictor of the location of ragweed plants has to do with vacant lots and demolition of housing. Ragweed plants being more common in neighborhoods with vacant lots will disproportionately affect disadvantaged neighborhoods, she said. Another study found in Baltimore that mouse allergens — specifically urine — were a bigger cause of asthma in low-income children than were cockroach allergens. 

“It’s important to consider context,” including age, gender and social and behavioral context, she said. “We as pediatricians know that children are particularly vulnerable, and what happens to them has an effect across the lifespan.” 

Furthermore, pediatricians are aware that disadvantaged and minoritized communities lack infrastructure; often live in areas with greater air pollution; often have heat islands in their communities without protection, such as tree canopy; and may be at greater flooding risk. “Poverty is also associated with increased vulnerability” because of poorer housing and infrastructure, less education, less access to care, more preexisting health conditions and greater discrimination, she said.
 

Three Cornerstone Interventions

Interventions fall into three main buckets, Matsui said: mitigation, adaption, and resilience. 

“Mitigation means reducing greenhouse gas and air pollution production and trying to enhance sinks for greenhouse gases,” she said. Mitigation strategies primarily occur at the policy level, with improved regulation, treaties, and market-based approaches, such as carbon tax and cap and trade. 

Adaptation includes actions that lessen the impact on health and environment, such as infrastructure changes and implementation of air conditioning. Examples of climate change adaptation strategies also mostly come from policy but largely at state and local levels, where individual pediatricians have a greater voice and influence. These can include changes in urban planning to address heat islands, flooding risk, and public transportation’s contribution to air pollution and climate change. It can also include changes in housing regulation and policy and investments in healthcare, such as expanded Medicaid and health insurance and investing in disaster planning and readiness. 

“Resilience is a more holistic concept,” Matsui said, “which advocates for system-wide, multilevel changes and involves a range of strategies to enhance social, human, natural, physical, and financial capacities.”
 

What Pediatricians Can Do

Pediatricians have an important role to play when it comes to climate change and health impacts. 

“The first step is sort of understanding the complexity of climate change in terms of its potential health effects, but also being prepared to talk with our patients and their families about it,” Matsui said. “The second step is advocacy.” She drew attention to the February policy statement in Pediatrics that discusses precisely the ways in which pediatricians can leverage their expertise and credibility. 

“Pediatricians are ideal advocates with whom to partner and uplift youth and community voices working to advance zero-carbon energy policy and climate justice,” she said. “There are many opportunities to advocate for climate solution policies at the local, state, national, and even international level.” 

These roles can include educating elected officials and health insurance entities about the risks that climate change poses to allergies, asthma, and child health more broadly, as well as the benefits of local solutions, including improved air quality, tree canopy, and green space. “There are lots of opportunities to engage with the community, including speaking at public hearings, serving as an expert testimony, and writing letters to the editor,” she said. 

The impact of these efforts can be further maximized by working with other healthcare professionals. Lori Byron, MD, a pediatrician from Red Lodge, Montana, who heads the AAP Chapter Climate Advocates program, noted during Q&A that every AAP chapter in the country has climate advocates. She added that the AAP is the first medical board to have climate modules in their maintenance of certification specifically designed to incorporate climate change education into well visits.
 

Adjusting Clinical Care

Meanwhile, in patient care, Matsui acknowledged it can be frustrating to think about what a massive impact climate has and simultaneously challenging to engage families in discussions about it. However, a wide range of resources are available that can be provided to patients. 

“For a patient in front of you, being informed and prepared to talk about it is the first step to being able to assess their climate change risk and provide tailored guidance,” she said. Tailored guidance takes into account the child’s specific health situation and the risks they’re most likely to encounter, such as wildfire smoke, air pollution, longer pollen seasons, environmental allergens, or disruption of infrastructure. 

“If I am seeing a patient with asthma who is allergic to a particular pollen, I can anticipate that pollen may be present in higher levels of the future, and that the season for that pollen may be longer,” Matsui said. “So if I’m thinking about allergen immunotherapy for that patient, future risk may be something that would push the conversation and the shared decision-making” from possible consideration to more serious consideration, depending on the child’s age. 

“Another example is a patient with asthma, thinking about wildfire risk and having them prepared, because we know from data that wildfire air pollution is going to be worse for that child than pollution from other sources, and there are ways for them to be prepared,” Matsui said. For instance, having an HVAC system with a high-grade air filter (at least a MERV 13) will filter the air better if a wildfire causes smoke to descend over an area. Portable, less expensive HEPA filters are also an option if a family cannot upgrade their system, and wearing an N95 or N95-equivalent mask can also reduce the impact of high air pollution levels. 

An example of thinking about the impact of potential infrastructure disruption could be ensuring patients have enough of all their medications if they’re close to running out. “It’s important for them to always have think about their medications and get those refills ahead of a storm,” she said.
 

Additional Resources 

Understanding that pediatricians may not have time to discuss all these issues or have broader conversations about climate change during visits, Matsui highlighted the AAP website of resources on climate change. In addition to resources for pediatricians, such as a basic fact sheet about climate change impacts on children’s health and the technical report that informed the policy statement, the site has multiple resources for families:

• Climate Change Impact: Safeguarding Your Family’s Health and Well-being (video), How to Talk With Children About Climate Change, Climate Change & Children’s Health: AAP Policy Explained, Climate Checkup for Children’s Health: Little Changes With Big Impact, How Climate Change Can Make Children Sick: What Parents Need to Know, Climate Change & Wildfires: Why Kids Are Most at Risk, Climate Change, Extreme Weather & Children: What Families Need to Know, Extreme Heat & Air Pollution: Health Effects on Babies & Pregnant People, and

The following resources can also be helpful to pediatricians and/or families:

• Ready.gov, AirNow, Patient Exposure and the Air Quality Index, Protecting Vulnerable Patient Populations from Climate Hazards: A Referral Guide for Health Professionals from the US Department of Health and Human Services, Low Income Home Energy Assistance Program (LIHEAP), Weatherization Assistance Program, and the Disaster Supplemental Nutrition Assistance Program (D-SNAP)

In some states, Medicaid will provide or cover the cost of air conditioning and/or air filters.

The presentation did not involve external funding. Drs. Matsui and Moon had no disclosures. 
 

A version of this article first appeared on Medscape.com.

ORLANDO — More than half of youth improved after receiving a dose of naloxone by emergency medical services (EMS) after an emergency dispatch call, according to research presented at the American Academy of Pediatrics 2024 National Conference.

“Emergency responders or EMS are often the first to arrive to an opioid poisoning, and they’re often the first to give naloxone, a potentially lifesaving medication,” said Christopher E. Gaw, MD, MPH, MBE, assistant professor of pediatrics at The Ohio State University College of Medicine and an emergency medicine physician at Nationwide Children’s Hospital in Columbus, Ohio.

Ohio State University

“Our study highlights and underscores its safety of use in the prehospital setting, and this is also supported by other data,” Gaw said. “Efforts to support public distribution and education on naloxone can really help each and every one of us as individual citizens prevent pediatric harm from the opioid crisis.”

Additional research at the meeting showed that teens’ knowledge, attitudes, and confidence about recognizing overdoses and assisting with naloxone administration improved following a peer-to-peer training program, suggesting that teens can play an important role in reducing youth mortality from overdoses.

An average of 22 American teens died from overdose every week in 2022, and as counterfeit pill use has increased among youth, research has found that fentanyl was detected in 93% of overdose deaths with counterfeit pills, according to Talia Puzantian, PharmD, BCPP, of the Keck Graduate Institute School of Pharmacy, Claremont, California, who led the study on peer education. Yet a recent survey had found that less than a third of teens (30%) knew what naloxone was, and only 14% knew how to administer it.

“Ensuring that adolescents have easy and confidential access to naloxone is important and can save lives,” said Taylor Nichols, MD, assistant clinical professor at the University of California San Francisco and an emergency medicine and addiction medicine–certified physician. “I have had teen patients who have told me that they have had to use naloxone obtained from our clinic on friends when they have accidentally overdosed.”

University of California

EMS Naloxone Administration to Youth

EMS clinicians are often the first healthcare providers to respond to an opioid overdose or poisoning event, and evidence-based guidelines for EMS naloxone administration were developed in 2019 to support this intervention. Gaw’s team investigated the frequency and demographics of pediatric administration of naloxone.

They analyzed data from the National Emergency Medical Services Information System on EMS activations for administration of at least one dose of naloxone during 2022 to those aged 0-17. There were 6215 EMS pediatric administrations of naloxone that year, and in the vast majority of cases (82%), the patient had not received a naloxone injection prior to EMS’s arrival.

Most patients (79%) were aged 13-17 years, but 10% were in the 6-12 age group. The remaining patients included 6% infants younger than 1 year and 4% aged 6-12 years. Just over half were for males (55%), and most were dispatched to a home or residential setting (61%). One in five incidents (22%) occurred at a non-healthcare business, 9% on a street or highway, and the rest at a healthcare facility or another location.

Most of the incidents occurred in urban areas (86%), followed by rural (7%), suburban (6%), and wilderness (1.4%). More occurred in the US South (42%) than in the West (29%), Midwest (22%), or Northeast (7.5%).

A key takeaway of those demographic findings is that ingestions and accidental poisonings with opioids can occur in children of any age, Nichols said. “Every single home that has any opioids in the home should absolutely have naloxone immediately available as well,” he said. “Every single person who is prescribed opioids should also have naloxone available and accessible and to be sure that the naloxone is not expired or otherwise tampered with and update that every few years.” He noted that Narcan expiration was recently extended from 3 years to 4 years by the US Food and Drug Administration (FDA).

“I always advise that people who have opioid medications keep them stored safely and securely,” Nichols said. “However, I also acknowledge that even perfect systems fail and that people make mistakes and may accidentally leave medication out, within reach, or otherwise unsecured. If that happens, and someone were to intentionally or unintentionally get into that medication and potentially overdose as a result, we want to have that reversal medication immediately available to reverse the overdose.”

In nearly all cases (91%), EMS provided advanced life support, with only 7.5% patients receiving basic life support and 1.5% receiving specialty critical care. Just under a third (29%) of the dispatch calls were for “overdose/poisoning/ingestion.” Other dispatch calls included “unconscious/fainting/near-fainting” (21%) or “cardiac arrest/death” (17%), but the frequency of each dispatch label varied by age groups.

For example, 38% of calls for infants were for cardiac arrest, compared with 15% of calls for older teens and 18% of calls for 6-12 year olds. An overdose/poisoning dispatch was meanwhile more common for teens (32%) than for infants (13%), younger children (23%), and older children/tweens (18%). Other dispatch complaints included “sick person/person down/unknown problem” (12%) and “breathing problem” (5%).

A possible reason for these variations is that “an overdose might be mistaken for another medical emergency, or vice versa, because opioid poisonings can be challenging to recognize, especially in young children and in the pediatric population,” Gaw said. “Both the public and emergency responders should maintain a high level of suspicion” of possible overdose for children with the signs or symptoms of it, such as low breathing, unresponsiveness, or small pupils.

In most cases (87%), the patient was not in cardiac arrest, though the patient had entered cardiac arrest before EMS’s arrival in 11.5% of cases. Two thirds of cases only involved one dose of naloxone, while the other 33% involved two doses.

Ryan Marino, MD, an addiction medicine specialist and an associate professor of emergency medicine at Case Western Reserve University School of Medicine in Cleveland, Ohio, who was not involved in the study, took note of the high proportion of cases in which two doses were administered.

“While there is, in my professional opinion, almost no downside to giving naloxone in situations like this, and everybody should have it available and know how to use it, I would caution people on the risk of anchor bias, especially when more than two doses of naloxone are given, since we know that one should be an effective amount for any known opioid overdose,” Marino said. Anchoring bias refers to the tendency for individuals to rely more heavily on the first piece of information they receive about a topic or situation.

“For first responders and healthcare professionals, the importance of additional resuscitation measures like oxygenation and ventilation are just as crucial,” Marino said. “People should not be discouraged if someone doesn’t immediately respond to naloxone as overdose physiology can cause mental status to stay impaired for other reasons beyond direct drug effect, such as hypercarbia, but continue to seek and/or provide additional emergency care in these situations.”

Patients improved after one dose in just over half the cases (54%), and their conditions were unchanged in 46% of cases. There were only 11 cases in which the patient’s condition worsened after a naloxone dose (0.2%). Most of the cases (88%) were transported by EMS, and there were 13 total deaths at the scene (0.2%).

Nichols found the low incidence of worsening clinical status particularly striking. “This is further evidence of a critically important point — naloxone is purely an opioid antagonist, and only binds to opioid receptors, such that if a person has not overdosed on opioids or does not otherwise have opioids in their system, naloxone will not have a significant effect and will not cause them harm,” Nichols said.

“The most common causes of harm are due to rapid reversal of overdose and the potential risks involved in the rapid reversal of opioid effects and potentially precipitating withdrawal, and as this paper demonstrates, these are exceedingly rare,” he said. “Given that, we should have an incredibly low barrier to administer naloxone appropriately.”

The study was limited by inability to know how many true pediatric opioid poisonings are managed by EMS, so future research could look at linking EMS and emergency room hospital databases.
 

Improved Self-Efficacy in Teens

Another study showed that a peer-to-peer training program increased teens’ knowledge about overdoses from 34% before training to 79% after (P < .0001), and it substantially improved their confidence in recognizing an overdose and administering naloxone.

Nichols said the study shows the importance of ensuring “that adolescents know how to keep themselves and their friends safe in the case that they or anyone they know does end up using illicit substances which either intentionally or unintentionally contain opioids.”

This study assessed a training program with 206 students in a Los Angeles County high school who were trained by their peers between November 2023 and March 2024. The training included trends in teen overdose deaths, defining what opioids and fentanyl are, recognizing an overdose, and responding to one with naloxone.

The teens were an average 16 years old, about evenly split between boys and girls, and mostly in 11th (40%) or 12th (28%) grade, though nearly a third (29%) were 9th graders.

The students’ knowledge about fentanyl’s presence in counterfeit pills increased from 21% before the training to 68% afterward, and their correct identification of an overdose increased from 47% of participants to 90%.

The students’ confidence and attitudes toward helping with an overdose also improved substantially after the training. About two thirds agreed that non-medical people should be able to carry naloxone before the training, and that rose to 88% agreeing after the training. The proportion who agreed they would be willing to assist in an overdose rose from 77% before to 89% after training.

More dramatically, the teens’ confidence after training more than doubled in recognizing an overdose (from 31% to 81%) and more than tripled in their ability to give naloxone during an overdose (from 26% to 83%).

“The critical piece to keep in mind is that the concern about opioid overdose is respiratory depression leading to a lack of oxygen getting to the brain,” Nichols explained. “In the event of an overdose, time is brain — the longer the brain is deprived of oxygen, the lower the chance of survival. There is no specific time at which naloxone would become less effective at reversing an overdose.”

Therefore, people do not need to know the exact time that someone may have overdosed or how long they have been passed out in order to administer naloxone, he said. “The sooner naloxone is administered to someone who is unresponsive and who may have overdosed on opioids, the higher the likelihood of a successful reversal of an overdose and of saving a life.”

The peer-to-peer program was sponsored by the CARLOW Center for Medical Innovation, and the EMS study used no external funding. The authors of both studies and Marino had no disclosures. Nichols has consulted or clinically advised TV shows and health tech startup companies and has no disclosures related to naloxone or the pharmaceutical industry.
 

A version of this article first appeared on Medscape.com.

ORLANDO — More than half of youth improved after receiving a dose of naloxone by emergency medical services (EMS) after an emergency dispatch call, according to research presented at the American Academy of Pediatrics 2024 National Conference.

“Emergency responders or EMS are often the first to arrive to an opioid poisoning, and they’re often the first to give naloxone, a potentially lifesaving medication,” said Christopher E. Gaw, MD, MPH, MBE, assistant professor of pediatrics at The Ohio State University College of Medicine and an emergency medicine physician at Nationwide Children’s Hospital in Columbus, Ohio.

Ohio State University

“Our study highlights and underscores its safety of use in the prehospital setting, and this is also supported by other data,” Gaw said. “Efforts to support public distribution and education on naloxone can really help each and every one of us as individual citizens prevent pediatric harm from the opioid crisis.”

Additional research at the meeting showed that teens’ knowledge, attitudes, and confidence about recognizing overdoses and assisting with naloxone administration improved following a peer-to-peer training program, suggesting that teens can play an important role in reducing youth mortality from overdoses.

An average of 22 American teens died from overdose every week in 2022, and as counterfeit pill use has increased among youth, research has found that fentanyl was detected in 93% of overdose deaths with counterfeit pills, according to Talia Puzantian, PharmD, BCPP, of the Keck Graduate Institute School of Pharmacy, Claremont, California, who led the study on peer education. Yet a recent survey had found that less than a third of teens (30%) knew what naloxone was, and only 14% knew how to administer it.

“Ensuring that adolescents have easy and confidential access to naloxone is important and can save lives,” said Taylor Nichols, MD, assistant clinical professor at the University of California San Francisco and an emergency medicine and addiction medicine–certified physician. “I have had teen patients who have told me that they have had to use naloxone obtained from our clinic on friends when they have accidentally overdosed.”

University of California

EMS Naloxone Administration to Youth

EMS clinicians are often the first healthcare providers to respond to an opioid overdose or poisoning event, and evidence-based guidelines for EMS naloxone administration were developed in 2019 to support this intervention. Gaw’s team investigated the frequency and demographics of pediatric administration of naloxone.

They analyzed data from the National Emergency Medical Services Information System on EMS activations for administration of at least one dose of naloxone during 2022 to those aged 0-17. There were 6215 EMS pediatric administrations of naloxone that year, and in the vast majority of cases (82%), the patient had not received a naloxone injection prior to EMS’s arrival.

Most patients (79%) were aged 13-17 years, but 10% were in the 6-12 age group. The remaining patients included 6% infants younger than 1 year and 4% aged 6-12 years. Just over half were for males (55%), and most were dispatched to a home or residential setting (61%). One in five incidents (22%) occurred at a non-healthcare business, 9% on a street or highway, and the rest at a healthcare facility or another location.

Most of the incidents occurred in urban areas (86%), followed by rural (7%), suburban (6%), and wilderness (1.4%). More occurred in the US South (42%) than in the West (29%), Midwest (22%), or Northeast (7.5%).

A key takeaway of those demographic findings is that ingestions and accidental poisonings with opioids can occur in children of any age, Nichols said. “Every single home that has any opioids in the home should absolutely have naloxone immediately available as well,” he said. “Every single person who is prescribed opioids should also have naloxone available and accessible and to be sure that the naloxone is not expired or otherwise tampered with and update that every few years.” He noted that Narcan expiration was recently extended from 3 years to 4 years by the US Food and Drug Administration (FDA).

“I always advise that people who have opioid medications keep them stored safely and securely,” Nichols said. “However, I also acknowledge that even perfect systems fail and that people make mistakes and may accidentally leave medication out, within reach, or otherwise unsecured. If that happens, and someone were to intentionally or unintentionally get into that medication and potentially overdose as a result, we want to have that reversal medication immediately available to reverse the overdose.”

In nearly all cases (91%), EMS provided advanced life support, with only 7.5% patients receiving basic life support and 1.5% receiving specialty critical care. Just under a third (29%) of the dispatch calls were for “overdose/poisoning/ingestion.” Other dispatch calls included “unconscious/fainting/near-fainting” (21%) or “cardiac arrest/death” (17%), but the frequency of each dispatch label varied by age groups.

For example, 38% of calls for infants were for cardiac arrest, compared with 15% of calls for older teens and 18% of calls for 6-12 year olds. An overdose/poisoning dispatch was meanwhile more common for teens (32%) than for infants (13%), younger children (23%), and older children/tweens (18%). Other dispatch complaints included “sick person/person down/unknown problem” (12%) and “breathing problem” (5%).

A possible reason for these variations is that “an overdose might be mistaken for another medical emergency, or vice versa, because opioid poisonings can be challenging to recognize, especially in young children and in the pediatric population,” Gaw said. “Both the public and emergency responders should maintain a high level of suspicion” of possible overdose for children with the signs or symptoms of it, such as low breathing, unresponsiveness, or small pupils.

In most cases (87%), the patient was not in cardiac arrest, though the patient had entered cardiac arrest before EMS’s arrival in 11.5% of cases. Two thirds of cases only involved one dose of naloxone, while the other 33% involved two doses.

Ryan Marino, MD, an addiction medicine specialist and an associate professor of emergency medicine at Case Western Reserve University School of Medicine in Cleveland, Ohio, who was not involved in the study, took note of the high proportion of cases in which two doses were administered.

“While there is, in my professional opinion, almost no downside to giving naloxone in situations like this, and everybody should have it available and know how to use it, I would caution people on the risk of anchor bias, especially when more than two doses of naloxone are given, since we know that one should be an effective amount for any known opioid overdose,” Marino said. Anchoring bias refers to the tendency for individuals to rely more heavily on the first piece of information they receive about a topic or situation.

“For first responders and healthcare professionals, the importance of additional resuscitation measures like oxygenation and ventilation are just as crucial,” Marino said. “People should not be discouraged if someone doesn’t immediately respond to naloxone as overdose physiology can cause mental status to stay impaired for other reasons beyond direct drug effect, such as hypercarbia, but continue to seek and/or provide additional emergency care in these situations.”

Patients improved after one dose in just over half the cases (54%), and their conditions were unchanged in 46% of cases. There were only 11 cases in which the patient’s condition worsened after a naloxone dose (0.2%). Most of the cases (88%) were transported by EMS, and there were 13 total deaths at the scene (0.2%).

Nichols found the low incidence of worsening clinical status particularly striking. “This is further evidence of a critically important point — naloxone is purely an opioid antagonist, and only binds to opioid receptors, such that if a person has not overdosed on opioids or does not otherwise have opioids in their system, naloxone will not have a significant effect and will not cause them harm,” Nichols said.

“The most common causes of harm are due to rapid reversal of overdose and the potential risks involved in the rapid reversal of opioid effects and potentially precipitating withdrawal, and as this paper demonstrates, these are exceedingly rare,” he said. “Given that, we should have an incredibly low barrier to administer naloxone appropriately.”

The study was limited by inability to know how many true pediatric opioid poisonings are managed by EMS, so future research could look at linking EMS and emergency room hospital databases.
 

Improved Self-Efficacy in Teens

Another study showed that a peer-to-peer training program increased teens’ knowledge about overdoses from 34% before training to 79% after (P < .0001), and it substantially improved their confidence in recognizing an overdose and administering naloxone.

Nichols said the study shows the importance of ensuring “that adolescents know how to keep themselves and their friends safe in the case that they or anyone they know does end up using illicit substances which either intentionally or unintentionally contain opioids.”

This study assessed a training program with 206 students in a Los Angeles County high school who were trained by their peers between November 2023 and March 2024. The training included trends in teen overdose deaths, defining what opioids and fentanyl are, recognizing an overdose, and responding to one with naloxone.

The teens were an average 16 years old, about evenly split between boys and girls, and mostly in 11th (40%) or 12th (28%) grade, though nearly a third (29%) were 9th graders.

The students’ knowledge about fentanyl’s presence in counterfeit pills increased from 21% before the training to 68% afterward, and their correct identification of an overdose increased from 47% of participants to 90%.

The students’ confidence and attitudes toward helping with an overdose also improved substantially after the training. About two thirds agreed that non-medical people should be able to carry naloxone before the training, and that rose to 88% agreeing after the training. The proportion who agreed they would be willing to assist in an overdose rose from 77% before to 89% after training.

More dramatically, the teens’ confidence after training more than doubled in recognizing an overdose (from 31% to 81%) and more than tripled in their ability to give naloxone during an overdose (from 26% to 83%).

“The critical piece to keep in mind is that the concern about opioid overdose is respiratory depression leading to a lack of oxygen getting to the brain,” Nichols explained. “In the event of an overdose, time is brain — the longer the brain is deprived of oxygen, the lower the chance of survival. There is no specific time at which naloxone would become less effective at reversing an overdose.”

Therefore, people do not need to know the exact time that someone may have overdosed or how long they have been passed out in order to administer naloxone, he said. “The sooner naloxone is administered to someone who is unresponsive and who may have overdosed on opioids, the higher the likelihood of a successful reversal of an overdose and of saving a life.”

The peer-to-peer program was sponsored by the CARLOW Center for Medical Innovation, and the EMS study used no external funding. The authors of both studies and Marino had no disclosures. Nichols has consulted or clinically advised TV shows and health tech startup companies and has no disclosures related to naloxone or the pharmaceutical industry.
 

A version of this article first appeared on Medscape.com.

ORLANDO — More than half of youth improved after receiving a dose of naloxone by emergency medical services (EMS) after an emergency dispatch call, according to research presented at the American Academy of Pediatrics 2024 National Conference.

“Emergency responders or EMS are often the first to arrive to an opioid poisoning, and they’re often the first to give naloxone, a potentially lifesaving medication,” said Christopher E. Gaw, MD, MPH, MBE, assistant professor of pediatrics at The Ohio State University College of Medicine and an emergency medicine physician at Nationwide Children’s Hospital in Columbus, Ohio.

Ohio State University

“Our study highlights and underscores its safety of use in the prehospital setting, and this is also supported by other data,” Gaw said. “Efforts to support public distribution and education on naloxone can really help each and every one of us as individual citizens prevent pediatric harm from the opioid crisis.”

Additional research at the meeting showed that teens’ knowledge, attitudes, and confidence about recognizing overdoses and assisting with naloxone administration improved following a peer-to-peer training program, suggesting that teens can play an important role in reducing youth mortality from overdoses.

An average of 22 American teens died from overdose every week in 2022, and as counterfeit pill use has increased among youth, research has found that fentanyl was detected in 93% of overdose deaths with counterfeit pills, according to Talia Puzantian, PharmD, BCPP, of the Keck Graduate Institute School of Pharmacy, Claremont, California, who led the study on peer education. Yet a recent survey had found that less than a third of teens (30%) knew what naloxone was, and only 14% knew how to administer it.

“Ensuring that adolescents have easy and confidential access to naloxone is important and can save lives,” said Taylor Nichols, MD, assistant clinical professor at the University of California San Francisco and an emergency medicine and addiction medicine–certified physician. “I have had teen patients who have told me that they have had to use naloxone obtained from our clinic on friends when they have accidentally overdosed.”

University of California

EMS Naloxone Administration to Youth

EMS clinicians are often the first healthcare providers to respond to an opioid overdose or poisoning event, and evidence-based guidelines for EMS naloxone administration were developed in 2019 to support this intervention. Gaw’s team investigated the frequency and demographics of pediatric administration of naloxone.

They analyzed data from the National Emergency Medical Services Information System on EMS activations for administration of at least one dose of naloxone during 2022 to those aged 0-17. There were 6215 EMS pediatric administrations of naloxone that year, and in the vast majority of cases (82%), the patient had not received a naloxone injection prior to EMS’s arrival.

Most patients (79%) were aged 13-17 years, but 10% were in the 6-12 age group. The remaining patients included 6% infants younger than 1 year and 4% aged 6-12 years. Just over half were for males (55%), and most were dispatched to a home or residential setting (61%). One in five incidents (22%) occurred at a non-healthcare business, 9% on a street or highway, and the rest at a healthcare facility or another location.

Most of the incidents occurred in urban areas (86%), followed by rural (7%), suburban (6%), and wilderness (1.4%). More occurred in the US South (42%) than in the West (29%), Midwest (22%), or Northeast (7.5%).

A key takeaway of those demographic findings is that ingestions and accidental poisonings with opioids can occur in children of any age, Nichols said. “Every single home that has any opioids in the home should absolutely have naloxone immediately available as well,” he said. “Every single person who is prescribed opioids should also have naloxone available and accessible and to be sure that the naloxone is not expired or otherwise tampered with and update that every few years.” He noted that Narcan expiration was recently extended from 3 years to 4 years by the US Food and Drug Administration (FDA).

“I always advise that people who have opioid medications keep them stored safely and securely,” Nichols said. “However, I also acknowledge that even perfect systems fail and that people make mistakes and may accidentally leave medication out, within reach, or otherwise unsecured. If that happens, and someone were to intentionally or unintentionally get into that medication and potentially overdose as a result, we want to have that reversal medication immediately available to reverse the overdose.”

In nearly all cases (91%), EMS provided advanced life support, with only 7.5% patients receiving basic life support and 1.5% receiving specialty critical care. Just under a third (29%) of the dispatch calls were for “overdose/poisoning/ingestion.” Other dispatch calls included “unconscious/fainting/near-fainting” (21%) or “cardiac arrest/death” (17%), but the frequency of each dispatch label varied by age groups.

For example, 38% of calls for infants were for cardiac arrest, compared with 15% of calls for older teens and 18% of calls for 6-12 year olds. An overdose/poisoning dispatch was meanwhile more common for teens (32%) than for infants (13%), younger children (23%), and older children/tweens (18%). Other dispatch complaints included “sick person/person down/unknown problem” (12%) and “breathing problem” (5%).

A possible reason for these variations is that “an overdose might be mistaken for another medical emergency, or vice versa, because opioid poisonings can be challenging to recognize, especially in young children and in the pediatric population,” Gaw said. “Both the public and emergency responders should maintain a high level of suspicion” of possible overdose for children with the signs or symptoms of it, such as low breathing, unresponsiveness, or small pupils.

In most cases (87%), the patient was not in cardiac arrest, though the patient had entered cardiac arrest before EMS’s arrival in 11.5% of cases. Two thirds of cases only involved one dose of naloxone, while the other 33% involved two doses.

Ryan Marino, MD, an addiction medicine specialist and an associate professor of emergency medicine at Case Western Reserve University School of Medicine in Cleveland, Ohio, who was not involved in the study, took note of the high proportion of cases in which two doses were administered.

“While there is, in my professional opinion, almost no downside to giving naloxone in situations like this, and everybody should have it available and know how to use it, I would caution people on the risk of anchor bias, especially when more than two doses of naloxone are given, since we know that one should be an effective amount for any known opioid overdose,” Marino said. Anchoring bias refers to the tendency for individuals to rely more heavily on the first piece of information they receive about a topic or situation.

“For first responders and healthcare professionals, the importance of additional resuscitation measures like oxygenation and ventilation are just as crucial,” Marino said. “People should not be discouraged if someone doesn’t immediately respond to naloxone as overdose physiology can cause mental status to stay impaired for other reasons beyond direct drug effect, such as hypercarbia, but continue to seek and/or provide additional emergency care in these situations.”

Patients improved after one dose in just over half the cases (54%), and their conditions were unchanged in 46% of cases. There were only 11 cases in which the patient’s condition worsened after a naloxone dose (0.2%). Most of the cases (88%) were transported by EMS, and there were 13 total deaths at the scene (0.2%).

Nichols found the low incidence of worsening clinical status particularly striking. “This is further evidence of a critically important point — naloxone is purely an opioid antagonist, and only binds to opioid receptors, such that if a person has not overdosed on opioids or does not otherwise have opioids in their system, naloxone will not have a significant effect and will not cause them harm,” Nichols said.

“The most common causes of harm are due to rapid reversal of overdose and the potential risks involved in the rapid reversal of opioid effects and potentially precipitating withdrawal, and as this paper demonstrates, these are exceedingly rare,” he said. “Given that, we should have an incredibly low barrier to administer naloxone appropriately.”

The study was limited by inability to know how many true pediatric opioid poisonings are managed by EMS, so future research could look at linking EMS and emergency room hospital databases.
 

Improved Self-Efficacy in Teens

Another study showed that a peer-to-peer training program increased teens’ knowledge about overdoses from 34% before training to 79% after (P < .0001), and it substantially improved their confidence in recognizing an overdose and administering naloxone.

Nichols said the study shows the importance of ensuring “that adolescents know how to keep themselves and their friends safe in the case that they or anyone they know does end up using illicit substances which either intentionally or unintentionally contain opioids.”

This study assessed a training program with 206 students in a Los Angeles County high school who were trained by their peers between November 2023 and March 2024. The training included trends in teen overdose deaths, defining what opioids and fentanyl are, recognizing an overdose, and responding to one with naloxone.

The teens were an average 16 years old, about evenly split between boys and girls, and mostly in 11th (40%) or 12th (28%) grade, though nearly a third (29%) were 9th graders.

The students’ knowledge about fentanyl’s presence in counterfeit pills increased from 21% before the training to 68% afterward, and their correct identification of an overdose increased from 47% of participants to 90%.

The students’ confidence and attitudes toward helping with an overdose also improved substantially after the training. About two thirds agreed that non-medical people should be able to carry naloxone before the training, and that rose to 88% agreeing after the training. The proportion who agreed they would be willing to assist in an overdose rose from 77% before to 89% after training.

More dramatically, the teens’ confidence after training more than doubled in recognizing an overdose (from 31% to 81%) and more than tripled in their ability to give naloxone during an overdose (from 26% to 83%).

“The critical piece to keep in mind is that the concern about opioid overdose is respiratory depression leading to a lack of oxygen getting to the brain,” Nichols explained. “In the event of an overdose, time is brain — the longer the brain is deprived of oxygen, the lower the chance of survival. There is no specific time at which naloxone would become less effective at reversing an overdose.”

Therefore, people do not need to know the exact time that someone may have overdosed or how long they have been passed out in order to administer naloxone, he said. “The sooner naloxone is administered to someone who is unresponsive and who may have overdosed on opioids, the higher the likelihood of a successful reversal of an overdose and of saving a life.”

The peer-to-peer program was sponsored by the CARLOW Center for Medical Innovation, and the EMS study used no external funding. The authors of both studies and Marino had no disclosures. Nichols has consulted or clinically advised TV shows and health tech startup companies and has no disclosures related to naloxone or the pharmaceutical industry.
 

A version of this article first appeared on Medscape.com.

ORLANDO, FLORIDA — Pediatricians and other healthcare providers have a valuable role to play in screening parents for firearm ownership and offering counseling on safe storage practices, according to researchers who presented their findings at the American Academy of Pediatrics (AAP) 2024 National Conference.

An estimated 4.6 million US homes with children have firearms that are loaded and unlocked, a risk factor for youth suicide, yet only about half of parents of suicidal children had been screened for gun ownership in the hospital even as most would be receptive to both firearm screening and counseling, found one study in Texas.

In another study in Colorado, nearly all firearm owners believed that securely storing guns reduces the risk for firearm injury or death, but owners were less likely than non-owners to believe suicide is preventable or that removing a gun from the home reduces the risk for injury or death.

“Previous studies have shown that when pediatricians discuss the importance of armed safe storage guidance with families, families are actually more likely to go home and store firearms safely — storing them locked, unloaded, and separate from the ammunition,” said study author Taylor Rosenbaum, MD, a former pediatric fellow at Baylor College of Medicine/Texas Children’s Hospital in Houston and now an assistant professor at Children’s Hospital University of Miami. “However, previous studies have also shown that pediatricians really are not discussing firearm safe storage with our patients and their families, and we see this both in the outpatient setting, but especially in the inpatient setting for youth suicides, which have risen since 2020 and now are the second leading cause of death for those who are 10-24 years old in the United States.”

University of Miami

Firearm Safety Is a Necessary Conversation

The leading cause of death among children and teens aged 1-19 years is actually firearms, which are also the most fatal method for suicide. While only 4% of all suicide attempts in youth are fatal, 90% of those attempted with a firearm are fatal, Dr. Rosenbaum said. In addition, she said, 80% of the guns used in attempted suicide by children and teens belonged to a family member, and an estimated 70% of firearm-related suicides in youth can be prevented with safe storage of guns.

“This really gives us, as pediatricians, something actionable to do during these hospitalizations” for suicidal ideation or attempts, Dr. Rosenbaum said. “We know that when pediatricians discuss the importance of firearm safe storage guidance with families, they’re more likely to store their firearm safely,” Dr. Rosenbaum said. “We also know that families are not being screened for firearm ownership, that caregivers of youth who are in the hospital for suicidal thoughts or actions want their healthcare team to be screening for firearms, to be giving them information on how to safely secure their firearms, and to be providing free firearm blocks.”

Nathan Boonstra, MD, a general pediatrician at Blank Children’s Hospital, Des Moines, Iowa, said these findings are encouraging in terms of the opportunity pediatricians have.

“There is so much politicization around even basic firearm safety that pediatricians might shy away from the topic, but this research is reassuring that parents are receptive to our advice on safe gun storage,” said Dr. Boonstra, who was not involved in any of this presented research. “It’s especially important for pediatricians to address home firearms when their patient has a history of suicidal ideation or an attempt.”
 

Reducing the Risk

The Colorado findings similarly reinforce the opportunity physicians have to help caregivers reduce suicide risk, according to Maya Haasz, MD, an associate professor of pediatrics and emergency medicine at the University of Colorado Anschutz Medical Campus, Aurora, Colorado.

“Only 60% of firearm owners believed that removing firearms from the home in times of mental health crisis can decrease the risk of suicide,” she said. “These findings are really concerning, but what we found on the flip side was that 93% of firearm owners actually believe that secure storage can overall decrease the risk for firearm injury and death. So overall, we are underestimating the risk for suicide in our community, and we’re also underestimating our ability to prevent it.”

University of Colorado

Survey Results

Dr. Rosenbaum’s team conducted the survey in Houston with caregivers whose children were 8-21 years old and hospitalized for suicidal ideation or attempts at a large children’s hospital and two nearby community hospitals between June 2023 and May 2024. The respondents were 46% White and 23% Black, and 47% of the population were Hispanic, all but three of whom were not gun owners.

Among 244 potential participants, only 150 were eligible and approached, and 100 of these completed the surveys, including 26% firearm owners and 68% non-owners. Most of the youth (74%) were aged 14-17 years, and about three in four respondents were their mothers. Only half of the respondents (51%) said the healthcare provider had asked them whether they owned a gun.

One of the key findings Dr. Rosenbaum highlighted was the receptiveness of firearm-owning caregivers to advice from healthcare providers about ownership. If the healthcare team advised parents not to have any guns in the home for the safety of their child with self-arm, 58% of the firearm owners would follow the advice and 27% would consider it, with none saying they would be offended by it.

Among the firearm owners, 81% said their guns were safely secured where they did not believe their child could access it, which meant one in five youth had unsecured access to firearms. Most of the gun owners (77%), like the non-owners (70%), were “not at all worried” about their child getting ahold of a gun in the home, though 11.5% of the firearm owners were “very worried” about it. Interestingly, more gun owners (19%) were very worried about their children accessing a gun outside their home, a concern shared by 37% of non-owners. Nearly twice as many gun owners (46%) as non-owners (25%) were not at all worried about their child getting a gun outside the home.

The vast majority of respondents — 88% of gun owners and 91% of non-owners — felt it was “very important for the healthcare team to ask parents of children with suicidal ideation/attempts about firearms in the home.” Similarly, high proportions believed it was important for the healthcare team to counsel those parents on safe gun storage. Although only 69% of firearm owners believed it was important to distribute firearm locks in the hospital, 81% would be interested in receiving a free one. Significantly more of the non-owners (80%; P = .02) believed free lock distribution was important, and 72% of non-owners would also be interested in one.

About half the respondents (55%) preferred to hear firearm counseling one-on-one from a provider, whereas 31% would like written information and 27% would be interested in a video. In terms of what information parents preferred to receive, a little over half of owners (54%) and non-owners (56%) were interested in how or when (50% and 40%, respectively) to discuss the topic with their child. Only about a third (35% owners and 37% non-owners) wanted information on how to discuss the topic with the parents of their child’s friends.

The survey’s biggest limitations after its small size were the selection bias of those willing to complete the survey and potential response bias from the self-reported data.

The study of Colorado caregivers, just published in Pediatrics, surveyed 512 Colorado caregivers in April-May 2023 to learn about their beliefs and perceptions regarding firearms, firearm storage and risk, and youth suicide (2024 Oct 1;154[4]:e2024066930. doi: 10.1542/peds.2024-066930). Just over half the respondents (52%) had grown up in a household with firearms, and 44% currently lived in a household with a gun. The sample was 43% men and 88% White, predominantly non-Hispanic (75%), with 11% living in rural areas and 19% who currently or previously served in the military. Most (79%) had a child age 12 or younger in the home.

Only about one in four caregivers (24%) correctly answered that suicide is the leading cause of firearm death in Colorado, with similar rates of correct responses among both firearm owners and non-firearm owners. Both groups were also similarly likely (64% overall) to be concerned about youth suicide in their community, though those from homes with firearms were less likely to be concerned about youth suicide in their own family (28%) than those from homes without firearms (39%; P = .013).

In addition, caregivers from homes with versus without firearms were considerably less likely to believe suicide can be prevented (48% vs 69%) and were less likely to believe that temporarily removing a firearm from the home reduces the risk for gun injury or death (60% vs 78%; P < .001 for both comparisons).

Firearm owners were also much less likely than non-owners to believe keeping a gun in the home makes it more dangerous (7% vs 29%) and over twice as likely to think keeping a firearm makes their home safer (52% vs 22%; P < .001). The vast majority of respondents (89%) believed secure storage of guns reduces the risk for injury or death, though the response was higher for firearm owners (93%) than for non-owners (86%; P < .001).

“Our finding that most firearm owners believe that secure firearm storage is protective against firearm injury is a promising messaging strategy,” the authors wrote. “It presents a preventive education opportunity for adults living with children who have mental health concerns, who may benefit most from secure in-home storage and/or temporary and voluntary storage of firearms away from home.”
 

Firearm Injuries

A separate study at the AAP conference underscored the devastating impact of firearm injuries even among those who survive, whether self-inflicted or not, and the potential for reducing healthcare treatment and costs from effective prevention efforts. A national analysis of pediatric inpatient data from 2017 to 2020 calculated how much greater the burden of healthcare treatment and costs is for firearm injuries of any kind compared with penetrating traumas and blunt traumas.

“As a surgical resident, I have seen these patients who make it into the trauma bed that we are then faced to care for,” said Colleen Nofi, DO, PhD, MBA, a general surgery resident at Cohen Children’s Medical Center at Northwell Health in New York. “Anecdotally, we understand that the devastation and injury caused by bullets far outweighs the injuries caused by other trauma mechanisms,” but the actual calculation of the burden hasn’t been studied.

Northwell Health

A version of this article appeared on Medscape.com.

ORLANDO, FLORIDA — Pediatricians and other healthcare providers have a valuable role to play in screening parents for firearm ownership and offering counseling on safe storage practices, according to researchers who presented their findings at the American Academy of Pediatrics (AAP) 2024 National Conference.

An estimated 4.6 million US homes with children have firearms that are loaded and unlocked, a risk factor for youth suicide, yet only about half of parents of suicidal children had been screened for gun ownership in the hospital even as most would be receptive to both firearm screening and counseling, found one study in Texas.

In another study in Colorado, nearly all firearm owners believed that securely storing guns reduces the risk for firearm injury or death, but owners were less likely than non-owners to believe suicide is preventable or that removing a gun from the home reduces the risk for injury or death.

“Previous studies have shown that when pediatricians discuss the importance of armed safe storage guidance with families, families are actually more likely to go home and store firearms safely — storing them locked, unloaded, and separate from the ammunition,” said study author Taylor Rosenbaum, MD, a former pediatric fellow at Baylor College of Medicine/Texas Children’s Hospital in Houston and now an assistant professor at Children’s Hospital University of Miami. “However, previous studies have also shown that pediatricians really are not discussing firearm safe storage with our patients and their families, and we see this both in the outpatient setting, but especially in the inpatient setting for youth suicides, which have risen since 2020 and now are the second leading cause of death for those who are 10-24 years old in the United States.”

University of Miami

Firearm Safety Is a Necessary Conversation

The leading cause of death among children and teens aged 1-19 years is actually firearms, which are also the most fatal method for suicide. While only 4% of all suicide attempts in youth are fatal, 90% of those attempted with a firearm are fatal, Dr. Rosenbaum said. In addition, she said, 80% of the guns used in attempted suicide by children and teens belonged to a family member, and an estimated 70% of firearm-related suicides in youth can be prevented with safe storage of guns.

“This really gives us, as pediatricians, something actionable to do during these hospitalizations” for suicidal ideation or attempts, Dr. Rosenbaum said. “We know that when pediatricians discuss the importance of firearm safe storage guidance with families, they’re more likely to store their firearm safely,” Dr. Rosenbaum said. “We also know that families are not being screened for firearm ownership, that caregivers of youth who are in the hospital for suicidal thoughts or actions want their healthcare team to be screening for firearms, to be giving them information on how to safely secure their firearms, and to be providing free firearm blocks.”

Nathan Boonstra, MD, a general pediatrician at Blank Children’s Hospital, Des Moines, Iowa, said these findings are encouraging in terms of the opportunity pediatricians have.

“There is so much politicization around even basic firearm safety that pediatricians might shy away from the topic, but this research is reassuring that parents are receptive to our advice on safe gun storage,” said Dr. Boonstra, who was not involved in any of this presented research. “It’s especially important for pediatricians to address home firearms when their patient has a history of suicidal ideation or an attempt.”
 

Reducing the Risk

The Colorado findings similarly reinforce the opportunity physicians have to help caregivers reduce suicide risk, according to Maya Haasz, MD, an associate professor of pediatrics and emergency medicine at the University of Colorado Anschutz Medical Campus, Aurora, Colorado.

“Only 60% of firearm owners believed that removing firearms from the home in times of mental health crisis can decrease the risk of suicide,” she said. “These findings are really concerning, but what we found on the flip side was that 93% of firearm owners actually believe that secure storage can overall decrease the risk for firearm injury and death. So overall, we are underestimating the risk for suicide in our community, and we’re also underestimating our ability to prevent it.”

University of Colorado

Survey Results

Dr. Rosenbaum’s team conducted the survey in Houston with caregivers whose children were 8-21 years old and hospitalized for suicidal ideation or attempts at a large children’s hospital and two nearby community hospitals between June 2023 and May 2024. The respondents were 46% White and 23% Black, and 47% of the population were Hispanic, all but three of whom were not gun owners.

Among 244 potential participants, only 150 were eligible and approached, and 100 of these completed the surveys, including 26% firearm owners and 68% non-owners. Most of the youth (74%) were aged 14-17 years, and about three in four respondents were their mothers. Only half of the respondents (51%) said the healthcare provider had asked them whether they owned a gun.

One of the key findings Dr. Rosenbaum highlighted was the receptiveness of firearm-owning caregivers to advice from healthcare providers about ownership. If the healthcare team advised parents not to have any guns in the home for the safety of their child with self-arm, 58% of the firearm owners would follow the advice and 27% would consider it, with none saying they would be offended by it.

Among the firearm owners, 81% said their guns were safely secured where they did not believe their child could access it, which meant one in five youth had unsecured access to firearms. Most of the gun owners (77%), like the non-owners (70%), were “not at all worried” about their child getting ahold of a gun in the home, though 11.5% of the firearm owners were “very worried” about it. Interestingly, more gun owners (19%) were very worried about their children accessing a gun outside their home, a concern shared by 37% of non-owners. Nearly twice as many gun owners (46%) as non-owners (25%) were not at all worried about their child getting a gun outside the home.

The vast majority of respondents — 88% of gun owners and 91% of non-owners — felt it was “very important for the healthcare team to ask parents of children with suicidal ideation/attempts about firearms in the home.” Similarly, high proportions believed it was important for the healthcare team to counsel those parents on safe gun storage. Although only 69% of firearm owners believed it was important to distribute firearm locks in the hospital, 81% would be interested in receiving a free one. Significantly more of the non-owners (80%; P = .02) believed free lock distribution was important, and 72% of non-owners would also be interested in one.

About half the respondents (55%) preferred to hear firearm counseling one-on-one from a provider, whereas 31% would like written information and 27% would be interested in a video. In terms of what information parents preferred to receive, a little over half of owners (54%) and non-owners (56%) were interested in how or when (50% and 40%, respectively) to discuss the topic with their child. Only about a third (35% owners and 37% non-owners) wanted information on how to discuss the topic with the parents of their child’s friends.

The survey’s biggest limitations after its small size were the selection bias of those willing to complete the survey and potential response bias from the self-reported data.

The study of Colorado caregivers, just published in Pediatrics, surveyed 512 Colorado caregivers in April-May 2023 to learn about their beliefs and perceptions regarding firearms, firearm storage and risk, and youth suicide (2024 Oct 1;154[4]:e2024066930. doi: 10.1542/peds.2024-066930). Just over half the respondents (52%) had grown up in a household with firearms, and 44% currently lived in a household with a gun. The sample was 43% men and 88% White, predominantly non-Hispanic (75%), with 11% living in rural areas and 19% who currently or previously served in the military. Most (79%) had a child age 12 or younger in the home.

Only about one in four caregivers (24%) correctly answered that suicide is the leading cause of firearm death in Colorado, with similar rates of correct responses among both firearm owners and non-firearm owners. Both groups were also similarly likely (64% overall) to be concerned about youth suicide in their community, though those from homes with firearms were less likely to be concerned about youth suicide in their own family (28%) than those from homes without firearms (39%; P = .013).

In addition, caregivers from homes with versus without firearms were considerably less likely to believe suicide can be prevented (48% vs 69%) and were less likely to believe that temporarily removing a firearm from the home reduces the risk for gun injury or death (60% vs 78%; P < .001 for both comparisons).

Firearm owners were also much less likely than non-owners to believe keeping a gun in the home makes it more dangerous (7% vs 29%) and over twice as likely to think keeping a firearm makes their home safer (52% vs 22%; P < .001). The vast majority of respondents (89%) believed secure storage of guns reduces the risk for injury or death, though the response was higher for firearm owners (93%) than for non-owners (86%; P < .001).

“Our finding that most firearm owners believe that secure firearm storage is protective against firearm injury is a promising messaging strategy,” the authors wrote. “It presents a preventive education opportunity for adults living with children who have mental health concerns, who may benefit most from secure in-home storage and/or temporary and voluntary storage of firearms away from home.”
 

Firearm Injuries

A separate study at the AAP conference underscored the devastating impact of firearm injuries even among those who survive, whether self-inflicted or not, and the potential for reducing healthcare treatment and costs from effective prevention efforts. A national analysis of pediatric inpatient data from 2017 to 2020 calculated how much greater the burden of healthcare treatment and costs is for firearm injuries of any kind compared with penetrating traumas and blunt traumas.

“As a surgical resident, I have seen these patients who make it into the trauma bed that we are then faced to care for,” said Colleen Nofi, DO, PhD, MBA, a general surgery resident at Cohen Children’s Medical Center at Northwell Health in New York. “Anecdotally, we understand that the devastation and injury caused by bullets far outweighs the injuries caused by other trauma mechanisms,” but the actual calculation of the burden hasn’t been studied.

Northwell Health

A version of this article appeared on Medscape.com.

ORLANDO, FLORIDA — Pediatricians and other healthcare providers have a valuable role to play in screening parents for firearm ownership and offering counseling on safe storage practices, according to researchers who presented their findings at the American Academy of Pediatrics (AAP) 2024 National Conference.

An estimated 4.6 million US homes with children have firearms that are loaded and unlocked, a risk factor for youth suicide, yet only about half of parents of suicidal children had been screened for gun ownership in the hospital even as most would be receptive to both firearm screening and counseling, found one study in Texas.

In another study in Colorado, nearly all firearm owners believed that securely storing guns reduces the risk for firearm injury or death, but owners were less likely than non-owners to believe suicide is preventable or that removing a gun from the home reduces the risk for injury or death.

“Previous studies have shown that when pediatricians discuss the importance of armed safe storage guidance with families, families are actually more likely to go home and store firearms safely — storing them locked, unloaded, and separate from the ammunition,” said study author Taylor Rosenbaum, MD, a former pediatric fellow at Baylor College of Medicine/Texas Children’s Hospital in Houston and now an assistant professor at Children’s Hospital University of Miami. “However, previous studies have also shown that pediatricians really are not discussing firearm safe storage with our patients and their families, and we see this both in the outpatient setting, but especially in the inpatient setting for youth suicides, which have risen since 2020 and now are the second leading cause of death for those who are 10-24 years old in the United States.”

University of Miami

Firearm Safety Is a Necessary Conversation

The leading cause of death among children and teens aged 1-19 years is actually firearms, which are also the most fatal method for suicide. While only 4% of all suicide attempts in youth are fatal, 90% of those attempted with a firearm are fatal, Dr. Rosenbaum said. In addition, she said, 80% of the guns used in attempted suicide by children and teens belonged to a family member, and an estimated 70% of firearm-related suicides in youth can be prevented with safe storage of guns.

“This really gives us, as pediatricians, something actionable to do during these hospitalizations” for suicidal ideation or attempts, Dr. Rosenbaum said. “We know that when pediatricians discuss the importance of firearm safe storage guidance with families, they’re more likely to store their firearm safely,” Dr. Rosenbaum said. “We also know that families are not being screened for firearm ownership, that caregivers of youth who are in the hospital for suicidal thoughts or actions want their healthcare team to be screening for firearms, to be giving them information on how to safely secure their firearms, and to be providing free firearm blocks.”

Nathan Boonstra, MD, a general pediatrician at Blank Children’s Hospital, Des Moines, Iowa, said these findings are encouraging in terms of the opportunity pediatricians have.

“There is so much politicization around even basic firearm safety that pediatricians might shy away from the topic, but this research is reassuring that parents are receptive to our advice on safe gun storage,” said Dr. Boonstra, who was not involved in any of this presented research. “It’s especially important for pediatricians to address home firearms when their patient has a history of suicidal ideation or an attempt.”
 

Reducing the Risk

The Colorado findings similarly reinforce the opportunity physicians have to help caregivers reduce suicide risk, according to Maya Haasz, MD, an associate professor of pediatrics and emergency medicine at the University of Colorado Anschutz Medical Campus, Aurora, Colorado.

“Only 60% of firearm owners believed that removing firearms from the home in times of mental health crisis can decrease the risk of suicide,” she said. “These findings are really concerning, but what we found on the flip side was that 93% of firearm owners actually believe that secure storage can overall decrease the risk for firearm injury and death. So overall, we are underestimating the risk for suicide in our community, and we’re also underestimating our ability to prevent it.”

University of Colorado

Survey Results

Dr. Rosenbaum’s team conducted the survey in Houston with caregivers whose children were 8-21 years old and hospitalized for suicidal ideation or attempts at a large children’s hospital and two nearby community hospitals between June 2023 and May 2024. The respondents were 46% White and 23% Black, and 47% of the population were Hispanic, all but three of whom were not gun owners.

Among 244 potential participants, only 150 were eligible and approached, and 100 of these completed the surveys, including 26% firearm owners and 68% non-owners. Most of the youth (74%) were aged 14-17 years, and about three in four respondents were their mothers. Only half of the respondents (51%) said the healthcare provider had asked them whether they owned a gun.

One of the key findings Dr. Rosenbaum highlighted was the receptiveness of firearm-owning caregivers to advice from healthcare providers about ownership. If the healthcare team advised parents not to have any guns in the home for the safety of their child with self-arm, 58% of the firearm owners would follow the advice and 27% would consider it, with none saying they would be offended by it.

Among the firearm owners, 81% said their guns were safely secured where they did not believe their child could access it, which meant one in five youth had unsecured access to firearms. Most of the gun owners (77%), like the non-owners (70%), were “not at all worried” about their child getting ahold of a gun in the home, though 11.5% of the firearm owners were “very worried” about it. Interestingly, more gun owners (19%) were very worried about their children accessing a gun outside their home, a concern shared by 37% of non-owners. Nearly twice as many gun owners (46%) as non-owners (25%) were not at all worried about their child getting a gun outside the home.

The vast majority of respondents — 88% of gun owners and 91% of non-owners — felt it was “very important for the healthcare team to ask parents of children with suicidal ideation/attempts about firearms in the home.” Similarly, high proportions believed it was important for the healthcare team to counsel those parents on safe gun storage. Although only 69% of firearm owners believed it was important to distribute firearm locks in the hospital, 81% would be interested in receiving a free one. Significantly more of the non-owners (80%; P = .02) believed free lock distribution was important, and 72% of non-owners would also be interested in one.

About half the respondents (55%) preferred to hear firearm counseling one-on-one from a provider, whereas 31% would like written information and 27% would be interested in a video. In terms of what information parents preferred to receive, a little over half of owners (54%) and non-owners (56%) were interested in how or when (50% and 40%, respectively) to discuss the topic with their child. Only about a third (35% owners and 37% non-owners) wanted information on how to discuss the topic with the parents of their child’s friends.

The survey’s biggest limitations after its small size were the selection bias of those willing to complete the survey and potential response bias from the self-reported data.

The study of Colorado caregivers, just published in Pediatrics, surveyed 512 Colorado caregivers in April-May 2023 to learn about their beliefs and perceptions regarding firearms, firearm storage and risk, and youth suicide (2024 Oct 1;154[4]:e2024066930. doi: 10.1542/peds.2024-066930). Just over half the respondents (52%) had grown up in a household with firearms, and 44% currently lived in a household with a gun. The sample was 43% men and 88% White, predominantly non-Hispanic (75%), with 11% living in rural areas and 19% who currently or previously served in the military. Most (79%) had a child age 12 or younger in the home.

Only about one in four caregivers (24%) correctly answered that suicide is the leading cause of firearm death in Colorado, with similar rates of correct responses among both firearm owners and non-firearm owners. Both groups were also similarly likely (64% overall) to be concerned about youth suicide in their community, though those from homes with firearms were less likely to be concerned about youth suicide in their own family (28%) than those from homes without firearms (39%; P = .013).

In addition, caregivers from homes with versus without firearms were considerably less likely to believe suicide can be prevented (48% vs 69%) and were less likely to believe that temporarily removing a firearm from the home reduces the risk for gun injury or death (60% vs 78%; P < .001 for both comparisons).

Firearm owners were also much less likely than non-owners to believe keeping a gun in the home makes it more dangerous (7% vs 29%) and over twice as likely to think keeping a firearm makes their home safer (52% vs 22%; P < .001). The vast majority of respondents (89%) believed secure storage of guns reduces the risk for injury or death, though the response was higher for firearm owners (93%) than for non-owners (86%; P < .001).

“Our finding that most firearm owners believe that secure firearm storage is protective against firearm injury is a promising messaging strategy,” the authors wrote. “It presents a preventive education opportunity for adults living with children who have mental health concerns, who may benefit most from secure in-home storage and/or temporary and voluntary storage of firearms away from home.”
 

Firearm Injuries

A separate study at the AAP conference underscored the devastating impact of firearm injuries even among those who survive, whether self-inflicted or not, and the potential for reducing healthcare treatment and costs from effective prevention efforts. A national analysis of pediatric inpatient data from 2017 to 2020 calculated how much greater the burden of healthcare treatment and costs is for firearm injuries of any kind compared with penetrating traumas and blunt traumas.

“As a surgical resident, I have seen these patients who make it into the trauma bed that we are then faced to care for,” said Colleen Nofi, DO, PhD, MBA, a general surgery resident at Cohen Children’s Medical Center at Northwell Health in New York. “Anecdotally, we understand that the devastation and injury caused by bullets far outweighs the injuries caused by other trauma mechanisms,” but the actual calculation of the burden hasn’t been studied.

Northwell Health

A version of this article appeared on Medscape.com.

Newly issued guidelines for cancer screening in patients with dermatomyositis had 100% sensitivity in a single institution’s cohort, though most of the cancers found would have been detected with standard cancer screenings recommended for the general population, according to a research letter published in JAMA Dermatology.

“These early results emphasize the continued need to refine risk assessment and cancer screening for patients with dermatomyositis while balancing resource use and outcomes,” concluded Caroline J. Stone and her colleagues at the Department of Dermatology, Perelman School of Medicine, University of Pennsylvania, Philadelphia.

dermatology.cdlib.org/CC BY-SA 3.0/wikimedia

Patients with dermatomyositis have approximately a 4.7 times greater risk for cancer than those without it, according to a 2016 meta-analysis. Despite the well-established link between cancer and dermatomyositis, cancer in people with idiopathic inflammatory myopathies is commonly diagnosed at a later stage and is the leading cause of death in people with these conditions.
 

Guidelines First Presented in 2022 and Published in 2023

A wide variability in screening practices eventually led the International Myositis Assessment & Clinical Studies Group (IMACS) to present the first evidence-based and consensus-based guidelines for cancer screening of patients with idiopathic inflammatory myopathies, including those with dermatomyositis, at the 2022 annual meeting of the American College of Rheumatology and publish them in 2023 in Nature Reviews Rheumatology. The guidelines advise low-risk patients to undergo basic cancer screening with routine blood and urine studies, liver function tests, plain chest radiography, and age- and sex-appropriate cancer screening.

Intermediate- and high-risk patients are recommended to undergo enhanced screening that can include mammography, Pap tests, endoscopy/colonoscopy, pelvic and transvaginal ultrasonography, prostate-specific antigen or cancer antigen 125 blood tests, fecal occult blood tests, and CT of the neck, thorax, abdomen, and pelvis.

But because the guidelines are new, little evidence exists regarding their validation in real-world cohorts. Researchers, therefore, assessed the IMACS guidelines in 370 patients, aged 18-80 years, who visited the University of Pennsylvania rheumatology-dermatology specialty clinic between July 2008 and January 2024. All participants had dermatomyositis and at least 3 years of follow-up and were an average 48 years old. The vast majority were women (87%) and White participants (89%).

Most (68.6%) had myositis-specific autoantibody test results, one of the factors included in the guidelines for determining whether the patient should be classified as low, intermediate, or high risk. Other factors for risk stratification included myositis subtype, age at disease onset, and clinical features. About half (49.2%) had classic dermatomyositis, 42.4% had amyopathic dermatomyositis, 3.8% had juvenile dermatomyositis, 3.2% had hypomyopathic dermatomyositis, 0.8% had antisynthetase syndrome, and 0.5% had immune-mediated necrotizing myopathy.

Just over half the patients (54%) were classified as high risk, while 37.3% were classified as intermediate risk and 8.9% as low risk using the guidelines. Among the 18 patients (4.9%) with paraneoplastic dermatomyositis, 15 were classified as high risk and 3 as intermediate risk.

Of the patients diagnosed with cancer, 55% of cases were diagnosed about a year before their dermatomyositis diagnosis. In three patients, symptoms “suggestive of cancer at the time of dermatomyositis diagnosis, including lymphadenopathy and unexplained weight loss,” led to diagnostic testing that found an underlying cancer.

In the eight patients diagnosed with cancer after their dermatomyositis diagnosis, 75% of the cancers were identified during the first year of follow-up and 25% in the second year. Five were identified based on basic cancer screening and three on enhanced screening.

A total of 11 patients (3%) developed intravenous contrast allergies, and no other adverse events were reported to be associated with cancer screening, but the study was not designed to capture other types of adverse screening effects, such as cost, quality of life, or risk from radiation exposure.

The most common neoplasm identified was breast cancer, found in nine (50%) of the patients using mammography. Two patients had lung cancer identified with chest radiography and two had ovarian cancer identified with abdominal radiography and CT. The remaining five patients included one each with bladder cancer, papillary thyroid cancer, renal cell carcinoma, non-Hodgkin lymphoma, and adenocarcinoma with unknown primary.

The sensitivity of the guidelines in detecting cancer related to dermatomyositis was 100%, though the authors noted that the “IMACS risk-stratification scheme may overestimate cancer risk and encourage enhanced screening protocols of unclear benefit.” Most of the cancers found after dermatomyositis diagnosis were detected with routine age- and sex-related screening that already falls under basic cancer screening recommendations for the general population. Nonetheless, 90% of the participants fell into the intermediate- and high-risk groups, warranting a more comprehensive and costly enhanced screening protocol.
 

Will the Guidelines Lead to Overscreening? 

The 4.9% cancer prevalence is considerably lower than the typical 15%-25% prevalence among patients with dermatomyositis, but the findings, regardless, suggest the guidelines will lead to overscreening, wrote Andrea D. Maderal, MD, University of Miami Miller School of Medicine in Florida, and Alisa Femia, MD, New York University Grossman School of Medicine, New York City, in an accompanying editorial. Given that the median age in patients with cancer in the study was 58 years — 18 years older than the age cutoff for high-risk criteria — one way to refine the guidelines may be to increase the age for the high-risk category, they suggested.

“While these guidelines led to many ultimately unnecessary screening tests based on currently recommended designations of intermediate-risk and high-risk patients, these guidelines reflect a more conservative approach to screening than was previously performed,” Dr. Maderal and Dr. Femia wrote.

Jeff Gehlhausen, MD, PhD, an assistant professor of dermatology at Yale School of Medicine, New Haven, Connecticut, said he is not concerned about overscreening in patients, however, and is “very enthusiastic” about the findings.

“Patients are very anxious for good reason,” given the typical cancer prevalence of 25% in this population, he said in an interview. “I think therein lies the challenge — with that risk, what is ‘enough’ screening?” Yet this “incredibly impressive” study “provides real insights into the applicability of the IMACS screenings to our dermatomyositis management,” including relevance to his own patients. “Their findings are instructive for how to better evaluate these patients in a more mindful fashion,” he said, and they are particularly welcome, given how widely variable practice has historically been before the guidelines were issued.

“This question has been an outstanding one for decades, and nearly every doctor has a different answer,” Dr. Gehlhausen said. “The introduction of the guidelines alone are now much more actionable with this study, and that’s why it’s such an important one for our community.”

Benedict Wu, DO, PhD, director of Inpatient Dermatology and an assistant professor at Montefiore Einstein and a member of the Montefiore Einstein Comprehensive Cancer Center in New York City, similarly regarded the findings as reassuring, though he was surprised at the low prevalence of cancer in the patients.

“The most reassuring finding was that the detection of most malignancies was possible by using routine age- and sex-related screening combined with basic cancer screening,” Wu said in an interview. “Basic cancer screening can reduce costs while keeping patients safe.”

He also found it reassuring that all the paraneoplastic dermatomyositis was in intermediate- or high-risk patients, and while he does not see the IMACS guidelines as overestimating cancer risk, he does think “the risk stratification and recommended screening tests could be revised to be less ‘aggressive.’ ” 

The overall low rate of cancer in the group “calls into question the need for stringent and annual cancer screening,” he said. “In this large cohort of patients, the fact that malignancy was detected within 2 years of dermatomyositis diagnosis will help guide us with long-term screening recommendations.”

Despite the study’s small size and single-center design, the demographics of the patients nearly represents exactly what is found in the United States more broadly, Wu noted. He also drew attention to how many patients lacked the myositis antibody profile performed, and he agreed with the authors that more extensive and prospective studies need to be conducted. He also emphasized the need to keep in mind that “the primary goal of dermatomyositis management should focus on controlling/reducing the disease burden.”

The research was funded by the National Institutes of Health and the US Department of Veterans Affairs. The authors had no disclosures. Dr. Maderal reported personal fees from argenx. No disclosures were noted for Dr. Gehlhausen and Dr. Wu.

A version of this article appeared on Medscape.com.

Newly issued guidelines for cancer screening in patients with dermatomyositis had 100% sensitivity in a single institution’s cohort, though most of the cancers found would have been detected with standard cancer screenings recommended for the general population, according to a research letter published in JAMA Dermatology.

“These early results emphasize the continued need to refine risk assessment and cancer screening for patients with dermatomyositis while balancing resource use and outcomes,” concluded Caroline J. Stone and her colleagues at the Department of Dermatology, Perelman School of Medicine, University of Pennsylvania, Philadelphia.

dermatology.cdlib.org/CC BY-SA 3.0/wikimedia

Patients with dermatomyositis have approximately a 4.7 times greater risk for cancer than those without it, according to a 2016 meta-analysis. Despite the well-established link between cancer and dermatomyositis, cancer in people with idiopathic inflammatory myopathies is commonly diagnosed at a later stage and is the leading cause of death in people with these conditions.
 

Guidelines First Presented in 2022 and Published in 2023

A wide variability in screening practices eventually led the International Myositis Assessment & Clinical Studies Group (IMACS) to present the first evidence-based and consensus-based guidelines for cancer screening of patients with idiopathic inflammatory myopathies, including those with dermatomyositis, at the 2022 annual meeting of the American College of Rheumatology and publish them in 2023 in Nature Reviews Rheumatology. The guidelines advise low-risk patients to undergo basic cancer screening with routine blood and urine studies, liver function tests, plain chest radiography, and age- and sex-appropriate cancer screening.

Intermediate- and high-risk patients are recommended to undergo enhanced screening that can include mammography, Pap tests, endoscopy/colonoscopy, pelvic and transvaginal ultrasonography, prostate-specific antigen or cancer antigen 125 blood tests, fecal occult blood tests, and CT of the neck, thorax, abdomen, and pelvis.

But because the guidelines are new, little evidence exists regarding their validation in real-world cohorts. Researchers, therefore, assessed the IMACS guidelines in 370 patients, aged 18-80 years, who visited the University of Pennsylvania rheumatology-dermatology specialty clinic between July 2008 and January 2024. All participants had dermatomyositis and at least 3 years of follow-up and were an average 48 years old. The vast majority were women (87%) and White participants (89%).

Most (68.6%) had myositis-specific autoantibody test results, one of the factors included in the guidelines for determining whether the patient should be classified as low, intermediate, or high risk. Other factors for risk stratification included myositis subtype, age at disease onset, and clinical features. About half (49.2%) had classic dermatomyositis, 42.4% had amyopathic dermatomyositis, 3.8% had juvenile dermatomyositis, 3.2% had hypomyopathic dermatomyositis, 0.8% had antisynthetase syndrome, and 0.5% had immune-mediated necrotizing myopathy.

Just over half the patients (54%) were classified as high risk, while 37.3% were classified as intermediate risk and 8.9% as low risk using the guidelines. Among the 18 patients (4.9%) with paraneoplastic dermatomyositis, 15 were classified as high risk and 3 as intermediate risk.

Of the patients diagnosed with cancer, 55% of cases were diagnosed about a year before their dermatomyositis diagnosis. In three patients, symptoms “suggestive of cancer at the time of dermatomyositis diagnosis, including lymphadenopathy and unexplained weight loss,” led to diagnostic testing that found an underlying cancer.

In the eight patients diagnosed with cancer after their dermatomyositis diagnosis, 75% of the cancers were identified during the first year of follow-up and 25% in the second year. Five were identified based on basic cancer screening and three on enhanced screening.

A total of 11 patients (3%) developed intravenous contrast allergies, and no other adverse events were reported to be associated with cancer screening, but the study was not designed to capture other types of adverse screening effects, such as cost, quality of life, or risk from radiation exposure.

The most common neoplasm identified was breast cancer, found in nine (50%) of the patients using mammography. Two patients had lung cancer identified with chest radiography and two had ovarian cancer identified with abdominal radiography and CT. The remaining five patients included one each with bladder cancer, papillary thyroid cancer, renal cell carcinoma, non-Hodgkin lymphoma, and adenocarcinoma with unknown primary.

The sensitivity of the guidelines in detecting cancer related to dermatomyositis was 100%, though the authors noted that the “IMACS risk-stratification scheme may overestimate cancer risk and encourage enhanced screening protocols of unclear benefit.” Most of the cancers found after dermatomyositis diagnosis were detected with routine age- and sex-related screening that already falls under basic cancer screening recommendations for the general population. Nonetheless, 90% of the participants fell into the intermediate- and high-risk groups, warranting a more comprehensive and costly enhanced screening protocol.
 

Will the Guidelines Lead to Overscreening? 

The 4.9% cancer prevalence is considerably lower than the typical 15%-25% prevalence among patients with dermatomyositis, but the findings, regardless, suggest the guidelines will lead to overscreening, wrote Andrea D. Maderal, MD, University of Miami Miller School of Medicine in Florida, and Alisa Femia, MD, New York University Grossman School of Medicine, New York City, in an accompanying editorial. Given that the median age in patients with cancer in the study was 58 years — 18 years older than the age cutoff for high-risk criteria — one way to refine the guidelines may be to increase the age for the high-risk category, they suggested.

“While these guidelines led to many ultimately unnecessary screening tests based on currently recommended designations of intermediate-risk and high-risk patients, these guidelines reflect a more conservative approach to screening than was previously performed,” Dr. Maderal and Dr. Femia wrote.

Jeff Gehlhausen, MD, PhD, an assistant professor of dermatology at Yale School of Medicine, New Haven, Connecticut, said he is not concerned about overscreening in patients, however, and is “very enthusiastic” about the findings.

“Patients are very anxious for good reason,” given the typical cancer prevalence of 25% in this population, he said in an interview. “I think therein lies the challenge — with that risk, what is ‘enough’ screening?” Yet this “incredibly impressive” study “provides real insights into the applicability of the IMACS screenings to our dermatomyositis management,” including relevance to his own patients. “Their findings are instructive for how to better evaluate these patients in a more mindful fashion,” he said, and they are particularly welcome, given how widely variable practice has historically been before the guidelines were issued.

“This question has been an outstanding one for decades, and nearly every doctor has a different answer,” Dr. Gehlhausen said. “The introduction of the guidelines alone are now much more actionable with this study, and that’s why it’s such an important one for our community.”

Benedict Wu, DO, PhD, director of Inpatient Dermatology and an assistant professor at Montefiore Einstein and a member of the Montefiore Einstein Comprehensive Cancer Center in New York City, similarly regarded the findings as reassuring, though he was surprised at the low prevalence of cancer in the patients.

“The most reassuring finding was that the detection of most malignancies was possible by using routine age- and sex-related screening combined with basic cancer screening,” Wu said in an interview. “Basic cancer screening can reduce costs while keeping patients safe.”

He also found it reassuring that all the paraneoplastic dermatomyositis was in intermediate- or high-risk patients, and while he does not see the IMACS guidelines as overestimating cancer risk, he does think “the risk stratification and recommended screening tests could be revised to be less ‘aggressive.’ ” 

The overall low rate of cancer in the group “calls into question the need for stringent and annual cancer screening,” he said. “In this large cohort of patients, the fact that malignancy was detected within 2 years of dermatomyositis diagnosis will help guide us with long-term screening recommendations.”

Despite the study’s small size and single-center design, the demographics of the patients nearly represents exactly what is found in the United States more broadly, Wu noted. He also drew attention to how many patients lacked the myositis antibody profile performed, and he agreed with the authors that more extensive and prospective studies need to be conducted. He also emphasized the need to keep in mind that “the primary goal of dermatomyositis management should focus on controlling/reducing the disease burden.”

The research was funded by the National Institutes of Health and the US Department of Veterans Affairs. The authors had no disclosures. Dr. Maderal reported personal fees from argenx. No disclosures were noted for Dr. Gehlhausen and Dr. Wu.

A version of this article appeared on Medscape.com.

Newly issued guidelines for cancer screening in patients with dermatomyositis had 100% sensitivity in a single institution’s cohort, though most of the cancers found would have been detected with standard cancer screenings recommended for the general population, according to a research letter published in JAMA Dermatology.

“These early results emphasize the continued need to refine risk assessment and cancer screening for patients with dermatomyositis while balancing resource use and outcomes,” concluded Caroline J. Stone and her colleagues at the Department of Dermatology, Perelman School of Medicine, University of Pennsylvania, Philadelphia.

dermatology.cdlib.org/CC BY-SA 3.0/wikimedia

Patients with dermatomyositis have approximately a 4.7 times greater risk for cancer than those without it, according to a 2016 meta-analysis. Despite the well-established link between cancer and dermatomyositis, cancer in people with idiopathic inflammatory myopathies is commonly diagnosed at a later stage and is the leading cause of death in people with these conditions.
 

Guidelines First Presented in 2022 and Published in 2023

A wide variability in screening practices eventually led the International Myositis Assessment & Clinical Studies Group (IMACS) to present the first evidence-based and consensus-based guidelines for cancer screening of patients with idiopathic inflammatory myopathies, including those with dermatomyositis, at the 2022 annual meeting of the American College of Rheumatology and publish them in 2023 in Nature Reviews Rheumatology. The guidelines advise low-risk patients to undergo basic cancer screening with routine blood and urine studies, liver function tests, plain chest radiography, and age- and sex-appropriate cancer screening.

Intermediate- and high-risk patients are recommended to undergo enhanced screening that can include mammography, Pap tests, endoscopy/colonoscopy, pelvic and transvaginal ultrasonography, prostate-specific antigen or cancer antigen 125 blood tests, fecal occult blood tests, and CT of the neck, thorax, abdomen, and pelvis.

But because the guidelines are new, little evidence exists regarding their validation in real-world cohorts. Researchers, therefore, assessed the IMACS guidelines in 370 patients, aged 18-80 years, who visited the University of Pennsylvania rheumatology-dermatology specialty clinic between July 2008 and January 2024. All participants had dermatomyositis and at least 3 years of follow-up and were an average 48 years old. The vast majority were women (87%) and White participants (89%).

Most (68.6%) had myositis-specific autoantibody test results, one of the factors included in the guidelines for determining whether the patient should be classified as low, intermediate, or high risk. Other factors for risk stratification included myositis subtype, age at disease onset, and clinical features. About half (49.2%) had classic dermatomyositis, 42.4% had amyopathic dermatomyositis, 3.8% had juvenile dermatomyositis, 3.2% had hypomyopathic dermatomyositis, 0.8% had antisynthetase syndrome, and 0.5% had immune-mediated necrotizing myopathy.

Just over half the patients (54%) were classified as high risk, while 37.3% were classified as intermediate risk and 8.9% as low risk using the guidelines. Among the 18 patients (4.9%) with paraneoplastic dermatomyositis, 15 were classified as high risk and 3 as intermediate risk.

Of the patients diagnosed with cancer, 55% of cases were diagnosed about a year before their dermatomyositis diagnosis. In three patients, symptoms “suggestive of cancer at the time of dermatomyositis diagnosis, including lymphadenopathy and unexplained weight loss,” led to diagnostic testing that found an underlying cancer.

In the eight patients diagnosed with cancer after their dermatomyositis diagnosis, 75% of the cancers were identified during the first year of follow-up and 25% in the second year. Five were identified based on basic cancer screening and three on enhanced screening.

A total of 11 patients (3%) developed intravenous contrast allergies, and no other adverse events were reported to be associated with cancer screening, but the study was not designed to capture other types of adverse screening effects, such as cost, quality of life, or risk from radiation exposure.

The most common neoplasm identified was breast cancer, found in nine (50%) of the patients using mammography. Two patients had lung cancer identified with chest radiography and two had ovarian cancer identified with abdominal radiography and CT. The remaining five patients included one each with bladder cancer, papillary thyroid cancer, renal cell carcinoma, non-Hodgkin lymphoma, and adenocarcinoma with unknown primary.

The sensitivity of the guidelines in detecting cancer related to dermatomyositis was 100%, though the authors noted that the “IMACS risk-stratification scheme may overestimate cancer risk and encourage enhanced screening protocols of unclear benefit.” Most of the cancers found after dermatomyositis diagnosis were detected with routine age- and sex-related screening that already falls under basic cancer screening recommendations for the general population. Nonetheless, 90% of the participants fell into the intermediate- and high-risk groups, warranting a more comprehensive and costly enhanced screening protocol.
 

Will the Guidelines Lead to Overscreening? 

The 4.9% cancer prevalence is considerably lower than the typical 15%-25% prevalence among patients with dermatomyositis, but the findings, regardless, suggest the guidelines will lead to overscreening, wrote Andrea D. Maderal, MD, University of Miami Miller School of Medicine in Florida, and Alisa Femia, MD, New York University Grossman School of Medicine, New York City, in an accompanying editorial. Given that the median age in patients with cancer in the study was 58 years — 18 years older than the age cutoff for high-risk criteria — one way to refine the guidelines may be to increase the age for the high-risk category, they suggested.

“While these guidelines led to many ultimately unnecessary screening tests based on currently recommended designations of intermediate-risk and high-risk patients, these guidelines reflect a more conservative approach to screening than was previously performed,” Dr. Maderal and Dr. Femia wrote.

Jeff Gehlhausen, MD, PhD, an assistant professor of dermatology at Yale School of Medicine, New Haven, Connecticut, said he is not concerned about overscreening in patients, however, and is “very enthusiastic” about the findings.

“Patients are very anxious for good reason,” given the typical cancer prevalence of 25% in this population, he said in an interview. “I think therein lies the challenge — with that risk, what is ‘enough’ screening?” Yet this “incredibly impressive” study “provides real insights into the applicability of the IMACS screenings to our dermatomyositis management,” including relevance to his own patients. “Their findings are instructive for how to better evaluate these patients in a more mindful fashion,” he said, and they are particularly welcome, given how widely variable practice has historically been before the guidelines were issued.

“This question has been an outstanding one for decades, and nearly every doctor has a different answer,” Dr. Gehlhausen said. “The introduction of the guidelines alone are now much more actionable with this study, and that’s why it’s such an important one for our community.”

Benedict Wu, DO, PhD, director of Inpatient Dermatology and an assistant professor at Montefiore Einstein and a member of the Montefiore Einstein Comprehensive Cancer Center in New York City, similarly regarded the findings as reassuring, though he was surprised at the low prevalence of cancer in the patients.

“The most reassuring finding was that the detection of most malignancies was possible by using routine age- and sex-related screening combined with basic cancer screening,” Wu said in an interview. “Basic cancer screening can reduce costs while keeping patients safe.”

He also found it reassuring that all the paraneoplastic dermatomyositis was in intermediate- or high-risk patients, and while he does not see the IMACS guidelines as overestimating cancer risk, he does think “the risk stratification and recommended screening tests could be revised to be less ‘aggressive.’ ” 

The overall low rate of cancer in the group “calls into question the need for stringent and annual cancer screening,” he said. “In this large cohort of patients, the fact that malignancy was detected within 2 years of dermatomyositis diagnosis will help guide us with long-term screening recommendations.”

Despite the study’s small size and single-center design, the demographics of the patients nearly represents exactly what is found in the United States more broadly, Wu noted. He also drew attention to how many patients lacked the myositis antibody profile performed, and he agreed with the authors that more extensive and prospective studies need to be conducted. He also emphasized the need to keep in mind that “the primary goal of dermatomyositis management should focus on controlling/reducing the disease burden.”

The research was funded by the National Institutes of Health and the US Department of Veterans Affairs. The authors had no disclosures. Dr. Maderal reported personal fees from argenx. No disclosures were noted for Dr. Gehlhausen and Dr. Wu.

A version of this article appeared on Medscape.com.

ORLANDO – Heat-related emergency department visits in children and teens more than doubled over the past decade in two Texas children’s hospitals, according to research presented at the annual meeting of the American Academy of Pediatrics (AAP).

“Our study really highlights the adverse effects that can come from extreme heat, and how increasing heat-related illness is affecting our children,” Taylor Merritt, MD, a pediatric resident at the University of Texas Southwestern Medical Center and Children’s Health in Dallas, said during a press briefing.

University of Texas

Underestimating the Problem?

Lori Byron, MD, a pediatrician from Red Lodge, Montana, who heads the AAP Chapter Climate Advocates program and was not involved in this research, was not surprised by the findings. “If anything, we’re vastly underestimating it because when people come in with heat exhaustion or heat smoke, that gets coded correctly, but when people come in with heart attacks, asthma attacks, strokes, and other exacerbations of chronic disease, it very rarely gets coded as a heat-related illness.”

Record-breaking summer temperatures from the changing climate have led to increased heat-related morbidity and mortality. Past research suggests that children and teens make up nearly half of all those affected by heat-related illnesses, she noted. 2023, for example, was the hottest year on record, and 2024 is predicted to be hotter, Dr. Merritt said.
 

A Sharp Increase in Cases

The retrospective study examined emergency department diagnoses during May-September from 2012-2023 at two large children’s hospitals within a north Texas pediatric health care system. The researchers compared heat-specific conditions with rhabdomyolysis encounters based on ICD-10 coding.

Heat-specific conditions include heatstroke/sunstroke, exertion heatstroke, heat syncope, heat crap, heat exhaustion, heat fatigue, heat edema, and exposure to excessive natural heat. Rhabdomyolysis encounters included both exertional and nonexertional rhabdomyolysis as well as non-traumatic rhabdomyolysis and elevated creatine kinase (CK) levels.

Among 542 heat-related encounters, 77% had heat-specific diagnoses and 24% had a rhabdomyolysis diagnosis. Combined, heat-related encounters increased 170% from 2012 to 2023, from 4.3 per 10,000 to 11.6 per 10,000 (P = .1). Summer months with higher peak temperatures were also associated with higher heat-related volume in the emergency department (P < .001).

Teenage boys were most likely to have rhabdomyolysis, with 82% of the cases occurring in boys and 70% in ages 12-18 (P < .001). “Compared to the rhabdomyolysis group, the heat-specific group was more likely to be younger, Hispanic, use government-based insurance, and live in an area with a lower Child Opportunity Index,” Dr. Merritt reported. “Most heat-specific encounters resulted in an ED discharge (96%), while most rhabdomyolysis encounters resulted in hospital admission (63%)” (P < .001).

”Thankfully, pediatric heat-related illness is still relatively rare,” Dr. Merritt said. “However, given the context of increasing temperatures, this is important for us all to know, anyone who cares for children, whether that be families or parents or pediatricians.”
 

Prevention Is Key

Dr. Byron noted that about half of AAP chapters now have climate committees, many of which have created educational materials on heat and wildfire smoke and on talking with athletes about risk of heat-related illnesses.

“A lot of the state high school sports associations are actually now adopting guidelines on when it’s safe to practice and when it’s safe to play for heat and for smoke, so that’s definitely something that we can talk to parents about and kids about,” Dr. Byron said. “Otherwise, you still have a lot of coaches and a lot of kids out there that think you’re just supposed to be tough and barrel through it.”

Rhabdomyolysis and heat stroke are both potentially deadly illnesses, so the biggest focus needs to be on prevention, Dr. Byron said. “Not just working with individuals in your office, but working within your school or within your state high school sports association is totally within the lane of a pediatrician to get involved.”

The research had no external funding. Dr. Merritt and Dr. Byron had no disclosures.

ORLANDO – Heat-related emergency department visits in children and teens more than doubled over the past decade in two Texas children’s hospitals, according to research presented at the annual meeting of the American Academy of Pediatrics (AAP).

“Our study really highlights the adverse effects that can come from extreme heat, and how increasing heat-related illness is affecting our children,” Taylor Merritt, MD, a pediatric resident at the University of Texas Southwestern Medical Center and Children’s Health in Dallas, said during a press briefing.

University of Texas

Underestimating the Problem?

Lori Byron, MD, a pediatrician from Red Lodge, Montana, who heads the AAP Chapter Climate Advocates program and was not involved in this research, was not surprised by the findings. “If anything, we’re vastly underestimating it because when people come in with heat exhaustion or heat smoke, that gets coded correctly, but when people come in with heart attacks, asthma attacks, strokes, and other exacerbations of chronic disease, it very rarely gets coded as a heat-related illness.”

Record-breaking summer temperatures from the changing climate have led to increased heat-related morbidity and mortality. Past research suggests that children and teens make up nearly half of all those affected by heat-related illnesses, she noted. 2023, for example, was the hottest year on record, and 2024 is predicted to be hotter, Dr. Merritt said.
 

A Sharp Increase in Cases

The retrospective study examined emergency department diagnoses during May-September from 2012-2023 at two large children’s hospitals within a north Texas pediatric health care system. The researchers compared heat-specific conditions with rhabdomyolysis encounters based on ICD-10 coding.

Heat-specific conditions include heatstroke/sunstroke, exertion heatstroke, heat syncope, heat crap, heat exhaustion, heat fatigue, heat edema, and exposure to excessive natural heat. Rhabdomyolysis encounters included both exertional and nonexertional rhabdomyolysis as well as non-traumatic rhabdomyolysis and elevated creatine kinase (CK) levels.

Among 542 heat-related encounters, 77% had heat-specific diagnoses and 24% had a rhabdomyolysis diagnosis. Combined, heat-related encounters increased 170% from 2012 to 2023, from 4.3 per 10,000 to 11.6 per 10,000 (P = .1). Summer months with higher peak temperatures were also associated with higher heat-related volume in the emergency department (P < .001).

Teenage boys were most likely to have rhabdomyolysis, with 82% of the cases occurring in boys and 70% in ages 12-18 (P < .001). “Compared to the rhabdomyolysis group, the heat-specific group was more likely to be younger, Hispanic, use government-based insurance, and live in an area with a lower Child Opportunity Index,” Dr. Merritt reported. “Most heat-specific encounters resulted in an ED discharge (96%), while most rhabdomyolysis encounters resulted in hospital admission (63%)” (P < .001).

”Thankfully, pediatric heat-related illness is still relatively rare,” Dr. Merritt said. “However, given the context of increasing temperatures, this is important for us all to know, anyone who cares for children, whether that be families or parents or pediatricians.”
 

Prevention Is Key

Dr. Byron noted that about half of AAP chapters now have climate committees, many of which have created educational materials on heat and wildfire smoke and on talking with athletes about risk of heat-related illnesses.

“A lot of the state high school sports associations are actually now adopting guidelines on when it’s safe to practice and when it’s safe to play for heat and for smoke, so that’s definitely something that we can talk to parents about and kids about,” Dr. Byron said. “Otherwise, you still have a lot of coaches and a lot of kids out there that think you’re just supposed to be tough and barrel through it.”

Rhabdomyolysis and heat stroke are both potentially deadly illnesses, so the biggest focus needs to be on prevention, Dr. Byron said. “Not just working with individuals in your office, but working within your school or within your state high school sports association is totally within the lane of a pediatrician to get involved.”

The research had no external funding. Dr. Merritt and Dr. Byron had no disclosures.

ORLANDO – Heat-related emergency department visits in children and teens more than doubled over the past decade in two Texas children’s hospitals, according to research presented at the annual meeting of the American Academy of Pediatrics (AAP).

“Our study really highlights the adverse effects that can come from extreme heat, and how increasing heat-related illness is affecting our children,” Taylor Merritt, MD, a pediatric resident at the University of Texas Southwestern Medical Center and Children’s Health in Dallas, said during a press briefing.

University of Texas

Underestimating the Problem?

Lori Byron, MD, a pediatrician from Red Lodge, Montana, who heads the AAP Chapter Climate Advocates program and was not involved in this research, was not surprised by the findings. “If anything, we’re vastly underestimating it because when people come in with heat exhaustion or heat smoke, that gets coded correctly, but when people come in with heart attacks, asthma attacks, strokes, and other exacerbations of chronic disease, it very rarely gets coded as a heat-related illness.”

Record-breaking summer temperatures from the changing climate have led to increased heat-related morbidity and mortality. Past research suggests that children and teens make up nearly half of all those affected by heat-related illnesses, she noted. 2023, for example, was the hottest year on record, and 2024 is predicted to be hotter, Dr. Merritt said.
 

A Sharp Increase in Cases

The retrospective study examined emergency department diagnoses during May-September from 2012-2023 at two large children’s hospitals within a north Texas pediatric health care system. The researchers compared heat-specific conditions with rhabdomyolysis encounters based on ICD-10 coding.

Heat-specific conditions include heatstroke/sunstroke, exertion heatstroke, heat syncope, heat crap, heat exhaustion, heat fatigue, heat edema, and exposure to excessive natural heat. Rhabdomyolysis encounters included both exertional and nonexertional rhabdomyolysis as well as non-traumatic rhabdomyolysis and elevated creatine kinase (CK) levels.

Among 542 heat-related encounters, 77% had heat-specific diagnoses and 24% had a rhabdomyolysis diagnosis. Combined, heat-related encounters increased 170% from 2012 to 2023, from 4.3 per 10,000 to 11.6 per 10,000 (P = .1). Summer months with higher peak temperatures were also associated with higher heat-related volume in the emergency department (P < .001).

Teenage boys were most likely to have rhabdomyolysis, with 82% of the cases occurring in boys and 70% in ages 12-18 (P < .001). “Compared to the rhabdomyolysis group, the heat-specific group was more likely to be younger, Hispanic, use government-based insurance, and live in an area with a lower Child Opportunity Index,” Dr. Merritt reported. “Most heat-specific encounters resulted in an ED discharge (96%), while most rhabdomyolysis encounters resulted in hospital admission (63%)” (P < .001).

”Thankfully, pediatric heat-related illness is still relatively rare,” Dr. Merritt said. “However, given the context of increasing temperatures, this is important for us all to know, anyone who cares for children, whether that be families or parents or pediatricians.”
 

Prevention Is Key

Dr. Byron noted that about half of AAP chapters now have climate committees, many of which have created educational materials on heat and wildfire smoke and on talking with athletes about risk of heat-related illnesses.

“A lot of the state high school sports associations are actually now adopting guidelines on when it’s safe to practice and when it’s safe to play for heat and for smoke, so that’s definitely something that we can talk to parents about and kids about,” Dr. Byron said. “Otherwise, you still have a lot of coaches and a lot of kids out there that think you’re just supposed to be tough and barrel through it.”

Rhabdomyolysis and heat stroke are both potentially deadly illnesses, so the biggest focus needs to be on prevention, Dr. Byron said. “Not just working with individuals in your office, but working within your school or within your state high school sports association is totally within the lane of a pediatrician to get involved.”

The research had no external funding. Dr. Merritt and Dr. Byron had no disclosures.

When Jennifer Welsh, a 40-year-old from New Britain, Connecticut, visited her doctor about pain in her joints and neck, her doctor sent her to the emergency department (ED) to rule out meningitis. The ED did rule that out, as well as strep, so Ms. Welsh went to her follow-up appointment a few days later, hoping for answers or at least more tests to get those answers.

Instead, the doctor — a different one from the same practice as her primary care physician (PCP) — wouldn’t even talk to Ms. Welsh about her symptoms because she couldn’t see the ED’s results and refused to view the results that Ms. Welsh could pull up online.

“She just completely shut me down,” Ms. Welsh recalled. “It was a really awful appointment, and I left in tears. I was in physical pain, I had just been to the ER, nothing is really resolved, I’m stressed out about it, and this woman is completely dismissing me.”

She had been able to schedule an appointment with her regular PCP later that week, but after the harrowing experience with this doctor, she wondered if her PCP would order the rheumatoid arthritis (RA) test that Ms. Welsh suspected she needed. So, she took matters into her own hands.

“I was searching for what test to ask for from my doctor,” she said, and she found that she could order it on her own from a major lab company she was already familiar with. For around $100, “I could get it done and see what it says on my own,” she said.

But that’s not how it worked out. Her regular PCP apologized for the other doctor’s behavior and ordered the RA test as well as additional tests — and got results while Ms. Welsh still waited for the one she ordered to arrive over a week later.

At first, Ms. Welsh was grateful she could order the RA test without her doctor’s referral. “I felt it gave me a sense of control over the situation that I felt really not in control of, until the system failed me, and I didn’t get the results,” she said. But then, “not having someone I could call and get an answer about why my tests were delayed, why I wasn’t able to access them, why it was taking so long — it was definitely anxiety-inducing.”
 

A Growing Market

Ms. Welsh is one of a growing number of patients who are ordering direct-to-consumer (DTC) lab tests without the recommendation or guidance of a doctor. They’re offered online by labs ranging from well-established giants like Quest and Labcorp to smaller, potentially less vetted companies, although some smaller companies contract with larger companies like Quest. Combined, the DTC market is projected to be worth $2 billion by 2025.

Yet the burgeoning industry has also drawn critiques from both bioethicists and privacy experts. A research letter in JAMA in 2023, for example, found that less than half of the 21 companies identified in an online search declared Health Insurance Portability and Accountability Act compliance, while more than half “indicated the potential use of consumer data for research purposes either internally or through third-party sharing.” That study found the most commonly offered tests were related to diabetes, the thyroid, and vitamin levels, and hormone tests for men and women, such as testosterone or estradiol.

But a number of companies also offer tests related to rheumatologic conditions. A handful of tests offered by Labcorp, for example, could be used in rheumatology, such as tests for celiac antibodies or high-sensitivity C-reactive protein. Quest similarly offers a handful of autoimmune-related tests. But other companies offer a long slate of autoimmune or antibody tests.

The antinuclear antibody (ANA) test and RA panel offered by Quest are the same tests, run and analyzed in the same labs, as those ordered by physicians and hospitals, according to James Faix, MD, the medical director of immunology at Quest Diagnostics. Their RA panel includes rheumatoid factor and anti-cyclic citrullinated peptide as well as antibody to mutated citrullinated vimentin, “which may detect approximately 10%-15%” of patients who test negative to the first two.

Quest’s ANA test with reflex costs $112, and its RA panel costs $110, price points that are similar across other companies’ offerings. Labcorp declined to respond to questions about its DTC tests, and several smaller companies did not respond to queries about their offerings. It can therefore be hard to assess what’s included or what the quality is of many DTC tests, particularly from smaller, less established companies.
 

Oversight and Quality Control

Anthony Killeen, MD, PhD, president of the Association for Diagnostics & Laboratory Medicine (ADLM) and director of Clinical Laboratories at the University of Minnesota Medical Center in Minneapolis, said via email that the ADLM supports “expanding consumer access to direct-to-consumer laboratory testing services that have demonstrated analytical and clinical validity and clinical utility,” given the importance of individuals learning about their health status and becoming more involved in health decisions. But the ADLM also recommends “that only CLIA-certified laboratories perform direct-to-consumer testing,” he said.

Association for Diagnostics &amp; Laboratory Medicine

“There are direct-to-consumer tests on the market that are not medical-grade laboratory tests and that may be performed in nonaccredited laboratories,” Dr. Killeen said. “We advise consumers to steer clear of such tests.” The ADLM also encourages consumers to “work with qualified healthcare providers when making decisions based off the results they receive from any direct-to-consumer tests” and recommends that DTC test companies “provide consumers with sufficient information and/or access to expert help to assist them in ordering tests and interpreting the results.”

Yet it’s unclear how much support, if any, consumers can receive in terms of understanding what their tests mean. Most of the companies in the 2023 study offered optional follow-up with a healthcare professional, but these professionals ranged from physicians to “health coaches,” and all the companies had disclaimers that “test results did not constitute medical advice.”

At Quest, the only company to respond to this news organization’s request for comment, consumer-initiated tests ordered online are first reviewed by a physician at PWNHealth, an independent, third-party physician network, to determine that it’s appropriate before the lab order is actually placed.

“Once results are available, individuals have the option to discuss their results with an independent physician at no extra cost,” Dr. Faix said. ANA or RA results outside the normal ranges may trigger a “call from a PWNHealth healthcare coordinator, who can help provide information, suggestions on next steps, and set up time for the individual to speak with an independent physician to discuss questions or concerns regarding the results,” he said.

“Our goal is not to replace the role of a healthcare provider,” Dr. Faix said. “We are providing an alternate way for people to engage with the healthcare system that offers convenience, gives people more control over their own healthcare journeys, and meets them where they are, supporting both consumers and their care teams.” The company has expanded its offerings from an initial 30 tests made available in 2018 to over 130 today, deciding which to offer “based on consumer research and expertise of clinical experts.” The company has also “seen steady interest in our two consumer rheumatology offerings,” Dr. Faix said.
 

The DTC Landscape in Rheumatology

Within rheumatology, among the most popular tests is for ANA, based on the experience of Alfred Kim, MD, PhD, associate professor of medicine at Washington University School of Medicine in St Louis, Missouri.

Dr. Kim

“For a lot of people, losing control over their health is maybe the most frightening experience they can have, so I think a lot of patients use this as a way to kind of have ownership over their health,” Dr. Kim said. “Let’s say they’ve been to four doctors. No one can explain what’s going on. They’re getting frustrated, and so they just turn to solutions where they feel like they have ownership over the situation.”

Though the market is undoubtedly growing, the growth appears uneven across geography and institution types. Kim has seen a “fair number of referrals,” with patients coming in with results from a DTC test. Michael Putman, MD, MSci, assistant professor of medicine at the Medical College of Wisconsin in Milwaukee, hasn’t seen it much. “I know that patients can get testing done themselves independently, but I don’t have people routinely coming in with tests they’ve ordered in advance of our appointment,” Dr. Putman said, but, like Dr. Kim, he recognizes why patients might seek them out.

Dr. Putman

Ron Hester Photography

Risks vs Benefits

DTC testing is not an answer to the national shortage of rheumatologists, however, especially given the risks that Dr. Niewold, Dr. Putman, and Dr. Kim worry outweigh potential benefits. On the one hand, getting online test results may help expedite a referral to a specialist, Dr. Niewold said. But a long wait for that appointment could then easily become a bigger source of anxiety than comfort, Dr. Putman said.

“It’s a trade-off where you are accepting a lot more people getting false-positive diagnoses and spending months thinking they have some disease where they might not, in exchange for a couple people who would have had a delayed diagnosis,” Dr. Putman said. “There’s an enormous amount of existential suffering,” that’s familiar to rheumatologists because some patients may dread the diagnosis of a rheumatic disease the way they might fear a cancer diagnosis, especially if they have lost a family member to a condition that they suspect they share, he said. “To put yourself into an existential catastrophe — that’s not a small harm.”

Dr. Niewold agreed, pointing out that patients with a positive ANA test may “get unnecessarily worried and stay up all night reading about lupus, getting scared for weeks on end before seeing a specialist.” And there are financial harms as well for patients who may order the same test multiple times, or a whole slate of tests, that they don’t need for hundreds or thousands of dollars. There’s also the lost time and effort of researching a condition or even seeking out support groups that patients may pursue, Dr. Niewold said.

The likely biggest risk to individuals, however, is the potential for overdiagnosis or misdiagnosis.

“If someone comes in and they’ve read the textbook on lupus and they have a positive ANA, it’s really hard as a rheumatologist to walk that back,” Dr. Putman said. “The human mind is a powerful thing,” he added, and people who get a positive test will likely start to notice things like joint pain or a rash on their cheeks and begin attributing it to a diagnosis they risk convincing themselves they have. “When people come into your clinic not knowing what a disease would look like and they just tell you how they’re feeling, it’s a much cleaner and more honest way to approach diagnosis.”

Most patients likely don’t realize, for example, that none of the tests rheumatologists usually order are diagnostic in and of themselves, Dr. Niewold said. “They’re all kind of like stars in the constellation of a diagnosis,” he said. “They’re helpful, but none of them is sufficient by itself.”

Dr. Killeen agreed, noting that “consumers might not understand the nuances of these tests well enough to know whether it is appropriate to order them or how to interpret the results correctly.” Given the long-term implications of a diagnosis for a rheumatologic disease, “I would have concerns about consumers ordering and interpreting rheumatologic tests without working closely with their physicians,” Dr. Killeen said. “The main concern that lab experts have about direct-to-consumer tests is the potential for people to get misleading results and/or to misinterpret their results, which in turn could lead to people not getting the treatment they need or getting treatment when they don’t need any at all.”

It’s one thing for patients to come in asking for a particular treatment they may not need but which a doctor may be able to dissuade them from seeking. But Dr. Kim also pointed out the risk that patients may decide to treat themselves with therapies that haven’t undergone rigorous testing or haven’t been recommended by a physician.

“We tend to have people who come in with a pretty clear idea of what they want done, but the problem is, we don’t know if their reasoning is correct from a clinical perspective,” Dr. Kim said. Companies offer these tests with the belief that they’re “providing patients a choice, an option to take ownership,” he said, “but the potential harm can be realized very quickly because there are going to be people who are misdiagnosing themselves and, worse yet, may then pursue their own treatment plan that’s going in the opposite direction of where we think it needs to go.”

Or, on the flip side, if a patient erroneously believes they have the answer to what ails them, it may delay diagnosis of a more serious condition that’s rarer or harder to detect. Kim pointed to, for example, intravascular lymphoma, which is notoriously as difficult to identify as it is rare and aggressive. If a patient’s confirmation bias has led them to believe they have an autoimmune condition, they may not receive the more serious diagnosis until it’s advanced too far to treat.
 

Patient-Provider Relationship Friction

Another concern is how these tests may lead to confusion and frustration that can erode the patient-provider relationship, particularly because most patients don’t know how to interpret the results or understand the bigger context in which the results have to be interpreted. Many patients may think a test can come back with a binary answer, a positive or negative, and that means they do or don’t have a condition. That’s generally true for pregnancy tests, COVID tests, and sexually transmitted infection tests — the kinds of tests that have long been available to consumers and which have fairly straightforward answers.

But physicians know that’s not the case for many conditions, particularly those in rheumatology.

“In rheumatic diseases, because the tests have such marginal value in terms of diagnosis, almost always we develop a suspicion before we even think about ordering the tests, and then that dictates whether or not we cross that threshold,” Dr. Kim said. “A negative test doesn’t exclude the fact that you may have disease X, but a positive test also doesn’t mean you have disease X. All they provide is an idea of the risk.”

But some patients who come in with DTC test results have “already made the decision in their mind that they have a certain condition,” Dr. Kim said. “This is obviously dangerous because the majority of these patients do not have the condition they think they have, and it leaves a very uncomfortable feeling after the visit because they feel like they’ve been either betrayed by me or by the test, and they leave more confused.”

Patients may also come in with tests that a doctor isn’t familiar with or isn’t sure how to interpret on its own, at least for that particular patient.

“For ANA testing, we have a pretty good idea of its positive and negative predictive value because it’s ordered so much, but for many of these tests being offered, there are specific autoantibodies, and we tend to only get them in people where there’s a clinical suspicion,” Dr. Kim said. “Within that very specific context, we kind of understand what that value means, but if you give it to the general public, then those numbers aren’t as applicable and most likely overestimate the risk of disease.”

Even if providers consider the results of a DTC test in their differential, they may want to be sure it’s from a trustworthy source. “If a provider is uncertain about whether a direct-to-consumer testing company is reputable or about whether a direct-to-consumer test result is reliable, I would encourage them to consult with their laboratory medicine colleagues,” Dr. Killeen said.
 

Responding to Patients

Like any other patient coming to a clinical visit, the most common reason patients are likely ordering these tests is that they’re seeking answers. Kim doesn’t typically see patients doing their own monitoring for diagnosed conditions between visits — the expense would add up too quickly — or testing for genetic markers, which likely wouldn’t be very helpful either.

“Even though most of our diseases probably have a genetic underpinning, how much it contributes is always unclear,” Dr. Kim said. Even conditions with clear genetic variants, such as familial Mediterranean fever, spondyloarthritis, and Behçet disease, can only support a diagnosis, not diagnose it on its own, Dr. Killeen said. And these are not among the tests currently available on most DTC company sites.

While there are also tests that can offer information about genetic risks for certain medications, such as a thiopurine methyltransferase test to find out if a patient lacks the enzyme needed to break down the immunosuppressant drug azathioprine, Kim hasn’t seen patients seeking these out either.

“The more global and more compassionate way to think about this is that we have a lot of people who are struggling to understand what’s going on with their bodies, and most physicians really don’t know what the next steps are for these people,” Dr. Kim said. “They’re desperate, and their quality of life is so poor that they’re going to take extreme steps to try to manage their own frustration with this condition.”

That means clinicians’ most powerful tools when patients come in with DTC test results are their listening skills.

“Empathy is the most important thing, just being able to share the patient’s frustration to the point where they had to take matters into their own hands,” Dr. Kim said. “I think a lot of rheumatologists are actually pretty comfortable being in this position.”

Additionally, doctors should know that some patients may be engaging in attempts to self-diagnose, self-treat, or otherwise self-manage their symptoms or perceived condition. “They just need to be aware and try to make sure there’s no harm being done,” Dr. Kim said.

Ms. Welsh didn’t seek treatment or diagnosis on her own, but getting her test also did not give her the control she was seeking. “Looking back, it was kind of a waste of money, but it felt good in the moment,” Ms. Welsh said. “I was so upset, and I wanted that control, and in the end, it didn’t get me results any sooner, and it didn’t give me peace of mind.”

It was Ms. Welsh’s primary care doctor listening to her concerns, ordering the same test she had ordered with several others, and working with her to seek answers that reassured her that her provider cared about her well-being.

“A lot of what I do in my business is reassure people that you know what they have is treatable or not going to end their life as they know it,” Dr. Putman said. “And you certainly can’t reassure them if they’re not in your clinic yet.”

Dr. Putman has participated in clinical trials with AbbVie, consulting with Novartis and GSK, and clinical trials and consulting with Amgen and AstraZeneca. Dr. Niewold reported receiving research grants from EMD Serono and Zenas BioPharma and consulting for Thermo Fisher Scientific, Progentec Diagnostics, Roivant Sciences, AstraZeneca, S3 Connected Health, Flagship Pioneering, and Guidepoint. Dr. Kim reported sponsored research agreements with AstraZeneca, Bristol-Myers Squibb, Novartis, and CRISPR Therapeutics; royalties from Kypha; and consulting/speaking for Amgen, ANI Pharmaceuticals, Atara Biotherapeutics, Aurinia Pharmaceuticals, CARGO Therapeutics, Exagen Diagnostics, GSK, Hinge Bio, Kypha, Progentec Diagnostics, Synthekine, and UpToDate. Dr. Killeen had no relevant financial relationships to disclose.
 

A version of this article first appeared on Medscape.com.

When Jennifer Welsh, a 40-year-old from New Britain, Connecticut, visited her doctor about pain in her joints and neck, her doctor sent her to the emergency department (ED) to rule out meningitis. The ED did rule that out, as well as strep, so Ms. Welsh went to her follow-up appointment a few days later, hoping for answers or at least more tests to get those answers.

Instead, the doctor — a different one from the same practice as her primary care physician (PCP) — wouldn’t even talk to Ms. Welsh about her symptoms because she couldn’t see the ED’s results and refused to view the results that Ms. Welsh could pull up online.

“She just completely shut me down,” Ms. Welsh recalled. “It was a really awful appointment, and I left in tears. I was in physical pain, I had just been to the ER, nothing is really resolved, I’m stressed out about it, and this woman is completely dismissing me.”

She had been able to schedule an appointment with her regular PCP later that week, but after the harrowing experience with this doctor, she wondered if her PCP would order the rheumatoid arthritis (RA) test that Ms. Welsh suspected she needed. So, she took matters into her own hands.

“I was searching for what test to ask for from my doctor,” she said, and she found that she could order it on her own from a major lab company she was already familiar with. For around $100, “I could get it done and see what it says on my own,” she said.

But that’s not how it worked out. Her regular PCP apologized for the other doctor’s behavior and ordered the RA test as well as additional tests — and got results while Ms. Welsh still waited for the one she ordered to arrive over a week later.

At first, Ms. Welsh was grateful she could order the RA test without her doctor’s referral. “I felt it gave me a sense of control over the situation that I felt really not in control of, until the system failed me, and I didn’t get the results,” she said. But then, “not having someone I could call and get an answer about why my tests were delayed, why I wasn’t able to access them, why it was taking so long — it was definitely anxiety-inducing.”
 

A Growing Market

Ms. Welsh is one of a growing number of patients who are ordering direct-to-consumer (DTC) lab tests without the recommendation or guidance of a doctor. They’re offered online by labs ranging from well-established giants like Quest and Labcorp to smaller, potentially less vetted companies, although some smaller companies contract with larger companies like Quest. Combined, the DTC market is projected to be worth $2 billion by 2025.

Yet the burgeoning industry has also drawn critiques from both bioethicists and privacy experts. A research letter in JAMA in 2023, for example, found that less than half of the 21 companies identified in an online search declared Health Insurance Portability and Accountability Act compliance, while more than half “indicated the potential use of consumer data for research purposes either internally or through third-party sharing.” That study found the most commonly offered tests were related to diabetes, the thyroid, and vitamin levels, and hormone tests for men and women, such as testosterone or estradiol.

But a number of companies also offer tests related to rheumatologic conditions. A handful of tests offered by Labcorp, for example, could be used in rheumatology, such as tests for celiac antibodies or high-sensitivity C-reactive protein. Quest similarly offers a handful of autoimmune-related tests. But other companies offer a long slate of autoimmune or antibody tests.

The antinuclear antibody (ANA) test and RA panel offered by Quest are the same tests, run and analyzed in the same labs, as those ordered by physicians and hospitals, according to James Faix, MD, the medical director of immunology at Quest Diagnostics. Their RA panel includes rheumatoid factor and anti-cyclic citrullinated peptide as well as antibody to mutated citrullinated vimentin, “which may detect approximately 10%-15%” of patients who test negative to the first two.

Quest’s ANA test with reflex costs $112, and its RA panel costs $110, price points that are similar across other companies’ offerings. Labcorp declined to respond to questions about its DTC tests, and several smaller companies did not respond to queries about their offerings. It can therefore be hard to assess what’s included or what the quality is of many DTC tests, particularly from smaller, less established companies.
 

Oversight and Quality Control

Anthony Killeen, MD, PhD, president of the Association for Diagnostics & Laboratory Medicine (ADLM) and director of Clinical Laboratories at the University of Minnesota Medical Center in Minneapolis, said via email that the ADLM supports “expanding consumer access to direct-to-consumer laboratory testing services that have demonstrated analytical and clinical validity and clinical utility,” given the importance of individuals learning about their health status and becoming more involved in health decisions. But the ADLM also recommends “that only CLIA-certified laboratories perform direct-to-consumer testing,” he said.

Association for Diagnostics &amp; Laboratory Medicine

“There are direct-to-consumer tests on the market that are not medical-grade laboratory tests and that may be performed in nonaccredited laboratories,” Dr. Killeen said. “We advise consumers to steer clear of such tests.” The ADLM also encourages consumers to “work with qualified healthcare providers when making decisions based off the results they receive from any direct-to-consumer tests” and recommends that DTC test companies “provide consumers with sufficient information and/or access to expert help to assist them in ordering tests and interpreting the results.”

Yet it’s unclear how much support, if any, consumers can receive in terms of understanding what their tests mean. Most of the companies in the 2023 study offered optional follow-up with a healthcare professional, but these professionals ranged from physicians to “health coaches,” and all the companies had disclaimers that “test results did not constitute medical advice.”

At Quest, the only company to respond to this news organization’s request for comment, consumer-initiated tests ordered online are first reviewed by a physician at PWNHealth, an independent, third-party physician network, to determine that it’s appropriate before the lab order is actually placed.

“Once results are available, individuals have the option to discuss their results with an independent physician at no extra cost,” Dr. Faix said. ANA or RA results outside the normal ranges may trigger a “call from a PWNHealth healthcare coordinator, who can help provide information, suggestions on next steps, and set up time for the individual to speak with an independent physician to discuss questions or concerns regarding the results,” he said.

“Our goal is not to replace the role of a healthcare provider,” Dr. Faix said. “We are providing an alternate way for people to engage with the healthcare system that offers convenience, gives people more control over their own healthcare journeys, and meets them where they are, supporting both consumers and their care teams.” The company has expanded its offerings from an initial 30 tests made available in 2018 to over 130 today, deciding which to offer “based on consumer research and expertise of clinical experts.” The company has also “seen steady interest in our two consumer rheumatology offerings,” Dr. Faix said.
 

The DTC Landscape in Rheumatology

Within rheumatology, among the most popular tests is for ANA, based on the experience of Alfred Kim, MD, PhD, associate professor of medicine at Washington University School of Medicine in St Louis, Missouri.

Dr. Kim

“For a lot of people, losing control over their health is maybe the most frightening experience they can have, so I think a lot of patients use this as a way to kind of have ownership over their health,” Dr. Kim said. “Let’s say they’ve been to four doctors. No one can explain what’s going on. They’re getting frustrated, and so they just turn to solutions where they feel like they have ownership over the situation.”

Though the market is undoubtedly growing, the growth appears uneven across geography and institution types. Kim has seen a “fair number of referrals,” with patients coming in with results from a DTC test. Michael Putman, MD, MSci, assistant professor of medicine at the Medical College of Wisconsin in Milwaukee, hasn’t seen it much. “I know that patients can get testing done themselves independently, but I don’t have people routinely coming in with tests they’ve ordered in advance of our appointment,” Dr. Putman said, but, like Dr. Kim, he recognizes why patients might seek them out.

Dr. Putman

Ron Hester Photography

Risks vs Benefits

DTC testing is not an answer to the national shortage of rheumatologists, however, especially given the risks that Dr. Niewold, Dr. Putman, and Dr. Kim worry outweigh potential benefits. On the one hand, getting online test results may help expedite a referral to a specialist, Dr. Niewold said. But a long wait for that appointment could then easily become a bigger source of anxiety than comfort, Dr. Putman said.

“It’s a trade-off where you are accepting a lot more people getting false-positive diagnoses and spending months thinking they have some disease where they might not, in exchange for a couple people who would have had a delayed diagnosis,” Dr. Putman said. “There’s an enormous amount of existential suffering,” that’s familiar to rheumatologists because some patients may dread the diagnosis of a rheumatic disease the way they might fear a cancer diagnosis, especially if they have lost a family member to a condition that they suspect they share, he said. “To put yourself into an existential catastrophe — that’s not a small harm.”

Dr. Niewold agreed, pointing out that patients with a positive ANA test may “get unnecessarily worried and stay up all night reading about lupus, getting scared for weeks on end before seeing a specialist.” And there are financial harms as well for patients who may order the same test multiple times, or a whole slate of tests, that they don’t need for hundreds or thousands of dollars. There’s also the lost time and effort of researching a condition or even seeking out support groups that patients may pursue, Dr. Niewold said.

The likely biggest risk to individuals, however, is the potential for overdiagnosis or misdiagnosis.

“If someone comes in and they’ve read the textbook on lupus and they have a positive ANA, it’s really hard as a rheumatologist to walk that back,” Dr. Putman said. “The human mind is a powerful thing,” he added, and people who get a positive test will likely start to notice things like joint pain or a rash on their cheeks and begin attributing it to a diagnosis they risk convincing themselves they have. “When people come into your clinic not knowing what a disease would look like and they just tell you how they’re feeling, it’s a much cleaner and more honest way to approach diagnosis.”

Most patients likely don’t realize, for example, that none of the tests rheumatologists usually order are diagnostic in and of themselves, Dr. Niewold said. “They’re all kind of like stars in the constellation of a diagnosis,” he said. “They’re helpful, but none of them is sufficient by itself.”

Dr. Killeen agreed, noting that “consumers might not understand the nuances of these tests well enough to know whether it is appropriate to order them or how to interpret the results correctly.” Given the long-term implications of a diagnosis for a rheumatologic disease, “I would have concerns about consumers ordering and interpreting rheumatologic tests without working closely with their physicians,” Dr. Killeen said. “The main concern that lab experts have about direct-to-consumer tests is the potential for people to get misleading results and/or to misinterpret their results, which in turn could lead to people not getting the treatment they need or getting treatment when they don’t need any at all.”

It’s one thing for patients to come in asking for a particular treatment they may not need but which a doctor may be able to dissuade them from seeking. But Dr. Kim also pointed out the risk that patients may decide to treat themselves with therapies that haven’t undergone rigorous testing or haven’t been recommended by a physician.

“We tend to have people who come in with a pretty clear idea of what they want done, but the problem is, we don’t know if their reasoning is correct from a clinical perspective,” Dr. Kim said. Companies offer these tests with the belief that they’re “providing patients a choice, an option to take ownership,” he said, “but the potential harm can be realized very quickly because there are going to be people who are misdiagnosing themselves and, worse yet, may then pursue their own treatment plan that’s going in the opposite direction of where we think it needs to go.”

Or, on the flip side, if a patient erroneously believes they have the answer to what ails them, it may delay diagnosis of a more serious condition that’s rarer or harder to detect. Kim pointed to, for example, intravascular lymphoma, which is notoriously as difficult to identify as it is rare and aggressive. If a patient’s confirmation bias has led them to believe they have an autoimmune condition, they may not receive the more serious diagnosis until it’s advanced too far to treat.
 

Patient-Provider Relationship Friction

Another concern is how these tests may lead to confusion and frustration that can erode the patient-provider relationship, particularly because most patients don’t know how to interpret the results or understand the bigger context in which the results have to be interpreted. Many patients may think a test can come back with a binary answer, a positive or negative, and that means they do or don’t have a condition. That’s generally true for pregnancy tests, COVID tests, and sexually transmitted infection tests — the kinds of tests that have long been available to consumers and which have fairly straightforward answers.

But physicians know that’s not the case for many conditions, particularly those in rheumatology.

“In rheumatic diseases, because the tests have such marginal value in terms of diagnosis, almost always we develop a suspicion before we even think about ordering the tests, and then that dictates whether or not we cross that threshold,” Dr. Kim said. “A negative test doesn’t exclude the fact that you may have disease X, but a positive test also doesn’t mean you have disease X. All they provide is an idea of the risk.”

But some patients who come in with DTC test results have “already made the decision in their mind that they have a certain condition,” Dr. Kim said. “This is obviously dangerous because the majority of these patients do not have the condition they think they have, and it leaves a very uncomfortable feeling after the visit because they feel like they’ve been either betrayed by me or by the test, and they leave more confused.”

Patients may also come in with tests that a doctor isn’t familiar with or isn’t sure how to interpret on its own, at least for that particular patient.

“For ANA testing, we have a pretty good idea of its positive and negative predictive value because it’s ordered so much, but for many of these tests being offered, there are specific autoantibodies, and we tend to only get them in people where there’s a clinical suspicion,” Dr. Kim said. “Within that very specific context, we kind of understand what that value means, but if you give it to the general public, then those numbers aren’t as applicable and most likely overestimate the risk of disease.”

Even if providers consider the results of a DTC test in their differential, they may want to be sure it’s from a trustworthy source. “If a provider is uncertain about whether a direct-to-consumer testing company is reputable or about whether a direct-to-consumer test result is reliable, I would encourage them to consult with their laboratory medicine colleagues,” Dr. Killeen said.
 

Responding to Patients

Like any other patient coming to a clinical visit, the most common reason patients are likely ordering these tests is that they’re seeking answers. Kim doesn’t typically see patients doing their own monitoring for diagnosed conditions between visits — the expense would add up too quickly — or testing for genetic markers, which likely wouldn’t be very helpful either.

“Even though most of our diseases probably have a genetic underpinning, how much it contributes is always unclear,” Dr. Kim said. Even conditions with clear genetic variants, such as familial Mediterranean fever, spondyloarthritis, and Behçet disease, can only support a diagnosis, not diagnose it on its own, Dr. Killeen said. And these are not among the tests currently available on most DTC company sites.

While there are also tests that can offer information about genetic risks for certain medications, such as a thiopurine methyltransferase test to find out if a patient lacks the enzyme needed to break down the immunosuppressant drug azathioprine, Kim hasn’t seen patients seeking these out either.

“The more global and more compassionate way to think about this is that we have a lot of people who are struggling to understand what’s going on with their bodies, and most physicians really don’t know what the next steps are for these people,” Dr. Kim said. “They’re desperate, and their quality of life is so poor that they’re going to take extreme steps to try to manage their own frustration with this condition.”

That means clinicians’ most powerful tools when patients come in with DTC test results are their listening skills.

“Empathy is the most important thing, just being able to share the patient’s frustration to the point where they had to take matters into their own hands,” Dr. Kim said. “I think a lot of rheumatologists are actually pretty comfortable being in this position.”

Additionally, doctors should know that some patients may be engaging in attempts to self-diagnose, self-treat, or otherwise self-manage their symptoms or perceived condition. “They just need to be aware and try to make sure there’s no harm being done,” Dr. Kim said.

Ms. Welsh didn’t seek treatment or diagnosis on her own, but getting her test also did not give her the control she was seeking. “Looking back, it was kind of a waste of money, but it felt good in the moment,” Ms. Welsh said. “I was so upset, and I wanted that control, and in the end, it didn’t get me results any sooner, and it didn’t give me peace of mind.”

It was Ms. Welsh’s primary care doctor listening to her concerns, ordering the same test she had ordered with several others, and working with her to seek answers that reassured her that her provider cared about her well-being.

“A lot of what I do in my business is reassure people that you know what they have is treatable or not going to end their life as they know it,” Dr. Putman said. “And you certainly can’t reassure them if they’re not in your clinic yet.”

Dr. Putman has participated in clinical trials with AbbVie, consulting with Novartis and GSK, and clinical trials and consulting with Amgen and AstraZeneca. Dr. Niewold reported receiving research grants from EMD Serono and Zenas BioPharma and consulting for Thermo Fisher Scientific, Progentec Diagnostics, Roivant Sciences, AstraZeneca, S3 Connected Health, Flagship Pioneering, and Guidepoint. Dr. Kim reported sponsored research agreements with AstraZeneca, Bristol-Myers Squibb, Novartis, and CRISPR Therapeutics; royalties from Kypha; and consulting/speaking for Amgen, ANI Pharmaceuticals, Atara Biotherapeutics, Aurinia Pharmaceuticals, CARGO Therapeutics, Exagen Diagnostics, GSK, Hinge Bio, Kypha, Progentec Diagnostics, Synthekine, and UpToDate. Dr. Killeen had no relevant financial relationships to disclose.
 

A version of this article first appeared on Medscape.com.

When Jennifer Welsh, a 40-year-old from New Britain, Connecticut, visited her doctor about pain in her joints and neck, her doctor sent her to the emergency department (ED) to rule out meningitis. The ED did rule that out, as well as strep, so Ms. Welsh went to her follow-up appointment a few days later, hoping for answers or at least more tests to get those answers.

Instead, the doctor — a different one from the same practice as her primary care physician (PCP) — wouldn’t even talk to Ms. Welsh about her symptoms because she couldn’t see the ED’s results and refused to view the results that Ms. Welsh could pull up online.

“She just completely shut me down,” Ms. Welsh recalled. “It was a really awful appointment, and I left in tears. I was in physical pain, I had just been to the ER, nothing is really resolved, I’m stressed out about it, and this woman is completely dismissing me.”

She had been able to schedule an appointment with her regular PCP later that week, but after the harrowing experience with this doctor, she wondered if her PCP would order the rheumatoid arthritis (RA) test that Ms. Welsh suspected she needed. So, she took matters into her own hands.

“I was searching for what test to ask for from my doctor,” she said, and she found that she could order it on her own from a major lab company she was already familiar with. For around $100, “I could get it done and see what it says on my own,” she said.

But that’s not how it worked out. Her regular PCP apologized for the other doctor’s behavior and ordered the RA test as well as additional tests — and got results while Ms. Welsh still waited for the one she ordered to arrive over a week later.

At first, Ms. Welsh was grateful she could order the RA test without her doctor’s referral. “I felt it gave me a sense of control over the situation that I felt really not in control of, until the system failed me, and I didn’t get the results,” she said. But then, “not having someone I could call and get an answer about why my tests were delayed, why I wasn’t able to access them, why it was taking so long — it was definitely anxiety-inducing.”
 

A Growing Market

Ms. Welsh is one of a growing number of patients who are ordering direct-to-consumer (DTC) lab tests without the recommendation or guidance of a doctor. They’re offered online by labs ranging from well-established giants like Quest and Labcorp to smaller, potentially less vetted companies, although some smaller companies contract with larger companies like Quest. Combined, the DTC market is projected to be worth $2 billion by 2025.

Yet the burgeoning industry has also drawn critiques from both bioethicists and privacy experts. A research letter in JAMA in 2023, for example, found that less than half of the 21 companies identified in an online search declared Health Insurance Portability and Accountability Act compliance, while more than half “indicated the potential use of consumer data for research purposes either internally or through third-party sharing.” That study found the most commonly offered tests were related to diabetes, the thyroid, and vitamin levels, and hormone tests for men and women, such as testosterone or estradiol.

But a number of companies also offer tests related to rheumatologic conditions. A handful of tests offered by Labcorp, for example, could be used in rheumatology, such as tests for celiac antibodies or high-sensitivity C-reactive protein. Quest similarly offers a handful of autoimmune-related tests. But other companies offer a long slate of autoimmune or antibody tests.

The antinuclear antibody (ANA) test and RA panel offered by Quest are the same tests, run and analyzed in the same labs, as those ordered by physicians and hospitals, according to James Faix, MD, the medical director of immunology at Quest Diagnostics. Their RA panel includes rheumatoid factor and anti-cyclic citrullinated peptide as well as antibody to mutated citrullinated vimentin, “which may detect approximately 10%-15%” of patients who test negative to the first two.

Quest’s ANA test with reflex costs $112, and its RA panel costs $110, price points that are similar across other companies’ offerings. Labcorp declined to respond to questions about its DTC tests, and several smaller companies did not respond to queries about their offerings. It can therefore be hard to assess what’s included or what the quality is of many DTC tests, particularly from smaller, less established companies.
 

Oversight and Quality Control

Anthony Killeen, MD, PhD, president of the Association for Diagnostics & Laboratory Medicine (ADLM) and director of Clinical Laboratories at the University of Minnesota Medical Center in Minneapolis, said via email that the ADLM supports “expanding consumer access to direct-to-consumer laboratory testing services that have demonstrated analytical and clinical validity and clinical utility,” given the importance of individuals learning about their health status and becoming more involved in health decisions. But the ADLM also recommends “that only CLIA-certified laboratories perform direct-to-consumer testing,” he said.

Association for Diagnostics &amp; Laboratory Medicine

“There are direct-to-consumer tests on the market that are not medical-grade laboratory tests and that may be performed in nonaccredited laboratories,” Dr. Killeen said. “We advise consumers to steer clear of such tests.” The ADLM also encourages consumers to “work with qualified healthcare providers when making decisions based off the results they receive from any direct-to-consumer tests” and recommends that DTC test companies “provide consumers with sufficient information and/or access to expert help to assist them in ordering tests and interpreting the results.”

Yet it’s unclear how much support, if any, consumers can receive in terms of understanding what their tests mean. Most of the companies in the 2023 study offered optional follow-up with a healthcare professional, but these professionals ranged from physicians to “health coaches,” and all the companies had disclaimers that “test results did not constitute medical advice.”

At Quest, the only company to respond to this news organization’s request for comment, consumer-initiated tests ordered online are first reviewed by a physician at PWNHealth, an independent, third-party physician network, to determine that it’s appropriate before the lab order is actually placed.

“Once results are available, individuals have the option to discuss their results with an independent physician at no extra cost,” Dr. Faix said. ANA or RA results outside the normal ranges may trigger a “call from a PWNHealth healthcare coordinator, who can help provide information, suggestions on next steps, and set up time for the individual to speak with an independent physician to discuss questions or concerns regarding the results,” he said.

“Our goal is not to replace the role of a healthcare provider,” Dr. Faix said. “We are providing an alternate way for people to engage with the healthcare system that offers convenience, gives people more control over their own healthcare journeys, and meets them where they are, supporting both consumers and their care teams.” The company has expanded its offerings from an initial 30 tests made available in 2018 to over 130 today, deciding which to offer “based on consumer research and expertise of clinical experts.” The company has also “seen steady interest in our two consumer rheumatology offerings,” Dr. Faix said.
 

The DTC Landscape in Rheumatology

Within rheumatology, among the most popular tests is for ANA, based on the experience of Alfred Kim, MD, PhD, associate professor of medicine at Washington University School of Medicine in St Louis, Missouri.

Dr. Kim

“For a lot of people, losing control over their health is maybe the most frightening experience they can have, so I think a lot of patients use this as a way to kind of have ownership over their health,” Dr. Kim said. “Let’s say they’ve been to four doctors. No one can explain what’s going on. They’re getting frustrated, and so they just turn to solutions where they feel like they have ownership over the situation.”

Though the market is undoubtedly growing, the growth appears uneven across geography and institution types. Kim has seen a “fair number of referrals,” with patients coming in with results from a DTC test. Michael Putman, MD, MSci, assistant professor of medicine at the Medical College of Wisconsin in Milwaukee, hasn’t seen it much. “I know that patients can get testing done themselves independently, but I don’t have people routinely coming in with tests they’ve ordered in advance of our appointment,” Dr. Putman said, but, like Dr. Kim, he recognizes why patients might seek them out.

Dr. Putman

Ron Hester Photography

Risks vs Benefits

DTC testing is not an answer to the national shortage of rheumatologists, however, especially given the risks that Dr. Niewold, Dr. Putman, and Dr. Kim worry outweigh potential benefits. On the one hand, getting online test results may help expedite a referral to a specialist, Dr. Niewold said. But a long wait for that appointment could then easily become a bigger source of anxiety than comfort, Dr. Putman said.

“It’s a trade-off where you are accepting a lot more people getting false-positive diagnoses and spending months thinking they have some disease where they might not, in exchange for a couple people who would have had a delayed diagnosis,” Dr. Putman said. “There’s an enormous amount of existential suffering,” that’s familiar to rheumatologists because some patients may dread the diagnosis of a rheumatic disease the way they might fear a cancer diagnosis, especially if they have lost a family member to a condition that they suspect they share, he said. “To put yourself into an existential catastrophe — that’s not a small harm.”

Dr. Niewold agreed, pointing out that patients with a positive ANA test may “get unnecessarily worried and stay up all night reading about lupus, getting scared for weeks on end before seeing a specialist.” And there are financial harms as well for patients who may order the same test multiple times, or a whole slate of tests, that they don’t need for hundreds or thousands of dollars. There’s also the lost time and effort of researching a condition or even seeking out support groups that patients may pursue, Dr. Niewold said.

The likely biggest risk to individuals, however, is the potential for overdiagnosis or misdiagnosis.

“If someone comes in and they’ve read the textbook on lupus and they have a positive ANA, it’s really hard as a rheumatologist to walk that back,” Dr. Putman said. “The human mind is a powerful thing,” he added, and people who get a positive test will likely start to notice things like joint pain or a rash on their cheeks and begin attributing it to a diagnosis they risk convincing themselves they have. “When people come into your clinic not knowing what a disease would look like and they just tell you how they’re feeling, it’s a much cleaner and more honest way to approach diagnosis.”

Most patients likely don’t realize, for example, that none of the tests rheumatologists usually order are diagnostic in and of themselves, Dr. Niewold said. “They’re all kind of like stars in the constellation of a diagnosis,” he said. “They’re helpful, but none of them is sufficient by itself.”

Dr. Killeen agreed, noting that “consumers might not understand the nuances of these tests well enough to know whether it is appropriate to order them or how to interpret the results correctly.” Given the long-term implications of a diagnosis for a rheumatologic disease, “I would have concerns about consumers ordering and interpreting rheumatologic tests without working closely with their physicians,” Dr. Killeen said. “The main concern that lab experts have about direct-to-consumer tests is the potential for people to get misleading results and/or to misinterpret their results, which in turn could lead to people not getting the treatment they need or getting treatment when they don’t need any at all.”

It’s one thing for patients to come in asking for a particular treatment they may not need but which a doctor may be able to dissuade them from seeking. But Dr. Kim also pointed out the risk that patients may decide to treat themselves with therapies that haven’t undergone rigorous testing or haven’t been recommended by a physician.

“We tend to have people who come in with a pretty clear idea of what they want done, but the problem is, we don’t know if their reasoning is correct from a clinical perspective,” Dr. Kim said. Companies offer these tests with the belief that they’re “providing patients a choice, an option to take ownership,” he said, “but the potential harm can be realized very quickly because there are going to be people who are misdiagnosing themselves and, worse yet, may then pursue their own treatment plan that’s going in the opposite direction of where we think it needs to go.”

Or, on the flip side, if a patient erroneously believes they have the answer to what ails them, it may delay diagnosis of a more serious condition that’s rarer or harder to detect. Kim pointed to, for example, intravascular lymphoma, which is notoriously as difficult to identify as it is rare and aggressive. If a patient’s confirmation bias has led them to believe they have an autoimmune condition, they may not receive the more serious diagnosis until it’s advanced too far to treat.
 

Patient-Provider Relationship Friction

Another concern is how these tests may lead to confusion and frustration that can erode the patient-provider relationship, particularly because most patients don’t know how to interpret the results or understand the bigger context in which the results have to be interpreted. Many patients may think a test can come back with a binary answer, a positive or negative, and that means they do or don’t have a condition. That’s generally true for pregnancy tests, COVID tests, and sexually transmitted infection tests — the kinds of tests that have long been available to consumers and which have fairly straightforward answers.

But physicians know that’s not the case for many conditions, particularly those in rheumatology.

“In rheumatic diseases, because the tests have such marginal value in terms of diagnosis, almost always we develop a suspicion before we even think about ordering the tests, and then that dictates whether or not we cross that threshold,” Dr. Kim said. “A negative test doesn’t exclude the fact that you may have disease X, but a positive test also doesn’t mean you have disease X. All they provide is an idea of the risk.”

But some patients who come in with DTC test results have “already made the decision in their mind that they have a certain condition,” Dr. Kim said. “This is obviously dangerous because the majority of these patients do not have the condition they think they have, and it leaves a very uncomfortable feeling after the visit because they feel like they’ve been either betrayed by me or by the test, and they leave more confused.”

Patients may also come in with tests that a doctor isn’t familiar with or isn’t sure how to interpret on its own, at least for that particular patient.

“For ANA testing, we have a pretty good idea of its positive and negative predictive value because it’s ordered so much, but for many of these tests being offered, there are specific autoantibodies, and we tend to only get them in people where there’s a clinical suspicion,” Dr. Kim said. “Within that very specific context, we kind of understand what that value means, but if you give it to the general public, then those numbers aren’t as applicable and most likely overestimate the risk of disease.”

Even if providers consider the results of a DTC test in their differential, they may want to be sure it’s from a trustworthy source. “If a provider is uncertain about whether a direct-to-consumer testing company is reputable or about whether a direct-to-consumer test result is reliable, I would encourage them to consult with their laboratory medicine colleagues,” Dr. Killeen said.
 

Responding to Patients

Like any other patient coming to a clinical visit, the most common reason patients are likely ordering these tests is that they’re seeking answers. Kim doesn’t typically see patients doing their own monitoring for diagnosed conditions between visits — the expense would add up too quickly — or testing for genetic markers, which likely wouldn’t be very helpful either.

“Even though most of our diseases probably have a genetic underpinning, how much it contributes is always unclear,” Dr. Kim said. Even conditions with clear genetic variants, such as familial Mediterranean fever, spondyloarthritis, and Behçet disease, can only support a diagnosis, not diagnose it on its own, Dr. Killeen said. And these are not among the tests currently available on most DTC company sites.

While there are also tests that can offer information about genetic risks for certain medications, such as a thiopurine methyltransferase test to find out if a patient lacks the enzyme needed to break down the immunosuppressant drug azathioprine, Kim hasn’t seen patients seeking these out either.

“The more global and more compassionate way to think about this is that we have a lot of people who are struggling to understand what’s going on with their bodies, and most physicians really don’t know what the next steps are for these people,” Dr. Kim said. “They’re desperate, and their quality of life is so poor that they’re going to take extreme steps to try to manage their own frustration with this condition.”

That means clinicians’ most powerful tools when patients come in with DTC test results are their listening skills.

“Empathy is the most important thing, just being able to share the patient’s frustration to the point where they had to take matters into their own hands,” Dr. Kim said. “I think a lot of rheumatologists are actually pretty comfortable being in this position.”

Additionally, doctors should know that some patients may be engaging in attempts to self-diagnose, self-treat, or otherwise self-manage their symptoms or perceived condition. “They just need to be aware and try to make sure there’s no harm being done,” Dr. Kim said.

Ms. Welsh didn’t seek treatment or diagnosis on her own, but getting her test also did not give her the control she was seeking. “Looking back, it was kind of a waste of money, but it felt good in the moment,” Ms. Welsh said. “I was so upset, and I wanted that control, and in the end, it didn’t get me results any sooner, and it didn’t give me peace of mind.”

It was Ms. Welsh’s primary care doctor listening to her concerns, ordering the same test she had ordered with several others, and working with her to seek answers that reassured her that her provider cared about her well-being.

“A lot of what I do in my business is reassure people that you know what they have is treatable or not going to end their life as they know it,” Dr. Putman said. “And you certainly can’t reassure them if they’re not in your clinic yet.”

Dr. Putman has participated in clinical trials with AbbVie, consulting with Novartis and GSK, and clinical trials and consulting with Amgen and AstraZeneca. Dr. Niewold reported receiving research grants from EMD Serono and Zenas BioPharma and consulting for Thermo Fisher Scientific, Progentec Diagnostics, Roivant Sciences, AstraZeneca, S3 Connected Health, Flagship Pioneering, and Guidepoint. Dr. Kim reported sponsored research agreements with AstraZeneca, Bristol-Myers Squibb, Novartis, and CRISPR Therapeutics; royalties from Kypha; and consulting/speaking for Amgen, ANI Pharmaceuticals, Atara Biotherapeutics, Aurinia Pharmaceuticals, CARGO Therapeutics, Exagen Diagnostics, GSK, Hinge Bio, Kypha, Progentec Diagnostics, Synthekine, and UpToDate. Dr. Killeen had no relevant financial relationships to disclose.
 

A version of this article first appeared on Medscape.com.

CHICAGO — The nonhormonal investigational drug elinzanetant led to significant improvement in hot flashes as well as sleep disturbance and quality of life, according to data from three randomized controlled trials presented at The Menopause Society 2024 Annual Meeting in Chicago. Two phase 3 trials, OASIS 1 and 2, were also published in JAMA, and the longer-term OASIS 3 trial was presented as a poster at the conference.

Elinzanetant is a selective neurokinin (NK) receptor antagonist, similar to fezolinetant, the first drug in this class approved by the US Food and Drug Administration (FDA) for vasomotor symptoms in May 2023. This class of medications targets the estrogen-sensitive kisspeptin/NK B/dynorphin (KNDy) neurons thought to play a role in thermoregulation and hot flashes during menopause. While fezolinetant targets only the NK-3 receptor, elinzanetant is a dual NK receptor antagonist that targets both NK-1 and NK-3. Bayer submitted a New Drug Application for elinzanetant to the FDA on August 1.

For those in whom hormone therapy is contraindicated, “it’s always been difficult for women with really severe symptoms to have a safe and effective therapy,” lead author JoAnn Pinkerton, MD, a professor of ob.gyn. at the University of Virginia in Charlottesville, Virginia, told this news organization. “The nonhormonal therapies we’ve used mostly off-label — the antidepressants, gabapentin, clonidine, oxybutynin — do help the hot flashes, but they don’t work nearly as effectively as these new NK receptor antagonists, and having one that looks like it might have a broader use for hot flashes, night sweats, mood, and sleep is just really exciting.”

Dr. Pinkerton said approximately 80% of the women in the OASIS 1 and 2 studies had at least a 50% reduction in hot flashes. “It was a very strong, dramatic positive finding, but the improvements in sleep and mood have really encouraged us to go further,” she said.

Declining estrogen levels during and after menopause can cause hypertrophy and hyperactivity of the KNDy neurons, which has been linked to thermoregulation disruptions that may trigger hot flashes, James Simon, MD, a clinical professor of ob.gyn. at The George Washington University School of Medicine & Health Sciences and medical director of IntimMedicine in Washington, DC, told attendees. He presented pooled data from OASIS 1 and 2. The NK-1 receptor, targeted by elinzanetant but not fezolinetant, is also thought to play a role in insomnia and possibly in mood.

“Oftentimes the focus on a lot of these drugs is hot flashes, hot flashes, hot flashes, but we know hot flashes do not occur in isolation,” Chrisandra Shufelt, MD, professor and chair of general internal medicine and associate director of the Women’s Health Research Center at Mayo Clinic in Jacksonville, Florida, told this news organization. Elinzanetant is “an interesting compound because it actually works on sleep, and that was critical because sleep disturbance precedes” many other menopausal symptoms, said Dr. Shufelt, who was not involved in the study.

“I think it is an outstanding option for women who don’t have the opportunity to get hormones,” Dr. Shufelt said, and she was particularly pleased to see there were no safety concerns for the liver in the trial data. The FDA issued a warning on September 12 about the risk for rare liver injury with fezolinetant, but the early signals that had been seen in fezolinetant data were not seen in these elinzanetant data.

The OASIS 1 and 2 trials enrolled postmenopausal women, aged 40-65 years, who had at least 50 moderate to severe vasomotor occurrences per week.

“A moderate hot flash is a hot flash that is also associated with sweating, and a severe hot flash is a moderate hot flash that stops a woman in her tracks,” Dr. Simon said. “Namely, it’s severe enough with sweating and central nervous system effects that she is interrupted in whatever it is that she’s doing at the time.”

Exclusion criteria for the trials included a history of arrhythmias, heart block, or QT prolongation; abnormal lab results; history of malignancy within the past 5 years; uncontrolled or treatment-resistant hypertension, hypothyroidism, or hyperthyroidism; unexplained postmenopausal bleeding; clinically relevant abnormal mammogram findings; or disordered proliferative endometrium, endometrial hyperplasia, polyp, or endometrial cancer.

The predominantly White (80%) women were an average 54 years old, with an average body mass index (BMI) of 27.8, and were an average 3.5 years from their last period. For the first 12 weeks of the trials, 399 women were assigned to receive 120 mg once daily of oral elinzanetant and 397 were assigned to once daily placebo. Then the women taking placebo switched to elinzanetant for the final 14 weeks of the study.

The endpoints included mean change in frequency and severity of vasomotor symptoms at weeks 1, 4, and 12 as well as change in sleep disturbance and quality of life at week 12. Sleep was assessed with the Patient-Reported Outcomes Measurement Information System Sleep Disturbance–Short Form score, which ranges from 28.9 to 76.5, with a higher number denoting greater sleep disturbance. The Menopause-Specific Quality-of-Life score ranges from 1 to 8, with a higher score indicating poorer quality of life.

Daily frequency of vasomotor symptoms was 14 per day at baseline in the elinzanetant group, decreasing by 4.8 per day at week 1, 8 per day at week 4, and 9.4 per day at week 12. In the placebo group, women had an average 15.2 occurrences per day at baseline, which decreased by 3.2 at week 1, 5.2 at week 4, and 6.4 at week 12. Comparing the groups at 12 weeks, those receiving elinzanetant had 3.2 fewer daily vasomotor symptoms than those receiving placebo (P < .0001).

The severity of vasomotor symptoms also improved more in the elinzanetant group than in the placebo group over 12 weeks, after which severity improved further in those who switched from placebo to elinzanetant (P < .0001).

Sleep disturbance scores, starting at a mean 61.5 in the elinzanetant group and 60.5 in the placebo group, fell 10.7 points in the elinzanetant group and 5.3 points in the placebo group at 12 weeks, for a difference of 4.9 points (P < .0001). Sleep then further improved in those who switched from placebo to elinzanetant. Quality-of-life scores improved 1.37 points (from 4.52 at baseline) in the elinzanetant group and 0.96 points (from 4.49 at baseline) in the placebo group, for a mean difference at 12 weeks of 0.36 (P < .0001).

Though no head-to-head data exist comparing elinzanetant and fezolinetant, Dr. Simon told this news organization the side effects with fezolinetant “tend to be gastrointestinal, whereas the side effects for elinzanetant tend to be central nervous system,” such as drowsiness and lethargy.

The women who are the best candidates for elinzanetant, Dr. Pinkerton told this news organization, include those who have had an estrogen-sensitive cancer, such as breast or endometrial cancer, or who have fear of it, a family history, or are otherwise high risk. Other ideal candidates include those with a history of venous thromboembolism, people who have migraine with aura (due to concerns about increased risk for stroke), and those who have endometriosis or large fibroids.

“Then the last group might be women who took hormone therapy in their 50s and want to continue, but they’re trying to go off, and they have a recurrence of their hot flashes or night sweats or sleep issues,” Dr. Pinkerton said. “This might be a great group to switch over.”

OASIS 3 assessed the drug for 1 year and “supported the results of OASIS 1 and 2, demonstrating efficacy over a longer study duration and in a population with a vasomotor symptom profile representative of that seen in clinical practice,” Nick Panay, BSc, MBBS, director of the Menopause & PMS Centre at Queen Charlotte’s Hospital & Imperial College London, London, England, and his colleague reported.

Among 628 postmenopausal women aged 40-65, the predominantly White (78.5%) women were an average 54 years old, with an average BMI of 27.6, and were an average 5 years past their last period. Half received 120 mg elinzanetant and half received a placebo for 52 weeks.

At 12 weeks, the women receiving elinzanetant reported an average 1.6 moderate to severe vasomotor symptoms per day, down from 6.7 at baseline. Daily average symptoms in the placebo group fell from 6.8 at baseline to 3.4 at 12 weeks, for a difference of 1.6 fewer occurrences per day in the elinzanetant group (P < .0001).

Sleep disturbances also improved, falling 9.4 points from a baseline 57.4 in the elinzanetant group and 5.7 points from a baseline 58 in the placebo group. Quality-of-life scores improved from 4.1 to 2.8 (−1.3 change) in the elinzanetant group and from 4.4 to 3.3 (−1.1 change) in the placebo group.

In addition to looking at treatment-emergent adverse events, the safety assessments also included endometrial biopsies; bone mineral density in the femoral neck, hip, and lumbar spine; weight; and labs. Adverse events related to the study drug occurred in 30.4% of those in the elinzanetant group and 14.6% of those in the placebo group. The most commonly reported adverse events were headache (9.6% elinzanetant vs 7% placebo), fatigue (7% vs 10.2%), and sleepiness (5.1% vs 1.3%). A higher proportion of women taking elinzanetant (12.5%) than those taking placebo (4.1%) discontinued the study.

No serious adverse events deemed to be treatment-related occurred in either group, and no endometrial hyperplasia or malignant neoplasm occurred in either group. Bone mineral density changes in both groups were within the expected range for the women’s age, and their weight remained stable over the 52 weeks.

Six women taking elinzanetant and four taking placebo met predefined criteria for close liver observation, but none showed hepatotoxicity or evidence of possible drug-induced liver injury.

The research was funded by Bayer. Dr. Pinkerton has run a trial funded by Bayer and is a consultant for Bayer and Pfizer. Dr. Shufelt had no disclosures. Dr. Simon had grant/research support, consulting/advisory board participation, and/or speaking disclosures with AbbVie, Bayer Healthcare, Besins Healthcare, California Institute of Integral Studies, Camargo Pharmaceutical Services, Covance, Daré Bioscience, DEKA M.E.L.A S.r.l., Femasys, Ipsen, KaNDy/NeRRe Therapeutics, Khyria, Madorra, Mayne Pharma, Mitsubishi Tanabe Pharma Development America, Mylan/Viatris Inc, Myovant Sciences, ObsEva SA, Pfizer, Pharmavite, QUE Oncology, Scynexis, Sebela Pharmaceuticals, Sprout Pharmaceuticals, TherapeuticsMD, Vella Bioscience, and Viveve Medical, and he is a stockholder in Sermonix Pharmaceuticals.

A version of this article first appeared on Medscape.com.

CHICAGO — The nonhormonal investigational drug elinzanetant led to significant improvement in hot flashes as well as sleep disturbance and quality of life, according to data from three randomized controlled trials presented at The Menopause Society 2024 Annual Meeting in Chicago. Two phase 3 trials, OASIS 1 and 2, were also published in JAMA, and the longer-term OASIS 3 trial was presented as a poster at the conference.

Elinzanetant is a selective neurokinin (NK) receptor antagonist, similar to fezolinetant, the first drug in this class approved by the US Food and Drug Administration (FDA) for vasomotor symptoms in May 2023. This class of medications targets the estrogen-sensitive kisspeptin/NK B/dynorphin (KNDy) neurons thought to play a role in thermoregulation and hot flashes during menopause. While fezolinetant targets only the NK-3 receptor, elinzanetant is a dual NK receptor antagonist that targets both NK-1 and NK-3. Bayer submitted a New Drug Application for elinzanetant to the FDA on August 1.

For those in whom hormone therapy is contraindicated, “it’s always been difficult for women with really severe symptoms to have a safe and effective therapy,” lead author JoAnn Pinkerton, MD, a professor of ob.gyn. at the University of Virginia in Charlottesville, Virginia, told this news organization. “The nonhormonal therapies we’ve used mostly off-label — the antidepressants, gabapentin, clonidine, oxybutynin — do help the hot flashes, but they don’t work nearly as effectively as these new NK receptor antagonists, and having one that looks like it might have a broader use for hot flashes, night sweats, mood, and sleep is just really exciting.”

Dr. Pinkerton said approximately 80% of the women in the OASIS 1 and 2 studies had at least a 50% reduction in hot flashes. “It was a very strong, dramatic positive finding, but the improvements in sleep and mood have really encouraged us to go further,” she said.

Declining estrogen levels during and after menopause can cause hypertrophy and hyperactivity of the KNDy neurons, which has been linked to thermoregulation disruptions that may trigger hot flashes, James Simon, MD, a clinical professor of ob.gyn. at The George Washington University School of Medicine & Health Sciences and medical director of IntimMedicine in Washington, DC, told attendees. He presented pooled data from OASIS 1 and 2. The NK-1 receptor, targeted by elinzanetant but not fezolinetant, is also thought to play a role in insomnia and possibly in mood.

“Oftentimes the focus on a lot of these drugs is hot flashes, hot flashes, hot flashes, but we know hot flashes do not occur in isolation,” Chrisandra Shufelt, MD, professor and chair of general internal medicine and associate director of the Women’s Health Research Center at Mayo Clinic in Jacksonville, Florida, told this news organization. Elinzanetant is “an interesting compound because it actually works on sleep, and that was critical because sleep disturbance precedes” many other menopausal symptoms, said Dr. Shufelt, who was not involved in the study.

“I think it is an outstanding option for women who don’t have the opportunity to get hormones,” Dr. Shufelt said, and she was particularly pleased to see there were no safety concerns for the liver in the trial data. The FDA issued a warning on September 12 about the risk for rare liver injury with fezolinetant, but the early signals that had been seen in fezolinetant data were not seen in these elinzanetant data.

The OASIS 1 and 2 trials enrolled postmenopausal women, aged 40-65 years, who had at least 50 moderate to severe vasomotor occurrences per week.

“A moderate hot flash is a hot flash that is also associated with sweating, and a severe hot flash is a moderate hot flash that stops a woman in her tracks,” Dr. Simon said. “Namely, it’s severe enough with sweating and central nervous system effects that she is interrupted in whatever it is that she’s doing at the time.”

Exclusion criteria for the trials included a history of arrhythmias, heart block, or QT prolongation; abnormal lab results; history of malignancy within the past 5 years; uncontrolled or treatment-resistant hypertension, hypothyroidism, or hyperthyroidism; unexplained postmenopausal bleeding; clinically relevant abnormal mammogram findings; or disordered proliferative endometrium, endometrial hyperplasia, polyp, or endometrial cancer.

The predominantly White (80%) women were an average 54 years old, with an average body mass index (BMI) of 27.8, and were an average 3.5 years from their last period. For the first 12 weeks of the trials, 399 women were assigned to receive 120 mg once daily of oral elinzanetant and 397 were assigned to once daily placebo. Then the women taking placebo switched to elinzanetant for the final 14 weeks of the study.

The endpoints included mean change in frequency and severity of vasomotor symptoms at weeks 1, 4, and 12 as well as change in sleep disturbance and quality of life at week 12. Sleep was assessed with the Patient-Reported Outcomes Measurement Information System Sleep Disturbance–Short Form score, which ranges from 28.9 to 76.5, with a higher number denoting greater sleep disturbance. The Menopause-Specific Quality-of-Life score ranges from 1 to 8, with a higher score indicating poorer quality of life.

Daily frequency of vasomotor symptoms was 14 per day at baseline in the elinzanetant group, decreasing by 4.8 per day at week 1, 8 per day at week 4, and 9.4 per day at week 12. In the placebo group, women had an average 15.2 occurrences per day at baseline, which decreased by 3.2 at week 1, 5.2 at week 4, and 6.4 at week 12. Comparing the groups at 12 weeks, those receiving elinzanetant had 3.2 fewer daily vasomotor symptoms than those receiving placebo (P < .0001).

The severity of vasomotor symptoms also improved more in the elinzanetant group than in the placebo group over 12 weeks, after which severity improved further in those who switched from placebo to elinzanetant (P < .0001).

Sleep disturbance scores, starting at a mean 61.5 in the elinzanetant group and 60.5 in the placebo group, fell 10.7 points in the elinzanetant group and 5.3 points in the placebo group at 12 weeks, for a difference of 4.9 points (P < .0001). Sleep then further improved in those who switched from placebo to elinzanetant. Quality-of-life scores improved 1.37 points (from 4.52 at baseline) in the elinzanetant group and 0.96 points (from 4.49 at baseline) in the placebo group, for a mean difference at 12 weeks of 0.36 (P < .0001).

Though no head-to-head data exist comparing elinzanetant and fezolinetant, Dr. Simon told this news organization the side effects with fezolinetant “tend to be gastrointestinal, whereas the side effects for elinzanetant tend to be central nervous system,” such as drowsiness and lethargy.

The women who are the best candidates for elinzanetant, Dr. Pinkerton told this news organization, include those who have had an estrogen-sensitive cancer, such as breast or endometrial cancer, or who have fear of it, a family history, or are otherwise high risk. Other ideal candidates include those with a history of venous thromboembolism, people who have migraine with aura (due to concerns about increased risk for stroke), and those who have endometriosis or large fibroids.

“Then the last group might be women who took hormone therapy in their 50s and want to continue, but they’re trying to go off, and they have a recurrence of their hot flashes or night sweats or sleep issues,” Dr. Pinkerton said. “This might be a great group to switch over.”

OASIS 3 assessed the drug for 1 year and “supported the results of OASIS 1 and 2, demonstrating efficacy over a longer study duration and in a population with a vasomotor symptom profile representative of that seen in clinical practice,” Nick Panay, BSc, MBBS, director of the Menopause & PMS Centre at Queen Charlotte’s Hospital & Imperial College London, London, England, and his colleague reported.

Among 628 postmenopausal women aged 40-65, the predominantly White (78.5%) women were an average 54 years old, with an average BMI of 27.6, and were an average 5 years past their last period. Half received 120 mg elinzanetant and half received a placebo for 52 weeks.

At 12 weeks, the women receiving elinzanetant reported an average 1.6 moderate to severe vasomotor symptoms per day, down from 6.7 at baseline. Daily average symptoms in the placebo group fell from 6.8 at baseline to 3.4 at 12 weeks, for a difference of 1.6 fewer occurrences per day in the elinzanetant group (P < .0001).

Sleep disturbances also improved, falling 9.4 points from a baseline 57.4 in the elinzanetant group and 5.7 points from a baseline 58 in the placebo group. Quality-of-life scores improved from 4.1 to 2.8 (−1.3 change) in the elinzanetant group and from 4.4 to 3.3 (−1.1 change) in the placebo group.

In addition to looking at treatment-emergent adverse events, the safety assessments also included endometrial biopsies; bone mineral density in the femoral neck, hip, and lumbar spine; weight; and labs. Adverse events related to the study drug occurred in 30.4% of those in the elinzanetant group and 14.6% of those in the placebo group. The most commonly reported adverse events were headache (9.6% elinzanetant vs 7% placebo), fatigue (7% vs 10.2%), and sleepiness (5.1% vs 1.3%). A higher proportion of women taking elinzanetant (12.5%) than those taking placebo (4.1%) discontinued the study.

No serious adverse events deemed to be treatment-related occurred in either group, and no endometrial hyperplasia or malignant neoplasm occurred in either group. Bone mineral density changes in both groups were within the expected range for the women’s age, and their weight remained stable over the 52 weeks.

Six women taking elinzanetant and four taking placebo met predefined criteria for close liver observation, but none showed hepatotoxicity or evidence of possible drug-induced liver injury.

The research was funded by Bayer. Dr. Pinkerton has run a trial funded by Bayer and is a consultant for Bayer and Pfizer. Dr. Shufelt had no disclosures. Dr. Simon had grant/research support, consulting/advisory board participation, and/or speaking disclosures with AbbVie, Bayer Healthcare, Besins Healthcare, California Institute of Integral Studies, Camargo Pharmaceutical Services, Covance, Daré Bioscience, DEKA M.E.L.A S.r.l., Femasys, Ipsen, KaNDy/NeRRe Therapeutics, Khyria, Madorra, Mayne Pharma, Mitsubishi Tanabe Pharma Development America, Mylan/Viatris Inc, Myovant Sciences, ObsEva SA, Pfizer, Pharmavite, QUE Oncology, Scynexis, Sebela Pharmaceuticals, Sprout Pharmaceuticals, TherapeuticsMD, Vella Bioscience, and Viveve Medical, and he is a stockholder in Sermonix Pharmaceuticals.

A version of this article first appeared on Medscape.com.

CHICAGO — The nonhormonal investigational drug elinzanetant led to significant improvement in hot flashes as well as sleep disturbance and quality of life, according to data from three randomized controlled trials presented at The Menopause Society 2024 Annual Meeting in Chicago. Two phase 3 trials, OASIS 1 and 2, were also published in JAMA, and the longer-term OASIS 3 trial was presented as a poster at the conference.

Elinzanetant is a selective neurokinin (NK) receptor antagonist, similar to fezolinetant, the first drug in this class approved by the US Food and Drug Administration (FDA) for vasomotor symptoms in May 2023. This class of medications targets the estrogen-sensitive kisspeptin/NK B/dynorphin (KNDy) neurons thought to play a role in thermoregulation and hot flashes during menopause. While fezolinetant targets only the NK-3 receptor, elinzanetant is a dual NK receptor antagonist that targets both NK-1 and NK-3. Bayer submitted a New Drug Application for elinzanetant to the FDA on August 1.

For those in whom hormone therapy is contraindicated, “it’s always been difficult for women with really severe symptoms to have a safe and effective therapy,” lead author JoAnn Pinkerton, MD, a professor of ob.gyn. at the University of Virginia in Charlottesville, Virginia, told this news organization. “The nonhormonal therapies we’ve used mostly off-label — the antidepressants, gabapentin, clonidine, oxybutynin — do help the hot flashes, but they don’t work nearly as effectively as these new NK receptor antagonists, and having one that looks like it might have a broader use for hot flashes, night sweats, mood, and sleep is just really exciting.”

Dr. Pinkerton said approximately 80% of the women in the OASIS 1 and 2 studies had at least a 50% reduction in hot flashes. “It was a very strong, dramatic positive finding, but the improvements in sleep and mood have really encouraged us to go further,” she said.

Declining estrogen levels during and after menopause can cause hypertrophy and hyperactivity of the KNDy neurons, which has been linked to thermoregulation disruptions that may trigger hot flashes, James Simon, MD, a clinical professor of ob.gyn. at The George Washington University School of Medicine & Health Sciences and medical director of IntimMedicine in Washington, DC, told attendees. He presented pooled data from OASIS 1 and 2. The NK-1 receptor, targeted by elinzanetant but not fezolinetant, is also thought to play a role in insomnia and possibly in mood.

“Oftentimes the focus on a lot of these drugs is hot flashes, hot flashes, hot flashes, but we know hot flashes do not occur in isolation,” Chrisandra Shufelt, MD, professor and chair of general internal medicine and associate director of the Women’s Health Research Center at Mayo Clinic in Jacksonville, Florida, told this news organization. Elinzanetant is “an interesting compound because it actually works on sleep, and that was critical because sleep disturbance precedes” many other menopausal symptoms, said Dr. Shufelt, who was not involved in the study.

“I think it is an outstanding option for women who don’t have the opportunity to get hormones,” Dr. Shufelt said, and she was particularly pleased to see there were no safety concerns for the liver in the trial data. The FDA issued a warning on September 12 about the risk for rare liver injury with fezolinetant, but the early signals that had been seen in fezolinetant data were not seen in these elinzanetant data.

The OASIS 1 and 2 trials enrolled postmenopausal women, aged 40-65 years, who had at least 50 moderate to severe vasomotor occurrences per week.

“A moderate hot flash is a hot flash that is also associated with sweating, and a severe hot flash is a moderate hot flash that stops a woman in her tracks,” Dr. Simon said. “Namely, it’s severe enough with sweating and central nervous system effects that she is interrupted in whatever it is that she’s doing at the time.”

Exclusion criteria for the trials included a history of arrhythmias, heart block, or QT prolongation; abnormal lab results; history of malignancy within the past 5 years; uncontrolled or treatment-resistant hypertension, hypothyroidism, or hyperthyroidism; unexplained postmenopausal bleeding; clinically relevant abnormal mammogram findings; or disordered proliferative endometrium, endometrial hyperplasia, polyp, or endometrial cancer.

The predominantly White (80%) women were an average 54 years old, with an average body mass index (BMI) of 27.8, and were an average 3.5 years from their last period. For the first 12 weeks of the trials, 399 women were assigned to receive 120 mg once daily of oral elinzanetant and 397 were assigned to once daily placebo. Then the women taking placebo switched to elinzanetant for the final 14 weeks of the study.

The endpoints included mean change in frequency and severity of vasomotor symptoms at weeks 1, 4, and 12 as well as change in sleep disturbance and quality of life at week 12. Sleep was assessed with the Patient-Reported Outcomes Measurement Information System Sleep Disturbance–Short Form score, which ranges from 28.9 to 76.5, with a higher number denoting greater sleep disturbance. The Menopause-Specific Quality-of-Life score ranges from 1 to 8, with a higher score indicating poorer quality of life.

Daily frequency of vasomotor symptoms was 14 per day at baseline in the elinzanetant group, decreasing by 4.8 per day at week 1, 8 per day at week 4, and 9.4 per day at week 12. In the placebo group, women had an average 15.2 occurrences per day at baseline, which decreased by 3.2 at week 1, 5.2 at week 4, and 6.4 at week 12. Comparing the groups at 12 weeks, those receiving elinzanetant had 3.2 fewer daily vasomotor symptoms than those receiving placebo (P < .0001).

The severity of vasomotor symptoms also improved more in the elinzanetant group than in the placebo group over 12 weeks, after which severity improved further in those who switched from placebo to elinzanetant (P < .0001).

Sleep disturbance scores, starting at a mean 61.5 in the elinzanetant group and 60.5 in the placebo group, fell 10.7 points in the elinzanetant group and 5.3 points in the placebo group at 12 weeks, for a difference of 4.9 points (P < .0001). Sleep then further improved in those who switched from placebo to elinzanetant. Quality-of-life scores improved 1.37 points (from 4.52 at baseline) in the elinzanetant group and 0.96 points (from 4.49 at baseline) in the placebo group, for a mean difference at 12 weeks of 0.36 (P < .0001).

Though no head-to-head data exist comparing elinzanetant and fezolinetant, Dr. Simon told this news organization the side effects with fezolinetant “tend to be gastrointestinal, whereas the side effects for elinzanetant tend to be central nervous system,” such as drowsiness and lethargy.

The women who are the best candidates for elinzanetant, Dr. Pinkerton told this news organization, include those who have had an estrogen-sensitive cancer, such as breast or endometrial cancer, or who have fear of it, a family history, or are otherwise high risk. Other ideal candidates include those with a history of venous thromboembolism, people who have migraine with aura (due to concerns about increased risk for stroke), and those who have endometriosis or large fibroids.

“Then the last group might be women who took hormone therapy in their 50s and want to continue, but they’re trying to go off, and they have a recurrence of their hot flashes or night sweats or sleep issues,” Dr. Pinkerton said. “This might be a great group to switch over.”

OASIS 3 assessed the drug for 1 year and “supported the results of OASIS 1 and 2, demonstrating efficacy over a longer study duration and in a population with a vasomotor symptom profile representative of that seen in clinical practice,” Nick Panay, BSc, MBBS, director of the Menopause & PMS Centre at Queen Charlotte’s Hospital & Imperial College London, London, England, and his colleague reported.

Among 628 postmenopausal women aged 40-65, the predominantly White (78.5%) women were an average 54 years old, with an average BMI of 27.6, and were an average 5 years past their last period. Half received 120 mg elinzanetant and half received a placebo for 52 weeks.

At 12 weeks, the women receiving elinzanetant reported an average 1.6 moderate to severe vasomotor symptoms per day, down from 6.7 at baseline. Daily average symptoms in the placebo group fell from 6.8 at baseline to 3.4 at 12 weeks, for a difference of 1.6 fewer occurrences per day in the elinzanetant group (P < .0001).

Sleep disturbances also improved, falling 9.4 points from a baseline 57.4 in the elinzanetant group and 5.7 points from a baseline 58 in the placebo group. Quality-of-life scores improved from 4.1 to 2.8 (−1.3 change) in the elinzanetant group and from 4.4 to 3.3 (−1.1 change) in the placebo group.

In addition to looking at treatment-emergent adverse events, the safety assessments also included endometrial biopsies; bone mineral density in the femoral neck, hip, and lumbar spine; weight; and labs. Adverse events related to the study drug occurred in 30.4% of those in the elinzanetant group and 14.6% of those in the placebo group. The most commonly reported adverse events were headache (9.6% elinzanetant vs 7% placebo), fatigue (7% vs 10.2%), and sleepiness (5.1% vs 1.3%). A higher proportion of women taking elinzanetant (12.5%) than those taking placebo (4.1%) discontinued the study.

No serious adverse events deemed to be treatment-related occurred in either group, and no endometrial hyperplasia or malignant neoplasm occurred in either group. Bone mineral density changes in both groups were within the expected range for the women’s age, and their weight remained stable over the 52 weeks.

Six women taking elinzanetant and four taking placebo met predefined criteria for close liver observation, but none showed hepatotoxicity or evidence of possible drug-induced liver injury.

The research was funded by Bayer. Dr. Pinkerton has run a trial funded by Bayer and is a consultant for Bayer and Pfizer. Dr. Shufelt had no disclosures. Dr. Simon had grant/research support, consulting/advisory board participation, and/or speaking disclosures with AbbVie, Bayer Healthcare, Besins Healthcare, California Institute of Integral Studies, Camargo Pharmaceutical Services, Covance, Daré Bioscience, DEKA M.E.L.A S.r.l., Femasys, Ipsen, KaNDy/NeRRe Therapeutics, Khyria, Madorra, Mayne Pharma, Mitsubishi Tanabe Pharma Development America, Mylan/Viatris Inc, Myovant Sciences, ObsEva SA, Pfizer, Pharmavite, QUE Oncology, Scynexis, Sebela Pharmaceuticals, Sprout Pharmaceuticals, TherapeuticsMD, Vella Bioscience, and Viveve Medical, and he is a stockholder in Sermonix Pharmaceuticals.

A version of this article first appeared on Medscape.com.

CHICAGO — The vast majority of women experiencing genitourinary syndrome of menopause (GSM) symptoms did not receive a prescription for hormonal vaginal therapies prior to seeking care at a specialized menopause clinic, according to research presented at the annual meeting of The Menopause Society.

“GSM symptoms are very common and affect women’s health and quality of life, often worsening without effective therapy,” Leticia Hernández Galán, PhD, of the Department of Obstetrics & Gynecology, McMaster University, Hamilton, Ontario, Canada, and colleagues reported. “We have demonstrated that most women seeking specialty care in an urban center with GSM symptoms have not been given a trial of local vaginal therapies by referring providers despite guidelines about safety and lack of contraindications. Given very long wait times for menopause providers in Canada, improved education for both women and their providers is needed to reduce needless suffering and improve care.”

Stephanie Faubion, MD, MBA, director of the Mayo Clinic Women’s Health in Jacksonville, Florida, and medical director of The Menopause Society, was not involved with the study but agreed with the authors’ assessment of the findings.

“This study highlights the treatment gap for women with genitourinary syndrome of menopause,” Dr. Faubion told this news organization. “Clearly, there is underutilization of low-dose vaginal hormonal therapies, which are known to be safe and effective. We still have work to do in terms of educating both women and providers on established treatment options for this common concern in menopausal women.” 

The findings match previous ones that found a majority of women with GSM do not receive treatment. A 2017 study, which was cited in the 2020 Menopause Society position statement on the condition, found that half of women with GSM had never used any treatment.

GSM is the current term that replaces previously used “vulvovaginal atrophy” and “atrophic vaginitis” because it encompasses all the menopause symptoms and signs associated with menopause that affect the vagina, vulva, and urinary tract. Anywhere from 50% to 84% of postmenopausal women experience GSM, the authors noted, with symptoms that include “burning, itching, or irritation of the vulva” and “lack of lubrication and discomfort or pain with sexual activity as well as dysuria, increased frequency or urgency of urination, and increased risk for urinary tract infections.”

First-line treatment of mild GSM often includes nonhormonal vaginal lubricants and moisturizers, but vaginal estrogen is considered the most effective treatment for more severe or bothersome cases. Other treatments include systematic hormone therapy and ospemifene or other selective estrogen receptor modulators.
 

Increased Risk for Urinary Tract Infections (UTIs)

Untreated GSM is not simply a quality of life issue; it increases the risk of developing serious UTIs, explained JoAnn Pinkerton, MD, a professor of obstetrics and gynecology at the University of Virginia, Charlottesville, who was not involved in the study.

“Estrogen depletion alters the vaginal epithelium, with distinct impairments in lubrication, elasticity, pH, and blood flow,” Dr. Pinkerton said. “The vaginal microbiome changes, with increasing pH following menopause and loss of lactobacillus predominance. These alterations allow a more hospitable environment for bacterial growth and increase the risk of UTI.”

Vaginal estrogen, meanwhile, reduces UTI risk because it “increases the presence of lactobacillus in the vagina due to improvements in vaginal pH, rebuilding superficial cells, elasticity, and connectivity,” she said.

The study assessed the incidence of GSM among patients at a single specialized Canadian institution, St. Joseph’s Healthcare Menopause Clinic in Hamilton, Ontario, between January 2021 and August 2024. Patients completed a Menopause Rating Scale that quantified two sets of GSM symptoms relating to “dryness of the vagina” and “bladder problems.” Patients also answered questions about the provider they had seen before coming to the specialized clinic and whether they had been prescribed local vaginal products before their visit.

Among 529 patients, the average age was 51, and the vast majority (88%) had some amount of tertiary education beyond high school. Only 21.5% were still menstruating, whereas the other respondents had stopped menstruating. The patient population was mostly White (85.6%), with Black, Hispanic, Asian, Middle Eastern, and Indigenous patients making up most of the other patient groups.

Among the 521 patients who answered the question on vaginal dryness, answers were similarly split between none (26%), mild (23%), moderate (21%), severe (15%), and very severe (15%). One third of the 526 women (34%) who answered the question on bladder problems said they had none, whereas the remainder reported their problems as mild (24%), moderate (24%), severe (11%), or very severe (7%).

Despite about half the participants reporting moderate to very severe vaginal dryness, 85% of them had not been prescribed local vaginal hormone therapies before their visit to the menopause clinic. Women were more likely to have been prescribed a localized therapy if they were older, were postmenopausal instead of perimenopausal, or had a female healthcare provider prior to this visit.

The survey also asked about the specialty and years in practice for the providers women had seen before visiting the clinic, but neither of these were predictors for receiving a hormone prescription. The patient’s education, partner status, and ethnicity were also not associated with the likelihood of a prescription.

Among 62 women who had been prescribed a vaginal hormone treatment, most were prescribed Vagifem (29%) or Premarin Vaginal cream (26%), followed by Intrarosa (19%), Estragyn cream (16%), Estring (3%), or something else (18%).
 

Serious Complications of GSM

Dr. Pinkerton described how GSM, particularly in older women, can run the risk of becoming life-threatening if untreated and unrecognized.

“For some women, UTIs can lead to urosepsis, as both the vaginal tissues and bladder tissues are thin with blood vessels close to the surface,” Dr. Pinkerton said. “What may have started as a UTI, can ascend to the kidneys or get into the bloodstream, which, in some, can develop into urosepsis, which can be life-threatening. The bacterial pathogen initiates the disease process, but host immune responses drive whether sepsis develops and its severity.”

The research by Dr. Hernández Galán was funded by the Canadian Institutes of Health Research, the Canadian Menopause Society, and Pfizer. Dr. Faubion had no disclosures, and Dr. Pinkerton has run a trial funded by Bayer and is a consultant for Bayer and Pfizer.

A version of this article first appeared on Medscape.com.

CHICAGO — The vast majority of women experiencing genitourinary syndrome of menopause (GSM) symptoms did not receive a prescription for hormonal vaginal therapies prior to seeking care at a specialized menopause clinic, according to research presented at the annual meeting of The Menopause Society.

“GSM symptoms are very common and affect women’s health and quality of life, often worsening without effective therapy,” Leticia Hernández Galán, PhD, of the Department of Obstetrics & Gynecology, McMaster University, Hamilton, Ontario, Canada, and colleagues reported. “We have demonstrated that most women seeking specialty care in an urban center with GSM symptoms have not been given a trial of local vaginal therapies by referring providers despite guidelines about safety and lack of contraindications. Given very long wait times for menopause providers in Canada, improved education for both women and their providers is needed to reduce needless suffering and improve care.”

Stephanie Faubion, MD, MBA, director of the Mayo Clinic Women’s Health in Jacksonville, Florida, and medical director of The Menopause Society, was not involved with the study but agreed with the authors’ assessment of the findings.

“This study highlights the treatment gap for women with genitourinary syndrome of menopause,” Dr. Faubion told this news organization. “Clearly, there is underutilization of low-dose vaginal hormonal therapies, which are known to be safe and effective. We still have work to do in terms of educating both women and providers on established treatment options for this common concern in menopausal women.” 

The findings match previous ones that found a majority of women with GSM do not receive treatment. A 2017 study, which was cited in the 2020 Menopause Society position statement on the condition, found that half of women with GSM had never used any treatment.

GSM is the current term that replaces previously used “vulvovaginal atrophy” and “atrophic vaginitis” because it encompasses all the menopause symptoms and signs associated with menopause that affect the vagina, vulva, and urinary tract. Anywhere from 50% to 84% of postmenopausal women experience GSM, the authors noted, with symptoms that include “burning, itching, or irritation of the vulva” and “lack of lubrication and discomfort or pain with sexual activity as well as dysuria, increased frequency or urgency of urination, and increased risk for urinary tract infections.”

First-line treatment of mild GSM often includes nonhormonal vaginal lubricants and moisturizers, but vaginal estrogen is considered the most effective treatment for more severe or bothersome cases. Other treatments include systematic hormone therapy and ospemifene or other selective estrogen receptor modulators.
 

Increased Risk for Urinary Tract Infections (UTIs)

Untreated GSM is not simply a quality of life issue; it increases the risk of developing serious UTIs, explained JoAnn Pinkerton, MD, a professor of obstetrics and gynecology at the University of Virginia, Charlottesville, who was not involved in the study.

“Estrogen depletion alters the vaginal epithelium, with distinct impairments in lubrication, elasticity, pH, and blood flow,” Dr. Pinkerton said. “The vaginal microbiome changes, with increasing pH following menopause and loss of lactobacillus predominance. These alterations allow a more hospitable environment for bacterial growth and increase the risk of UTI.”

Vaginal estrogen, meanwhile, reduces UTI risk because it “increases the presence of lactobacillus in the vagina due to improvements in vaginal pH, rebuilding superficial cells, elasticity, and connectivity,” she said.

The study assessed the incidence of GSM among patients at a single specialized Canadian institution, St. Joseph’s Healthcare Menopause Clinic in Hamilton, Ontario, between January 2021 and August 2024. Patients completed a Menopause Rating Scale that quantified two sets of GSM symptoms relating to “dryness of the vagina” and “bladder problems.” Patients also answered questions about the provider they had seen before coming to the specialized clinic and whether they had been prescribed local vaginal products before their visit.

Among 529 patients, the average age was 51, and the vast majority (88%) had some amount of tertiary education beyond high school. Only 21.5% were still menstruating, whereas the other respondents had stopped menstruating. The patient population was mostly White (85.6%), with Black, Hispanic, Asian, Middle Eastern, and Indigenous patients making up most of the other patient groups.

Among the 521 patients who answered the question on vaginal dryness, answers were similarly split between none (26%), mild (23%), moderate (21%), severe (15%), and very severe (15%). One third of the 526 women (34%) who answered the question on bladder problems said they had none, whereas the remainder reported their problems as mild (24%), moderate (24%), severe (11%), or very severe (7%).

Despite about half the participants reporting moderate to very severe vaginal dryness, 85% of them had not been prescribed local vaginal hormone therapies before their visit to the menopause clinic. Women were more likely to have been prescribed a localized therapy if they were older, were postmenopausal instead of perimenopausal, or had a female healthcare provider prior to this visit.

The survey also asked about the specialty and years in practice for the providers women had seen before visiting the clinic, but neither of these were predictors for receiving a hormone prescription. The patient’s education, partner status, and ethnicity were also not associated with the likelihood of a prescription.

Among 62 women who had been prescribed a vaginal hormone treatment, most were prescribed Vagifem (29%) or Premarin Vaginal cream (26%), followed by Intrarosa (19%), Estragyn cream (16%), Estring (3%), or something else (18%).
 

Serious Complications of GSM

Dr. Pinkerton described how GSM, particularly in older women, can run the risk of becoming life-threatening if untreated and unrecognized.

“For some women, UTIs can lead to urosepsis, as both the vaginal tissues and bladder tissues are thin with blood vessels close to the surface,” Dr. Pinkerton said. “What may have started as a UTI, can ascend to the kidneys or get into the bloodstream, which, in some, can develop into urosepsis, which can be life-threatening. The bacterial pathogen initiates the disease process, but host immune responses drive whether sepsis develops and its severity.”

The research by Dr. Hernández Galán was funded by the Canadian Institutes of Health Research, the Canadian Menopause Society, and Pfizer. Dr. Faubion had no disclosures, and Dr. Pinkerton has run a trial funded by Bayer and is a consultant for Bayer and Pfizer.

A version of this article first appeared on Medscape.com.

CHICAGO — The vast majority of women experiencing genitourinary syndrome of menopause (GSM) symptoms did not receive a prescription for hormonal vaginal therapies prior to seeking care at a specialized menopause clinic, according to research presented at the annual meeting of The Menopause Society.

“GSM symptoms are very common and affect women’s health and quality of life, often worsening without effective therapy,” Leticia Hernández Galán, PhD, of the Department of Obstetrics & Gynecology, McMaster University, Hamilton, Ontario, Canada, and colleagues reported. “We have demonstrated that most women seeking specialty care in an urban center with GSM symptoms have not been given a trial of local vaginal therapies by referring providers despite guidelines about safety and lack of contraindications. Given very long wait times for menopause providers in Canada, improved education for both women and their providers is needed to reduce needless suffering and improve care.”

Stephanie Faubion, MD, MBA, director of the Mayo Clinic Women’s Health in Jacksonville, Florida, and medical director of The Menopause Society, was not involved with the study but agreed with the authors’ assessment of the findings.

“This study highlights the treatment gap for women with genitourinary syndrome of menopause,” Dr. Faubion told this news organization. “Clearly, there is underutilization of low-dose vaginal hormonal therapies, which are known to be safe and effective. We still have work to do in terms of educating both women and providers on established treatment options for this common concern in menopausal women.” 

The findings match previous ones that found a majority of women with GSM do not receive treatment. A 2017 study, which was cited in the 2020 Menopause Society position statement on the condition, found that half of women with GSM had never used any treatment.

GSM is the current term that replaces previously used “vulvovaginal atrophy” and “atrophic vaginitis” because it encompasses all the menopause symptoms and signs associated with menopause that affect the vagina, vulva, and urinary tract. Anywhere from 50% to 84% of postmenopausal women experience GSM, the authors noted, with symptoms that include “burning, itching, or irritation of the vulva” and “lack of lubrication and discomfort or pain with sexual activity as well as dysuria, increased frequency or urgency of urination, and increased risk for urinary tract infections.”

First-line treatment of mild GSM often includes nonhormonal vaginal lubricants and moisturizers, but vaginal estrogen is considered the most effective treatment for more severe or bothersome cases. Other treatments include systematic hormone therapy and ospemifene or other selective estrogen receptor modulators.
 

Increased Risk for Urinary Tract Infections (UTIs)

Untreated GSM is not simply a quality of life issue; it increases the risk of developing serious UTIs, explained JoAnn Pinkerton, MD, a professor of obstetrics and gynecology at the University of Virginia, Charlottesville, who was not involved in the study.

“Estrogen depletion alters the vaginal epithelium, with distinct impairments in lubrication, elasticity, pH, and blood flow,” Dr. Pinkerton said. “The vaginal microbiome changes, with increasing pH following menopause and loss of lactobacillus predominance. These alterations allow a more hospitable environment for bacterial growth and increase the risk of UTI.”

Vaginal estrogen, meanwhile, reduces UTI risk because it “increases the presence of lactobacillus in the vagina due to improvements in vaginal pH, rebuilding superficial cells, elasticity, and connectivity,” she said.

The study assessed the incidence of GSM among patients at a single specialized Canadian institution, St. Joseph’s Healthcare Menopause Clinic in Hamilton, Ontario, between January 2021 and August 2024. Patients completed a Menopause Rating Scale that quantified two sets of GSM symptoms relating to “dryness of the vagina” and “bladder problems.” Patients also answered questions about the provider they had seen before coming to the specialized clinic and whether they had been prescribed local vaginal products before their visit.

Among 529 patients, the average age was 51, and the vast majority (88%) had some amount of tertiary education beyond high school. Only 21.5% were still menstruating, whereas the other respondents had stopped menstruating. The patient population was mostly White (85.6%), with Black, Hispanic, Asian, Middle Eastern, and Indigenous patients making up most of the other patient groups.

Among the 521 patients who answered the question on vaginal dryness, answers were similarly split between none (26%), mild (23%), moderate (21%), severe (15%), and very severe (15%). One third of the 526 women (34%) who answered the question on bladder problems said they had none, whereas the remainder reported their problems as mild (24%), moderate (24%), severe (11%), or very severe (7%).

Despite about half the participants reporting moderate to very severe vaginal dryness, 85% of them had not been prescribed local vaginal hormone therapies before their visit to the menopause clinic. Women were more likely to have been prescribed a localized therapy if they were older, were postmenopausal instead of perimenopausal, or had a female healthcare provider prior to this visit.

The survey also asked about the specialty and years in practice for the providers women had seen before visiting the clinic, but neither of these were predictors for receiving a hormone prescription. The patient’s education, partner status, and ethnicity were also not associated with the likelihood of a prescription.

Among 62 women who had been prescribed a vaginal hormone treatment, most were prescribed Vagifem (29%) or Premarin Vaginal cream (26%), followed by Intrarosa (19%), Estragyn cream (16%), Estring (3%), or something else (18%).
 

Serious Complications of GSM

Dr. Pinkerton described how GSM, particularly in older women, can run the risk of becoming life-threatening if untreated and unrecognized.

“For some women, UTIs can lead to urosepsis, as both the vaginal tissues and bladder tissues are thin with blood vessels close to the surface,” Dr. Pinkerton said. “What may have started as a UTI, can ascend to the kidneys or get into the bloodstream, which, in some, can develop into urosepsis, which can be life-threatening. The bacterial pathogen initiates the disease process, but host immune responses drive whether sepsis develops and its severity.”

The research by Dr. Hernández Galán was funded by the Canadian Institutes of Health Research, the Canadian Menopause Society, and Pfizer. Dr. Faubion had no disclosures, and Dr. Pinkerton has run a trial funded by Bayer and is a consultant for Bayer and Pfizer.

A version of this article first appeared on Medscape.com.