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LOS ANGELES — Use of preexposure prophylaxis (PrEP) to prevent HIV is increasing overall, but both the rate of increase for starting PrEP and the rate of unmet need differ widely by demographic group, according to new data from a large study.

An analysis by Li Tao, MD, MS, PhD, director of real-world evidence at Gilead Sciences, and colleagues looked at statistical trends from 2019 to 2023 and found that Black, Hispanic, and Medicaid-insured populations continue to lack equitable access to PrEP.

Among the findings were that most new PrEP users were men with HIV risk factors who are commercially insured and live in predominantly non-Hispanic White areas (53% in 2019 and 43% in 2023). For comparison, men living in predominantly Black or Hispanic neighborhoods, or who are insured by Medicaid, saw lower proportions of PrEP use (16% in 2019 and 17% in 2023) despite higher annual increases in PrEP use (11% per year) and higher unmet needs.
 

Half a Million Real-World Participants

Tao presented her team’s findings at the Infectious Disease Week (IDWeek) 2024 Annual Meeting. The study included “more than half a million real-world PrEP users over the past 5 years,” she said.

The group with the lowest growth in initiation of PrEP in the study period (an annual percentage increase of 2%) and the lowest unmet need included men with HIV risk factors, who were using commercial insurance and living in White-dominant neighborhoods.

HIV risk factors included diagnosis of any sexually transmitted disease, contact with and exposure to communicable diseases, high-risk sexual behavior, contact with a hypodermic needle, long-term prophylaxis, HIV prevention counseling, and HIV screening.

Other men with HIV risk factors (those who were commercially insured, living in Black/Hispanic neighborhoods, or those on Medicaid across all neighborhoods) had a moderate increase in PrEP initiation (an annual percentage increase of 11%-16%) and higher unmet needs.

Researchers gathered data on PrEP prescriptions and new HIV diagnoses (from 2019 to 2023) through the IQVIA pharmacy claims database. PrEP-to-need ratio (PNR) is the number of individuals using PrEP in a year divided by new HIV diagnoses in the previous year. It was calculated for subgroups defined by five PNR-associated factors: Sex, insurance, recorded HIV risk factors (identified by diagnosis or procedure codes), “Ending the HIV Epidemic” jurisdictions, and neighborhood race/ethnicity mix.
 

Disparities Persist

While PrEP use improved across all the groups studied in the 5 years, “disparities still persist and the need remains very significant,” Tao said. “It’s very crucial for guiding the future HIV prevention options.”

“Long-acting PrEP options may help to address some social determinants structural factors in HIV acquisition,” she added.
 

What Programs Are Helping?

Some guidelines and programs are helping increase uptake, Tao said.

The United States Preventive Services Task Force (USPSTF) guidelines “reinforce more accessible PrEP programs to individuals like zero-cost sharing or same-day dispensing,” Tao said in a press briefing. “Those kinds of policies are really effective. We can see that after the implementation of the USPSTF guidelines, the copay sharing is really decreasing and is coinciding with the HIV rates declining.”

The Medicaid coverage expansion in 40 states “has been really effective” in PrEP uptake, she added.

Colleen Kelley, MD, MPH, with the Division of Infectious Diseases at the Rollins School of Public Health, Emory University, in Atlanta, who was not part of the research, said there has been a slow but improving uptake of PrEP across the board in the United States, “but the issue is that the uptake has been inequitable.”
 

Large Study With Recent Data

“This is an extremely large study with very recent data,” Kelley said. “Additionally, they were able to couple (the uptake) with unmet need. People who are at higher risk of acquiring HIV or who live in high-risk areas for HIV should have greater access to PrEP. They have a greater need for PrEP. What we really need to do from an equity perspective is match the PrEP use with the PrEP need and we have not been successful in doing that.”

Kelley added that the finding that the group that had the highest unmet need for PrEP in the study also had no recorded HIV risk factors. “It’s an interesting time to start thinking about beyond risk factor coverage for PrEP,” she said.

Another issue, Kelley said, is that “people are using (PrEP) but they’re also stopping it. People will need to take PrEP many years for protection, but about half discontinue in the first 6-12 months.

“We need to look at how people will persist on PrEP over the long term. That’s the next frontier,” she said. “We hope the long-acting injectables will help overcome some of the PrEP fatigue. But some may just tire of taking medication repeatedly for an infection they don’t have,” she said.

The study was funded by Gilead Sciences. Tao is employed by and is a shareholder of Gilead Sciences. All relevant financial disclosures have been mitigated, according to the paper. Kelley has research grants to her institution from Gilead, Moderna, Novavax, ViiV, and Humanigen.
 

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

LOS ANGELES — Use of preexposure prophylaxis (PrEP) to prevent HIV is increasing overall, but both the rate of increase for starting PrEP and the rate of unmet need differ widely by demographic group, according to new data from a large study.

An analysis by Li Tao, MD, MS, PhD, director of real-world evidence at Gilead Sciences, and colleagues looked at statistical trends from 2019 to 2023 and found that Black, Hispanic, and Medicaid-insured populations continue to lack equitable access to PrEP.

Among the findings were that most new PrEP users were men with HIV risk factors who are commercially insured and live in predominantly non-Hispanic White areas (53% in 2019 and 43% in 2023). For comparison, men living in predominantly Black or Hispanic neighborhoods, or who are insured by Medicaid, saw lower proportions of PrEP use (16% in 2019 and 17% in 2023) despite higher annual increases in PrEP use (11% per year) and higher unmet needs.
 

Half a Million Real-World Participants

Tao presented her team’s findings at the Infectious Disease Week (IDWeek) 2024 Annual Meeting. The study included “more than half a million real-world PrEP users over the past 5 years,” she said.

The group with the lowest growth in initiation of PrEP in the study period (an annual percentage increase of 2%) and the lowest unmet need included men with HIV risk factors, who were using commercial insurance and living in White-dominant neighborhoods.

HIV risk factors included diagnosis of any sexually transmitted disease, contact with and exposure to communicable diseases, high-risk sexual behavior, contact with a hypodermic needle, long-term prophylaxis, HIV prevention counseling, and HIV screening.

Other men with HIV risk factors (those who were commercially insured, living in Black/Hispanic neighborhoods, or those on Medicaid across all neighborhoods) had a moderate increase in PrEP initiation (an annual percentage increase of 11%-16%) and higher unmet needs.

Researchers gathered data on PrEP prescriptions and new HIV diagnoses (from 2019 to 2023) through the IQVIA pharmacy claims database. PrEP-to-need ratio (PNR) is the number of individuals using PrEP in a year divided by new HIV diagnoses in the previous year. It was calculated for subgroups defined by five PNR-associated factors: Sex, insurance, recorded HIV risk factors (identified by diagnosis or procedure codes), “Ending the HIV Epidemic” jurisdictions, and neighborhood race/ethnicity mix.
 

Disparities Persist

While PrEP use improved across all the groups studied in the 5 years, “disparities still persist and the need remains very significant,” Tao said. “It’s very crucial for guiding the future HIV prevention options.”

“Long-acting PrEP options may help to address some social determinants structural factors in HIV acquisition,” she added.
 

What Programs Are Helping?

Some guidelines and programs are helping increase uptake, Tao said.

The United States Preventive Services Task Force (USPSTF) guidelines “reinforce more accessible PrEP programs to individuals like zero-cost sharing or same-day dispensing,” Tao said in a press briefing. “Those kinds of policies are really effective. We can see that after the implementation of the USPSTF guidelines, the copay sharing is really decreasing and is coinciding with the HIV rates declining.”

The Medicaid coverage expansion in 40 states “has been really effective” in PrEP uptake, she added.

Colleen Kelley, MD, MPH, with the Division of Infectious Diseases at the Rollins School of Public Health, Emory University, in Atlanta, who was not part of the research, said there has been a slow but improving uptake of PrEP across the board in the United States, “but the issue is that the uptake has been inequitable.”
 

Large Study With Recent Data

“This is an extremely large study with very recent data,” Kelley said. “Additionally, they were able to couple (the uptake) with unmet need. People who are at higher risk of acquiring HIV or who live in high-risk areas for HIV should have greater access to PrEP. They have a greater need for PrEP. What we really need to do from an equity perspective is match the PrEP use with the PrEP need and we have not been successful in doing that.”

Kelley added that the finding that the group that had the highest unmet need for PrEP in the study also had no recorded HIV risk factors. “It’s an interesting time to start thinking about beyond risk factor coverage for PrEP,” she said.

Another issue, Kelley said, is that “people are using (PrEP) but they’re also stopping it. People will need to take PrEP many years for protection, but about half discontinue in the first 6-12 months.

“We need to look at how people will persist on PrEP over the long term. That’s the next frontier,” she said. “We hope the long-acting injectables will help overcome some of the PrEP fatigue. But some may just tire of taking medication repeatedly for an infection they don’t have,” she said.

The study was funded by Gilead Sciences. Tao is employed by and is a shareholder of Gilead Sciences. All relevant financial disclosures have been mitigated, according to the paper. Kelley has research grants to her institution from Gilead, Moderna, Novavax, ViiV, and Humanigen.
 

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

LOS ANGELES — Use of preexposure prophylaxis (PrEP) to prevent HIV is increasing overall, but both the rate of increase for starting PrEP and the rate of unmet need differ widely by demographic group, according to new data from a large study.

An analysis by Li Tao, MD, MS, PhD, director of real-world evidence at Gilead Sciences, and colleagues looked at statistical trends from 2019 to 2023 and found that Black, Hispanic, and Medicaid-insured populations continue to lack equitable access to PrEP.

Among the findings were that most new PrEP users were men with HIV risk factors who are commercially insured and live in predominantly non-Hispanic White areas (53% in 2019 and 43% in 2023). For comparison, men living in predominantly Black or Hispanic neighborhoods, or who are insured by Medicaid, saw lower proportions of PrEP use (16% in 2019 and 17% in 2023) despite higher annual increases in PrEP use (11% per year) and higher unmet needs.
 

Half a Million Real-World Participants

Tao presented her team’s findings at the Infectious Disease Week (IDWeek) 2024 Annual Meeting. The study included “more than half a million real-world PrEP users over the past 5 years,” she said.

The group with the lowest growth in initiation of PrEP in the study period (an annual percentage increase of 2%) and the lowest unmet need included men with HIV risk factors, who were using commercial insurance and living in White-dominant neighborhoods.

HIV risk factors included diagnosis of any sexually transmitted disease, contact with and exposure to communicable diseases, high-risk sexual behavior, contact with a hypodermic needle, long-term prophylaxis, HIV prevention counseling, and HIV screening.

Other men with HIV risk factors (those who were commercially insured, living in Black/Hispanic neighborhoods, or those on Medicaid across all neighborhoods) had a moderate increase in PrEP initiation (an annual percentage increase of 11%-16%) and higher unmet needs.

Researchers gathered data on PrEP prescriptions and new HIV diagnoses (from 2019 to 2023) through the IQVIA pharmacy claims database. PrEP-to-need ratio (PNR) is the number of individuals using PrEP in a year divided by new HIV diagnoses in the previous year. It was calculated for subgroups defined by five PNR-associated factors: Sex, insurance, recorded HIV risk factors (identified by diagnosis or procedure codes), “Ending the HIV Epidemic” jurisdictions, and neighborhood race/ethnicity mix.
 

Disparities Persist

While PrEP use improved across all the groups studied in the 5 years, “disparities still persist and the need remains very significant,” Tao said. “It’s very crucial for guiding the future HIV prevention options.”

“Long-acting PrEP options may help to address some social determinants structural factors in HIV acquisition,” she added.
 

What Programs Are Helping?

Some guidelines and programs are helping increase uptake, Tao said.

The United States Preventive Services Task Force (USPSTF) guidelines “reinforce more accessible PrEP programs to individuals like zero-cost sharing or same-day dispensing,” Tao said in a press briefing. “Those kinds of policies are really effective. We can see that after the implementation of the USPSTF guidelines, the copay sharing is really decreasing and is coinciding with the HIV rates declining.”

The Medicaid coverage expansion in 40 states “has been really effective” in PrEP uptake, she added.

Colleen Kelley, MD, MPH, with the Division of Infectious Diseases at the Rollins School of Public Health, Emory University, in Atlanta, who was not part of the research, said there has been a slow but improving uptake of PrEP across the board in the United States, “but the issue is that the uptake has been inequitable.”
 

Large Study With Recent Data

“This is an extremely large study with very recent data,” Kelley said. “Additionally, they were able to couple (the uptake) with unmet need. People who are at higher risk of acquiring HIV or who live in high-risk areas for HIV should have greater access to PrEP. They have a greater need for PrEP. What we really need to do from an equity perspective is match the PrEP use with the PrEP need and we have not been successful in doing that.”

Kelley added that the finding that the group that had the highest unmet need for PrEP in the study also had no recorded HIV risk factors. “It’s an interesting time to start thinking about beyond risk factor coverage for PrEP,” she said.

Another issue, Kelley said, is that “people are using (PrEP) but they’re also stopping it. People will need to take PrEP many years for protection, but about half discontinue in the first 6-12 months.

“We need to look at how people will persist on PrEP over the long term. That’s the next frontier,” she said. “We hope the long-acting injectables will help overcome some of the PrEP fatigue. But some may just tire of taking medication repeatedly for an infection they don’t have,” she said.

The study was funded by Gilead Sciences. Tao is employed by and is a shareholder of Gilead Sciences. All relevant financial disclosures have been mitigated, according to the paper. Kelley has research grants to her institution from Gilead, Moderna, Novavax, ViiV, and Humanigen.
 

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

SAVANNAH, GEORGIA — At a pathophysiological level, juvenile myasthenia gravis (MG) seems to be identical to the adult form, neuromuscular specialists learned. But there are still important differences between children and their elders that affect pediatric care.

For example, “we have to think a little bit differently about the side effect profiles of the medications and their toxicity because children may react to medications differently,” said Matthew Ginsberg, MD, a pediatric neurologist based in Akron, Ohio, in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.

And then there’s the matter of adherence. “It’s hard to get adults to take medication, but a teenager is sometimes an exceptional challenge,” Ginsberg said.
 

Case In Point: A 13-Year-Old With MG

Pediatric MG is rare. Cases in children are estimated to account for 10% of MG cases diagnosed each year. According to a 2020 report, “the majority will present with ptosis and a variable degree of ophthalmoplegia [paralysis of eye muscles].”

Ginsberg highlighted a case of a 13-year-old girl who’d been healthy but developed fatigable ptosis and mild restriction of extraocular movements. The patient’s acetylcholine receptor antibodies were very elevated, but she didn’t have MuSK antibodies.

“This isn’t a diagnostic conundrum. She has autoimmune myasthenia gravis with ocular manifestations,” Ginsberg said. “For someone like this, whether it’s an adult or a child, many people would start symptomatic treatment with an acetylcholinesterase inhibitor like pyridostigmine.”

The use of the drug in children is similar to that in adults, he said, although weight-based dosing is used. “Usually it’s around 3-7 mg/kg/d, but it’s still very individualized based on patient response.” The timing of symptoms can affect the distribution of doses throughout the day, he said.

“There are extended-release formulations of the medication, and I think some people use them more than I do,” he said. “The side effects are basically similar to adults. Most of the patients I have on it tolerate it really well and don’t have a lot of the muscarinic side effects that you would expect.”
 

Consider Prescription Eye Drops for Ptosis

Alpha-1A agonists oxymetazoline and apraclonidine in the form of topical eye drops can help with ptosis. “They potentially avoid some of the systemic toxicity of the other medications,” Ginsberg said. “So they might be an option if you’re really just trying to target ptosis as a symptom.”

However, it can be difficult to get insurers to cover these medications, he said.

The 13-year-old patient initially improved but developed difficulty walking. “Her hands began to feel heavy, and she had difficulty chewing and nasal regurgitation. On her exam, she still had fatigable ptosis plus hypernasal speech and generalized weakness. At this point, we’re starting to see that she has generalized myasthenia gravis that may be an impending crisis.”
 

The Young Patient Worsens. Now What?

The patient was admitted and given intravenous immunoglobulin at 2 g/kg over a couple days. But her symptoms worsened following initial improvement.

Glucocorticoids can play a larger role in treatment at this stage, and the patient was initially on prednisone. But there are reasons for caution, including effects on bone growth and interference with live vaccines.

However, live vaccines aren’t common in children, with the exception of the MMRV vaccine, he said. “It’s worth noting that you can give that second dose as early as 3 months after the initial one, so most patients really should be able to complete a course before they start on immunosuppression,” he said.

Another option is immunotherapy. “There’s a really large menu of options for immunotherapy in myasthenia gravis right now,” Ginsberg said. “It’s great that we have all these options, but it adds to the complexity.”

Rituximab may be considered based on early data, he said. And thymectomy — removal of the thymus gland — should be considered early.
 

Don’t Neglect Supportive Care

Ginsberg urged colleagues to consider supportive care measures. Advocacy groups such as the Myasthenia Gravis Foundation of America can help with weight management and diet/exercise counseling, especially in patients taking glucocorticoids.

He added that “school accommodations are very important in this age group. They might need a plan, for example, to have modified gym class or an excuse not to carry a book bag between classes.”

How did the 13-year-old do? She underwent thymectomy, and her disease remained stable after 6 months. “Her rituximab was discontinued,” Ginsberg said. “She considered participating in a clinical trial but then started seeing improvements. About a year after the thymectomy, she just stopped her steroids on her own, and she was fine.”

Ginsberg had no disclosures.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — At a pathophysiological level, juvenile myasthenia gravis (MG) seems to be identical to the adult form, neuromuscular specialists learned. But there are still important differences between children and their elders that affect pediatric care.

For example, “we have to think a little bit differently about the side effect profiles of the medications and their toxicity because children may react to medications differently,” said Matthew Ginsberg, MD, a pediatric neurologist based in Akron, Ohio, in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.

And then there’s the matter of adherence. “It’s hard to get adults to take medication, but a teenager is sometimes an exceptional challenge,” Ginsberg said.
 

Case In Point: A 13-Year-Old With MG

Pediatric MG is rare. Cases in children are estimated to account for 10% of MG cases diagnosed each year. According to a 2020 report, “the majority will present with ptosis and a variable degree of ophthalmoplegia [paralysis of eye muscles].”

Ginsberg highlighted a case of a 13-year-old girl who’d been healthy but developed fatigable ptosis and mild restriction of extraocular movements. The patient’s acetylcholine receptor antibodies were very elevated, but she didn’t have MuSK antibodies.

“This isn’t a diagnostic conundrum. She has autoimmune myasthenia gravis with ocular manifestations,” Ginsberg said. “For someone like this, whether it’s an adult or a child, many people would start symptomatic treatment with an acetylcholinesterase inhibitor like pyridostigmine.”

The use of the drug in children is similar to that in adults, he said, although weight-based dosing is used. “Usually it’s around 3-7 mg/kg/d, but it’s still very individualized based on patient response.” The timing of symptoms can affect the distribution of doses throughout the day, he said.

“There are extended-release formulations of the medication, and I think some people use them more than I do,” he said. “The side effects are basically similar to adults. Most of the patients I have on it tolerate it really well and don’t have a lot of the muscarinic side effects that you would expect.”
 

Consider Prescription Eye Drops for Ptosis

Alpha-1A agonists oxymetazoline and apraclonidine in the form of topical eye drops can help with ptosis. “They potentially avoid some of the systemic toxicity of the other medications,” Ginsberg said. “So they might be an option if you’re really just trying to target ptosis as a symptom.”

However, it can be difficult to get insurers to cover these medications, he said.

The 13-year-old patient initially improved but developed difficulty walking. “Her hands began to feel heavy, and she had difficulty chewing and nasal regurgitation. On her exam, she still had fatigable ptosis plus hypernasal speech and generalized weakness. At this point, we’re starting to see that she has generalized myasthenia gravis that may be an impending crisis.”
 

The Young Patient Worsens. Now What?

The patient was admitted and given intravenous immunoglobulin at 2 g/kg over a couple days. But her symptoms worsened following initial improvement.

Glucocorticoids can play a larger role in treatment at this stage, and the patient was initially on prednisone. But there are reasons for caution, including effects on bone growth and interference with live vaccines.

However, live vaccines aren’t common in children, with the exception of the MMRV vaccine, he said. “It’s worth noting that you can give that second dose as early as 3 months after the initial one, so most patients really should be able to complete a course before they start on immunosuppression,” he said.

Another option is immunotherapy. “There’s a really large menu of options for immunotherapy in myasthenia gravis right now,” Ginsberg said. “It’s great that we have all these options, but it adds to the complexity.”

Rituximab may be considered based on early data, he said. And thymectomy — removal of the thymus gland — should be considered early.
 

Don’t Neglect Supportive Care

Ginsberg urged colleagues to consider supportive care measures. Advocacy groups such as the Myasthenia Gravis Foundation of America can help with weight management and diet/exercise counseling, especially in patients taking glucocorticoids.

He added that “school accommodations are very important in this age group. They might need a plan, for example, to have modified gym class or an excuse not to carry a book bag between classes.”

How did the 13-year-old do? She underwent thymectomy, and her disease remained stable after 6 months. “Her rituximab was discontinued,” Ginsberg said. “She considered participating in a clinical trial but then started seeing improvements. About a year after the thymectomy, she just stopped her steroids on her own, and she was fine.”

Ginsberg had no disclosures.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — At a pathophysiological level, juvenile myasthenia gravis (MG) seems to be identical to the adult form, neuromuscular specialists learned. But there are still important differences between children and their elders that affect pediatric care.

For example, “we have to think a little bit differently about the side effect profiles of the medications and their toxicity because children may react to medications differently,” said Matthew Ginsberg, MD, a pediatric neurologist based in Akron, Ohio, in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.

And then there’s the matter of adherence. “It’s hard to get adults to take medication, but a teenager is sometimes an exceptional challenge,” Ginsberg said.
 

Case In Point: A 13-Year-Old With MG

Pediatric MG is rare. Cases in children are estimated to account for 10% of MG cases diagnosed each year. According to a 2020 report, “the majority will present with ptosis and a variable degree of ophthalmoplegia [paralysis of eye muscles].”

Ginsberg highlighted a case of a 13-year-old girl who’d been healthy but developed fatigable ptosis and mild restriction of extraocular movements. The patient’s acetylcholine receptor antibodies were very elevated, but she didn’t have MuSK antibodies.

“This isn’t a diagnostic conundrum. She has autoimmune myasthenia gravis with ocular manifestations,” Ginsberg said. “For someone like this, whether it’s an adult or a child, many people would start symptomatic treatment with an acetylcholinesterase inhibitor like pyridostigmine.”

The use of the drug in children is similar to that in adults, he said, although weight-based dosing is used. “Usually it’s around 3-7 mg/kg/d, but it’s still very individualized based on patient response.” The timing of symptoms can affect the distribution of doses throughout the day, he said.

“There are extended-release formulations of the medication, and I think some people use them more than I do,” he said. “The side effects are basically similar to adults. Most of the patients I have on it tolerate it really well and don’t have a lot of the muscarinic side effects that you would expect.”
 

Consider Prescription Eye Drops for Ptosis

Alpha-1A agonists oxymetazoline and apraclonidine in the form of topical eye drops can help with ptosis. “They potentially avoid some of the systemic toxicity of the other medications,” Ginsberg said. “So they might be an option if you’re really just trying to target ptosis as a symptom.”

However, it can be difficult to get insurers to cover these medications, he said.

The 13-year-old patient initially improved but developed difficulty walking. “Her hands began to feel heavy, and she had difficulty chewing and nasal regurgitation. On her exam, she still had fatigable ptosis plus hypernasal speech and generalized weakness. At this point, we’re starting to see that she has generalized myasthenia gravis that may be an impending crisis.”
 

The Young Patient Worsens. Now What?

The patient was admitted and given intravenous immunoglobulin at 2 g/kg over a couple days. But her symptoms worsened following initial improvement.

Glucocorticoids can play a larger role in treatment at this stage, and the patient was initially on prednisone. But there are reasons for caution, including effects on bone growth and interference with live vaccines.

However, live vaccines aren’t common in children, with the exception of the MMRV vaccine, he said. “It’s worth noting that you can give that second dose as early as 3 months after the initial one, so most patients really should be able to complete a course before they start on immunosuppression,” he said.

Another option is immunotherapy. “There’s a really large menu of options for immunotherapy in myasthenia gravis right now,” Ginsberg said. “It’s great that we have all these options, but it adds to the complexity.”

Rituximab may be considered based on early data, he said. And thymectomy — removal of the thymus gland — should be considered early.
 

Don’t Neglect Supportive Care

Ginsberg urged colleagues to consider supportive care measures. Advocacy groups such as the Myasthenia Gravis Foundation of America can help with weight management and diet/exercise counseling, especially in patients taking glucocorticoids.

He added that “school accommodations are very important in this age group. They might need a plan, for example, to have modified gym class or an excuse not to carry a book bag between classes.”

How did the 13-year-old do? She underwent thymectomy, and her disease remained stable after 6 months. “Her rituximab was discontinued,” Ginsberg said. “She considered participating in a clinical trial but then started seeing improvements. About a year after the thymectomy, she just stopped her steroids on her own, and she was fine.”

Ginsberg had no disclosures.
 

A version of this article appeared on Medscape.com.

Photodynamic therapy (PDT) — a treatment most commonly thought of for field cancerization — is an effective tool for reducing rhytides and lentigines, results from a small prospective study showed.

“Our study helps capture and quantify a phenomenon that clinicians who use PDT in their practice have already noticed: Patients experience a visible improvement across several cosmetically important metrics including but not limited to fine lines, wrinkles, and skin tightness following PDT,” one of the study authors, Luke Horton, MD, a fourth-year dermatology resident at the University of California, Irvine, said in an interview following the annual meeting of the American Society for Dermatologic Surgery, where he presented the results during an oral abstract session.

Dr. Horton

For the study, 11 patients underwent a 120-minute incubation period with 17% 5-aminolevulinic acid over the face, followed by visible blue light PDT exposure for 16 minutes, to reduce rhytides. The researchers used a Vectra imaging system to capture three-dimensional images of the patients before the procedure and during the follow-up. Three dermatologists analyzed the pre-procedure and post-procedure images and used a validated five-point Merz wrinkle severity scale to grade various regions of the face including the forehead, glabella, lateral canthal rhytides, melolabial folds, nasolabial folds, and perioral rhytides.

They also used a five-point solar lentigines scale to evaluate the change in degree of pigmentation and quantity of age spots as well as the change in rhytid severity before and after PDT and the change in the seven-point Global Aesthetic Improvement Scale (GAIS) to gauge overall improvement of fine lines and wrinkles.

After a mean follow-up of 4.25 months, rhytid severity among the 11 patients was reduced by an average of 0.65 points on the Merz scale, with an SD of 0.20. Broken down by region, rhytid severity scores decreased by 0.2 points (SD, 0.42) for the forehead, 0.7 points (SD, 0.48) for the glabella and lateral canthal rhytides, 0.88 points (SD, 0.35) for the melolabial folds and perioral rhytides, and 0.8 points (SD, 0.42) for the nasolabial folds. (The researchers excluded ratings for the melolabial folds and perioral rhytides in two patients with beards.)

In other findings, solar lentigines grading showed an average reduction of 1 point (SD, 0.45), while the GAIS score improved by 1 or more for every patient, with an average of score of 1.45 (SD, 0.52), showing that some degree of improvement in facial rhytides was noted for all patients following PDT.

“The degree of improvement as measured by our independent physician graders was impressive and not far off from those reported with CO₂ ablative laser,” Horton said. “Further, the effect was not isolated to actinic keratoses but extended to improved appearance of fine lines, some deep lines, and lentigines. Although we are not implying that PDT is superior to and should replace lasers or other energy-based devices, it does provide a real, measurable cosmetic benefit.”

Clinicians, he added, can use these findings “to counsel their patients when discussing field cancerization treatment options, especially for patients who may be hesitant to undergo PDT as it can be a painful therapy with a considerable downtime for some.”

Lawrence J. Green, MD, clinical professor of dermatology, The George Washington University, Washington, DC, who was asked to comment on the study results, said that the findings “shine more light on the long-standing off-label use of PDT for lessening signs of photoaging. Like studies done before it, I think this adds an additional benefit to discuss for those who are considering PDT treatment for their actinic keratoses.”

Horton acknowledged certain limitations of the study including its small sample size and the fact that physician graders were not blinded to which images were pre- and post-treatment, “which could introduce an element of bias in the data,” he said. “But this being an unfunded project born out of clinical observation, we hope to later expand its size. Furthermore, we invite other physicians to join us to better study these effects and to design protocols that minimize adverse effects and maximize clinical outcomes.”

His co-authors were Milan Hirpara; Sarah Choe; Joel Cohen, MD; and Natasha A. Mesinkovska, MD, PhD.

No relevant disclosures were reported. Green had no relevant disclosures.

A version of this article appeared on Medscape.com.

Photodynamic therapy (PDT) — a treatment most commonly thought of for field cancerization — is an effective tool for reducing rhytides and lentigines, results from a small prospective study showed.

“Our study helps capture and quantify a phenomenon that clinicians who use PDT in their practice have already noticed: Patients experience a visible improvement across several cosmetically important metrics including but not limited to fine lines, wrinkles, and skin tightness following PDT,” one of the study authors, Luke Horton, MD, a fourth-year dermatology resident at the University of California, Irvine, said in an interview following the annual meeting of the American Society for Dermatologic Surgery, where he presented the results during an oral abstract session.

Dr. Horton

For the study, 11 patients underwent a 120-minute incubation period with 17% 5-aminolevulinic acid over the face, followed by visible blue light PDT exposure for 16 minutes, to reduce rhytides. The researchers used a Vectra imaging system to capture three-dimensional images of the patients before the procedure and during the follow-up. Three dermatologists analyzed the pre-procedure and post-procedure images and used a validated five-point Merz wrinkle severity scale to grade various regions of the face including the forehead, glabella, lateral canthal rhytides, melolabial folds, nasolabial folds, and perioral rhytides.

They also used a five-point solar lentigines scale to evaluate the change in degree of pigmentation and quantity of age spots as well as the change in rhytid severity before and after PDT and the change in the seven-point Global Aesthetic Improvement Scale (GAIS) to gauge overall improvement of fine lines and wrinkles.

After a mean follow-up of 4.25 months, rhytid severity among the 11 patients was reduced by an average of 0.65 points on the Merz scale, with an SD of 0.20. Broken down by region, rhytid severity scores decreased by 0.2 points (SD, 0.42) for the forehead, 0.7 points (SD, 0.48) for the glabella and lateral canthal rhytides, 0.88 points (SD, 0.35) for the melolabial folds and perioral rhytides, and 0.8 points (SD, 0.42) for the nasolabial folds. (The researchers excluded ratings for the melolabial folds and perioral rhytides in two patients with beards.)

In other findings, solar lentigines grading showed an average reduction of 1 point (SD, 0.45), while the GAIS score improved by 1 or more for every patient, with an average of score of 1.45 (SD, 0.52), showing that some degree of improvement in facial rhytides was noted for all patients following PDT.

“The degree of improvement as measured by our independent physician graders was impressive and not far off from those reported with CO₂ ablative laser,” Horton said. “Further, the effect was not isolated to actinic keratoses but extended to improved appearance of fine lines, some deep lines, and lentigines. Although we are not implying that PDT is superior to and should replace lasers or other energy-based devices, it does provide a real, measurable cosmetic benefit.”

Clinicians, he added, can use these findings “to counsel their patients when discussing field cancerization treatment options, especially for patients who may be hesitant to undergo PDT as it can be a painful therapy with a considerable downtime for some.”

Lawrence J. Green, MD, clinical professor of dermatology, The George Washington University, Washington, DC, who was asked to comment on the study results, said that the findings “shine more light on the long-standing off-label use of PDT for lessening signs of photoaging. Like studies done before it, I think this adds an additional benefit to discuss for those who are considering PDT treatment for their actinic keratoses.”

Horton acknowledged certain limitations of the study including its small sample size and the fact that physician graders were not blinded to which images were pre- and post-treatment, “which could introduce an element of bias in the data,” he said. “But this being an unfunded project born out of clinical observation, we hope to later expand its size. Furthermore, we invite other physicians to join us to better study these effects and to design protocols that minimize adverse effects and maximize clinical outcomes.”

His co-authors were Milan Hirpara; Sarah Choe; Joel Cohen, MD; and Natasha A. Mesinkovska, MD, PhD.

No relevant disclosures were reported. Green had no relevant disclosures.

A version of this article appeared on Medscape.com.

Photodynamic therapy (PDT) — a treatment most commonly thought of for field cancerization — is an effective tool for reducing rhytides and lentigines, results from a small prospective study showed.

“Our study helps capture and quantify a phenomenon that clinicians who use PDT in their practice have already noticed: Patients experience a visible improvement across several cosmetically important metrics including but not limited to fine lines, wrinkles, and skin tightness following PDT,” one of the study authors, Luke Horton, MD, a fourth-year dermatology resident at the University of California, Irvine, said in an interview following the annual meeting of the American Society for Dermatologic Surgery, where he presented the results during an oral abstract session.

Dr. Horton

For the study, 11 patients underwent a 120-minute incubation period with 17% 5-aminolevulinic acid over the face, followed by visible blue light PDT exposure for 16 minutes, to reduce rhytides. The researchers used a Vectra imaging system to capture three-dimensional images of the patients before the procedure and during the follow-up. Three dermatologists analyzed the pre-procedure and post-procedure images and used a validated five-point Merz wrinkle severity scale to grade various regions of the face including the forehead, glabella, lateral canthal rhytides, melolabial folds, nasolabial folds, and perioral rhytides.

They also used a five-point solar lentigines scale to evaluate the change in degree of pigmentation and quantity of age spots as well as the change in rhytid severity before and after PDT and the change in the seven-point Global Aesthetic Improvement Scale (GAIS) to gauge overall improvement of fine lines and wrinkles.

After a mean follow-up of 4.25 months, rhytid severity among the 11 patients was reduced by an average of 0.65 points on the Merz scale, with an SD of 0.20. Broken down by region, rhytid severity scores decreased by 0.2 points (SD, 0.42) for the forehead, 0.7 points (SD, 0.48) for the glabella and lateral canthal rhytides, 0.88 points (SD, 0.35) for the melolabial folds and perioral rhytides, and 0.8 points (SD, 0.42) for the nasolabial folds. (The researchers excluded ratings for the melolabial folds and perioral rhytides in two patients with beards.)

In other findings, solar lentigines grading showed an average reduction of 1 point (SD, 0.45), while the GAIS score improved by 1 or more for every patient, with an average of score of 1.45 (SD, 0.52), showing that some degree of improvement in facial rhytides was noted for all patients following PDT.

“The degree of improvement as measured by our independent physician graders was impressive and not far off from those reported with CO₂ ablative laser,” Horton said. “Further, the effect was not isolated to actinic keratoses but extended to improved appearance of fine lines, some deep lines, and lentigines. Although we are not implying that PDT is superior to and should replace lasers or other energy-based devices, it does provide a real, measurable cosmetic benefit.”

Clinicians, he added, can use these findings “to counsel their patients when discussing field cancerization treatment options, especially for patients who may be hesitant to undergo PDT as it can be a painful therapy with a considerable downtime for some.”

Lawrence J. Green, MD, clinical professor of dermatology, The George Washington University, Washington, DC, who was asked to comment on the study results, said that the findings “shine more light on the long-standing off-label use of PDT for lessening signs of photoaging. Like studies done before it, I think this adds an additional benefit to discuss for those who are considering PDT treatment for their actinic keratoses.”

Horton acknowledged certain limitations of the study including its small sample size and the fact that physician graders were not blinded to which images were pre- and post-treatment, “which could introduce an element of bias in the data,” he said. “But this being an unfunded project born out of clinical observation, we hope to later expand its size. Furthermore, we invite other physicians to join us to better study these effects and to design protocols that minimize adverse effects and maximize clinical outcomes.”

His co-authors were Milan Hirpara; Sarah Choe; Joel Cohen, MD; and Natasha A. Mesinkovska, MD, PhD.

No relevant disclosures were reported. Green had no relevant disclosures.

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — A new clinical education tool aims to speed the diagnosis of amyotrophic lateral sclerosis (ALS), which often goes undetected for months even in neurologist offices.

The one-page “thinkALS” tool, designed for clinicians who don’t specialize in neuromuscular disorders, offers a guide to recognize ALS symptoms and determine when it’s time to refer patients to ALS clinics.

“Time is of the essence. It’s really important because the paradigm of looking at ALS is shifting from this being a fatal disease that nobody can do anything about,” said Suma Babu, MBBS, MPH, assistant professor of neurology at Massachusetts General Hospital/Harvard Medical School in Boston, in a presentation at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024. “As a community, we need to think about how can get to the diagnosis point early and get patients started on therapies.”
 

On Average, ALS Diagnosis Takes 12-15 Months

As Babu noted, the percentage of patients initially diagnosed with something else may be as high as 52%. The time to diagnosis in ALS remained steady at a mean 12-15 months from 1996-1998 to 2000-2018.

“If you keep in mind that an average ALS patient lives only 3-5 years from symptom onset, they’re spending one third of their survival time in just trying to figure out what the diagnosis is,” Babu said. “Often, they may even undergo unnecessary testing and unnecessary surgeries — carpal tunnel releases, spinal surgeries, and so on.”

Babu’s own research, which is under review for publication, examined 2011-2021 Medicare claims to determine the typical time from first neurologist consult to confirmed ALS diagnosis. The mean for ALS/neuromuscular specialists is 9.6 months, while it’s 16.7 months for nonspecialist neurologists.

“It’s a hard pill to swallow,” Babu said, referring to the fact that neurologists are contributing to some of this situation. “But it is a challenge because ALS does not have a definitive diagnostic test, and you’re ruling out other possibilities.”
 

A ‘Sense of Nihilism’ About Prognoses

She added that “unless you’re seeing a lot of ALS patients, this is not going to be on a neurologist’s or a nurse practitioner’s radar to think about ALS early and then refer them to the right place.”

There’s also an unwarranted “sense of nihilism” about prognoses for patients, she said. “Sometimes people do not understand what’s going on within the ALS field in terms of ‘What are we going to do about it if it’s diagnosed?’ ”

The new one-page tool will be helpful in making diagnoses, she said. “If you have a patient who has asymmetric, progressive weakness, there is an instrument you can turn to that will walk you through the most common symptoms. It’ll also walk you through what to do next.”

The tool lists features of ALS and factors that support — or don’t support — an ALS diagnosis. Users are told to “think ALS” if features in two categories are present and no features in a third category are present.
 

Referral Wording Is Crucial

Babu added that the “important key feature of this instrument” is guidance for non-neurologists regarding what to write on a referral to neurology so the patient is channeled directly to an ALS clinic. The recommended wording: “CLINICAL SUSPICION FOR ALS.”

Neurologist Ximena Arcila-Londono, MD, of Henry Ford Health in Detroit, spoke after Babu’s presentation and agreed that wording is crucial in referrals. “Please include in your words ‘Rule out motor neuron disorder’ or ‘Rule out ALS,’ ” she said. “Some people in the community are very reluctant to use those words in their referral. If you don’t use the referral and you send them [regarding] weakness, that person is going to get stuck in the general neurology pile. The moment you use the word ‘motor neuron disorder’ or ALS, most of us will get to those patients within a month.”

The tool’s wording adds that “most ALS centers can accommodate urgent ALS referrals within 2 weeks.”

Babu disclosed receiving research funding from the AANEM Foundation, American Academy of Neurology, Muscular Dystrophy Association, OrphAI, Biogen, Ionis, Novartis, Denali, uniQure, and MarvelBiome. Arcila-Londono had no disclosures.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — A new clinical education tool aims to speed the diagnosis of amyotrophic lateral sclerosis (ALS), which often goes undetected for months even in neurologist offices.

The one-page “thinkALS” tool, designed for clinicians who don’t specialize in neuromuscular disorders, offers a guide to recognize ALS symptoms and determine when it’s time to refer patients to ALS clinics.

“Time is of the essence. It’s really important because the paradigm of looking at ALS is shifting from this being a fatal disease that nobody can do anything about,” said Suma Babu, MBBS, MPH, assistant professor of neurology at Massachusetts General Hospital/Harvard Medical School in Boston, in a presentation at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024. “As a community, we need to think about how can get to the diagnosis point early and get patients started on therapies.”
 

On Average, ALS Diagnosis Takes 12-15 Months

As Babu noted, the percentage of patients initially diagnosed with something else may be as high as 52%. The time to diagnosis in ALS remained steady at a mean 12-15 months from 1996-1998 to 2000-2018.

“If you keep in mind that an average ALS patient lives only 3-5 years from symptom onset, they’re spending one third of their survival time in just trying to figure out what the diagnosis is,” Babu said. “Often, they may even undergo unnecessary testing and unnecessary surgeries — carpal tunnel releases, spinal surgeries, and so on.”

Babu’s own research, which is under review for publication, examined 2011-2021 Medicare claims to determine the typical time from first neurologist consult to confirmed ALS diagnosis. The mean for ALS/neuromuscular specialists is 9.6 months, while it’s 16.7 months for nonspecialist neurologists.

“It’s a hard pill to swallow,” Babu said, referring to the fact that neurologists are contributing to some of this situation. “But it is a challenge because ALS does not have a definitive diagnostic test, and you’re ruling out other possibilities.”
 

A ‘Sense of Nihilism’ About Prognoses

She added that “unless you’re seeing a lot of ALS patients, this is not going to be on a neurologist’s or a nurse practitioner’s radar to think about ALS early and then refer them to the right place.”

There’s also an unwarranted “sense of nihilism” about prognoses for patients, she said. “Sometimes people do not understand what’s going on within the ALS field in terms of ‘What are we going to do about it if it’s diagnosed?’ ”

The new one-page tool will be helpful in making diagnoses, she said. “If you have a patient who has asymmetric, progressive weakness, there is an instrument you can turn to that will walk you through the most common symptoms. It’ll also walk you through what to do next.”

The tool lists features of ALS and factors that support — or don’t support — an ALS diagnosis. Users are told to “think ALS” if features in two categories are present and no features in a third category are present.
 

Referral Wording Is Crucial

Babu added that the “important key feature of this instrument” is guidance for non-neurologists regarding what to write on a referral to neurology so the patient is channeled directly to an ALS clinic. The recommended wording: “CLINICAL SUSPICION FOR ALS.”

Neurologist Ximena Arcila-Londono, MD, of Henry Ford Health in Detroit, spoke after Babu’s presentation and agreed that wording is crucial in referrals. “Please include in your words ‘Rule out motor neuron disorder’ or ‘Rule out ALS,’ ” she said. “Some people in the community are very reluctant to use those words in their referral. If you don’t use the referral and you send them [regarding] weakness, that person is going to get stuck in the general neurology pile. The moment you use the word ‘motor neuron disorder’ or ALS, most of us will get to those patients within a month.”

The tool’s wording adds that “most ALS centers can accommodate urgent ALS referrals within 2 weeks.”

Babu disclosed receiving research funding from the AANEM Foundation, American Academy of Neurology, Muscular Dystrophy Association, OrphAI, Biogen, Ionis, Novartis, Denali, uniQure, and MarvelBiome. Arcila-Londono had no disclosures.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — A new clinical education tool aims to speed the diagnosis of amyotrophic lateral sclerosis (ALS), which often goes undetected for months even in neurologist offices.

The one-page “thinkALS” tool, designed for clinicians who don’t specialize in neuromuscular disorders, offers a guide to recognize ALS symptoms and determine when it’s time to refer patients to ALS clinics.

“Time is of the essence. It’s really important because the paradigm of looking at ALS is shifting from this being a fatal disease that nobody can do anything about,” said Suma Babu, MBBS, MPH, assistant professor of neurology at Massachusetts General Hospital/Harvard Medical School in Boston, in a presentation at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024. “As a community, we need to think about how can get to the diagnosis point early and get patients started on therapies.”
 

On Average, ALS Diagnosis Takes 12-15 Months

As Babu noted, the percentage of patients initially diagnosed with something else may be as high as 52%. The time to diagnosis in ALS remained steady at a mean 12-15 months from 1996-1998 to 2000-2018.

“If you keep in mind that an average ALS patient lives only 3-5 years from symptom onset, they’re spending one third of their survival time in just trying to figure out what the diagnosis is,” Babu said. “Often, they may even undergo unnecessary testing and unnecessary surgeries — carpal tunnel releases, spinal surgeries, and so on.”

Babu’s own research, which is under review for publication, examined 2011-2021 Medicare claims to determine the typical time from first neurologist consult to confirmed ALS diagnosis. The mean for ALS/neuromuscular specialists is 9.6 months, while it’s 16.7 months for nonspecialist neurologists.

“It’s a hard pill to swallow,” Babu said, referring to the fact that neurologists are contributing to some of this situation. “But it is a challenge because ALS does not have a definitive diagnostic test, and you’re ruling out other possibilities.”
 

A ‘Sense of Nihilism’ About Prognoses

She added that “unless you’re seeing a lot of ALS patients, this is not going to be on a neurologist’s or a nurse practitioner’s radar to think about ALS early and then refer them to the right place.”

There’s also an unwarranted “sense of nihilism” about prognoses for patients, she said. “Sometimes people do not understand what’s going on within the ALS field in terms of ‘What are we going to do about it if it’s diagnosed?’ ”

The new one-page tool will be helpful in making diagnoses, she said. “If you have a patient who has asymmetric, progressive weakness, there is an instrument you can turn to that will walk you through the most common symptoms. It’ll also walk you through what to do next.”

The tool lists features of ALS and factors that support — or don’t support — an ALS diagnosis. Users are told to “think ALS” if features in two categories are present and no features in a third category are present.
 

Referral Wording Is Crucial

Babu added that the “important key feature of this instrument” is guidance for non-neurologists regarding what to write on a referral to neurology so the patient is channeled directly to an ALS clinic. The recommended wording: “CLINICAL SUSPICION FOR ALS.”

Neurologist Ximena Arcila-Londono, MD, of Henry Ford Health in Detroit, spoke after Babu’s presentation and agreed that wording is crucial in referrals. “Please include in your words ‘Rule out motor neuron disorder’ or ‘Rule out ALS,’ ” she said. “Some people in the community are very reluctant to use those words in their referral. If you don’t use the referral and you send them [regarding] weakness, that person is going to get stuck in the general neurology pile. The moment you use the word ‘motor neuron disorder’ or ALS, most of us will get to those patients within a month.”

The tool’s wording adds that “most ALS centers can accommodate urgent ALS referrals within 2 weeks.”

Babu disclosed receiving research funding from the AANEM Foundation, American Academy of Neurology, Muscular Dystrophy Association, OrphAI, Biogen, Ionis, Novartis, Denali, uniQure, and MarvelBiome. Arcila-Londono had no disclosures.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Heart problems are common in the vast majority of neuromuscular disorders, and cardiac monitoring of patients is crucial, even at younger ages, a neurologist told an audience of nerve/muscle specialists.

The cardiac conditions can range from asymptomatic to potentially lethal, Nicholas J. Silvestri, MD, professor of neurology at the Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, in New York, said in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024. “It’s really important to know when to do tests and refer to cardiology, and it’s really important to find a cardiologist who can work in concert in taking care of these patients.”
 

Protein Alterations May Disrupt Heart Muscles

In muscular dystrophies, a prevailing theory suggests that alterations to proteins such as dystrophin disrupt structural integrity in both muscle and cardiac cells, he said.

In Duchenne muscular dystrophy (DMD), cardiomyopathy, cardiac conduction abnormalities, or both usually appear in patients by age 10. “It’s important to know that it’s probably present to some degree before that, and it’s not going to get better over time,” he said.

Cardiac problems are universal in DMD by age 18, he said. “Men and boys are living longer, so they have the opportunity to develop the cardiac abnormalities that accrue with time.” Conduction abnormalities typically appear first. “In a lot of these boys, you’ll typically see persistent sinus tachycardia. But they can also develop atrial arrhythmias and bundle branch blocks.”

Sudden cardiac death is responsible for mortality in an estimated 15% patients with DMD. “Very sadly, I lost a patient this way just a few months ago,” Silvestri said.
 

ECGs and Echos Are Recommended

Screening is crucial. “Make sure that patients get that referral and get these tests done,” he said. “You need an ECG and echo by diagnosis or age 6. This is usually repeated annually or biannually, typically by the cardiologist you’re working with.”

The good news is that there’s evidence of survival benefits from treatment with angiotensin-converting enzyme inhibitors for dilated cardiomyopathy. “Some cardiac experts feel treatment with angiotensin receptor blockers (ARBs) is equivalent.”

Most boys will get echocardiograms, he said, “but there’s a lot of evidence showing that cardiac MRI is probably preferable for a number of reasons,” including better visualization. But the need for sedation limits access, he said, and cardiac MRI may not be available at some facilities.
 

Worse Outcomes in Becker Muscular Dystrophy (BMD)

Cardiac involvement is more common and more severe in BMD than in DMD. About 50% of deaths in BMD are attributed to malignant arrhythmias or congestive heart failure, he said.

Screening requirements and treatment options in BMD are similar to those in DMD, with the added option of heart transplantation.

Silvestri cautioned that up to 40% of female carriers of dystrophin mutations can develop cardiac dysfunction similar to that seen in DMD and BMD. Cardiac assessments are recommended every 5 years. “It’s important to genotype Mom,” he said, especially in light of the fact that two thirds of DMD cases may be inherited.

“When I send genetic testing on the mother and find her to be a carrier, I send her to a cardiologist so she has the appropriate screening done,” he said.
 

Pacemakers May Be Considered in Type 1 Myotonic Dystrophy

In type 1 myotonic dystrophy, cardiac conduction abnormalities are seen in two thirds of patients, and sudden cardiac death in up to 30% of patients. “When it is diagnosed, patients do need an ECG at that time, as well as annually,” he said.

Holter monitoring or implantable loop recorders may be recommended, and permanent pacing via an implantable cardioverter-defibrillator might be appropriate.

“Based on the literature to date, the exact timing is not is not clear,” Silvestri said. “The electrophysiologists in my area tend to be very aggressive, thankfully, and treat them fairly soon with pacemakers when we see the first sign of trouble.”

Silvestri disclosed consultant/advisory relationships with argenx, Alexion, Amgen, UCB, Immunovant, and Janssen.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Heart problems are common in the vast majority of neuromuscular disorders, and cardiac monitoring of patients is crucial, even at younger ages, a neurologist told an audience of nerve/muscle specialists.

The cardiac conditions can range from asymptomatic to potentially lethal, Nicholas J. Silvestri, MD, professor of neurology at the Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, in New York, said in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024. “It’s really important to know when to do tests and refer to cardiology, and it’s really important to find a cardiologist who can work in concert in taking care of these patients.”
 

Protein Alterations May Disrupt Heart Muscles

In muscular dystrophies, a prevailing theory suggests that alterations to proteins such as dystrophin disrupt structural integrity in both muscle and cardiac cells, he said.

In Duchenne muscular dystrophy (DMD), cardiomyopathy, cardiac conduction abnormalities, or both usually appear in patients by age 10. “It’s important to know that it’s probably present to some degree before that, and it’s not going to get better over time,” he said.

Cardiac problems are universal in DMD by age 18, he said. “Men and boys are living longer, so they have the opportunity to develop the cardiac abnormalities that accrue with time.” Conduction abnormalities typically appear first. “In a lot of these boys, you’ll typically see persistent sinus tachycardia. But they can also develop atrial arrhythmias and bundle branch blocks.”

Sudden cardiac death is responsible for mortality in an estimated 15% patients with DMD. “Very sadly, I lost a patient this way just a few months ago,” Silvestri said.
 

ECGs and Echos Are Recommended

Screening is crucial. “Make sure that patients get that referral and get these tests done,” he said. “You need an ECG and echo by diagnosis or age 6. This is usually repeated annually or biannually, typically by the cardiologist you’re working with.”

The good news is that there’s evidence of survival benefits from treatment with angiotensin-converting enzyme inhibitors for dilated cardiomyopathy. “Some cardiac experts feel treatment with angiotensin receptor blockers (ARBs) is equivalent.”

Most boys will get echocardiograms, he said, “but there’s a lot of evidence showing that cardiac MRI is probably preferable for a number of reasons,” including better visualization. But the need for sedation limits access, he said, and cardiac MRI may not be available at some facilities.
 

Worse Outcomes in Becker Muscular Dystrophy (BMD)

Cardiac involvement is more common and more severe in BMD than in DMD. About 50% of deaths in BMD are attributed to malignant arrhythmias or congestive heart failure, he said.

Screening requirements and treatment options in BMD are similar to those in DMD, with the added option of heart transplantation.

Silvestri cautioned that up to 40% of female carriers of dystrophin mutations can develop cardiac dysfunction similar to that seen in DMD and BMD. Cardiac assessments are recommended every 5 years. “It’s important to genotype Mom,” he said, especially in light of the fact that two thirds of DMD cases may be inherited.

“When I send genetic testing on the mother and find her to be a carrier, I send her to a cardiologist so she has the appropriate screening done,” he said.
 

Pacemakers May Be Considered in Type 1 Myotonic Dystrophy

In type 1 myotonic dystrophy, cardiac conduction abnormalities are seen in two thirds of patients, and sudden cardiac death in up to 30% of patients. “When it is diagnosed, patients do need an ECG at that time, as well as annually,” he said.

Holter monitoring or implantable loop recorders may be recommended, and permanent pacing via an implantable cardioverter-defibrillator might be appropriate.

“Based on the literature to date, the exact timing is not is not clear,” Silvestri said. “The electrophysiologists in my area tend to be very aggressive, thankfully, and treat them fairly soon with pacemakers when we see the first sign of trouble.”

Silvestri disclosed consultant/advisory relationships with argenx, Alexion, Amgen, UCB, Immunovant, and Janssen.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Heart problems are common in the vast majority of neuromuscular disorders, and cardiac monitoring of patients is crucial, even at younger ages, a neurologist told an audience of nerve/muscle specialists.

The cardiac conditions can range from asymptomatic to potentially lethal, Nicholas J. Silvestri, MD, professor of neurology at the Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, in New York, said in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024. “It’s really important to know when to do tests and refer to cardiology, and it’s really important to find a cardiologist who can work in concert in taking care of these patients.”
 

Protein Alterations May Disrupt Heart Muscles

In muscular dystrophies, a prevailing theory suggests that alterations to proteins such as dystrophin disrupt structural integrity in both muscle and cardiac cells, he said.

In Duchenne muscular dystrophy (DMD), cardiomyopathy, cardiac conduction abnormalities, or both usually appear in patients by age 10. “It’s important to know that it’s probably present to some degree before that, and it’s not going to get better over time,” he said.

Cardiac problems are universal in DMD by age 18, he said. “Men and boys are living longer, so they have the opportunity to develop the cardiac abnormalities that accrue with time.” Conduction abnormalities typically appear first. “In a lot of these boys, you’ll typically see persistent sinus tachycardia. But they can also develop atrial arrhythmias and bundle branch blocks.”

Sudden cardiac death is responsible for mortality in an estimated 15% patients with DMD. “Very sadly, I lost a patient this way just a few months ago,” Silvestri said.
 

ECGs and Echos Are Recommended

Screening is crucial. “Make sure that patients get that referral and get these tests done,” he said. “You need an ECG and echo by diagnosis or age 6. This is usually repeated annually or biannually, typically by the cardiologist you’re working with.”

The good news is that there’s evidence of survival benefits from treatment with angiotensin-converting enzyme inhibitors for dilated cardiomyopathy. “Some cardiac experts feel treatment with angiotensin receptor blockers (ARBs) is equivalent.”

Most boys will get echocardiograms, he said, “but there’s a lot of evidence showing that cardiac MRI is probably preferable for a number of reasons,” including better visualization. But the need for sedation limits access, he said, and cardiac MRI may not be available at some facilities.
 

Worse Outcomes in Becker Muscular Dystrophy (BMD)

Cardiac involvement is more common and more severe in BMD than in DMD. About 50% of deaths in BMD are attributed to malignant arrhythmias or congestive heart failure, he said.

Screening requirements and treatment options in BMD are similar to those in DMD, with the added option of heart transplantation.

Silvestri cautioned that up to 40% of female carriers of dystrophin mutations can develop cardiac dysfunction similar to that seen in DMD and BMD. Cardiac assessments are recommended every 5 years. “It’s important to genotype Mom,” he said, especially in light of the fact that two thirds of DMD cases may be inherited.

“When I send genetic testing on the mother and find her to be a carrier, I send her to a cardiologist so she has the appropriate screening done,” he said.
 

Pacemakers May Be Considered in Type 1 Myotonic Dystrophy

In type 1 myotonic dystrophy, cardiac conduction abnormalities are seen in two thirds of patients, and sudden cardiac death in up to 30% of patients. “When it is diagnosed, patients do need an ECG at that time, as well as annually,” he said.

Holter monitoring or implantable loop recorders may be recommended, and permanent pacing via an implantable cardioverter-defibrillator might be appropriate.

“Based on the literature to date, the exact timing is not is not clear,” Silvestri said. “The electrophysiologists in my area tend to be very aggressive, thankfully, and treat them fairly soon with pacemakers when we see the first sign of trouble.”

Silvestri disclosed consultant/advisory relationships with argenx, Alexion, Amgen, UCB, Immunovant, and Janssen.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Current disease-modifying therapies for amyotrophic lateral sclerosis (ALS) don’t extend lifespans by much, but several drug options are available, nerve specialists learned.

The glutamate blocker riluzole (Rilutek), which became the first ALS drug to receive US Food and Drug Administration (FDA) approval in 1995, continues to be used, Michael D. Weiss, MD, professor of neurology at University of Washington School of Medicine, Seattle, said in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.

Weiss highlighted a 2012 Cochrane Library review that examined research and found the drug is “reasonably safe” and prolongs median survival by about 2-3 months. “About 12% develop liver disease. It’s pretty rare that we stop the medicine due to liver toxicity.”
 

Earlier Treatment Could Pay Dividends

A recent study “suggests we might be able to get more bang for our buck from riluzole” by initiating treatment earlier, Weiss said.

Researchers tracked 4778 patients with ALS, including 3446 (72.1%) who took riluzole. Those who took the drug survived a median 2 extra months (22.6 vs 20.2 months; P < .001). The data suggested that delaying riluzole initiation by 1 year (from 6 months to 18 months after diagnosis) reduced the median survival by 1.9 months (from 40.1 to 38.2 months).

There’s “a relatively significant additional benefit” to earlier treatment, Weiss said, although patients will vary on whether they think it’s meaningful. As for limitations, “there’s no clear impact on disease progression, and there’s a need for periodic monitoring of liver function profile.” 

He added that there’s an out-of-pocket co-pay. “Even as a generic, it’s not that cheap. Depending on the source, it could cost anywhere from $1800 to $8400 a year.”
 

Edaravone Could Lack Relevant Benefit

No other ALS treatment appeared until 2017, when the FDA approved the novel antioxidant edaravone (Radicava). In 2022, the agency approved an oral suspension version, but a study published that year suggested there may not be a clinically relevant benefit.

The University of Washington, where Weiss works, offered the drug to 144 patients, according to an analysis. Eighty percent of the patients wanted it, but insurers refused to cover it for more than 20%. The average time to treatment with the drug was 28 days after patients said they wanted it.

That’s a “substantial delay,” Weiss said.

The cost is about $171,000 a year, he said, and assistance is limited for underinsured patients.*

Other Options

As Weiss noted, another drug, AMX0035 (Relyvrio), received FDA approval in 2022, but its manufacturer pulled it from the US/Canada market in April 2024 following poor phase 3 trial findings.

In 2023, the FDA approved another drug, the antisense oligonucleotide tofersen (Qalsody), in patients with ALS associated with a mutation in the superoxide dismutase 1 gene. According to the FDA, reductions in plasma neurofilament light concentration were “reasonably likely to predict a clinical benefit in patients.”

Only 1%-2% of patients with ALS fit the criteria to get the drug, Weiss said. He noted other limitations such as the cost ($180,000 a year), the need for lifelong monthly intrathecal injections, and serious neurological side effects in 7% of patients per a 2022 trial.

Weiss disclosed advisory board (Alexion, Ra [now UCB], argenx, Biogen, Mitsubishi Tanabe Pharma, Amylyx), data safety monitoring board (Sanofi, AI), consulting (Cytokinetics, CSL Behring), and speaker (Soleo) relationships.

*Correction, 10/23/2024: This story originally quoted Weiss as saying the maker of edaravone provides no assistance to underinsured patients. Weiss has clarified that he should have said this coverage is “limited.”

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Current disease-modifying therapies for amyotrophic lateral sclerosis (ALS) don’t extend lifespans by much, but several drug options are available, nerve specialists learned.

The glutamate blocker riluzole (Rilutek), which became the first ALS drug to receive US Food and Drug Administration (FDA) approval in 1995, continues to be used, Michael D. Weiss, MD, professor of neurology at University of Washington School of Medicine, Seattle, said in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.

Weiss highlighted a 2012 Cochrane Library review that examined research and found the drug is “reasonably safe” and prolongs median survival by about 2-3 months. “About 12% develop liver disease. It’s pretty rare that we stop the medicine due to liver toxicity.”
 

Earlier Treatment Could Pay Dividends

A recent study “suggests we might be able to get more bang for our buck from riluzole” by initiating treatment earlier, Weiss said.

Researchers tracked 4778 patients with ALS, including 3446 (72.1%) who took riluzole. Those who took the drug survived a median 2 extra months (22.6 vs 20.2 months; P < .001). The data suggested that delaying riluzole initiation by 1 year (from 6 months to 18 months after diagnosis) reduced the median survival by 1.9 months (from 40.1 to 38.2 months).

There’s “a relatively significant additional benefit” to earlier treatment, Weiss said, although patients will vary on whether they think it’s meaningful. As for limitations, “there’s no clear impact on disease progression, and there’s a need for periodic monitoring of liver function profile.” 

He added that there’s an out-of-pocket co-pay. “Even as a generic, it’s not that cheap. Depending on the source, it could cost anywhere from $1800 to $8400 a year.”
 

Edaravone Could Lack Relevant Benefit

No other ALS treatment appeared until 2017, when the FDA approved the novel antioxidant edaravone (Radicava). In 2022, the agency approved an oral suspension version, but a study published that year suggested there may not be a clinically relevant benefit.

The University of Washington, where Weiss works, offered the drug to 144 patients, according to an analysis. Eighty percent of the patients wanted it, but insurers refused to cover it for more than 20%. The average time to treatment with the drug was 28 days after patients said they wanted it.

That’s a “substantial delay,” Weiss said.

The cost is about $171,000 a year, he said, and assistance is limited for underinsured patients.*

Other Options

As Weiss noted, another drug, AMX0035 (Relyvrio), received FDA approval in 2022, but its manufacturer pulled it from the US/Canada market in April 2024 following poor phase 3 trial findings.

In 2023, the FDA approved another drug, the antisense oligonucleotide tofersen (Qalsody), in patients with ALS associated with a mutation in the superoxide dismutase 1 gene. According to the FDA, reductions in plasma neurofilament light concentration were “reasonably likely to predict a clinical benefit in patients.”

Only 1%-2% of patients with ALS fit the criteria to get the drug, Weiss said. He noted other limitations such as the cost ($180,000 a year), the need for lifelong monthly intrathecal injections, and serious neurological side effects in 7% of patients per a 2022 trial.

Weiss disclosed advisory board (Alexion, Ra [now UCB], argenx, Biogen, Mitsubishi Tanabe Pharma, Amylyx), data safety monitoring board (Sanofi, AI), consulting (Cytokinetics, CSL Behring), and speaker (Soleo) relationships.

*Correction, 10/23/2024: This story originally quoted Weiss as saying the maker of edaravone provides no assistance to underinsured patients. Weiss has clarified that he should have said this coverage is “limited.”

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Current disease-modifying therapies for amyotrophic lateral sclerosis (ALS) don’t extend lifespans by much, but several drug options are available, nerve specialists learned.

The glutamate blocker riluzole (Rilutek), which became the first ALS drug to receive US Food and Drug Administration (FDA) approval in 1995, continues to be used, Michael D. Weiss, MD, professor of neurology at University of Washington School of Medicine, Seattle, said in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.

Weiss highlighted a 2012 Cochrane Library review that examined research and found the drug is “reasonably safe” and prolongs median survival by about 2-3 months. “About 12% develop liver disease. It’s pretty rare that we stop the medicine due to liver toxicity.”
 

Earlier Treatment Could Pay Dividends

A recent study “suggests we might be able to get more bang for our buck from riluzole” by initiating treatment earlier, Weiss said.

Researchers tracked 4778 patients with ALS, including 3446 (72.1%) who took riluzole. Those who took the drug survived a median 2 extra months (22.6 vs 20.2 months; P < .001). The data suggested that delaying riluzole initiation by 1 year (from 6 months to 18 months after diagnosis) reduced the median survival by 1.9 months (from 40.1 to 38.2 months).

There’s “a relatively significant additional benefit” to earlier treatment, Weiss said, although patients will vary on whether they think it’s meaningful. As for limitations, “there’s no clear impact on disease progression, and there’s a need for periodic monitoring of liver function profile.” 

He added that there’s an out-of-pocket co-pay. “Even as a generic, it’s not that cheap. Depending on the source, it could cost anywhere from $1800 to $8400 a year.”
 

Edaravone Could Lack Relevant Benefit

No other ALS treatment appeared until 2017, when the FDA approved the novel antioxidant edaravone (Radicava). In 2022, the agency approved an oral suspension version, but a study published that year suggested there may not be a clinically relevant benefit.

The University of Washington, where Weiss works, offered the drug to 144 patients, according to an analysis. Eighty percent of the patients wanted it, but insurers refused to cover it for more than 20%. The average time to treatment with the drug was 28 days after patients said they wanted it.

That’s a “substantial delay,” Weiss said.

The cost is about $171,000 a year, he said, and assistance is limited for underinsured patients.*

Other Options

As Weiss noted, another drug, AMX0035 (Relyvrio), received FDA approval in 2022, but its manufacturer pulled it from the US/Canada market in April 2024 following poor phase 3 trial findings.

In 2023, the FDA approved another drug, the antisense oligonucleotide tofersen (Qalsody), in patients with ALS associated with a mutation in the superoxide dismutase 1 gene. According to the FDA, reductions in plasma neurofilament light concentration were “reasonably likely to predict a clinical benefit in patients.”

Only 1%-2% of patients with ALS fit the criteria to get the drug, Weiss said. He noted other limitations such as the cost ($180,000 a year), the need for lifelong monthly intrathecal injections, and serious neurological side effects in 7% of patients per a 2022 trial.

Weiss disclosed advisory board (Alexion, Ra [now UCB], argenx, Biogen, Mitsubishi Tanabe Pharma, Amylyx), data safety monitoring board (Sanofi, AI), consulting (Cytokinetics, CSL Behring), and speaker (Soleo) relationships.

*Correction, 10/23/2024: This story originally quoted Weiss as saying the maker of edaravone provides no assistance to underinsured patients. Weiss has clarified that he should have said this coverage is “limited.”

A version of this article appeared on Medscape.com.

LOS ANGELES — Embedding a new discharge order set into electronic health records (EHRs) with a preselected 5-day antibiotic course for children aged 2 years or older diagnosed with acute otitis media (AOM) cut antibiotic duration sharply, according to new data presented at the Infectious Disease Week (IDWeek) 2024 Annual Meeting.

“We were effectively able to cut antibiotic use in half by shortening the duration of treatment,” said lead author Joana Dimo, DO, a Pediatric Infectious Diseases fellow at the University of Colorado Denver/Children’s Hospital Colorado.

In the United States, 80% of children will experience otitis media during their lifetime. Untreated ear infections can lead to symptoms ranging from mild ear discharge to life-threatening conditions such as mastoiditis and intracranial abscesses.
 

Most Cases Resolve Without Antibiotics

Ear infections “are the leading reason for antibiotic prescriptions in kids,” Dimo noted, adding that 24% of all pediatric antibiotic prescriptions are for AOM. Amoxicillin is the preferred first-line treatment. “Research supports that 75% of children get better on their own without antibiotics, and when needed, short courses of just 5 days are safe and effective.”

Antibiotics can cause side effects such as diarrhea and rashes. “Each additional day of antibiotics that are not needed leads to more side effects,” Dimo said, as well as contributing to antibiotic resistance.

Dimo’s team implemented new EHR order sets across the University of Colorado/Children’s Hospital Colorado health network’s four emergency departments and four urgent care centers and included 31,929 patients in the study.

Then they conducted a retrospective review of patients 61 days to 18 years old who entered those settings and had confirmed AOM between January 2019 through December 2023, before and after the April 2021 intervention. The researchers also developed a guideline on managing ear infections to support clinicians as part of the intervention in December 2022.
 

Compliance Grew From 3% to 83%

Dimo said they found very few clinicians in their study had been prescribing according to current guidelines. Their results showed a jump from 3% to 83% in providers prescribing 5-day durations of antibiotics for children aged 2 years or older after their intervention.

The intervention did not lead to increased treatment failures or complications, she added. The team looked for diagnostic codes for mastoiditis, subperiosteal abscess, petrositis, labyrinthitis, meningitis, and intracranial abscess, and “none of our patients” developed any of those complications, Dimo said.

Dimo said the overall rate of prescribing, however, increased. Finding out why prescribing rates remained high throughout the study, before and after their intervention, is a question they are investigating in future work, she said.
 

Cost-Effective and Scalable

“The benefit of this strategy to other institutions is that it’s not labor-intensive. It’s cost-effective, and it can result in dramatic changes in antibiotic use,” Dimo said.

“In the outpatient setting, there’s still a lot of antibiotics being given unnecessarily to children with acute otitis media,” said William Schaffner, MD, infectious disease specialist at Vanderbilt University School of Medicine in Nashville, Tennessee, who was not part of the research. “The American Academy of Pediatrics has been working on that for about a decade — to get pediatricians attuned to when you use them. Most of these episodes of acute otitis media — it’s now well-established — are due to viral infections.”

He said that some physicians may still be defaulting to the longer doses — up to 10 days — that they may have learned in medical school or residency.

“The data would indicate that 5 days of treatment — when treatment is appropriate — is, in the vast majority of instances, sufficient,” Schaffner said.

The researchers “were remarkably successful,” he said, adding that another question is ripe for research. “They still have to get to this issue of whether all of these antibiotic starts were necessary.”

Not knowing whether antibiotic prescriptions in this study were warranted is a limitation of the study, Dimo said, as was not being able to track whether patients presented to institutions outside their own for a return visit or for complications.

She said she thinks one of the reasons for such a sharp increase in compliance was that clinicians in their system routinely use order sets, so using the new order sets easily became part of their workflow.

Dimo and Schaffner reported no relevant financial relationships.

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

LOS ANGELES — Embedding a new discharge order set into electronic health records (EHRs) with a preselected 5-day antibiotic course for children aged 2 years or older diagnosed with acute otitis media (AOM) cut antibiotic duration sharply, according to new data presented at the Infectious Disease Week (IDWeek) 2024 Annual Meeting.

“We were effectively able to cut antibiotic use in half by shortening the duration of treatment,” said lead author Joana Dimo, DO, a Pediatric Infectious Diseases fellow at the University of Colorado Denver/Children’s Hospital Colorado.

In the United States, 80% of children will experience otitis media during their lifetime. Untreated ear infections can lead to symptoms ranging from mild ear discharge to life-threatening conditions such as mastoiditis and intracranial abscesses.
 

Most Cases Resolve Without Antibiotics

Ear infections “are the leading reason for antibiotic prescriptions in kids,” Dimo noted, adding that 24% of all pediatric antibiotic prescriptions are for AOM. Amoxicillin is the preferred first-line treatment. “Research supports that 75% of children get better on their own without antibiotics, and when needed, short courses of just 5 days are safe and effective.”

Antibiotics can cause side effects such as diarrhea and rashes. “Each additional day of antibiotics that are not needed leads to more side effects,” Dimo said, as well as contributing to antibiotic resistance.

Dimo’s team implemented new EHR order sets across the University of Colorado/Children’s Hospital Colorado health network’s four emergency departments and four urgent care centers and included 31,929 patients in the study.

Then they conducted a retrospective review of patients 61 days to 18 years old who entered those settings and had confirmed AOM between January 2019 through December 2023, before and after the April 2021 intervention. The researchers also developed a guideline on managing ear infections to support clinicians as part of the intervention in December 2022.
 

Compliance Grew From 3% to 83%

Dimo said they found very few clinicians in their study had been prescribing according to current guidelines. Their results showed a jump from 3% to 83% in providers prescribing 5-day durations of antibiotics for children aged 2 years or older after their intervention.

The intervention did not lead to increased treatment failures or complications, she added. The team looked for diagnostic codes for mastoiditis, subperiosteal abscess, petrositis, labyrinthitis, meningitis, and intracranial abscess, and “none of our patients” developed any of those complications, Dimo said.

Dimo said the overall rate of prescribing, however, increased. Finding out why prescribing rates remained high throughout the study, before and after their intervention, is a question they are investigating in future work, she said.
 

Cost-Effective and Scalable

“The benefit of this strategy to other institutions is that it’s not labor-intensive. It’s cost-effective, and it can result in dramatic changes in antibiotic use,” Dimo said.

“In the outpatient setting, there’s still a lot of antibiotics being given unnecessarily to children with acute otitis media,” said William Schaffner, MD, infectious disease specialist at Vanderbilt University School of Medicine in Nashville, Tennessee, who was not part of the research. “The American Academy of Pediatrics has been working on that for about a decade — to get pediatricians attuned to when you use them. Most of these episodes of acute otitis media — it’s now well-established — are due to viral infections.”

He said that some physicians may still be defaulting to the longer doses — up to 10 days — that they may have learned in medical school or residency.

“The data would indicate that 5 days of treatment — when treatment is appropriate — is, in the vast majority of instances, sufficient,” Schaffner said.

The researchers “were remarkably successful,” he said, adding that another question is ripe for research. “They still have to get to this issue of whether all of these antibiotic starts were necessary.”

Not knowing whether antibiotic prescriptions in this study were warranted is a limitation of the study, Dimo said, as was not being able to track whether patients presented to institutions outside their own for a return visit or for complications.

She said she thinks one of the reasons for such a sharp increase in compliance was that clinicians in their system routinely use order sets, so using the new order sets easily became part of their workflow.

Dimo and Schaffner reported no relevant financial relationships.

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

LOS ANGELES — Embedding a new discharge order set into electronic health records (EHRs) with a preselected 5-day antibiotic course for children aged 2 years or older diagnosed with acute otitis media (AOM) cut antibiotic duration sharply, according to new data presented at the Infectious Disease Week (IDWeek) 2024 Annual Meeting.

“We were effectively able to cut antibiotic use in half by shortening the duration of treatment,” said lead author Joana Dimo, DO, a Pediatric Infectious Diseases fellow at the University of Colorado Denver/Children’s Hospital Colorado.

In the United States, 80% of children will experience otitis media during their lifetime. Untreated ear infections can lead to symptoms ranging from mild ear discharge to life-threatening conditions such as mastoiditis and intracranial abscesses.
 

Most Cases Resolve Without Antibiotics

Ear infections “are the leading reason for antibiotic prescriptions in kids,” Dimo noted, adding that 24% of all pediatric antibiotic prescriptions are for AOM. Amoxicillin is the preferred first-line treatment. “Research supports that 75% of children get better on their own without antibiotics, and when needed, short courses of just 5 days are safe and effective.”

Antibiotics can cause side effects such as diarrhea and rashes. “Each additional day of antibiotics that are not needed leads to more side effects,” Dimo said, as well as contributing to antibiotic resistance.

Dimo’s team implemented new EHR order sets across the University of Colorado/Children’s Hospital Colorado health network’s four emergency departments and four urgent care centers and included 31,929 patients in the study.

Then they conducted a retrospective review of patients 61 days to 18 years old who entered those settings and had confirmed AOM between January 2019 through December 2023, before and after the April 2021 intervention. The researchers also developed a guideline on managing ear infections to support clinicians as part of the intervention in December 2022.
 

Compliance Grew From 3% to 83%

Dimo said they found very few clinicians in their study had been prescribing according to current guidelines. Their results showed a jump from 3% to 83% in providers prescribing 5-day durations of antibiotics for children aged 2 years or older after their intervention.

The intervention did not lead to increased treatment failures or complications, she added. The team looked for diagnostic codes for mastoiditis, subperiosteal abscess, petrositis, labyrinthitis, meningitis, and intracranial abscess, and “none of our patients” developed any of those complications, Dimo said.

Dimo said the overall rate of prescribing, however, increased. Finding out why prescribing rates remained high throughout the study, before and after their intervention, is a question they are investigating in future work, she said.
 

Cost-Effective and Scalable

“The benefit of this strategy to other institutions is that it’s not labor-intensive. It’s cost-effective, and it can result in dramatic changes in antibiotic use,” Dimo said.

“In the outpatient setting, there’s still a lot of antibiotics being given unnecessarily to children with acute otitis media,” said William Schaffner, MD, infectious disease specialist at Vanderbilt University School of Medicine in Nashville, Tennessee, who was not part of the research. “The American Academy of Pediatrics has been working on that for about a decade — to get pediatricians attuned to when you use them. Most of these episodes of acute otitis media — it’s now well-established — are due to viral infections.”

He said that some physicians may still be defaulting to the longer doses — up to 10 days — that they may have learned in medical school or residency.

“The data would indicate that 5 days of treatment — when treatment is appropriate — is, in the vast majority of instances, sufficient,” Schaffner said.

The researchers “were remarkably successful,” he said, adding that another question is ripe for research. “They still have to get to this issue of whether all of these antibiotic starts were necessary.”

Not knowing whether antibiotic prescriptions in this study were warranted is a limitation of the study, Dimo said, as was not being able to track whether patients presented to institutions outside their own for a return visit or for complications.

She said she thinks one of the reasons for such a sharp increase in compliance was that clinicians in their system routinely use order sets, so using the new order sets easily became part of their workflow.

Dimo and Schaffner reported no relevant financial relationships.

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

A new study provides more evidence that glucagon-like peptide 1 receptor agonists (GLP-1 RAs) used to treat diabetes and obesity could be repurposed for opioid use disorder (OUD) and alcohol use disorder (AUD).

Researchers found that patients with OUD or AUD who were taking semaglutide (Ozempic, Novo Nordisk) or similar medications for diabetes or weight-related conditions had a 40% lower rate of opioid overdose and a 50% lower rate of alcohol intoxication than their peers with OUD or AUD who were not taking these medications.

Their real-world study of more than 1 million adults with a history of OUD or AUD provide “foundational” estimates of the association between glucose-dependent insulinotropic polypeptide (GIP)/GLP-1 RA prescriptions and opioid overdose/alcohol intoxication “and introduce the idea that GLP-1 RA and other related drugs should be investigated as a novel pharmacotherapy treatment option for individuals with OUD or AUD,” wrote the investigators, led by Fares Qeadan, PhD, Parkinson School of Health Sciences and Public Health, Loyola University Chicago, Maywood, Illinois.

The study was published online in the journal Addiction.
 

Protective Effect?

As previously reported by Medscape Medical News, earlier studies have pointed to a link between weight loss drugs and reduced overdose risk in people with OUD and decreased alcohol intake in people with AUD.

Until now, most studies on GLP-1 RAs and GIP agonists like tirzepatide (Mounjaro) to treat substance use disorders consisted of animal studies and small-scale clinical trials, investigators noted.

This new retrospective cohort study analyzed de-identified electronic health record data from the Oracle Health Real-World Data.

Participants, all aged 18 years or older, included 503,747 patients with a history of OUD, of whom 8103 had a GLP-1 RA or GIP prescription, and 817,309 patients with a history of AUD, of whom 5621 had a GLP-1 RA or GIP prescription.

Patients with OUD who were prescribed GLP-1 RAs had a 40% lower rate of opioid overdose than those without such prescriptions (adjusted incidence rate ratio [aIRR], 0.60; 95% CI, 0.43-0.83), the study team found.

In addition, patients with AUD and a GLP-1 RA prescription exhibited a 50% lower rate of alcohol intoxication (aIRR, 0.50; 95% CI, 0.40-0.63).

The protective effect of GLP-1 RA on opioid overdose and alcohol intoxication was maintained across patients with comorbid conditions, such as type 2 diabetes and obesity.

“Future research should focus on prospective clinical trials to validate these findings, explore the underlying mechanisms, and determine the long-term efficacy and safety of GIP/GLP-1 RA medications in diverse populations,” Qeadan and colleagues concluded.

“Additionally, the study highlights the importance of interdisciplinary research in understanding the neurobiological links between metabolic disorders and problematic substance use, potentially leading to more effective treatment strategies within healthcare systems,” they added.
 

Questions Remain

In a statement from the UK nonprofit Science Media Centre, Matt Field, DPhil, professor of psychology, The University of Sheffield, in England, noted that the findings “add to those from other studies, particularly animal research, which suggest that this and similar drugs might one day be prescribed to help people with addiction.”

However, “a note of caution is that the outcomes are very extreme instances of substance intoxication,” added Field, who wasn’t involved in the study.

“Those outcomes are very different from the outcomes used when researchers test new treatments for addiction, in which case we might look at whether the treatment helps people to stop taking the substance altogether (complete abstinence), or if it helps people to reduce the amount of substance they consume, or how often they consume it. Those things could not be measured in this study,” he continued.

“This leaves open the possibility that while Ozempic may — for reasons currently unknown — prevent people from taking so much alcohol or heroin that they overdose and end up in hospital, it may not actually help them to reduce their substance use, or to abstain altogether,” Field said.

The study had no specific funding. The study authors and Field declared no relevant conflicts of interest.
 

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

A new study provides more evidence that glucagon-like peptide 1 receptor agonists (GLP-1 RAs) used to treat diabetes and obesity could be repurposed for opioid use disorder (OUD) and alcohol use disorder (AUD).

Researchers found that patients with OUD or AUD who were taking semaglutide (Ozempic, Novo Nordisk) or similar medications for diabetes or weight-related conditions had a 40% lower rate of opioid overdose and a 50% lower rate of alcohol intoxication than their peers with OUD or AUD who were not taking these medications.

Their real-world study of more than 1 million adults with a history of OUD or AUD provide “foundational” estimates of the association between glucose-dependent insulinotropic polypeptide (GIP)/GLP-1 RA prescriptions and opioid overdose/alcohol intoxication “and introduce the idea that GLP-1 RA and other related drugs should be investigated as a novel pharmacotherapy treatment option for individuals with OUD or AUD,” wrote the investigators, led by Fares Qeadan, PhD, Parkinson School of Health Sciences and Public Health, Loyola University Chicago, Maywood, Illinois.

The study was published online in the journal Addiction.
 

Protective Effect?

As previously reported by Medscape Medical News, earlier studies have pointed to a link between weight loss drugs and reduced overdose risk in people with OUD and decreased alcohol intake in people with AUD.

Until now, most studies on GLP-1 RAs and GIP agonists like tirzepatide (Mounjaro) to treat substance use disorders consisted of animal studies and small-scale clinical trials, investigators noted.

This new retrospective cohort study analyzed de-identified electronic health record data from the Oracle Health Real-World Data.

Participants, all aged 18 years or older, included 503,747 patients with a history of OUD, of whom 8103 had a GLP-1 RA or GIP prescription, and 817,309 patients with a history of AUD, of whom 5621 had a GLP-1 RA or GIP prescription.

Patients with OUD who were prescribed GLP-1 RAs had a 40% lower rate of opioid overdose than those without such prescriptions (adjusted incidence rate ratio [aIRR], 0.60; 95% CI, 0.43-0.83), the study team found.

In addition, patients with AUD and a GLP-1 RA prescription exhibited a 50% lower rate of alcohol intoxication (aIRR, 0.50; 95% CI, 0.40-0.63).

The protective effect of GLP-1 RA on opioid overdose and alcohol intoxication was maintained across patients with comorbid conditions, such as type 2 diabetes and obesity.

“Future research should focus on prospective clinical trials to validate these findings, explore the underlying mechanisms, and determine the long-term efficacy and safety of GIP/GLP-1 RA medications in diverse populations,” Qeadan and colleagues concluded.

“Additionally, the study highlights the importance of interdisciplinary research in understanding the neurobiological links between metabolic disorders and problematic substance use, potentially leading to more effective treatment strategies within healthcare systems,” they added.
 

Questions Remain

In a statement from the UK nonprofit Science Media Centre, Matt Field, DPhil, professor of psychology, The University of Sheffield, in England, noted that the findings “add to those from other studies, particularly animal research, which suggest that this and similar drugs might one day be prescribed to help people with addiction.”

However, “a note of caution is that the outcomes are very extreme instances of substance intoxication,” added Field, who wasn’t involved in the study.

“Those outcomes are very different from the outcomes used when researchers test new treatments for addiction, in which case we might look at whether the treatment helps people to stop taking the substance altogether (complete abstinence), or if it helps people to reduce the amount of substance they consume, or how often they consume it. Those things could not be measured in this study,” he continued.

“This leaves open the possibility that while Ozempic may — for reasons currently unknown — prevent people from taking so much alcohol or heroin that they overdose and end up in hospital, it may not actually help them to reduce their substance use, or to abstain altogether,” Field said.

The study had no specific funding. The study authors and Field declared no relevant conflicts of interest.
 

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

A new study provides more evidence that glucagon-like peptide 1 receptor agonists (GLP-1 RAs) used to treat diabetes and obesity could be repurposed for opioid use disorder (OUD) and alcohol use disorder (AUD).

Researchers found that patients with OUD or AUD who were taking semaglutide (Ozempic, Novo Nordisk) or similar medications for diabetes or weight-related conditions had a 40% lower rate of opioid overdose and a 50% lower rate of alcohol intoxication than their peers with OUD or AUD who were not taking these medications.

Their real-world study of more than 1 million adults with a history of OUD or AUD provide “foundational” estimates of the association between glucose-dependent insulinotropic polypeptide (GIP)/GLP-1 RA prescriptions and opioid overdose/alcohol intoxication “and introduce the idea that GLP-1 RA and other related drugs should be investigated as a novel pharmacotherapy treatment option for individuals with OUD or AUD,” wrote the investigators, led by Fares Qeadan, PhD, Parkinson School of Health Sciences and Public Health, Loyola University Chicago, Maywood, Illinois.

The study was published online in the journal Addiction.
 

Protective Effect?

As previously reported by Medscape Medical News, earlier studies have pointed to a link between weight loss drugs and reduced overdose risk in people with OUD and decreased alcohol intake in people with AUD.

Until now, most studies on GLP-1 RAs and GIP agonists like tirzepatide (Mounjaro) to treat substance use disorders consisted of animal studies and small-scale clinical trials, investigators noted.

This new retrospective cohort study analyzed de-identified electronic health record data from the Oracle Health Real-World Data.

Participants, all aged 18 years or older, included 503,747 patients with a history of OUD, of whom 8103 had a GLP-1 RA or GIP prescription, and 817,309 patients with a history of AUD, of whom 5621 had a GLP-1 RA or GIP prescription.

Patients with OUD who were prescribed GLP-1 RAs had a 40% lower rate of opioid overdose than those without such prescriptions (adjusted incidence rate ratio [aIRR], 0.60; 95% CI, 0.43-0.83), the study team found.

In addition, patients with AUD and a GLP-1 RA prescription exhibited a 50% lower rate of alcohol intoxication (aIRR, 0.50; 95% CI, 0.40-0.63).

The protective effect of GLP-1 RA on opioid overdose and alcohol intoxication was maintained across patients with comorbid conditions, such as type 2 diabetes and obesity.

“Future research should focus on prospective clinical trials to validate these findings, explore the underlying mechanisms, and determine the long-term efficacy and safety of GIP/GLP-1 RA medications in diverse populations,” Qeadan and colleagues concluded.

“Additionally, the study highlights the importance of interdisciplinary research in understanding the neurobiological links between metabolic disorders and problematic substance use, potentially leading to more effective treatment strategies within healthcare systems,” they added.
 

Questions Remain

In a statement from the UK nonprofit Science Media Centre, Matt Field, DPhil, professor of psychology, The University of Sheffield, in England, noted that the findings “add to those from other studies, particularly animal research, which suggest that this and similar drugs might one day be prescribed to help people with addiction.”

However, “a note of caution is that the outcomes are very extreme instances of substance intoxication,” added Field, who wasn’t involved in the study.

“Those outcomes are very different from the outcomes used when researchers test new treatments for addiction, in which case we might look at whether the treatment helps people to stop taking the substance altogether (complete abstinence), or if it helps people to reduce the amount of substance they consume, or how often they consume it. Those things could not be measured in this study,” he continued.

“This leaves open the possibility that while Ozempic may — for reasons currently unknown — prevent people from taking so much alcohol or heroin that they overdose and end up in hospital, it may not actually help them to reduce their substance use, or to abstain altogether,” Field said.

The study had no specific funding. The study authors and Field declared no relevant conflicts of interest.
 

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.

A new serum containing 2-mercaptonicotinoyl glycine (Melasyl) as its main ingredient was at least as good as, if not better than, cysteamine 5% cream in treating facial melasma in a randomized controlled study presented at the European Academy of Dermatology and Venereology (EADV) 2024 Congress.

“Melasyl is a new potent melanogenesis inhibitor that exhibits a unique mode of action while preserving melanocyte integrity,” Mukta Sachdev, MD, head of the Department of Dermatology at Manipal Hospital in Bangalore, India, said at a late-breaking news session.

Both the serum and the cysteamine cream lightened participants’ skin to a similar extent, according to the modified Melasma Area and Severity Index (mMASI), with respective reductions of 4.19 and 3.81 points over a period of 4 months from baseline values of 11.15 and 10.93. 

Toa55/iStock/Getty Images

The mMASI score ranges from 0 to 24, with the lowest score representing the least and the highest score the most severe hyperpigmentation of the skin.

But the serum performed better than the cream by another measure. Judged by investigators blinded to which preparation study participants had been using, there was a significantly higher reduction in the Investigator Global Assessment (IGA) score from baseline among those treated with the serum than among those treated with the cream (−51.85% vs −39.06%; P = .0163). 

Moreover, after 4 months of treatment, there were significantly more participants with clear or almost clear skin with the serum than with the cream (17.46% vs 7.81%; P = .0163), Sachdev reported.

Other skin parameters relative to melasma, such as the brightness of skin tone and evenness of the improvement, improved more in the participants using the serum vs cream, she said. 

With “no side effects, no local skin reactions,” Sachdev said, “quality of life improved significantly and similarly, and almost all subjects in both groups were very satisfied with their treatment options.”
 

Active Ingredients

Margarida Gonçalo, MD, PhD, professor of dermatology at the University of Coimbra, in Portugal, who co-chaired the late-breaking news session, commented: “It’s really nice to have new products to treat such a devastating disease.”

Session co-chair, Lidia Rudnicka, MD, head of the Department of Dermatology, Medical University of Warsaw, in Poland, and president of the Polish Dermatological Society, wanted to know more about the active ingredients of the serum and the study’s design. 

Sachdev replied that the serum also contains other ingredients that provide “antioxidant protection” and moisturization. These include retinyl palmitate, which works on the dermal-epidermal junction, and hyaluronic acid, as well as “soothing agents,” such as the medicinal herb Centella asiatica, she said.
 

Study Design

Conducted at a single center in India, the study involved 127 adults aged 20-50 years with melasma. For inclusion, the participants had to have facial epidermal or mixed melasma (phototypes III-V) for more than 1 year; those with dermal melasma were excluded. 

Participants were randomly allocated to receive either the serum, which was applied topically to the areas of interest twice a day in the morning and then at bedtime (n = 63), or cysteamine cream (n = 64), which was applied once a day in addition to a neutral moisturizer. Treatment was for 4 months, with an on-site visit every month. 

All participants were supplied with the same sunscreen/ultraviolet protector applied twice a day (once in the morning and again at midday) and a neutral hydrating cleanser that was used in the morning and evening. 
 

Practical Implications

Over 4 months, both products showed significant improvement in melasma without reaching a plateau, Sachdev reported, with the serum demonstrating superior efficacy and tolerability, as judged by the investigators. 

The study suggests that the serum is a promising non-hydroquinone treatment for melasma, she said. Hydroquinone-containing topical preparations are used to depigment the skin, but their long-term use can be limited for safety reasons. 

“When products like this demonstrate improvement, it is something for the dermatologist to think about because we now have newer ingredients, which are safer and well tolerated,” she continued, noting that there appeared to be no risk for exogenous ochronosis, which can occur with long-term application of hydroquinone.

“So, I think the armamentarium of non-hydroquinone products for the treatment of melasma is rapidly expanding, and there are studies now with clinically proven efficacy,” Sachdev concluded. 

The study was supported by L’Oréal France La Roche-Posay, which launched Melasyl in March 2024. Sachdev reported receipt of research support and honoraria from the company. Gonçalo and Rudnicka were not involved in the study and had no relevant conflicts of interest to report. 
 

A version of this article appeared on Medscape.com.

A new serum containing 2-mercaptonicotinoyl glycine (Melasyl) as its main ingredient was at least as good as, if not better than, cysteamine 5% cream in treating facial melasma in a randomized controlled study presented at the European Academy of Dermatology and Venereology (EADV) 2024 Congress.

“Melasyl is a new potent melanogenesis inhibitor that exhibits a unique mode of action while preserving melanocyte integrity,” Mukta Sachdev, MD, head of the Department of Dermatology at Manipal Hospital in Bangalore, India, said at a late-breaking news session.

Both the serum and the cysteamine cream lightened participants’ skin to a similar extent, according to the modified Melasma Area and Severity Index (mMASI), with respective reductions of 4.19 and 3.81 points over a period of 4 months from baseline values of 11.15 and 10.93. 

Toa55/iStock/Getty Images

The mMASI score ranges from 0 to 24, with the lowest score representing the least and the highest score the most severe hyperpigmentation of the skin.

But the serum performed better than the cream by another measure. Judged by investigators blinded to which preparation study participants had been using, there was a significantly higher reduction in the Investigator Global Assessment (IGA) score from baseline among those treated with the serum than among those treated with the cream (−51.85% vs −39.06%; P = .0163). 

Moreover, after 4 months of treatment, there were significantly more participants with clear or almost clear skin with the serum than with the cream (17.46% vs 7.81%; P = .0163), Sachdev reported.

Other skin parameters relative to melasma, such as the brightness of skin tone and evenness of the improvement, improved more in the participants using the serum vs cream, she said. 

With “no side effects, no local skin reactions,” Sachdev said, “quality of life improved significantly and similarly, and almost all subjects in both groups were very satisfied with their treatment options.”
 

Active Ingredients

Margarida Gonçalo, MD, PhD, professor of dermatology at the University of Coimbra, in Portugal, who co-chaired the late-breaking news session, commented: “It’s really nice to have new products to treat such a devastating disease.”

Session co-chair, Lidia Rudnicka, MD, head of the Department of Dermatology, Medical University of Warsaw, in Poland, and president of the Polish Dermatological Society, wanted to know more about the active ingredients of the serum and the study’s design. 

Sachdev replied that the serum also contains other ingredients that provide “antioxidant protection” and moisturization. These include retinyl palmitate, which works on the dermal-epidermal junction, and hyaluronic acid, as well as “soothing agents,” such as the medicinal herb Centella asiatica, she said.
 

Study Design

Conducted at a single center in India, the study involved 127 adults aged 20-50 years with melasma. For inclusion, the participants had to have facial epidermal or mixed melasma (phototypes III-V) for more than 1 year; those with dermal melasma were excluded. 

Participants were randomly allocated to receive either the serum, which was applied topically to the areas of interest twice a day in the morning and then at bedtime (n = 63), or cysteamine cream (n = 64), which was applied once a day in addition to a neutral moisturizer. Treatment was for 4 months, with an on-site visit every month. 

All participants were supplied with the same sunscreen/ultraviolet protector applied twice a day (once in the morning and again at midday) and a neutral hydrating cleanser that was used in the morning and evening. 
 

Practical Implications

Over 4 months, both products showed significant improvement in melasma without reaching a plateau, Sachdev reported, with the serum demonstrating superior efficacy and tolerability, as judged by the investigators. 

The study suggests that the serum is a promising non-hydroquinone treatment for melasma, she said. Hydroquinone-containing topical preparations are used to depigment the skin, but their long-term use can be limited for safety reasons. 

“When products like this demonstrate improvement, it is something for the dermatologist to think about because we now have newer ingredients, which are safer and well tolerated,” she continued, noting that there appeared to be no risk for exogenous ochronosis, which can occur with long-term application of hydroquinone.

“So, I think the armamentarium of non-hydroquinone products for the treatment of melasma is rapidly expanding, and there are studies now with clinically proven efficacy,” Sachdev concluded. 

The study was supported by L’Oréal France La Roche-Posay, which launched Melasyl in March 2024. Sachdev reported receipt of research support and honoraria from the company. Gonçalo and Rudnicka were not involved in the study and had no relevant conflicts of interest to report. 
 

A version of this article appeared on Medscape.com.

A new serum containing 2-mercaptonicotinoyl glycine (Melasyl) as its main ingredient was at least as good as, if not better than, cysteamine 5% cream in treating facial melasma in a randomized controlled study presented at the European Academy of Dermatology and Venereology (EADV) 2024 Congress.

“Melasyl is a new potent melanogenesis inhibitor that exhibits a unique mode of action while preserving melanocyte integrity,” Mukta Sachdev, MD, head of the Department of Dermatology at Manipal Hospital in Bangalore, India, said at a late-breaking news session.

Both the serum and the cysteamine cream lightened participants’ skin to a similar extent, according to the modified Melasma Area and Severity Index (mMASI), with respective reductions of 4.19 and 3.81 points over a period of 4 months from baseline values of 11.15 and 10.93. 

Toa55/iStock/Getty Images

The mMASI score ranges from 0 to 24, with the lowest score representing the least and the highest score the most severe hyperpigmentation of the skin.

But the serum performed better than the cream by another measure. Judged by investigators blinded to which preparation study participants had been using, there was a significantly higher reduction in the Investigator Global Assessment (IGA) score from baseline among those treated with the serum than among those treated with the cream (−51.85% vs −39.06%; P = .0163). 

Moreover, after 4 months of treatment, there were significantly more participants with clear or almost clear skin with the serum than with the cream (17.46% vs 7.81%; P = .0163), Sachdev reported.

Other skin parameters relative to melasma, such as the brightness of skin tone and evenness of the improvement, improved more in the participants using the serum vs cream, she said. 

With “no side effects, no local skin reactions,” Sachdev said, “quality of life improved significantly and similarly, and almost all subjects in both groups were very satisfied with their treatment options.”
 

Active Ingredients

Margarida Gonçalo, MD, PhD, professor of dermatology at the University of Coimbra, in Portugal, who co-chaired the late-breaking news session, commented: “It’s really nice to have new products to treat such a devastating disease.”

Session co-chair, Lidia Rudnicka, MD, head of the Department of Dermatology, Medical University of Warsaw, in Poland, and president of the Polish Dermatological Society, wanted to know more about the active ingredients of the serum and the study’s design. 

Sachdev replied that the serum also contains other ingredients that provide “antioxidant protection” and moisturization. These include retinyl palmitate, which works on the dermal-epidermal junction, and hyaluronic acid, as well as “soothing agents,” such as the medicinal herb Centella asiatica, she said.
 

Study Design

Conducted at a single center in India, the study involved 127 adults aged 20-50 years with melasma. For inclusion, the participants had to have facial epidermal or mixed melasma (phototypes III-V) for more than 1 year; those with dermal melasma were excluded. 

Participants were randomly allocated to receive either the serum, which was applied topically to the areas of interest twice a day in the morning and then at bedtime (n = 63), or cysteamine cream (n = 64), which was applied once a day in addition to a neutral moisturizer. Treatment was for 4 months, with an on-site visit every month. 

All participants were supplied with the same sunscreen/ultraviolet protector applied twice a day (once in the morning and again at midday) and a neutral hydrating cleanser that was used in the morning and evening. 
 

Practical Implications

Over 4 months, both products showed significant improvement in melasma without reaching a plateau, Sachdev reported, with the serum demonstrating superior efficacy and tolerability, as judged by the investigators. 

The study suggests that the serum is a promising non-hydroquinone treatment for melasma, she said. Hydroquinone-containing topical preparations are used to depigment the skin, but their long-term use can be limited for safety reasons. 

“When products like this demonstrate improvement, it is something for the dermatologist to think about because we now have newer ingredients, which are safer and well tolerated,” she continued, noting that there appeared to be no risk for exogenous ochronosis, which can occur with long-term application of hydroquinone.

“So, I think the armamentarium of non-hydroquinone products for the treatment of melasma is rapidly expanding, and there are studies now with clinically proven efficacy,” Sachdev concluded. 

The study was supported by L’Oréal France La Roche-Posay, which launched Melasyl in March 2024. Sachdev reported receipt of research support and honoraria from the company. Gonçalo and Rudnicka were not involved in the study and had no relevant conflicts of interest to report. 
 

A version of this article appeared on Medscape.com.