Adolescent Medicine

Pediatric concussion is one of those rare phenomena in which we may be witnessing its emergence and clarification in a generation. When I was serving as the game doctor for our local high school football team in the 1970s, I and many other physicians had a very simplistic view of concussion. If the patient never lost conscious and had a reasonably intact short-term memory, we didn’t seriously entertain concussion as a diagnosis. “What’s the score and who is the president?” Were my favorite screening questions.

Obviously, we were underdiagnosing and mismanaging concussion. In part thanks to some high-profile athletes who suffered multiple concussions and eventually chronic traumatic encephalopathy (CTE) physicians began to realize that they should be looking more closely at children who sustained a head injury. The diagnostic criteria were expanded to include any injury that even temporarily effected brain function.

With the new appreciation for the risk of multiple concussions, the focus broadened to include the question of when is it safe for the athlete to return to competition. What signs or symptoms can the patient offer us so we can be sure his or her brain is sufficiently recovered? Here we stepped off into a deep abyss of ignorance. Fortunately, it became obvious fairly quickly that imaging studies weren’t going to help us, as they were invariably normal or at least didn’t tell us anything that wasn’t obvious on a physical exam.

If the patient had a headache, complained of dizziness, or manifested amnesia, monitoring the patient was fairly straightforward. But, in the absence of symptoms and no obvious way to determine the pace of recovery of an organ we couldn’t visualize, clinicians were pulling criteria and time tables out of thin air. Guessing that the concussed brain was in some ways like a torn muscle or overstretched tendon, “brain rest” was often suggested. So no TV, no reading, and certainly none of the cerebral challenging activity of school. Fortunately, we don’t hear much about the notion of brain rest anymore and there is at least one study that suggests that patients kept home from school recover more slowly.

But there remains a significant number of patients who have persistent symptoms and are unable to resume their usual activities, including school and sports. Sometimes they describe headache or dizziness but often they complain of a vague mental unwellness. “Brain fog,” a term that has emerged in the wake of the COVID pandemic, might be an apt descriptor. Management of these slow recoverers has been a challenge.

However, two recent articles in the journal Pediatrics may provide some clarity and offer guidance in their management. In a study coming from the psychology department at Georgia State University, researchers reported that they have been able to find “no evidence of clinical meaningful differences in IQ after pediatric concussion.” In their words there is “strong evidence against reduced intelligence in the first few weeks to month after pediatric concussion.”

While their findings may simply toss the IQ onto the pile of worthless measures of healing, a companion commentary by Talin Babikian, PhD, a psychologist at the Semel Institute for Neuroscience and Human Behavior at UCLA, provides a more nuanced interpretation. He writes that if we are looking for an explanation when a patient’s recovery is taking longer than we might expect we need to look beyond some structural damage. Maybe the patient has a previously undiagnosed premorbid condition effecting his or her intellectual, cognitive, or learning abilities. Could the stall in improvement be the result of other symptoms? Here fatigue and sleep deprivation may be the culprits. Could some underlying emotional factor such as anxiety or depression be the problem? For example, I have seen patients whose fear of re-injury has prevented their return to full function. And, finally, the patient may be avoiding a “nonpreferred or challenging situation” unrelated to the injury.

In other words, the concussion may simply be the most obvious rip in a fabric that was already frayed and under stress. This kind of broad holistic (a word I usually like to avoid) thinking may be what is lacking as we struggle to understand other mysterious and chronic conditions such as Lyme disease and chronic fatigue syndrome.

While these two papers help provide some clarity in the management of pediatric concussion, what they fail to address is the bigger question of the relationship between head injury and CTE. The answers to that conundrum are enshrouded in a mix of politics and publicity that I doubt will clear in the near future.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

Pediatric concussion is one of those rare phenomena in which we may be witnessing its emergence and clarification in a generation. When I was serving as the game doctor for our local high school football team in the 1970s, I and many other physicians had a very simplistic view of concussion. If the patient never lost conscious and had a reasonably intact short-term memory, we didn’t seriously entertain concussion as a diagnosis. “What’s the score and who is the president?” Were my favorite screening questions.

Obviously, we were underdiagnosing and mismanaging concussion. In part thanks to some high-profile athletes who suffered multiple concussions and eventually chronic traumatic encephalopathy (CTE) physicians began to realize that they should be looking more closely at children who sustained a head injury. The diagnostic criteria were expanded to include any injury that even temporarily effected brain function.

With the new appreciation for the risk of multiple concussions, the focus broadened to include the question of when is it safe for the athlete to return to competition. What signs or symptoms can the patient offer us so we can be sure his or her brain is sufficiently recovered? Here we stepped off into a deep abyss of ignorance. Fortunately, it became obvious fairly quickly that imaging studies weren’t going to help us, as they were invariably normal or at least didn’t tell us anything that wasn’t obvious on a physical exam.

If the patient had a headache, complained of dizziness, or manifested amnesia, monitoring the patient was fairly straightforward. But, in the absence of symptoms and no obvious way to determine the pace of recovery of an organ we couldn’t visualize, clinicians were pulling criteria and time tables out of thin air. Guessing that the concussed brain was in some ways like a torn muscle or overstretched tendon, “brain rest” was often suggested. So no TV, no reading, and certainly none of the cerebral challenging activity of school. Fortunately, we don’t hear much about the notion of brain rest anymore and there is at least one study that suggests that patients kept home from school recover more slowly.

But there remains a significant number of patients who have persistent symptoms and are unable to resume their usual activities, including school and sports. Sometimes they describe headache or dizziness but often they complain of a vague mental unwellness. “Brain fog,” a term that has emerged in the wake of the COVID pandemic, might be an apt descriptor. Management of these slow recoverers has been a challenge.

However, two recent articles in the journal Pediatrics may provide some clarity and offer guidance in their management. In a study coming from the psychology department at Georgia State University, researchers reported that they have been able to find “no evidence of clinical meaningful differences in IQ after pediatric concussion.” In their words there is “strong evidence against reduced intelligence in the first few weeks to month after pediatric concussion.”

While their findings may simply toss the IQ onto the pile of worthless measures of healing, a companion commentary by Talin Babikian, PhD, a psychologist at the Semel Institute for Neuroscience and Human Behavior at UCLA, provides a more nuanced interpretation. He writes that if we are looking for an explanation when a patient’s recovery is taking longer than we might expect we need to look beyond some structural damage. Maybe the patient has a previously undiagnosed premorbid condition effecting his or her intellectual, cognitive, or learning abilities. Could the stall in improvement be the result of other symptoms? Here fatigue and sleep deprivation may be the culprits. Could some underlying emotional factor such as anxiety or depression be the problem? For example, I have seen patients whose fear of re-injury has prevented their return to full function. And, finally, the patient may be avoiding a “nonpreferred or challenging situation” unrelated to the injury.

In other words, the concussion may simply be the most obvious rip in a fabric that was already frayed and under stress. This kind of broad holistic (a word I usually like to avoid) thinking may be what is lacking as we struggle to understand other mysterious and chronic conditions such as Lyme disease and chronic fatigue syndrome.

While these two papers help provide some clarity in the management of pediatric concussion, what they fail to address is the bigger question of the relationship between head injury and CTE. The answers to that conundrum are enshrouded in a mix of politics and publicity that I doubt will clear in the near future.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

Pediatric concussion is one of those rare phenomena in which we may be witnessing its emergence and clarification in a generation. When I was serving as the game doctor for our local high school football team in the 1970s, I and many other physicians had a very simplistic view of concussion. If the patient never lost conscious and had a reasonably intact short-term memory, we didn’t seriously entertain concussion as a diagnosis. “What’s the score and who is the president?” Were my favorite screening questions.

Obviously, we were underdiagnosing and mismanaging concussion. In part thanks to some high-profile athletes who suffered multiple concussions and eventually chronic traumatic encephalopathy (CTE) physicians began to realize that they should be looking more closely at children who sustained a head injury. The diagnostic criteria were expanded to include any injury that even temporarily effected brain function.

With the new appreciation for the risk of multiple concussions, the focus broadened to include the question of when is it safe for the athlete to return to competition. What signs or symptoms can the patient offer us so we can be sure his or her brain is sufficiently recovered? Here we stepped off into a deep abyss of ignorance. Fortunately, it became obvious fairly quickly that imaging studies weren’t going to help us, as they were invariably normal or at least didn’t tell us anything that wasn’t obvious on a physical exam.

If the patient had a headache, complained of dizziness, or manifested amnesia, monitoring the patient was fairly straightforward. But, in the absence of symptoms and no obvious way to determine the pace of recovery of an organ we couldn’t visualize, clinicians were pulling criteria and time tables out of thin air. Guessing that the concussed brain was in some ways like a torn muscle or overstretched tendon, “brain rest” was often suggested. So no TV, no reading, and certainly none of the cerebral challenging activity of school. Fortunately, we don’t hear much about the notion of brain rest anymore and there is at least one study that suggests that patients kept home from school recover more slowly.

But there remains a significant number of patients who have persistent symptoms and are unable to resume their usual activities, including school and sports. Sometimes they describe headache or dizziness but often they complain of a vague mental unwellness. “Brain fog,” a term that has emerged in the wake of the COVID pandemic, might be an apt descriptor. Management of these slow recoverers has been a challenge.

However, two recent articles in the journal Pediatrics may provide some clarity and offer guidance in their management. In a study coming from the psychology department at Georgia State University, researchers reported that they have been able to find “no evidence of clinical meaningful differences in IQ after pediatric concussion.” In their words there is “strong evidence against reduced intelligence in the first few weeks to month after pediatric concussion.”

While their findings may simply toss the IQ onto the pile of worthless measures of healing, a companion commentary by Talin Babikian, PhD, a psychologist at the Semel Institute for Neuroscience and Human Behavior at UCLA, provides a more nuanced interpretation. He writes that if we are looking for an explanation when a patient’s recovery is taking longer than we might expect we need to look beyond some structural damage. Maybe the patient has a previously undiagnosed premorbid condition effecting his or her intellectual, cognitive, or learning abilities. Could the stall in improvement be the result of other symptoms? Here fatigue and sleep deprivation may be the culprits. Could some underlying emotional factor such as anxiety or depression be the problem? For example, I have seen patients whose fear of re-injury has prevented their return to full function. And, finally, the patient may be avoiding a “nonpreferred or challenging situation” unrelated to the injury.

In other words, the concussion may simply be the most obvious rip in a fabric that was already frayed and under stress. This kind of broad holistic (a word I usually like to avoid) thinking may be what is lacking as we struggle to understand other mysterious and chronic conditions such as Lyme disease and chronic fatigue syndrome.

While these two papers help provide some clarity in the management of pediatric concussion, what they fail to address is the bigger question of the relationship between head injury and CTE. The answers to that conundrum are enshrouded in a mix of politics and publicity that I doubt will clear in the near future.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

Adolescents and young adults who smoked and vaped were more likely to report ocular problems including dryness, redness, pain, blurry vision, light sensitivity, and headaches, according to an observational study published in JAMA Ophthalmology.

Eye symptoms were significantly worse among young people who reported using both cigarettes and e-cigarettes than for those who said they used only one of the products, according to researchers. Symptoms were particularly frequent and severe among those who had used both products in the prior week. 

“In ophthalmology clinics, I’ve increasingly noticed patients, particularly adolescents and young adults, presenting with eye-related symptoms such as dryness, irritation, and even vision disturbances,” said Anne Xuan-Lan Nguyen, MDCM, an ophthalmology resident at the University of Toronto, who led the study. 

Many of these patients said they did not use contact lenses or take medications associated with eye problems, but they did report a history of using e-cigarettes and cigarettes. 

This “sparked my curiosity about the possible link between smoking or vaping and ocular symptoms,” Dr. Nguyen, who conducted the research as a medical student at McGill University in Montreal, told this news organization. 

E-cigarettes are the most popular tobacco product among young people. Public health data show an increasing trend toward both vaping and smoking cigarettes, known as dual use. An estimated 40% of middle- and high school–aged tobacco users report using two or more tobacco products, according to the Centers for Disease Control and Prevention. Cigarette use has been linked to ocular damage, but the effects of e-cigarettes on eyesight and the combined effect with cigarettes are not as well known. 

Dr. Nguyen and her colleagues surveyed more than 4,000 people aged 13-24 about their use of cigarettes or e-cigarettes in the last 30 days, the last 7 days, or ever. Half said they had never used any tobacco product and one quarter reported having used cigarettes, vapes, or both in the last month. More than 900 respondents said they had used one or both tobacco products in the last week. 

Of the respondents who had ever vaped, 55.9% said they also used cigarettes. These dual users reported more severe and frequent eye symptoms compared with users of either product alone. Up to 4% of respondents who had ever been a dual user reported daily, severe, or very severe ocular symptoms – more than in the cigarette-only or e-cigarette-only groups. 

More frequent tobacco use also was associated with more ocular symptoms. Young people who smoked or vaped in the previous week reported more symptoms than did the 30-day group, who reported more symptoms than the ever-user group (those who had taken at least a puff but not in the last month).

“All these conditions we know are worse as you get older,” said Laura B. Enyedi, MD, pediatric ophthalmologist at the Duke Eye Center in Durham, N.C., who was not associated with the study. “So if young people are having symptoms, it doesn’t bode well for them as they age.”

E-cigarette use alone did not appear to be linked to eye ailments, according to the findings. But to Dr. Nguyen’s surprise the survey results showed users of vaping products spent the most time worried about their eye health compared with all other participants. Users who smoked only cigarettes reported ocular symptoms, but not as severe or frequent as those of dual users. 

The researchers hypothesized that ocular problems caused by vapes and cigarettes could be classified as oxidative damage. The combustion of the cigarette and the e-cigarette solvent (propylene glycol) potentially generates free radicals that can cause oxidative stress, damaging the ocular surface and film, Dr. Nguyen said. 

Ophthalmologists are “always asking about contact lens use, lid hygiene, and screen time. Here’s another thing to consider when we get those common, nonspecific complaints of symptoms like dryness, redness, and burning,” Dr. Enyedi said.

Given the observational nature of the study, the researchers cannot confirm that dual use causes ocular symptoms. But given the public health challenge that tobacco use already presents for young people, the findings provide yet another reason to counsel against tobacco use and provide cessation options, Dr. Nguyen said. 

“This study is just one of many, many studies showing a significant relationship among smoking, e-cigarette use, and health outcomes,” said Bonnie Halpern-Felsher, PhD, professor of pediatrics at Stanford (Calif.) University and a coauthor of the study. “We clearly need to help young people not use at all, or quit or cut back if using.” 

This study was supported by the Taube Research Faculty Scholar Endowment; the National Heart, Lung, and Blood Institute; the Food and Drug Administration Center for Tobacco Products; the National Cancer Institute; the Stanford Maternal and Child Health Research Institute; and the Research to Prevent Blindness and National Eye Institute. Dr. Halpern-Felsher reported receiving personal fees as an expert scientist in litigation against some e-cigarette companies. The other study authors and Dr. Enyedi reported no relevant financial relationships.

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

Adolescents and young adults who smoked and vaped were more likely to report ocular problems including dryness, redness, pain, blurry vision, light sensitivity, and headaches, according to an observational study published in JAMA Ophthalmology.

Eye symptoms were significantly worse among young people who reported using both cigarettes and e-cigarettes than for those who said they used only one of the products, according to researchers. Symptoms were particularly frequent and severe among those who had used both products in the prior week. 

“In ophthalmology clinics, I’ve increasingly noticed patients, particularly adolescents and young adults, presenting with eye-related symptoms such as dryness, irritation, and even vision disturbances,” said Anne Xuan-Lan Nguyen, MDCM, an ophthalmology resident at the University of Toronto, who led the study. 

Many of these patients said they did not use contact lenses or take medications associated with eye problems, but they did report a history of using e-cigarettes and cigarettes. 

This “sparked my curiosity about the possible link between smoking or vaping and ocular symptoms,” Dr. Nguyen, who conducted the research as a medical student at McGill University in Montreal, told this news organization. 

E-cigarettes are the most popular tobacco product among young people. Public health data show an increasing trend toward both vaping and smoking cigarettes, known as dual use. An estimated 40% of middle- and high school–aged tobacco users report using two or more tobacco products, according to the Centers for Disease Control and Prevention. Cigarette use has been linked to ocular damage, but the effects of e-cigarettes on eyesight and the combined effect with cigarettes are not as well known. 

Dr. Nguyen and her colleagues surveyed more than 4,000 people aged 13-24 about their use of cigarettes or e-cigarettes in the last 30 days, the last 7 days, or ever. Half said they had never used any tobacco product and one quarter reported having used cigarettes, vapes, or both in the last month. More than 900 respondents said they had used one or both tobacco products in the last week. 

Of the respondents who had ever vaped, 55.9% said they also used cigarettes. These dual users reported more severe and frequent eye symptoms compared with users of either product alone. Up to 4% of respondents who had ever been a dual user reported daily, severe, or very severe ocular symptoms – more than in the cigarette-only or e-cigarette-only groups. 

More frequent tobacco use also was associated with more ocular symptoms. Young people who smoked or vaped in the previous week reported more symptoms than did the 30-day group, who reported more symptoms than the ever-user group (those who had taken at least a puff but not in the last month).

“All these conditions we know are worse as you get older,” said Laura B. Enyedi, MD, pediatric ophthalmologist at the Duke Eye Center in Durham, N.C., who was not associated with the study. “So if young people are having symptoms, it doesn’t bode well for them as they age.”

E-cigarette use alone did not appear to be linked to eye ailments, according to the findings. But to Dr. Nguyen’s surprise the survey results showed users of vaping products spent the most time worried about their eye health compared with all other participants. Users who smoked only cigarettes reported ocular symptoms, but not as severe or frequent as those of dual users. 

The researchers hypothesized that ocular problems caused by vapes and cigarettes could be classified as oxidative damage. The combustion of the cigarette and the e-cigarette solvent (propylene glycol) potentially generates free radicals that can cause oxidative stress, damaging the ocular surface and film, Dr. Nguyen said. 

Ophthalmologists are “always asking about contact lens use, lid hygiene, and screen time. Here’s another thing to consider when we get those common, nonspecific complaints of symptoms like dryness, redness, and burning,” Dr. Enyedi said.

Given the observational nature of the study, the researchers cannot confirm that dual use causes ocular symptoms. But given the public health challenge that tobacco use already presents for young people, the findings provide yet another reason to counsel against tobacco use and provide cessation options, Dr. Nguyen said. 

“This study is just one of many, many studies showing a significant relationship among smoking, e-cigarette use, and health outcomes,” said Bonnie Halpern-Felsher, PhD, professor of pediatrics at Stanford (Calif.) University and a coauthor of the study. “We clearly need to help young people not use at all, or quit or cut back if using.” 

This study was supported by the Taube Research Faculty Scholar Endowment; the National Heart, Lung, and Blood Institute; the Food and Drug Administration Center for Tobacco Products; the National Cancer Institute; the Stanford Maternal and Child Health Research Institute; and the Research to Prevent Blindness and National Eye Institute. Dr. Halpern-Felsher reported receiving personal fees as an expert scientist in litigation against some e-cigarette companies. The other study authors and Dr. Enyedi reported no relevant financial relationships.

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

Adolescents and young adults who smoked and vaped were more likely to report ocular problems including dryness, redness, pain, blurry vision, light sensitivity, and headaches, according to an observational study published in JAMA Ophthalmology.

Eye symptoms were significantly worse among young people who reported using both cigarettes and e-cigarettes than for those who said they used only one of the products, according to researchers. Symptoms were particularly frequent and severe among those who had used both products in the prior week. 

“In ophthalmology clinics, I’ve increasingly noticed patients, particularly adolescents and young adults, presenting with eye-related symptoms such as dryness, irritation, and even vision disturbances,” said Anne Xuan-Lan Nguyen, MDCM, an ophthalmology resident at the University of Toronto, who led the study. 

Many of these patients said they did not use contact lenses or take medications associated with eye problems, but they did report a history of using e-cigarettes and cigarettes. 

This “sparked my curiosity about the possible link between smoking or vaping and ocular symptoms,” Dr. Nguyen, who conducted the research as a medical student at McGill University in Montreal, told this news organization. 

E-cigarettes are the most popular tobacco product among young people. Public health data show an increasing trend toward both vaping and smoking cigarettes, known as dual use. An estimated 40% of middle- and high school–aged tobacco users report using two or more tobacco products, according to the Centers for Disease Control and Prevention. Cigarette use has been linked to ocular damage, but the effects of e-cigarettes on eyesight and the combined effect with cigarettes are not as well known. 

Dr. Nguyen and her colleagues surveyed more than 4,000 people aged 13-24 about their use of cigarettes or e-cigarettes in the last 30 days, the last 7 days, or ever. Half said they had never used any tobacco product and one quarter reported having used cigarettes, vapes, or both in the last month. More than 900 respondents said they had used one or both tobacco products in the last week. 

Of the respondents who had ever vaped, 55.9% said they also used cigarettes. These dual users reported more severe and frequent eye symptoms compared with users of either product alone. Up to 4% of respondents who had ever been a dual user reported daily, severe, or very severe ocular symptoms – more than in the cigarette-only or e-cigarette-only groups. 

More frequent tobacco use also was associated with more ocular symptoms. Young people who smoked or vaped in the previous week reported more symptoms than did the 30-day group, who reported more symptoms than the ever-user group (those who had taken at least a puff but not in the last month).

“All these conditions we know are worse as you get older,” said Laura B. Enyedi, MD, pediatric ophthalmologist at the Duke Eye Center in Durham, N.C., who was not associated with the study. “So if young people are having symptoms, it doesn’t bode well for them as they age.”

E-cigarette use alone did not appear to be linked to eye ailments, according to the findings. But to Dr. Nguyen’s surprise the survey results showed users of vaping products spent the most time worried about their eye health compared with all other participants. Users who smoked only cigarettes reported ocular symptoms, but not as severe or frequent as those of dual users. 

The researchers hypothesized that ocular problems caused by vapes and cigarettes could be classified as oxidative damage. The combustion of the cigarette and the e-cigarette solvent (propylene glycol) potentially generates free radicals that can cause oxidative stress, damaging the ocular surface and film, Dr. Nguyen said. 

Ophthalmologists are “always asking about contact lens use, lid hygiene, and screen time. Here’s another thing to consider when we get those common, nonspecific complaints of symptoms like dryness, redness, and burning,” Dr. Enyedi said.

Given the observational nature of the study, the researchers cannot confirm that dual use causes ocular symptoms. But given the public health challenge that tobacco use already presents for young people, the findings provide yet another reason to counsel against tobacco use and provide cessation options, Dr. Nguyen said. 

“This study is just one of many, many studies showing a significant relationship among smoking, e-cigarette use, and health outcomes,” said Bonnie Halpern-Felsher, PhD, professor of pediatrics at Stanford (Calif.) University and a coauthor of the study. “We clearly need to help young people not use at all, or quit or cut back if using.” 

This study was supported by the Taube Research Faculty Scholar Endowment; the National Heart, Lung, and Blood Institute; the Food and Drug Administration Center for Tobacco Products; the National Cancer Institute; the Stanford Maternal and Child Health Research Institute; and the Research to Prevent Blindness and National Eye Institute. Dr. Halpern-Felsher reported receiving personal fees as an expert scientist in litigation against some e-cigarette companies. The other study authors and Dr. Enyedi reported no relevant financial relationships.

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

The largest study to date of chronic traumatic encephalopathy (CTE) in young athletes shows that 41% had the neurodegenerative disease, caused by repetitive head impacts (RHIs).

Analysis of brain tissue from athletes who were exposed to RHIs and died before the age of 30 revealed neuropathological evidence of shrinkage of the brain and microscopic changes that indicate a breach of the blood-brain barrier. The case series also identified the first known American female athlete with CTE.

Nearly all of those with CTE had a mild form of the disease and 71% played only at the amateur level in youth, high school, or college sports.

“A lot of people think CTE is a result of high-level, professional play such as football, ice hockey, and boxing, but it can affect amateur athletes and can affect people at a young age,” lead author Ann McKee, MD, professor of neurology and pathology and director of the Chronic Traumatic Encephalopathy Center at Boston University, said in an interview.

The findings were published online in JAMA Neurology.
 

A rare look

Brain donation at younger ages is rare, so most of what is known about CTE comes from studies in older athletes.

“We’ve always known that young people could develop this disease early after just amateur high school, youth, and college exposure, but this is the largest study of donor brains at this age,” Dr. McKee said.

The case series included 152 brains of athletes who played contact sports, experienced RHIs, and died before age 30. The tissues are part of the Understanding Neurologic Injury and Traumatic Encephalopathy (UNITE) Brain Bank and were donated between February 2008 and September 2022.

Researchers reviewed the donors’ medical records and conducted retrospective interviews with the donors’ next of kin to assess cognitive symptoms, mood disturbances, and neurobehavioral issues.

Donors died between the ages of 13 and 29 years, 92.8% were male and 73% were White. In 57.2% of the cases, suicide was the cause of death, with no difference between those with or without CTE.

CTE was neuropathologically diagnosed in 41.4% of athletes, using diagnostic criteria developed by the National Institute of Neurological Disorders and Stroke. 

More than 95% had mild CTE. Diagnosis was associated with older age (mean difference, 3.92 years; P < .001) and significantly more years of exposure to contact sports (11.6 vs. 8.8 years).

Among those with CTE, 71.4% played amateur sports, including football (60.9%), soccer (17.2%), hockey (7.8%), and wrestling (7%).

The cohort includes the first known American female athlete with CTE. Recruiting female brain donors has always been a challenge, Dr. McKee said. In this study, females comprised about 7% of the entire cohort and tended to be younger and play fewer years of a sport, compared with their male counterparts. All of that could lower their risk for CTE, Dr. McKee said.

“We don’t have enough brain donations to make any comments about differences between the genders, but we’ve always known that women can develop CTE,” she said. “It’s been reported after domestic violence and in an autistic woman who was a headbanger, so it was just a matter of time before we found our first case.”
 

Early stage of CTE?

Neuropathological analysis revealed neuronal p-tau aggregates in all CTE cases, a hallmark of the disease.

Young athletes with CTE had significantly more ventricular dilatation, suggesting atrophy or shrinkage of the brain, and more cavum septum pellucidum.

“I was surprised that even at this very young age group we could see structural changes to the gross pathology,” Dr. McKee said.

Investigators also found evidence of perivascular macrophages in the deep white matter, a microscopic change that correlated with CTE and years of play and indicates a breach of the blood-brain barrier that could allow pro-inflammatory molecules to enter the brain, setting up a neuroinflammatory response.

“Neuroinflammation is a very early change after repetitive head impacts, as well as in CTE,” Dr. McKee said. “This may be one of the mechanisms by which the inflammation starts, meaning microvascular injury might be an integral part of the pathogenesis of CTE.”
 

A message for clinicians

All athletes had symptoms of mood and neurobehavioral dysfunction common in people with RHIs. There were no significant differences in those clinical symptoms based on CTE diagnosis, which is likely related to the retrospective nature of the clinical evaluations, Dr. McKee said.

While the study leaves many questions about CTE in younger athletes unanswered, there is a message for clinicians and for patients in the findings, she said.

For clinicians, it’s important to note that “this young population of amateur athletes can be very symptomatic, and in all likelihood, a lot of these symptoms are reversible with proper care and management,” Dr. McKee said.

“For individual athletes, it’s important to note that 58% of this cohort did not have CTE, so just because you have these symptoms is not an indication that you have a neurodegenerative disease,” she added.

The study was funded by Andlinger Foundation, the National Football League, Mac Parkman Foundation, National Operating Committee on Standards for Athletic Equipment, and the Nick and Lynn Buoniconti Foundation, World Wrestling Entertainment, Alzheimer’s Association, National Institutes of Health, Concussion Legacy Foundation, U.S. Department of Defense and the U.S. Department of Veterans Affairs. Dr. McKee is a member of the Mackey-White Health and Safety Committee of the National Football League Players Association and reported receiving grants from the NIH and Department of Veteran Affairs and other funding from the Buoniconti Foundation and Mac Parkman Foundation during the conduct of the study.

A version of this article appeared on Medscape.com.

The largest study to date of chronic traumatic encephalopathy (CTE) in young athletes shows that 41% had the neurodegenerative disease, caused by repetitive head impacts (RHIs).

Analysis of brain tissue from athletes who were exposed to RHIs and died before the age of 30 revealed neuropathological evidence of shrinkage of the brain and microscopic changes that indicate a breach of the blood-brain barrier. The case series also identified the first known American female athlete with CTE.

Nearly all of those with CTE had a mild form of the disease and 71% played only at the amateur level in youth, high school, or college sports.

“A lot of people think CTE is a result of high-level, professional play such as football, ice hockey, and boxing, but it can affect amateur athletes and can affect people at a young age,” lead author Ann McKee, MD, professor of neurology and pathology and director of the Chronic Traumatic Encephalopathy Center at Boston University, said in an interview.

The findings were published online in JAMA Neurology.
 

A rare look

Brain donation at younger ages is rare, so most of what is known about CTE comes from studies in older athletes.

“We’ve always known that young people could develop this disease early after just amateur high school, youth, and college exposure, but this is the largest study of donor brains at this age,” Dr. McKee said.

The case series included 152 brains of athletes who played contact sports, experienced RHIs, and died before age 30. The tissues are part of the Understanding Neurologic Injury and Traumatic Encephalopathy (UNITE) Brain Bank and were donated between February 2008 and September 2022.

Researchers reviewed the donors’ medical records and conducted retrospective interviews with the donors’ next of kin to assess cognitive symptoms, mood disturbances, and neurobehavioral issues.

Donors died between the ages of 13 and 29 years, 92.8% were male and 73% were White. In 57.2% of the cases, suicide was the cause of death, with no difference between those with or without CTE.

CTE was neuropathologically diagnosed in 41.4% of athletes, using diagnostic criteria developed by the National Institute of Neurological Disorders and Stroke. 

More than 95% had mild CTE. Diagnosis was associated with older age (mean difference, 3.92 years; P < .001) and significantly more years of exposure to contact sports (11.6 vs. 8.8 years).

Among those with CTE, 71.4% played amateur sports, including football (60.9%), soccer (17.2%), hockey (7.8%), and wrestling (7%).

The cohort includes the first known American female athlete with CTE. Recruiting female brain donors has always been a challenge, Dr. McKee said. In this study, females comprised about 7% of the entire cohort and tended to be younger and play fewer years of a sport, compared with their male counterparts. All of that could lower their risk for CTE, Dr. McKee said.

“We don’t have enough brain donations to make any comments about differences between the genders, but we’ve always known that women can develop CTE,” she said. “It’s been reported after domestic violence and in an autistic woman who was a headbanger, so it was just a matter of time before we found our first case.”
 

Early stage of CTE?

Neuropathological analysis revealed neuronal p-tau aggregates in all CTE cases, a hallmark of the disease.

Young athletes with CTE had significantly more ventricular dilatation, suggesting atrophy or shrinkage of the brain, and more cavum septum pellucidum.

“I was surprised that even at this very young age group we could see structural changes to the gross pathology,” Dr. McKee said.

Investigators also found evidence of perivascular macrophages in the deep white matter, a microscopic change that correlated with CTE and years of play and indicates a breach of the blood-brain barrier that could allow pro-inflammatory molecules to enter the brain, setting up a neuroinflammatory response.

“Neuroinflammation is a very early change after repetitive head impacts, as well as in CTE,” Dr. McKee said. “This may be one of the mechanisms by which the inflammation starts, meaning microvascular injury might be an integral part of the pathogenesis of CTE.”
 

A message for clinicians

All athletes had symptoms of mood and neurobehavioral dysfunction common in people with RHIs. There were no significant differences in those clinical symptoms based on CTE diagnosis, which is likely related to the retrospective nature of the clinical evaluations, Dr. McKee said.

While the study leaves many questions about CTE in younger athletes unanswered, there is a message for clinicians and for patients in the findings, she said.

For clinicians, it’s important to note that “this young population of amateur athletes can be very symptomatic, and in all likelihood, a lot of these symptoms are reversible with proper care and management,” Dr. McKee said.

“For individual athletes, it’s important to note that 58% of this cohort did not have CTE, so just because you have these symptoms is not an indication that you have a neurodegenerative disease,” she added.

The study was funded by Andlinger Foundation, the National Football League, Mac Parkman Foundation, National Operating Committee on Standards for Athletic Equipment, and the Nick and Lynn Buoniconti Foundation, World Wrestling Entertainment, Alzheimer’s Association, National Institutes of Health, Concussion Legacy Foundation, U.S. Department of Defense and the U.S. Department of Veterans Affairs. Dr. McKee is a member of the Mackey-White Health and Safety Committee of the National Football League Players Association and reported receiving grants from the NIH and Department of Veteran Affairs and other funding from the Buoniconti Foundation and Mac Parkman Foundation during the conduct of the study.

A version of this article appeared on Medscape.com.

The largest study to date of chronic traumatic encephalopathy (CTE) in young athletes shows that 41% had the neurodegenerative disease, caused by repetitive head impacts (RHIs).

Analysis of brain tissue from athletes who were exposed to RHIs and died before the age of 30 revealed neuropathological evidence of shrinkage of the brain and microscopic changes that indicate a breach of the blood-brain barrier. The case series also identified the first known American female athlete with CTE.

Nearly all of those with CTE had a mild form of the disease and 71% played only at the amateur level in youth, high school, or college sports.

“A lot of people think CTE is a result of high-level, professional play such as football, ice hockey, and boxing, but it can affect amateur athletes and can affect people at a young age,” lead author Ann McKee, MD, professor of neurology and pathology and director of the Chronic Traumatic Encephalopathy Center at Boston University, said in an interview.

The findings were published online in JAMA Neurology.
 

A rare look

Brain donation at younger ages is rare, so most of what is known about CTE comes from studies in older athletes.

“We’ve always known that young people could develop this disease early after just amateur high school, youth, and college exposure, but this is the largest study of donor brains at this age,” Dr. McKee said.

The case series included 152 brains of athletes who played contact sports, experienced RHIs, and died before age 30. The tissues are part of the Understanding Neurologic Injury and Traumatic Encephalopathy (UNITE) Brain Bank and were donated between February 2008 and September 2022.

Researchers reviewed the donors’ medical records and conducted retrospective interviews with the donors’ next of kin to assess cognitive symptoms, mood disturbances, and neurobehavioral issues.

Donors died between the ages of 13 and 29 years, 92.8% were male and 73% were White. In 57.2% of the cases, suicide was the cause of death, with no difference between those with or without CTE.

CTE was neuropathologically diagnosed in 41.4% of athletes, using diagnostic criteria developed by the National Institute of Neurological Disorders and Stroke. 

More than 95% had mild CTE. Diagnosis was associated with older age (mean difference, 3.92 years; P < .001) and significantly more years of exposure to contact sports (11.6 vs. 8.8 years).

Among those with CTE, 71.4% played amateur sports, including football (60.9%), soccer (17.2%), hockey (7.8%), and wrestling (7%).

The cohort includes the first known American female athlete with CTE. Recruiting female brain donors has always been a challenge, Dr. McKee said. In this study, females comprised about 7% of the entire cohort and tended to be younger and play fewer years of a sport, compared with their male counterparts. All of that could lower their risk for CTE, Dr. McKee said.

“We don’t have enough brain donations to make any comments about differences between the genders, but we’ve always known that women can develop CTE,” she said. “It’s been reported after domestic violence and in an autistic woman who was a headbanger, so it was just a matter of time before we found our first case.”
 

Early stage of CTE?

Neuropathological analysis revealed neuronal p-tau aggregates in all CTE cases, a hallmark of the disease.

Young athletes with CTE had significantly more ventricular dilatation, suggesting atrophy or shrinkage of the brain, and more cavum septum pellucidum.

“I was surprised that even at this very young age group we could see structural changes to the gross pathology,” Dr. McKee said.

Investigators also found evidence of perivascular macrophages in the deep white matter, a microscopic change that correlated with CTE and years of play and indicates a breach of the blood-brain barrier that could allow pro-inflammatory molecules to enter the brain, setting up a neuroinflammatory response.

“Neuroinflammation is a very early change after repetitive head impacts, as well as in CTE,” Dr. McKee said. “This may be one of the mechanisms by which the inflammation starts, meaning microvascular injury might be an integral part of the pathogenesis of CTE.”
 

A message for clinicians

All athletes had symptoms of mood and neurobehavioral dysfunction common in people with RHIs. There were no significant differences in those clinical symptoms based on CTE diagnosis, which is likely related to the retrospective nature of the clinical evaluations, Dr. McKee said.

While the study leaves many questions about CTE in younger athletes unanswered, there is a message for clinicians and for patients in the findings, she said.

For clinicians, it’s important to note that “this young population of amateur athletes can be very symptomatic, and in all likelihood, a lot of these symptoms are reversible with proper care and management,” Dr. McKee said.

“For individual athletes, it’s important to note that 58% of this cohort did not have CTE, so just because you have these symptoms is not an indication that you have a neurodegenerative disease,” she added.

The study was funded by Andlinger Foundation, the National Football League, Mac Parkman Foundation, National Operating Committee on Standards for Athletic Equipment, and the Nick and Lynn Buoniconti Foundation, World Wrestling Entertainment, Alzheimer’s Association, National Institutes of Health, Concussion Legacy Foundation, U.S. Department of Defense and the U.S. Department of Veterans Affairs. Dr. McKee is a member of the Mackey-White Health and Safety Committee of the National Football League Players Association and reported receiving grants from the NIH and Department of Veteran Affairs and other funding from the Buoniconti Foundation and Mac Parkman Foundation during the conduct of the study.

A version of this article appeared on Medscape.com.

The latest results of the Monitoring the Future (MTF) longitudinal survey show that American adults are consuming marijuana and hallucinogens, vaping, and binge drinking at historic levels.

“In 2022, we are seeing that marijuana and hallucinogen use, and vaping of nicotine and marijuana, are higher than ever among young adults ages 19-30,” said Megan Patrick, research professor and principal investigator of the MTF study. “In addition, midlife adults ages 35-50 have the highest level of binge drinking we have ever seen in that age group,” she said in a statement.

The survey, conducted annually since 1975 by the University of Michigan’s Institute for Social Research, Ann Arbor, queries nationally representative samples of 8th, 10th, and 12th graders and then follows a subset through adulthood to come up with longitudinal data. It is funded by the National Institute on Drug Abuse.

The adult data for 2022 were gathered by online and paper surveys from April to October 2022 and included responses from some 10,000 individuals. Participants were divided into two cohorts: those aged 19-30 years and those aged 35-50 years.

About a third of the older age group reported using marijuana in the past year, an all-time high, up from 25% in 2021 and more than double the users in 2012 (13%). Of this group, 4% reported past-year hallucinogen use, also a record high and double the reported use in 2021.

Alcohol use among adults aged 35-50 has gradually increased over the past decade. Of this group, 85% reported past-year drinking in 2022, up from 83% in 2012.

Binge drinking – defined as having five or more drinks in a row in the past 2 weeks – has also been on the rise in the past decade. One-third of older adults reported binge drinking in 2022. Binge drinking was highest among White (31.4%) and Hispanic (30.6%) midlife adults and was lower among Black (17.1%) midlife adults.

Vaping among the older age cohort has remained at similar levels since first measured in 2019; 9% vaped marijuana in the past year, while 7% vaped nicotine.
 

Marijuana popular among younger Americans

“In 2022, marijuana use among young adults reached the highest levels ever recorded since the indices were first available in 1988,” the study authors write. Both past-year and daily use hit record levels for the cohort of those aged 19-30.

Forty-four percent reported past-year marijuana use, up from 28% in 2012. The highest levels of use were in those aged 27-28. One in five reported daily use, up from 6% a decade ago; almost 14% of 23- to 24-year-olds reported daily use.

Past-year use of hallucinogens – including LSD, MDMA, mescaline, peyote, mushrooms or psilocybin, and PCP – was reported by 8% of this age group. Most of the increase was driven by use of hallucinogens other than LSD, which accounted for 7% of the reported use.

Young adults also reported record levels of vaping marijuana, with 21% reporting past-year use and 14% reporting past-month use. Vaping of nicotine has doubled in prevalence since the survey started asking about it, from 14% for past-year use in 2017 to 24% in 2022.

NIDA Director Nora Volkow, MD, noted in a statement that the survey results show that “substance use is not limited to teens and young adults,” adding that “these data help us understand how people use drugs across the lifespan.”

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

The latest results of the Monitoring the Future (MTF) longitudinal survey show that American adults are consuming marijuana and hallucinogens, vaping, and binge drinking at historic levels.

“In 2022, we are seeing that marijuana and hallucinogen use, and vaping of nicotine and marijuana, are higher than ever among young adults ages 19-30,” said Megan Patrick, research professor and principal investigator of the MTF study. “In addition, midlife adults ages 35-50 have the highest level of binge drinking we have ever seen in that age group,” she said in a statement.

The survey, conducted annually since 1975 by the University of Michigan’s Institute for Social Research, Ann Arbor, queries nationally representative samples of 8th, 10th, and 12th graders and then follows a subset through adulthood to come up with longitudinal data. It is funded by the National Institute on Drug Abuse.

The adult data for 2022 were gathered by online and paper surveys from April to October 2022 and included responses from some 10,000 individuals. Participants were divided into two cohorts: those aged 19-30 years and those aged 35-50 years.

About a third of the older age group reported using marijuana in the past year, an all-time high, up from 25% in 2021 and more than double the users in 2012 (13%). Of this group, 4% reported past-year hallucinogen use, also a record high and double the reported use in 2021.

Alcohol use among adults aged 35-50 has gradually increased over the past decade. Of this group, 85% reported past-year drinking in 2022, up from 83% in 2012.

Binge drinking – defined as having five or more drinks in a row in the past 2 weeks – has also been on the rise in the past decade. One-third of older adults reported binge drinking in 2022. Binge drinking was highest among White (31.4%) and Hispanic (30.6%) midlife adults and was lower among Black (17.1%) midlife adults.

Vaping among the older age cohort has remained at similar levels since first measured in 2019; 9% vaped marijuana in the past year, while 7% vaped nicotine.
 

Marijuana popular among younger Americans

“In 2022, marijuana use among young adults reached the highest levels ever recorded since the indices were first available in 1988,” the study authors write. Both past-year and daily use hit record levels for the cohort of those aged 19-30.

Forty-four percent reported past-year marijuana use, up from 28% in 2012. The highest levels of use were in those aged 27-28. One in five reported daily use, up from 6% a decade ago; almost 14% of 23- to 24-year-olds reported daily use.

Past-year use of hallucinogens – including LSD, MDMA, mescaline, peyote, mushrooms or psilocybin, and PCP – was reported by 8% of this age group. Most of the increase was driven by use of hallucinogens other than LSD, which accounted for 7% of the reported use.

Young adults also reported record levels of vaping marijuana, with 21% reporting past-year use and 14% reporting past-month use. Vaping of nicotine has doubled in prevalence since the survey started asking about it, from 14% for past-year use in 2017 to 24% in 2022.

NIDA Director Nora Volkow, MD, noted in a statement that the survey results show that “substance use is not limited to teens and young adults,” adding that “these data help us understand how people use drugs across the lifespan.”

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

The latest results of the Monitoring the Future (MTF) longitudinal survey show that American adults are consuming marijuana and hallucinogens, vaping, and binge drinking at historic levels.

“In 2022, we are seeing that marijuana and hallucinogen use, and vaping of nicotine and marijuana, are higher than ever among young adults ages 19-30,” said Megan Patrick, research professor and principal investigator of the MTF study. “In addition, midlife adults ages 35-50 have the highest level of binge drinking we have ever seen in that age group,” she said in a statement.

The survey, conducted annually since 1975 by the University of Michigan’s Institute for Social Research, Ann Arbor, queries nationally representative samples of 8th, 10th, and 12th graders and then follows a subset through adulthood to come up with longitudinal data. It is funded by the National Institute on Drug Abuse.

The adult data for 2022 were gathered by online and paper surveys from April to October 2022 and included responses from some 10,000 individuals. Participants were divided into two cohorts: those aged 19-30 years and those aged 35-50 years.

About a third of the older age group reported using marijuana in the past year, an all-time high, up from 25% in 2021 and more than double the users in 2012 (13%). Of this group, 4% reported past-year hallucinogen use, also a record high and double the reported use in 2021.

Alcohol use among adults aged 35-50 has gradually increased over the past decade. Of this group, 85% reported past-year drinking in 2022, up from 83% in 2012.

Binge drinking – defined as having five or more drinks in a row in the past 2 weeks – has also been on the rise in the past decade. One-third of older adults reported binge drinking in 2022. Binge drinking was highest among White (31.4%) and Hispanic (30.6%) midlife adults and was lower among Black (17.1%) midlife adults.

Vaping among the older age cohort has remained at similar levels since first measured in 2019; 9% vaped marijuana in the past year, while 7% vaped nicotine.
 

Marijuana popular among younger Americans

“In 2022, marijuana use among young adults reached the highest levels ever recorded since the indices were first available in 1988,” the study authors write. Both past-year and daily use hit record levels for the cohort of those aged 19-30.

Forty-four percent reported past-year marijuana use, up from 28% in 2012. The highest levels of use were in those aged 27-28. One in five reported daily use, up from 6% a decade ago; almost 14% of 23- to 24-year-olds reported daily use.

Past-year use of hallucinogens – including LSD, MDMA, mescaline, peyote, mushrooms or psilocybin, and PCP – was reported by 8% of this age group. Most of the increase was driven by use of hallucinogens other than LSD, which accounted for 7% of the reported use.

Young adults also reported record levels of vaping marijuana, with 21% reporting past-year use and 14% reporting past-month use. Vaping of nicotine has doubled in prevalence since the survey started asking about it, from 14% for past-year use in 2017 to 24% in 2022.

NIDA Director Nora Volkow, MD, noted in a statement that the survey results show that “substance use is not limited to teens and young adults,” adding that “these data help us understand how people use drugs across the lifespan.”

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

Pediatric oncologist Lena Winestone, MD, recalls treating a 2-year-old leukemia patient who underwent a bone marrow transplant as her only chance for a cure.

The girl’s family, who spoke only Spanish and struggled with literacy, could not pay their rent or afford the girl’s weekly transportation to the hospital for after-transplant care. The family had three other children and lived more than 2 hours from the transplant center, remembers Dr. Winestone, an assistant professor of pediatrics in the division of malignancies and bone & marrow transplant at the University of California, San Francisco.

The hospital’s social worker was able to secure grant support for the family’s housing and worked with the patient’s insurance to arrange for transportation. However, the departure times were rigid, Dr. Winestone said, and the family sometimes had to leave the hospital before the child’s graft vs. host disease (GvHD) treatment was complete for the day. 

“If we had not finished her treatment, we had to disconnect her from the machine early,” Dr. Winestone said. “Her mother also had to load her oxygen tanks [three of them], her BiPAP machine, and her tube feeds into the transportation every week in order to make sure she could be safely transported. She was treated for GvHD for almost 2 years, but unfortunately, her GvHD started to affect her lungs and ultimately, she passed away.”

Dr. Winestone says it’s difficult to know whether the girl’s death was directly related to her socioeconomic status, but that it certainly made all aspects of the child’s care more complicated and forced health care providers to adapt her cancer care to accommodate the family’s circumstances.

This story is one of countless cases where socioeconomic status impacted a young patient’s cancer care and likely contributed to a worse outcome. A plethora of data has demonstrated that children with cancer who are Black, Hispanic, or of lower socioeconomic status are more likely to relapse and die even when treated uniformly on clinical trials.

A 2022 study for example, found that children from marginalized racial/ethnic groups and those living in poverty were more likely to have inferior 5-year overall survival, compared with other children, even when assigned to receive the same initial treatment. Of 696 children with high-risk neuroblastoma, 47% of Hispanic children had a 5-year overall survival (OS), compared with 50% for other non-Hispanic children, and 61% for white non-Hispanic patients. Children on public health insurance (a proxy for household poverty) had a 53% 5-year OS, compared with 63% for children unexposed to household poverty. Pediatric patients exposed to neighborhood poverty had a 54% 5-year OS, compared with 62% for unexposed children.

In another study, children with acute lymphoblastic leukemia who lived in high-poverty areas were more likely to experience early relapse than other patients, despite having the same treatment. Of the 575 children studied, 92% of children from high-poverty areas who relapsed, experienced early relapse, defined as less than 36 months after remission. By comparison, only 48% of other children who relapsed experienced early relapse.

Reasons behind the relapse and survival disparities are multifold, which has led to challenges in addressing the gaps and improving cancer outcomes for poverty-stricken children. A research infrastructure that is largely based on biological, rather than social determinants of health, acts as another barrier, oncologists say.

Historically, interventions to address disparities in pediatric oncology have never been evaluated, said Kira Bona, MD, MPH, a pediatric oncologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. This is in large part because the body of literature illustrating the disparities is relatively new, said Dr. Bona, whose research focuses on poverty-associated outcome disparities in childhood cancer.

However, new efforts aim to change this landscape by using the growing data to develop and analyze possible interventions. A set of three novel interventions led by Dr. Bona and her research team are in the works, some of which have shown promise in early studies.

“Now is the time to begin to actively intervene on disparities in childhood cancer,” Dr. Bona said. “We’re really good at studying genetic mutations in cancer cells that might lead to a risk of relapse, and when we identify those mutations, what we do is intervene. We try new chemotherapy agents, new ways of delivering therapy. We are now at the point where we have identified that social determinants of health may be equally ‘risky’ but we haven’t taken the next step to begin intervening in the same way.”
 

What is causing disparities in pediatric cancer outcomes?

Lack of access to the health care system is a top contributor to the disparities, although there is no single root cause, said Sharon Castellino, MD, director of the Leukemia and Lymphoma Program at the Aflac Cancer & Blood Disorders Center of Children’s Healthcare of Atlanta, and a professor in the department of pediatrics at Emory University, Atlanta.

Even before cancer diagnosis, Dr. Castellino notes that many children of color and/or of lower socioeconomic status are not receiving regular health care, leading to sicker children and more advanced-stage cancer by the time they are diagnosed.

Lack of insurance is a primary barrier to this access, adds Xu Ji, PhD, MSPH, an assistant professor in the department of pediatrics at Emory University and a member of the Cancer Prevention and Control Research Program at the university’s Winship Cancer Institute.

Studies  have long shown that uninsured children are more likely to go without needed care, compared with those with private insurance. Patients of color are at much higher risk of being uninsured than White patients, with the uninsured rates for Hispanic, American Indian, and Alaska Native patients being more than 2.5 times higher than that of White patients.

“We all know that insurance is a strong predictor of health outcomes,” said Dr. Ji, whose research focuses on insurance disparities and gains among cancer patients. “Lack of insurance coverage and therefore lack of access to care along the pediatric cancer continuum from early detection to early diagnosis to timely initiation of treatment to receipt of high-quality treatment to access to recommended survivorship care and even access to palliative and end-of-life care are all very important constructs in the pathway from poverty to ultimate cancer outcomes for children.”

Unstable housing, employment difficulties, and lack of family support can also come into play. Dr. Castellino remembers the case of a 12-year-old cancer patient who entered treatment with advanced-stage Hodgkin Lymphoma. The girl came from a low-income, single-parent household without stable housing. Dr. Castellino said when the child was granted a wish from the Make-a-Wish Foundation, she asked for her own bed.

“We had been working with her every week for 6 months when that request came up,” she recalled. “We said, ‘You don’t have to wait for your make-a-wish, we can get you a bed now.’ We don’t even know the extent of what happens at home for many of these children.”

The impact of toxic stress on child cancer patients is an emerging area of research, said Dr. Winestone, whose research explores racial, ethnic, and socioeconomic disparities in access to care and outcomes of leukemia and lymphoma treatment. For example, Dr. Winestone’s research includes understanding how exposure to poverty or adverse experiences in childhood may influence a patient’s biological response to chemotherapy.

Other contributors to disparities include transportation issues, lack of childcare for other children, literacy, and language barriers. A 2016 study  suggests that language barriers negatively impact the quality of informed decision-making and the care experience for Spanish-speaking parents of pediatric cancer patients with limited English proficiency.

Such access issues are also compounded by systemic factors, including a shortage of physicians of color who may be able to forge better trust relationships with families of similar race and ethnicity, Dr. Castellino adds. Lower enrollment of pediatric cancer patients with higher social vulnerabilities in clinical trials is another problem.

“In childhood cancer, I believe our improvements have been built on the backs of prior generations of families and children who have enrolled in trials. We learn things, and the next generation of therapy improves,” Dr. Castellino said. “If you have a whole group of the population not represented in trials, you don’t know what’s driving the fact they may or may not improve.”
 

Working toward solutions  

With such a diverse set of factors fueling outcome gaps, a similarly diverse approach is needed to help bridge the divide, say disparity researchers.

To this end, Dr. Bona and her research team are currently building the first portfolio of health equity interventions, each designed to address a different adverse social determinant of health differently.

The Pediatric Cancer Resource Equity (PediCARE) intervention is a centrally delivered, household material hardship (HMH)–targeted intervention that provides transportation and groceries to low-income pediatric oncology families. The intervention was recently studied in a pilot, randomized, controlled trial at Dana-Farber Cancer Institute and the University of Alabama between May 2019 and August 2021.  

Families were first screened for HMH and randomized into receiving either the intervention or usual care for 6 months. The intervention group received groceries via Instacart and transportation to and from the hospital coordinated through the Ride Health platform using Uber or Lyft. For families with their own cars, gas cards were provided. Of the families offered the chance to participate, 100% agreed to participate in the program, and there was 0% attrition in either arm of the program during the 6 months, according to the study findings, which were presented at the 2023 American Society of Clinical Oncology annual meeting in June.   

Among families who received the PediCARE intervention, 100% successfully received grocery and transportation resources, 100% reported that it was “easier to buy food for my family,” 85% reported it was easier to get to and from the hospital, and 95% reported they would be “very likely to recommend the intervention to other families,” according to the results.

“The key takeaway is that we had excellent feasibility outcomes,” said Haley Newman, MD, lead author of the study and an attending physician in the division of oncology at The Children’s Hospital of Philadelphia. “From this study, we learned that PediCARE is accessible and feasible in very diverse settings. From this, what we really took away is that PediCARE could be successfully rolled out in a phase 3 randomized trial, which would be the best way to examine efficacy.”

Another initiative in its early stages is Pediatric RISE, a guaranteed income intervention being developed with support from the Children’s Cancer Research Fund, the American Cancer Society, and other donors. The intervention will provide unrestricted cash transfers to low-income families during the early months of chemotherapy, Dr. Bona said. Families are currently being enrolled in a pilot study with a goal of refining the intervention before it’s tested for feasibility and efficacy.

“The goal here is ultimately to evaluate the question: If we are able to successfully provide income support to low-income families going through childhood cancer treatment, might we be able to ameliorate some of the disparities associated with living in poverty that we have already described in childhood cancer,” Dr. Bona said.

Pediatric Assist, a developing intervention centering on benefits, is a third initiative that will soon be evaluated. The intervention will provide newly diagnosed families with systemic access to a centralized benefits counselor who can help them determine which existing government benefits they might be eligible for and assist them in navigating the application process.

“The idea here is that we know many lower-income families in the U.S. are eligible for existing supports, but may not be accessing them because of how incredibly difficult the system is to navigate,” Dr. Bona said. “For example, we know that low-income families may be eligible for SNAP benefits, but figuring out if you are eligible and then applying for SNAP involves multiple, complicated steps that are often infeasible for a parent when their child is admitted to the hospital with a newly diagnosed, life-threatening illness.”

Pilot refinement of the intervention is expected in the fall of 2023.
 

Overcoming barriers, addressing challenges

Investigators are also making headway in proving that collecting social determinants of health (SDoH) data during existing clinical trials is easily achievable.

Past Children’s Oncology Group trials have collected only race, ethnicity, insurance, and zip code data as proxies for exposure to adverse SDoH. Dr. Winestone and her colleagues recently investigated the feasibility and acceptability of the first COG trial to prospectively embed SDoH data collection.  

Of eligible participants, 360 of 413 opted-in to the embedded SDOH aim across 101 COG sites (87.2% consent rate). Among participants, 316 surveys (87.8%) were completed a median of 11 days post enrollment, according to the findings, which were presented at the ASCO annual meeting.

“We’ve come to realize the importance of the social determinants of health [as it pertains] to outcomes, but it has been a process to learn how to effectively collect that data in a large collaborative environment,” said Dr. Winestone. “This abstract demonstrates that patients are very willing to provide this data, and they’re able to do it in an efficient way. People think of these questions as very sensitive and that families may not want to share the answers, but this study demonstrates those presumptions are false.”

The authors hope the findings fuel incorporation of SDoH data collection in future National Clinical Trials Network trials to inform impactful health equity research.

While such research and intervention efforts are gaining momentum, challenges to do the work remain. A lack of research funding and support are among the obstacles, Dr. Winestone said.

To date, much of pediatric cancer work has focused on developing new therapeutic approaches to reach a cure for more patients, she explained.

“While that’s incredibly essential, if we’re creating these approaches that only work for a subset of patients that have resources, we’re contributing to the inequities in the system,” Dr. Winestone said. “Really, [we need] dedicated support to studying how to make sure the interventions we know are effective are reaching all populations, and that the patients are poised to benefit from those interventions by setting them up for success.”

A strong research infrastructure exists to evaluate and support clinical drug trials in pediatric oncology, but the same does not exist for health equity interventions, Dr. Bona adds. A significant question that needs to be addressed is how best to integrate health equity evaluation into existing infrastructure or whether to build a parallel infrastructure.

Despite the challenges, Dr. Bona believes now is exactly the right time to investigate and intervene in poverty as a risk factor for childhood cancer relapse and outcomes. What has led to success in childhood cancer is how pediatric oncology has collaborated across the country to operate clinical drug trials at various centers, all in the same way, to identify which treatments work best, she said.

“We have an opportunity now in pediatrics to take advantage of this highly successful clinical trials research infrastructure to integrate interventions to address disparities in a way that has not been done previously,” she said. “The opportunity to significantly improve survival in childhood cancer by reducing disparities exists if we take this head on from a research and funding perspective and approach social risk factors just as we already know how to approach tumor genomic risk factors.”

Pediatric oncologist Lena Winestone, MD, recalls treating a 2-year-old leukemia patient who underwent a bone marrow transplant as her only chance for a cure.

The girl’s family, who spoke only Spanish and struggled with literacy, could not pay their rent or afford the girl’s weekly transportation to the hospital for after-transplant care. The family had three other children and lived more than 2 hours from the transplant center, remembers Dr. Winestone, an assistant professor of pediatrics in the division of malignancies and bone & marrow transplant at the University of California, San Francisco.

The hospital’s social worker was able to secure grant support for the family’s housing and worked with the patient’s insurance to arrange for transportation. However, the departure times were rigid, Dr. Winestone said, and the family sometimes had to leave the hospital before the child’s graft vs. host disease (GvHD) treatment was complete for the day. 

“If we had not finished her treatment, we had to disconnect her from the machine early,” Dr. Winestone said. “Her mother also had to load her oxygen tanks [three of them], her BiPAP machine, and her tube feeds into the transportation every week in order to make sure she could be safely transported. She was treated for GvHD for almost 2 years, but unfortunately, her GvHD started to affect her lungs and ultimately, she passed away.”

Dr. Winestone says it’s difficult to know whether the girl’s death was directly related to her socioeconomic status, but that it certainly made all aspects of the child’s care more complicated and forced health care providers to adapt her cancer care to accommodate the family’s circumstances.

This story is one of countless cases where socioeconomic status impacted a young patient’s cancer care and likely contributed to a worse outcome. A plethora of data has demonstrated that children with cancer who are Black, Hispanic, or of lower socioeconomic status are more likely to relapse and die even when treated uniformly on clinical trials.

A 2022 study for example, found that children from marginalized racial/ethnic groups and those living in poverty were more likely to have inferior 5-year overall survival, compared with other children, even when assigned to receive the same initial treatment. Of 696 children with high-risk neuroblastoma, 47% of Hispanic children had a 5-year overall survival (OS), compared with 50% for other non-Hispanic children, and 61% for white non-Hispanic patients. Children on public health insurance (a proxy for household poverty) had a 53% 5-year OS, compared with 63% for children unexposed to household poverty. Pediatric patients exposed to neighborhood poverty had a 54% 5-year OS, compared with 62% for unexposed children.

In another study, children with acute lymphoblastic leukemia who lived in high-poverty areas were more likely to experience early relapse than other patients, despite having the same treatment. Of the 575 children studied, 92% of children from high-poverty areas who relapsed, experienced early relapse, defined as less than 36 months after remission. By comparison, only 48% of other children who relapsed experienced early relapse.

Reasons behind the relapse and survival disparities are multifold, which has led to challenges in addressing the gaps and improving cancer outcomes for poverty-stricken children. A research infrastructure that is largely based on biological, rather than social determinants of health, acts as another barrier, oncologists say.

Historically, interventions to address disparities in pediatric oncology have never been evaluated, said Kira Bona, MD, MPH, a pediatric oncologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. This is in large part because the body of literature illustrating the disparities is relatively new, said Dr. Bona, whose research focuses on poverty-associated outcome disparities in childhood cancer.

However, new efforts aim to change this landscape by using the growing data to develop and analyze possible interventions. A set of three novel interventions led by Dr. Bona and her research team are in the works, some of which have shown promise in early studies.

“Now is the time to begin to actively intervene on disparities in childhood cancer,” Dr. Bona said. “We’re really good at studying genetic mutations in cancer cells that might lead to a risk of relapse, and when we identify those mutations, what we do is intervene. We try new chemotherapy agents, new ways of delivering therapy. We are now at the point where we have identified that social determinants of health may be equally ‘risky’ but we haven’t taken the next step to begin intervening in the same way.”
 

What is causing disparities in pediatric cancer outcomes?

Lack of access to the health care system is a top contributor to the disparities, although there is no single root cause, said Sharon Castellino, MD, director of the Leukemia and Lymphoma Program at the Aflac Cancer & Blood Disorders Center of Children’s Healthcare of Atlanta, and a professor in the department of pediatrics at Emory University, Atlanta.

Even before cancer diagnosis, Dr. Castellino notes that many children of color and/or of lower socioeconomic status are not receiving regular health care, leading to sicker children and more advanced-stage cancer by the time they are diagnosed.

Lack of insurance is a primary barrier to this access, adds Xu Ji, PhD, MSPH, an assistant professor in the department of pediatrics at Emory University and a member of the Cancer Prevention and Control Research Program at the university’s Winship Cancer Institute.

Studies  have long shown that uninsured children are more likely to go without needed care, compared with those with private insurance. Patients of color are at much higher risk of being uninsured than White patients, with the uninsured rates for Hispanic, American Indian, and Alaska Native patients being more than 2.5 times higher than that of White patients.

“We all know that insurance is a strong predictor of health outcomes,” said Dr. Ji, whose research focuses on insurance disparities and gains among cancer patients. “Lack of insurance coverage and therefore lack of access to care along the pediatric cancer continuum from early detection to early diagnosis to timely initiation of treatment to receipt of high-quality treatment to access to recommended survivorship care and even access to palliative and end-of-life care are all very important constructs in the pathway from poverty to ultimate cancer outcomes for children.”

Unstable housing, employment difficulties, and lack of family support can also come into play. Dr. Castellino remembers the case of a 12-year-old cancer patient who entered treatment with advanced-stage Hodgkin Lymphoma. The girl came from a low-income, single-parent household without stable housing. Dr. Castellino said when the child was granted a wish from the Make-a-Wish Foundation, she asked for her own bed.

“We had been working with her every week for 6 months when that request came up,” she recalled. “We said, ‘You don’t have to wait for your make-a-wish, we can get you a bed now.’ We don’t even know the extent of what happens at home for many of these children.”

The impact of toxic stress on child cancer patients is an emerging area of research, said Dr. Winestone, whose research explores racial, ethnic, and socioeconomic disparities in access to care and outcomes of leukemia and lymphoma treatment. For example, Dr. Winestone’s research includes understanding how exposure to poverty or adverse experiences in childhood may influence a patient’s biological response to chemotherapy.

Other contributors to disparities include transportation issues, lack of childcare for other children, literacy, and language barriers. A 2016 study  suggests that language barriers negatively impact the quality of informed decision-making and the care experience for Spanish-speaking parents of pediatric cancer patients with limited English proficiency.

Such access issues are also compounded by systemic factors, including a shortage of physicians of color who may be able to forge better trust relationships with families of similar race and ethnicity, Dr. Castellino adds. Lower enrollment of pediatric cancer patients with higher social vulnerabilities in clinical trials is another problem.

“In childhood cancer, I believe our improvements have been built on the backs of prior generations of families and children who have enrolled in trials. We learn things, and the next generation of therapy improves,” Dr. Castellino said. “If you have a whole group of the population not represented in trials, you don’t know what’s driving the fact they may or may not improve.”
 

Working toward solutions  

With such a diverse set of factors fueling outcome gaps, a similarly diverse approach is needed to help bridge the divide, say disparity researchers.

To this end, Dr. Bona and her research team are currently building the first portfolio of health equity interventions, each designed to address a different adverse social determinant of health differently.

The Pediatric Cancer Resource Equity (PediCARE) intervention is a centrally delivered, household material hardship (HMH)–targeted intervention that provides transportation and groceries to low-income pediatric oncology families. The intervention was recently studied in a pilot, randomized, controlled trial at Dana-Farber Cancer Institute and the University of Alabama between May 2019 and August 2021.  

Families were first screened for HMH and randomized into receiving either the intervention or usual care for 6 months. The intervention group received groceries via Instacart and transportation to and from the hospital coordinated through the Ride Health platform using Uber or Lyft. For families with their own cars, gas cards were provided. Of the families offered the chance to participate, 100% agreed to participate in the program, and there was 0% attrition in either arm of the program during the 6 months, according to the study findings, which were presented at the 2023 American Society of Clinical Oncology annual meeting in June.   

Among families who received the PediCARE intervention, 100% successfully received grocery and transportation resources, 100% reported that it was “easier to buy food for my family,” 85% reported it was easier to get to and from the hospital, and 95% reported they would be “very likely to recommend the intervention to other families,” according to the results.

“The key takeaway is that we had excellent feasibility outcomes,” said Haley Newman, MD, lead author of the study and an attending physician in the division of oncology at The Children’s Hospital of Philadelphia. “From this study, we learned that PediCARE is accessible and feasible in very diverse settings. From this, what we really took away is that PediCARE could be successfully rolled out in a phase 3 randomized trial, which would be the best way to examine efficacy.”

Another initiative in its early stages is Pediatric RISE, a guaranteed income intervention being developed with support from the Children’s Cancer Research Fund, the American Cancer Society, and other donors. The intervention will provide unrestricted cash transfers to low-income families during the early months of chemotherapy, Dr. Bona said. Families are currently being enrolled in a pilot study with a goal of refining the intervention before it’s tested for feasibility and efficacy.

“The goal here is ultimately to evaluate the question: If we are able to successfully provide income support to low-income families going through childhood cancer treatment, might we be able to ameliorate some of the disparities associated with living in poverty that we have already described in childhood cancer,” Dr. Bona said.

Pediatric Assist, a developing intervention centering on benefits, is a third initiative that will soon be evaluated. The intervention will provide newly diagnosed families with systemic access to a centralized benefits counselor who can help them determine which existing government benefits they might be eligible for and assist them in navigating the application process.

“The idea here is that we know many lower-income families in the U.S. are eligible for existing supports, but may not be accessing them because of how incredibly difficult the system is to navigate,” Dr. Bona said. “For example, we know that low-income families may be eligible for SNAP benefits, but figuring out if you are eligible and then applying for SNAP involves multiple, complicated steps that are often infeasible for a parent when their child is admitted to the hospital with a newly diagnosed, life-threatening illness.”

Pilot refinement of the intervention is expected in the fall of 2023.
 

Overcoming barriers, addressing challenges

Investigators are also making headway in proving that collecting social determinants of health (SDoH) data during existing clinical trials is easily achievable.

Past Children’s Oncology Group trials have collected only race, ethnicity, insurance, and zip code data as proxies for exposure to adverse SDoH. Dr. Winestone and her colleagues recently investigated the feasibility and acceptability of the first COG trial to prospectively embed SDoH data collection.  

Of eligible participants, 360 of 413 opted-in to the embedded SDOH aim across 101 COG sites (87.2% consent rate). Among participants, 316 surveys (87.8%) were completed a median of 11 days post enrollment, according to the findings, which were presented at the ASCO annual meeting.

“We’ve come to realize the importance of the social determinants of health [as it pertains] to outcomes, but it has been a process to learn how to effectively collect that data in a large collaborative environment,” said Dr. Winestone. “This abstract demonstrates that patients are very willing to provide this data, and they’re able to do it in an efficient way. People think of these questions as very sensitive and that families may not want to share the answers, but this study demonstrates those presumptions are false.”

The authors hope the findings fuel incorporation of SDoH data collection in future National Clinical Trials Network trials to inform impactful health equity research.

While such research and intervention efforts are gaining momentum, challenges to do the work remain. A lack of research funding and support are among the obstacles, Dr. Winestone said.

To date, much of pediatric cancer work has focused on developing new therapeutic approaches to reach a cure for more patients, she explained.

“While that’s incredibly essential, if we’re creating these approaches that only work for a subset of patients that have resources, we’re contributing to the inequities in the system,” Dr. Winestone said. “Really, [we need] dedicated support to studying how to make sure the interventions we know are effective are reaching all populations, and that the patients are poised to benefit from those interventions by setting them up for success.”

A strong research infrastructure exists to evaluate and support clinical drug trials in pediatric oncology, but the same does not exist for health equity interventions, Dr. Bona adds. A significant question that needs to be addressed is how best to integrate health equity evaluation into existing infrastructure or whether to build a parallel infrastructure.

Despite the challenges, Dr. Bona believes now is exactly the right time to investigate and intervene in poverty as a risk factor for childhood cancer relapse and outcomes. What has led to success in childhood cancer is how pediatric oncology has collaborated across the country to operate clinical drug trials at various centers, all in the same way, to identify which treatments work best, she said.

“We have an opportunity now in pediatrics to take advantage of this highly successful clinical trials research infrastructure to integrate interventions to address disparities in a way that has not been done previously,” she said. “The opportunity to significantly improve survival in childhood cancer by reducing disparities exists if we take this head on from a research and funding perspective and approach social risk factors just as we already know how to approach tumor genomic risk factors.”

Pediatric oncologist Lena Winestone, MD, recalls treating a 2-year-old leukemia patient who underwent a bone marrow transplant as her only chance for a cure.

The girl’s family, who spoke only Spanish and struggled with literacy, could not pay their rent or afford the girl’s weekly transportation to the hospital for after-transplant care. The family had three other children and lived more than 2 hours from the transplant center, remembers Dr. Winestone, an assistant professor of pediatrics in the division of malignancies and bone & marrow transplant at the University of California, San Francisco.

The hospital’s social worker was able to secure grant support for the family’s housing and worked with the patient’s insurance to arrange for transportation. However, the departure times were rigid, Dr. Winestone said, and the family sometimes had to leave the hospital before the child’s graft vs. host disease (GvHD) treatment was complete for the day. 

“If we had not finished her treatment, we had to disconnect her from the machine early,” Dr. Winestone said. “Her mother also had to load her oxygen tanks [three of them], her BiPAP machine, and her tube feeds into the transportation every week in order to make sure she could be safely transported. She was treated for GvHD for almost 2 years, but unfortunately, her GvHD started to affect her lungs and ultimately, she passed away.”

Dr. Winestone says it’s difficult to know whether the girl’s death was directly related to her socioeconomic status, but that it certainly made all aspects of the child’s care more complicated and forced health care providers to adapt her cancer care to accommodate the family’s circumstances.

This story is one of countless cases where socioeconomic status impacted a young patient’s cancer care and likely contributed to a worse outcome. A plethora of data has demonstrated that children with cancer who are Black, Hispanic, or of lower socioeconomic status are more likely to relapse and die even when treated uniformly on clinical trials.

A 2022 study for example, found that children from marginalized racial/ethnic groups and those living in poverty were more likely to have inferior 5-year overall survival, compared with other children, even when assigned to receive the same initial treatment. Of 696 children with high-risk neuroblastoma, 47% of Hispanic children had a 5-year overall survival (OS), compared with 50% for other non-Hispanic children, and 61% for white non-Hispanic patients. Children on public health insurance (a proxy for household poverty) had a 53% 5-year OS, compared with 63% for children unexposed to household poverty. Pediatric patients exposed to neighborhood poverty had a 54% 5-year OS, compared with 62% for unexposed children.

In another study, children with acute lymphoblastic leukemia who lived in high-poverty areas were more likely to experience early relapse than other patients, despite having the same treatment. Of the 575 children studied, 92% of children from high-poverty areas who relapsed, experienced early relapse, defined as less than 36 months after remission. By comparison, only 48% of other children who relapsed experienced early relapse.

Reasons behind the relapse and survival disparities are multifold, which has led to challenges in addressing the gaps and improving cancer outcomes for poverty-stricken children. A research infrastructure that is largely based on biological, rather than social determinants of health, acts as another barrier, oncologists say.

Historically, interventions to address disparities in pediatric oncology have never been evaluated, said Kira Bona, MD, MPH, a pediatric oncologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. This is in large part because the body of literature illustrating the disparities is relatively new, said Dr. Bona, whose research focuses on poverty-associated outcome disparities in childhood cancer.

However, new efforts aim to change this landscape by using the growing data to develop and analyze possible interventions. A set of three novel interventions led by Dr. Bona and her research team are in the works, some of which have shown promise in early studies.

“Now is the time to begin to actively intervene on disparities in childhood cancer,” Dr. Bona said. “We’re really good at studying genetic mutations in cancer cells that might lead to a risk of relapse, and when we identify those mutations, what we do is intervene. We try new chemotherapy agents, new ways of delivering therapy. We are now at the point where we have identified that social determinants of health may be equally ‘risky’ but we haven’t taken the next step to begin intervening in the same way.”
 

What is causing disparities in pediatric cancer outcomes?

Lack of access to the health care system is a top contributor to the disparities, although there is no single root cause, said Sharon Castellino, MD, director of the Leukemia and Lymphoma Program at the Aflac Cancer & Blood Disorders Center of Children’s Healthcare of Atlanta, and a professor in the department of pediatrics at Emory University, Atlanta.

Even before cancer diagnosis, Dr. Castellino notes that many children of color and/or of lower socioeconomic status are not receiving regular health care, leading to sicker children and more advanced-stage cancer by the time they are diagnosed.

Lack of insurance is a primary barrier to this access, adds Xu Ji, PhD, MSPH, an assistant professor in the department of pediatrics at Emory University and a member of the Cancer Prevention and Control Research Program at the university’s Winship Cancer Institute.

Studies  have long shown that uninsured children are more likely to go without needed care, compared with those with private insurance. Patients of color are at much higher risk of being uninsured than White patients, with the uninsured rates for Hispanic, American Indian, and Alaska Native patients being more than 2.5 times higher than that of White patients.

“We all know that insurance is a strong predictor of health outcomes,” said Dr. Ji, whose research focuses on insurance disparities and gains among cancer patients. “Lack of insurance coverage and therefore lack of access to care along the pediatric cancer continuum from early detection to early diagnosis to timely initiation of treatment to receipt of high-quality treatment to access to recommended survivorship care and even access to palliative and end-of-life care are all very important constructs in the pathway from poverty to ultimate cancer outcomes for children.”

Unstable housing, employment difficulties, and lack of family support can also come into play. Dr. Castellino remembers the case of a 12-year-old cancer patient who entered treatment with advanced-stage Hodgkin Lymphoma. The girl came from a low-income, single-parent household without stable housing. Dr. Castellino said when the child was granted a wish from the Make-a-Wish Foundation, she asked for her own bed.

“We had been working with her every week for 6 months when that request came up,” she recalled. “We said, ‘You don’t have to wait for your make-a-wish, we can get you a bed now.’ We don’t even know the extent of what happens at home for many of these children.”

The impact of toxic stress on child cancer patients is an emerging area of research, said Dr. Winestone, whose research explores racial, ethnic, and socioeconomic disparities in access to care and outcomes of leukemia and lymphoma treatment. For example, Dr. Winestone’s research includes understanding how exposure to poverty or adverse experiences in childhood may influence a patient’s biological response to chemotherapy.

Other contributors to disparities include transportation issues, lack of childcare for other children, literacy, and language barriers. A 2016 study  suggests that language barriers negatively impact the quality of informed decision-making and the care experience for Spanish-speaking parents of pediatric cancer patients with limited English proficiency.

Such access issues are also compounded by systemic factors, including a shortage of physicians of color who may be able to forge better trust relationships with families of similar race and ethnicity, Dr. Castellino adds. Lower enrollment of pediatric cancer patients with higher social vulnerabilities in clinical trials is another problem.

“In childhood cancer, I believe our improvements have been built on the backs of prior generations of families and children who have enrolled in trials. We learn things, and the next generation of therapy improves,” Dr. Castellino said. “If you have a whole group of the population not represented in trials, you don’t know what’s driving the fact they may or may not improve.”
 

Working toward solutions  

With such a diverse set of factors fueling outcome gaps, a similarly diverse approach is needed to help bridge the divide, say disparity researchers.

To this end, Dr. Bona and her research team are currently building the first portfolio of health equity interventions, each designed to address a different adverse social determinant of health differently.

The Pediatric Cancer Resource Equity (PediCARE) intervention is a centrally delivered, household material hardship (HMH)–targeted intervention that provides transportation and groceries to low-income pediatric oncology families. The intervention was recently studied in a pilot, randomized, controlled trial at Dana-Farber Cancer Institute and the University of Alabama between May 2019 and August 2021.  

Families were first screened for HMH and randomized into receiving either the intervention or usual care for 6 months. The intervention group received groceries via Instacart and transportation to and from the hospital coordinated through the Ride Health platform using Uber or Lyft. For families with their own cars, gas cards were provided. Of the families offered the chance to participate, 100% agreed to participate in the program, and there was 0% attrition in either arm of the program during the 6 months, according to the study findings, which were presented at the 2023 American Society of Clinical Oncology annual meeting in June.   

Among families who received the PediCARE intervention, 100% successfully received grocery and transportation resources, 100% reported that it was “easier to buy food for my family,” 85% reported it was easier to get to and from the hospital, and 95% reported they would be “very likely to recommend the intervention to other families,” according to the results.

“The key takeaway is that we had excellent feasibility outcomes,” said Haley Newman, MD, lead author of the study and an attending physician in the division of oncology at The Children’s Hospital of Philadelphia. “From this study, we learned that PediCARE is accessible and feasible in very diverse settings. From this, what we really took away is that PediCARE could be successfully rolled out in a phase 3 randomized trial, which would be the best way to examine efficacy.”

Another initiative in its early stages is Pediatric RISE, a guaranteed income intervention being developed with support from the Children’s Cancer Research Fund, the American Cancer Society, and other donors. The intervention will provide unrestricted cash transfers to low-income families during the early months of chemotherapy, Dr. Bona said. Families are currently being enrolled in a pilot study with a goal of refining the intervention before it’s tested for feasibility and efficacy.

“The goal here is ultimately to evaluate the question: If we are able to successfully provide income support to low-income families going through childhood cancer treatment, might we be able to ameliorate some of the disparities associated with living in poverty that we have already described in childhood cancer,” Dr. Bona said.

Pediatric Assist, a developing intervention centering on benefits, is a third initiative that will soon be evaluated. The intervention will provide newly diagnosed families with systemic access to a centralized benefits counselor who can help them determine which existing government benefits they might be eligible for and assist them in navigating the application process.

“The idea here is that we know many lower-income families in the U.S. are eligible for existing supports, but may not be accessing them because of how incredibly difficult the system is to navigate,” Dr. Bona said. “For example, we know that low-income families may be eligible for SNAP benefits, but figuring out if you are eligible and then applying for SNAP involves multiple, complicated steps that are often infeasible for a parent when their child is admitted to the hospital with a newly diagnosed, life-threatening illness.”

Pilot refinement of the intervention is expected in the fall of 2023.
 

Overcoming barriers, addressing challenges

Investigators are also making headway in proving that collecting social determinants of health (SDoH) data during existing clinical trials is easily achievable.

Past Children’s Oncology Group trials have collected only race, ethnicity, insurance, and zip code data as proxies for exposure to adverse SDoH. Dr. Winestone and her colleagues recently investigated the feasibility and acceptability of the first COG trial to prospectively embed SDoH data collection.  

Of eligible participants, 360 of 413 opted-in to the embedded SDOH aim across 101 COG sites (87.2% consent rate). Among participants, 316 surveys (87.8%) were completed a median of 11 days post enrollment, according to the findings, which were presented at the ASCO annual meeting.

“We’ve come to realize the importance of the social determinants of health [as it pertains] to outcomes, but it has been a process to learn how to effectively collect that data in a large collaborative environment,” said Dr. Winestone. “This abstract demonstrates that patients are very willing to provide this data, and they’re able to do it in an efficient way. People think of these questions as very sensitive and that families may not want to share the answers, but this study demonstrates those presumptions are false.”

The authors hope the findings fuel incorporation of SDoH data collection in future National Clinical Trials Network trials to inform impactful health equity research.

While such research and intervention efforts are gaining momentum, challenges to do the work remain. A lack of research funding and support are among the obstacles, Dr. Winestone said.

To date, much of pediatric cancer work has focused on developing new therapeutic approaches to reach a cure for more patients, she explained.

“While that’s incredibly essential, if we’re creating these approaches that only work for a subset of patients that have resources, we’re contributing to the inequities in the system,” Dr. Winestone said. “Really, [we need] dedicated support to studying how to make sure the interventions we know are effective are reaching all populations, and that the patients are poised to benefit from those interventions by setting them up for success.”

A strong research infrastructure exists to evaluate and support clinical drug trials in pediatric oncology, but the same does not exist for health equity interventions, Dr. Bona adds. A significant question that needs to be addressed is how best to integrate health equity evaluation into existing infrastructure or whether to build a parallel infrastructure.

Despite the challenges, Dr. Bona believes now is exactly the right time to investigate and intervene in poverty as a risk factor for childhood cancer relapse and outcomes. What has led to success in childhood cancer is how pediatric oncology has collaborated across the country to operate clinical drug trials at various centers, all in the same way, to identify which treatments work best, she said.

“We have an opportunity now in pediatrics to take advantage of this highly successful clinical trials research infrastructure to integrate interventions to address disparities in a way that has not been done previously,” she said. “The opportunity to significantly improve survival in childhood cancer by reducing disparities exists if we take this head on from a research and funding perspective and approach social risk factors just as we already know how to approach tumor genomic risk factors.”

While oral antibiotics remained the most prescribed systemic treatment for women with acne, spironolactone use continued to grow and became nearly as common as oral antibiotics, results from an analysis of prescribing trends from 2017 through 2020 showed.

Notably, isotretinoin prescribing among men and women decreased slightly during the study period, “which may reflect ongoing administrative burdens associated with iPLEDGE,” study author John S. Barbieri, MD, MBA, of the department of dermatology, at Brigham and Women’s Hospital, Boston, told this news organization.

For the cross-sectional study, which was published online as a research letter in JAMA Dermatology, Dr. Barbieri drew from the Truven Health MarketScan Commercial Claims Database from Jan. 1, 2017, to Dec. 31, 2020, to identify individuals with an encounter for acne, prescriptions for oral tetracycline antibiotics (doxycycline, minocycline), other commonly prescribed oral antibiotics (trimethoprim-sulfamethoxazole, amoxicillin, cephalexin), spironolactone, and isotretinoin. Only drug courses greater than 28 days were included in the analysis, and Dr. Barbieri stratified them according to clinician type (dermatologist, nondermatology physician, and nurse-practitioner or physician assistant). To normalize prescribing rates (to address possible changes in the number of patients treated for acne over time), the number of treatment courses prescribed each year was standardized to the number of encounters for acne with that clinician type during the same calendar year.

The study period included a mean of 1.9 million acne encounters per year.

Dr. Barbieri found that dermatologists prescribed more oral antibiotics per clinician for acne than any other major medical specialty and that oral antibiotics remained frequently prescribed for treating acne by both dermatologists and nondermatologists. “Among oral antibiotics, minocycline and trimethoprim-sulfamethoxazole remain relatively commonly prescribed, despite potential safety concerns and a lack of evidence that they are any more effective than doxycycline,” he said in an interview.

“Patient outcomes could likely be improved by reducing use of minocycline and particularly trimethoprim-sulfamethoxazole given its high risk of serious side effects such as SJS/TEN [Stevens-Johnson syndrome/toxic epidermal necrolysis] and acute respiratory failure,” he added.

Dr. Barbieri noted that there are likely opportunities to consider nonantibiotic alternatives such as hormonal therapy (spironolactone, combined oral contraceptives) and isotretinoin. “There is also a need for continued research to identify nonantibiotic treatment options for patients with acne,” he said.

The analysis revealed that for women with acne prescriptions for spironolactone increased about three- to fourfold during the study period among all clinician types. In 2017, oral antibiotics were prescribed about two- to threefold more often than spironolactone, but by 2020 they were being prescribed at about the same frequency. “Given spironolactone may have similar effectiveness to oral antibiotics in the treatment of acne, this shift in practice has the potential to improve outcomes for patients by reducing the risk of antibiotic-associated complications,” Dr. Barbieri wrote. Still, in 2020, oral antibiotics were still slightly more commonly prescribed than spironolactone by nondermatology physicians and NP or PAs.

In other findings, isotretinoin prescribing decreased slightly among male and female patients during the study period. Among antibiotic prescriptions, prescribing for doxycycline increased at a higher rate than prescribing for minocycline, especially among dermatologists and NPs or PAs.

In the interview, Dr. Barbieri acknowledged certain limitations of the study, including the fact that the dataset “does not allow for evaluation of severity of acne and it is not possible to directly link prescriptions to diagnoses, so some prescriptions might not be for acne and others that are for acne might not have been included.”

Lawrence J. Green, MD, of the department of dermatology at George Washington University, Washington, who was asked to comment on the results, said that, while a course of antibiotic therapy was tied to an office visit in the analysis, the duration of each course of therapy was unclear. It would be interesting to see if antibiotic courses became shorter during the time period analyzed, such as 1-3 months versus 4 or more months, he added, “as this should reduce risks associated with long-term use of oral antibiotics.”

Dr. Barbieri reported personal fees from Dexcel Pharma for consulting outside the submitted work. Dr. Green disclosed that he is a speaker, consultant, or investigator for numerous pharmaceutical companies.

While oral antibiotics remained the most prescribed systemic treatment for women with acne, spironolactone use continued to grow and became nearly as common as oral antibiotics, results from an analysis of prescribing trends from 2017 through 2020 showed.

Notably, isotretinoin prescribing among men and women decreased slightly during the study period, “which may reflect ongoing administrative burdens associated with iPLEDGE,” study author John S. Barbieri, MD, MBA, of the department of dermatology, at Brigham and Women’s Hospital, Boston, told this news organization.

For the cross-sectional study, which was published online as a research letter in JAMA Dermatology, Dr. Barbieri drew from the Truven Health MarketScan Commercial Claims Database from Jan. 1, 2017, to Dec. 31, 2020, to identify individuals with an encounter for acne, prescriptions for oral tetracycline antibiotics (doxycycline, minocycline), other commonly prescribed oral antibiotics (trimethoprim-sulfamethoxazole, amoxicillin, cephalexin), spironolactone, and isotretinoin. Only drug courses greater than 28 days were included in the analysis, and Dr. Barbieri stratified them according to clinician type (dermatologist, nondermatology physician, and nurse-practitioner or physician assistant). To normalize prescribing rates (to address possible changes in the number of patients treated for acne over time), the number of treatment courses prescribed each year was standardized to the number of encounters for acne with that clinician type during the same calendar year.

The study period included a mean of 1.9 million acne encounters per year.

Dr. Barbieri found that dermatologists prescribed more oral antibiotics per clinician for acne than any other major medical specialty and that oral antibiotics remained frequently prescribed for treating acne by both dermatologists and nondermatologists. “Among oral antibiotics, minocycline and trimethoprim-sulfamethoxazole remain relatively commonly prescribed, despite potential safety concerns and a lack of evidence that they are any more effective than doxycycline,” he said in an interview.

“Patient outcomes could likely be improved by reducing use of minocycline and particularly trimethoprim-sulfamethoxazole given its high risk of serious side effects such as SJS/TEN [Stevens-Johnson syndrome/toxic epidermal necrolysis] and acute respiratory failure,” he added.

Dr. Barbieri noted that there are likely opportunities to consider nonantibiotic alternatives such as hormonal therapy (spironolactone, combined oral contraceptives) and isotretinoin. “There is also a need for continued research to identify nonantibiotic treatment options for patients with acne,” he said.

The analysis revealed that for women with acne prescriptions for spironolactone increased about three- to fourfold during the study period among all clinician types. In 2017, oral antibiotics were prescribed about two- to threefold more often than spironolactone, but by 2020 they were being prescribed at about the same frequency. “Given spironolactone may have similar effectiveness to oral antibiotics in the treatment of acne, this shift in practice has the potential to improve outcomes for patients by reducing the risk of antibiotic-associated complications,” Dr. Barbieri wrote. Still, in 2020, oral antibiotics were still slightly more commonly prescribed than spironolactone by nondermatology physicians and NP or PAs.

In other findings, isotretinoin prescribing decreased slightly among male and female patients during the study period. Among antibiotic prescriptions, prescribing for doxycycline increased at a higher rate than prescribing for minocycline, especially among dermatologists and NPs or PAs.

In the interview, Dr. Barbieri acknowledged certain limitations of the study, including the fact that the dataset “does not allow for evaluation of severity of acne and it is not possible to directly link prescriptions to diagnoses, so some prescriptions might not be for acne and others that are for acne might not have been included.”

Lawrence J. Green, MD, of the department of dermatology at George Washington University, Washington, who was asked to comment on the results, said that, while a course of antibiotic therapy was tied to an office visit in the analysis, the duration of each course of therapy was unclear. It would be interesting to see if antibiotic courses became shorter during the time period analyzed, such as 1-3 months versus 4 or more months, he added, “as this should reduce risks associated with long-term use of oral antibiotics.”

Dr. Barbieri reported personal fees from Dexcel Pharma for consulting outside the submitted work. Dr. Green disclosed that he is a speaker, consultant, or investigator for numerous pharmaceutical companies.

While oral antibiotics remained the most prescribed systemic treatment for women with acne, spironolactone use continued to grow and became nearly as common as oral antibiotics, results from an analysis of prescribing trends from 2017 through 2020 showed.

Notably, isotretinoin prescribing among men and women decreased slightly during the study period, “which may reflect ongoing administrative burdens associated with iPLEDGE,” study author John S. Barbieri, MD, MBA, of the department of dermatology, at Brigham and Women’s Hospital, Boston, told this news organization.

For the cross-sectional study, which was published online as a research letter in JAMA Dermatology, Dr. Barbieri drew from the Truven Health MarketScan Commercial Claims Database from Jan. 1, 2017, to Dec. 31, 2020, to identify individuals with an encounter for acne, prescriptions for oral tetracycline antibiotics (doxycycline, minocycline), other commonly prescribed oral antibiotics (trimethoprim-sulfamethoxazole, amoxicillin, cephalexin), spironolactone, and isotretinoin. Only drug courses greater than 28 days were included in the analysis, and Dr. Barbieri stratified them according to clinician type (dermatologist, nondermatology physician, and nurse-practitioner or physician assistant). To normalize prescribing rates (to address possible changes in the number of patients treated for acne over time), the number of treatment courses prescribed each year was standardized to the number of encounters for acne with that clinician type during the same calendar year.

The study period included a mean of 1.9 million acne encounters per year.

Dr. Barbieri found that dermatologists prescribed more oral antibiotics per clinician for acne than any other major medical specialty and that oral antibiotics remained frequently prescribed for treating acne by both dermatologists and nondermatologists. “Among oral antibiotics, minocycline and trimethoprim-sulfamethoxazole remain relatively commonly prescribed, despite potential safety concerns and a lack of evidence that they are any more effective than doxycycline,” he said in an interview.

“Patient outcomes could likely be improved by reducing use of minocycline and particularly trimethoprim-sulfamethoxazole given its high risk of serious side effects such as SJS/TEN [Stevens-Johnson syndrome/toxic epidermal necrolysis] and acute respiratory failure,” he added.

Dr. Barbieri noted that there are likely opportunities to consider nonantibiotic alternatives such as hormonal therapy (spironolactone, combined oral contraceptives) and isotretinoin. “There is also a need for continued research to identify nonantibiotic treatment options for patients with acne,” he said.

The analysis revealed that for women with acne prescriptions for spironolactone increased about three- to fourfold during the study period among all clinician types. In 2017, oral antibiotics were prescribed about two- to threefold more often than spironolactone, but by 2020 they were being prescribed at about the same frequency. “Given spironolactone may have similar effectiveness to oral antibiotics in the treatment of acne, this shift in practice has the potential to improve outcomes for patients by reducing the risk of antibiotic-associated complications,” Dr. Barbieri wrote. Still, in 2020, oral antibiotics were still slightly more commonly prescribed than spironolactone by nondermatology physicians and NP or PAs.

In other findings, isotretinoin prescribing decreased slightly among male and female patients during the study period. Among antibiotic prescriptions, prescribing for doxycycline increased at a higher rate than prescribing for minocycline, especially among dermatologists and NPs or PAs.

In the interview, Dr. Barbieri acknowledged certain limitations of the study, including the fact that the dataset “does not allow for evaluation of severity of acne and it is not possible to directly link prescriptions to diagnoses, so some prescriptions might not be for acne and others that are for acne might not have been included.”

Lawrence J. Green, MD, of the department of dermatology at George Washington University, Washington, who was asked to comment on the results, said that, while a course of antibiotic therapy was tied to an office visit in the analysis, the duration of each course of therapy was unclear. It would be interesting to see if antibiotic courses became shorter during the time period analyzed, such as 1-3 months versus 4 or more months, he added, “as this should reduce risks associated with long-term use of oral antibiotics.”

Dr. Barbieri reported personal fees from Dexcel Pharma for consulting outside the submitted work. Dr. Green disclosed that he is a speaker, consultant, or investigator for numerous pharmaceutical companies.

As the political targeting of transgender youth and families continues to play out on the national stage, it is more important than ever for pediatricians and other primary care providers to support this vulnerable population by defending the recommendations and guidelines of reputable medical organizations based in science and to show grace and humility in caring for their patients.

Guidelines and resources

All leading medical groups in the United States with statements or policies related to gender-affirming care (including the American Medical Association, the American Academy of Pediatrics, the Society for Adolescent Health and Medicine, the Pediatric Endocrine Society, the American Academy of Child and Adolescent Psychiatry, and many more) recognize that this care is medically necessary and that exclusions for gender-related services are harmful to patients and their families. As pediatricians, families and youth rely on our expertise and guidance related to childhood and adolescent development, including the development of gender identity and ways to create safe and supportive environments needed for youth to reach their full potential.¹

While pediatricians are experts in youth development, some may have had limited access to training specific to LGBTQ+ identity development and interventions related to gender-affirming care. There are, however, readily accessible resources to help guide pediatricians in providing support and recommendations to families with concerns around gender or sexuality (See Resources). The American Academy of Pediatrics and Bright Futures recommend discussing the differences between assigned sex at birth and gender identity development with parents of those younger than 12 months of age as well as beginning to discuss and explore gender identity with all youth beginning at 4-5 years of age. Beginning at 8 years of age, pediatricians are also recommended to assess for a patient’s understanding and feelings toward emerging puberty to identify any potential concerns for gender dysphoria.² If concerns or questions emerge from screening, the family can then be referred to a gender-affirming care specialist for more support.
 

Gender dysphoria and gender-affirming care

Gender dysphoria may present in different ways and at different times for each patient. Some patients may present early in childhood with gender-diverse behaviors or the assertion of a gender identity different than their assigned sex at birth. However, the most commonly seen presentation is just prior to or during puberty, when one’s physical body starts to change in ways that are not consistent with their gender identity. Many patients report distress around gender before this time, but the distress that comes with the physical changes of puberty often prompts patients to reach out to parents, friends, and/or medical providers for help. Other than youth specifically disclosing their gender identity, as with any life stressors, gender dysphoria may initially present with a decline in school or social functioning, increased mood irritability, depression, or anxiety.³

The goal of support prior to puberty is for youth to grow and thrive as any other child and to not have gender dysphoria get in the way of normal development and social functioning.⁴ Some families will pursue social transition, the process of making changes within different areas of social interaction (such as name, pronouns, clothing, hairstyle, etc.) to decrease distress around gender. The decision of whether to pursue social transition is unique to each patient and family. The goal of this process is to allow youth to explore these changes in an effort to decrease the distress they experience in social interactions. Youth should be centered in this process and be the leader of any potential changes with parents and schools providing safe and supportive environments.⁵ Social transition has been shown to decrease rates of depression in gender-diverse youth to the same level as that of their cisgender peers.⁶

It is important to note that there are no recommended medical interventions for gender-affirming care before the time of puberty and, once a patient reaches Sexual Maturity Rating II (early puberty), the first potential treatment option is the reversible suppression of puberty using gonadotropin-releasing hormone (GnRH) analogues. The goal of this type of medication is to allow youth more time to explore their gender and avoid the permanent physical changes that occur during their endogenous puberty that can have a significant negative impact on their gender dysphoria and psychosocial functioning. Youth and their families can then later decide to discontinue the medication and go through their endogenous puberty or to proceed with gender-affirming hormone treatment.⁷

With the growing number of states who have or are attempting to ban gender-affirming care for youth, more patients and families will be left with no options for accessing this potentially life-saving care and support within their home state. Some families have already been forced to relocate to more supportive environments or to travel significant distances to receive medically necessary care.⁸ This summer, the American Academy of Pediatrics reaffirmed their current policy stating, “The AAP opposes any laws or regulations that discriminate against transgender and gender-diverse individuals, or that interfere in the doctor-patient relationship,” and they “support giving transgender adolescents access to the health care they need.”⁹

Pediatricians should continue to utilize existing resources for recommended routine screening and subsequent referral for patients or families with concerns around gender identity. When possible, connect patients and families in need of more supportive services around gender-affirming care to appropriate specialty providers. If providers are uncertain about the current legal climate in their state, it is recommended to consult with legal counsel if needed. As pediatricians, we must strive to uphold the tenets of medicine, follow expert recommendations and guidelines based on the best available evidence to provide comprehensive care to all patients, and continue to advocate for our patients and families.
 

Dr. Warus is an adolescent medicine physician who specializes in care for transgender and gender-nonconforming youth, and LGBTQ health for youth at Children’s Hospital of Los Angeles. He is assistant professor of pediatrics at University of Southern California, Los Angeles.

Resources

Bright Futures – Promoting healthy development of sexuality and gender identity (Implementation tip sheet).

Rafferty J. AAP Committee on Psychosocial Aspects of Child and Family Health, AAP Committee on Adolescence, AAP Section on Lesbian, Gay, Bisexual, and Transgender Health and Wellness. Ensuring comprehensive care and support for transgender and gender-diverse children and adolescents.

References

1. Rafferty J. AAP Committee on Psychosocial Aspects of Child and Family Health, AAP Committee on Adolescence, AAP Section on Lesbian, Gay, Bisexual, and Transgender Health and Wellness. Ensuring comprehensive care and support for transgender and gender-diverse children and adolescents.

2. Bright Futures – Promoting healthy development of sexuality and gender identity (Implementation tip sheet).

3. Shumer DE et al. Advances in the care of transgender children and adolescents.

4. Vance SR et al. Psychological and medical care of gender nonconforming youth.

5. Ehrensaft D et al. Prepubertal social gender transitions: What we know; what we can learn – A view from a gender affirmative lens.

6. Olson KR et al. Mental health of transgender children who are supported in their identities.

7. Olson J et al. Management of the transgender adolescent.

8. Rodgers A and Goldberg M. New State laws force families with trans kids to seek gender-affirming care elsewhere.

9. Wyckoff AS, ed. AAP reaffirms gender-affirming care policy, authorizes systematic review of evidence to guide update.

As the political targeting of transgender youth and families continues to play out on the national stage, it is more important than ever for pediatricians and other primary care providers to support this vulnerable population by defending the recommendations and guidelines of reputable medical organizations based in science and to show grace and humility in caring for their patients.

Guidelines and resources

All leading medical groups in the United States with statements or policies related to gender-affirming care (including the American Medical Association, the American Academy of Pediatrics, the Society for Adolescent Health and Medicine, the Pediatric Endocrine Society, the American Academy of Child and Adolescent Psychiatry, and many more) recognize that this care is medically necessary and that exclusions for gender-related services are harmful to patients and their families. As pediatricians, families and youth rely on our expertise and guidance related to childhood and adolescent development, including the development of gender identity and ways to create safe and supportive environments needed for youth to reach their full potential.¹

While pediatricians are experts in youth development, some may have had limited access to training specific to LGBTQ+ identity development and interventions related to gender-affirming care. There are, however, readily accessible resources to help guide pediatricians in providing support and recommendations to families with concerns around gender or sexuality (See Resources). The American Academy of Pediatrics and Bright Futures recommend discussing the differences between assigned sex at birth and gender identity development with parents of those younger than 12 months of age as well as beginning to discuss and explore gender identity with all youth beginning at 4-5 years of age. Beginning at 8 years of age, pediatricians are also recommended to assess for a patient’s understanding and feelings toward emerging puberty to identify any potential concerns for gender dysphoria.² If concerns or questions emerge from screening, the family can then be referred to a gender-affirming care specialist for more support.
 

Gender dysphoria and gender-affirming care

Gender dysphoria may present in different ways and at different times for each patient. Some patients may present early in childhood with gender-diverse behaviors or the assertion of a gender identity different than their assigned sex at birth. However, the most commonly seen presentation is just prior to or during puberty, when one’s physical body starts to change in ways that are not consistent with their gender identity. Many patients report distress around gender before this time, but the distress that comes with the physical changes of puberty often prompts patients to reach out to parents, friends, and/or medical providers for help. Other than youth specifically disclosing their gender identity, as with any life stressors, gender dysphoria may initially present with a decline in school or social functioning, increased mood irritability, depression, or anxiety.³

The goal of support prior to puberty is for youth to grow and thrive as any other child and to not have gender dysphoria get in the way of normal development and social functioning.⁴ Some families will pursue social transition, the process of making changes within different areas of social interaction (such as name, pronouns, clothing, hairstyle, etc.) to decrease distress around gender. The decision of whether to pursue social transition is unique to each patient and family. The goal of this process is to allow youth to explore these changes in an effort to decrease the distress they experience in social interactions. Youth should be centered in this process and be the leader of any potential changes with parents and schools providing safe and supportive environments.⁵ Social transition has been shown to decrease rates of depression in gender-diverse youth to the same level as that of their cisgender peers.⁶

It is important to note that there are no recommended medical interventions for gender-affirming care before the time of puberty and, once a patient reaches Sexual Maturity Rating II (early puberty), the first potential treatment option is the reversible suppression of puberty using gonadotropin-releasing hormone (GnRH) analogues. The goal of this type of medication is to allow youth more time to explore their gender and avoid the permanent physical changes that occur during their endogenous puberty that can have a significant negative impact on their gender dysphoria and psychosocial functioning. Youth and their families can then later decide to discontinue the medication and go through their endogenous puberty or to proceed with gender-affirming hormone treatment.⁷

With the growing number of states who have or are attempting to ban gender-affirming care for youth, more patients and families will be left with no options for accessing this potentially life-saving care and support within their home state. Some families have already been forced to relocate to more supportive environments or to travel significant distances to receive medically necessary care.⁸ This summer, the American Academy of Pediatrics reaffirmed their current policy stating, “The AAP opposes any laws or regulations that discriminate against transgender and gender-diverse individuals, or that interfere in the doctor-patient relationship,” and they “support giving transgender adolescents access to the health care they need.”⁹

Pediatricians should continue to utilize existing resources for recommended routine screening and subsequent referral for patients or families with concerns around gender identity. When possible, connect patients and families in need of more supportive services around gender-affirming care to appropriate specialty providers. If providers are uncertain about the current legal climate in their state, it is recommended to consult with legal counsel if needed. As pediatricians, we must strive to uphold the tenets of medicine, follow expert recommendations and guidelines based on the best available evidence to provide comprehensive care to all patients, and continue to advocate for our patients and families.
 

Dr. Warus is an adolescent medicine physician who specializes in care for transgender and gender-nonconforming youth, and LGBTQ health for youth at Children’s Hospital of Los Angeles. He is assistant professor of pediatrics at University of Southern California, Los Angeles.

Resources

Bright Futures – Promoting healthy development of sexuality and gender identity (Implementation tip sheet).

Rafferty J. AAP Committee on Psychosocial Aspects of Child and Family Health, AAP Committee on Adolescence, AAP Section on Lesbian, Gay, Bisexual, and Transgender Health and Wellness. Ensuring comprehensive care and support for transgender and gender-diverse children and adolescents.

References

1. Rafferty J. AAP Committee on Psychosocial Aspects of Child and Family Health, AAP Committee on Adolescence, AAP Section on Lesbian, Gay, Bisexual, and Transgender Health and Wellness. Ensuring comprehensive care and support for transgender and gender-diverse children and adolescents.

2. Bright Futures – Promoting healthy development of sexuality and gender identity (Implementation tip sheet).

3. Shumer DE et al. Advances in the care of transgender children and adolescents.

4. Vance SR et al. Psychological and medical care of gender nonconforming youth.

5. Ehrensaft D et al. Prepubertal social gender transitions: What we know; what we can learn – A view from a gender affirmative lens.

6. Olson KR et al. Mental health of transgender children who are supported in their identities.

7. Olson J et al. Management of the transgender adolescent.

8. Rodgers A and Goldberg M. New State laws force families with trans kids to seek gender-affirming care elsewhere.

9. Wyckoff AS, ed. AAP reaffirms gender-affirming care policy, authorizes systematic review of evidence to guide update.

As the political targeting of transgender youth and families continues to play out on the national stage, it is more important than ever for pediatricians and other primary care providers to support this vulnerable population by defending the recommendations and guidelines of reputable medical organizations based in science and to show grace and humility in caring for their patients.

Guidelines and resources

All leading medical groups in the United States with statements or policies related to gender-affirming care (including the American Medical Association, the American Academy of Pediatrics, the Society for Adolescent Health and Medicine, the Pediatric Endocrine Society, the American Academy of Child and Adolescent Psychiatry, and many more) recognize that this care is medically necessary and that exclusions for gender-related services are harmful to patients and their families. As pediatricians, families and youth rely on our expertise and guidance related to childhood and adolescent development, including the development of gender identity and ways to create safe and supportive environments needed for youth to reach their full potential.¹

While pediatricians are experts in youth development, some may have had limited access to training specific to LGBTQ+ identity development and interventions related to gender-affirming care. There are, however, readily accessible resources to help guide pediatricians in providing support and recommendations to families with concerns around gender or sexuality (See Resources). The American Academy of Pediatrics and Bright Futures recommend discussing the differences between assigned sex at birth and gender identity development with parents of those younger than 12 months of age as well as beginning to discuss and explore gender identity with all youth beginning at 4-5 years of age. Beginning at 8 years of age, pediatricians are also recommended to assess for a patient’s understanding and feelings toward emerging puberty to identify any potential concerns for gender dysphoria.² If concerns or questions emerge from screening, the family can then be referred to a gender-affirming care specialist for more support.
 

Gender dysphoria and gender-affirming care

Gender dysphoria may present in different ways and at different times for each patient. Some patients may present early in childhood with gender-diverse behaviors or the assertion of a gender identity different than their assigned sex at birth. However, the most commonly seen presentation is just prior to or during puberty, when one’s physical body starts to change in ways that are not consistent with their gender identity. Many patients report distress around gender before this time, but the distress that comes with the physical changes of puberty often prompts patients to reach out to parents, friends, and/or medical providers for help. Other than youth specifically disclosing their gender identity, as with any life stressors, gender dysphoria may initially present with a decline in school or social functioning, increased mood irritability, depression, or anxiety.³

The goal of support prior to puberty is for youth to grow and thrive as any other child and to not have gender dysphoria get in the way of normal development and social functioning.⁴ Some families will pursue social transition, the process of making changes within different areas of social interaction (such as name, pronouns, clothing, hairstyle, etc.) to decrease distress around gender. The decision of whether to pursue social transition is unique to each patient and family. The goal of this process is to allow youth to explore these changes in an effort to decrease the distress they experience in social interactions. Youth should be centered in this process and be the leader of any potential changes with parents and schools providing safe and supportive environments.⁵ Social transition has been shown to decrease rates of depression in gender-diverse youth to the same level as that of their cisgender peers.⁶

It is important to note that there are no recommended medical interventions for gender-affirming care before the time of puberty and, once a patient reaches Sexual Maturity Rating II (early puberty), the first potential treatment option is the reversible suppression of puberty using gonadotropin-releasing hormone (GnRH) analogues. The goal of this type of medication is to allow youth more time to explore their gender and avoid the permanent physical changes that occur during their endogenous puberty that can have a significant negative impact on their gender dysphoria and psychosocial functioning. Youth and their families can then later decide to discontinue the medication and go through their endogenous puberty or to proceed with gender-affirming hormone treatment.⁷

With the growing number of states who have or are attempting to ban gender-affirming care for youth, more patients and families will be left with no options for accessing this potentially life-saving care and support within their home state. Some families have already been forced to relocate to more supportive environments or to travel significant distances to receive medically necessary care.⁸ This summer, the American Academy of Pediatrics reaffirmed their current policy stating, “The AAP opposes any laws or regulations that discriminate against transgender and gender-diverse individuals, or that interfere in the doctor-patient relationship,” and they “support giving transgender adolescents access to the health care they need.”⁹

Pediatricians should continue to utilize existing resources for recommended routine screening and subsequent referral for patients or families with concerns around gender identity. When possible, connect patients and families in need of more supportive services around gender-affirming care to appropriate specialty providers. If providers are uncertain about the current legal climate in their state, it is recommended to consult with legal counsel if needed. As pediatricians, we must strive to uphold the tenets of medicine, follow expert recommendations and guidelines based on the best available evidence to provide comprehensive care to all patients, and continue to advocate for our patients and families.
 

Dr. Warus is an adolescent medicine physician who specializes in care for transgender and gender-nonconforming youth, and LGBTQ health for youth at Children’s Hospital of Los Angeles. He is assistant professor of pediatrics at University of Southern California, Los Angeles.

Resources

Bright Futures – Promoting healthy development of sexuality and gender identity (Implementation tip sheet).

Rafferty J. AAP Committee on Psychosocial Aspects of Child and Family Health, AAP Committee on Adolescence, AAP Section on Lesbian, Gay, Bisexual, and Transgender Health and Wellness. Ensuring comprehensive care and support for transgender and gender-diverse children and adolescents.

References

1. Rafferty J. AAP Committee on Psychosocial Aspects of Child and Family Health, AAP Committee on Adolescence, AAP Section on Lesbian, Gay, Bisexual, and Transgender Health and Wellness. Ensuring comprehensive care and support for transgender and gender-diverse children and adolescents.

2. Bright Futures – Promoting healthy development of sexuality and gender identity (Implementation tip sheet).

3. Shumer DE et al. Advances in the care of transgender children and adolescents.

4. Vance SR et al. Psychological and medical care of gender nonconforming youth.

5. Ehrensaft D et al. Prepubertal social gender transitions: What we know; what we can learn – A view from a gender affirmative lens.

6. Olson KR et al. Mental health of transgender children who are supported in their identities.

7. Olson J et al. Management of the transgender adolescent.

8. Rodgers A and Goldberg M. New State laws force families with trans kids to seek gender-affirming care elsewhere.

9. Wyckoff AS, ed. AAP reaffirms gender-affirming care policy, authorizes systematic review of evidence to guide update.

A telehealth primary care visit more than doubled the well-visit show rate for a cohort of hard-to-reach adolescents, results of a small pilot study show.

Brian P. Jenssen, MD, MSHP, department of pediatrics, University of Pennsylvania, Philadelphia, led the pilot study and the project team, which included physicians, researchers, and experts in innovation, quality improvement, and data analytics.

Findings were published online in Annals of Family Medicine.

Keeping adolescents in consistent primary care can be challenging for many reasons. The study authors note, “Only 50% of adolescents have had a health supervision visit in the past year, missing a critical opportunity for clinicians to influence health, development, screening, and counseling.”

Interest high in hard-to-reach group

This study included a particularly hard-to-reach group of 18-year-old patients at an urban primary care clinic who were lost to follow-up and had Medicaid insurance. They had not completed a well visit in more than 2 years and had a history of no-show visits.

Interest in the pilot program was high. The authors write: “We contacted patients (or their caregivers) to gauge interest in a virtual well visit with a goal to fill five telehealth slots in one evening block with one clinician. Due to high patient interest and demand, we expanded to 15 slots over three evenings, filling the slots after contacting just 24 patients.”

Professional organizations have recommended a telehealth/in-person hybrid care model to meet hard-to-reach adolescents “wherever they are,” the authors note, but the concept has not been well studied.

Under the hybrid model, the first visit is through telehealth and in-person follow-up is scheduled as needed.

Navigators contacted patients to remind them of the appointment, and helped activate the patient portal and complete previsit screening questions for depression and other health risks.

Telehealth visits were billed as preventive visits and in-person follow-up visits as no-charge nurse visits, and these payments were supported by Medicaid.
 

Sharp increase in show rate

In the pilot study, of the 15 patients scheduled for the telehealth visit, 11 connected virtually (73% show rate). Of those, nine needed in-person follow-up, and five completed the follow-up.

Before the intervention, the average well-visit show rate for this patient group was 33%.

Clinicians counseled all the patients about substance use and safe sex. One patient screened positive for depression and was then connected to services. Two patients were started on birth control.

During the in-person follow-up, all patients received vaccinations (influenza, meningococcal, and/or COVID-19) and were screened for sexually transmitted infection. Eight patients completed the satisfaction survey and all said they liked the convenience of the telehealth visit.
 

Telehealth may reduce barriers for teens

Anthony Cheng, MD, a family medicine physician at Oregon Health & Science University in Portland, who was not part of the study, said he found the hybrid model promising.

One reason is that telehealth eliminates the need for transportation to medical appointments, which can be a barrier for adolescents.

Among the top causes of death for young people are mental health issues and addressing those, Dr. Cheng noted, is well-suited to a telehealth visit.

“There’s so much we can do if we can establish a relationship and maintain a relationship with our patients as young adults,” he said. “People do better when they have a regular source of care.”

He added that adolescents also have grown up communicating via screens so it’s often more comfortable for them to communicate with health care providers this way.

Dr. Cheng said adopting such a model may be difficult for providers reluctant to switch from the practice model with which they are most comfortable.

“We prefer to do things we have the most confidence in,” he said. “It does take an investment to train staff and build your own clinical comfort. If that experience wasn’t good over the past 3 years, you may be anxious to get back to your normal way of doing business.”

The authors and Dr. Cheng have no relevant financial relationships to disclose.

A telehealth primary care visit more than doubled the well-visit show rate for a cohort of hard-to-reach adolescents, results of a small pilot study show.

Brian P. Jenssen, MD, MSHP, department of pediatrics, University of Pennsylvania, Philadelphia, led the pilot study and the project team, which included physicians, researchers, and experts in innovation, quality improvement, and data analytics.

Findings were published online in Annals of Family Medicine.

Keeping adolescents in consistent primary care can be challenging for many reasons. The study authors note, “Only 50% of adolescents have had a health supervision visit in the past year, missing a critical opportunity for clinicians to influence health, development, screening, and counseling.”

Interest high in hard-to-reach group

This study included a particularly hard-to-reach group of 18-year-old patients at an urban primary care clinic who were lost to follow-up and had Medicaid insurance. They had not completed a well visit in more than 2 years and had a history of no-show visits.

Interest in the pilot program was high. The authors write: “We contacted patients (or their caregivers) to gauge interest in a virtual well visit with a goal to fill five telehealth slots in one evening block with one clinician. Due to high patient interest and demand, we expanded to 15 slots over three evenings, filling the slots after contacting just 24 patients.”

Professional organizations have recommended a telehealth/in-person hybrid care model to meet hard-to-reach adolescents “wherever they are,” the authors note, but the concept has not been well studied.

Under the hybrid model, the first visit is through telehealth and in-person follow-up is scheduled as needed.

Navigators contacted patients to remind them of the appointment, and helped activate the patient portal and complete previsit screening questions for depression and other health risks.

Telehealth visits were billed as preventive visits and in-person follow-up visits as no-charge nurse visits, and these payments were supported by Medicaid.
 

Sharp increase in show rate

In the pilot study, of the 15 patients scheduled for the telehealth visit, 11 connected virtually (73% show rate). Of those, nine needed in-person follow-up, and five completed the follow-up.

Before the intervention, the average well-visit show rate for this patient group was 33%.

Clinicians counseled all the patients about substance use and safe sex. One patient screened positive for depression and was then connected to services. Two patients were started on birth control.

During the in-person follow-up, all patients received vaccinations (influenza, meningococcal, and/or COVID-19) and were screened for sexually transmitted infection. Eight patients completed the satisfaction survey and all said they liked the convenience of the telehealth visit.
 

Telehealth may reduce barriers for teens

Anthony Cheng, MD, a family medicine physician at Oregon Health & Science University in Portland, who was not part of the study, said he found the hybrid model promising.

One reason is that telehealth eliminates the need for transportation to medical appointments, which can be a barrier for adolescents.

Among the top causes of death for young people are mental health issues and addressing those, Dr. Cheng noted, is well-suited to a telehealth visit.

“There’s so much we can do if we can establish a relationship and maintain a relationship with our patients as young adults,” he said. “People do better when they have a regular source of care.”

He added that adolescents also have grown up communicating via screens so it’s often more comfortable for them to communicate with health care providers this way.

Dr. Cheng said adopting such a model may be difficult for providers reluctant to switch from the practice model with which they are most comfortable.

“We prefer to do things we have the most confidence in,” he said. “It does take an investment to train staff and build your own clinical comfort. If that experience wasn’t good over the past 3 years, you may be anxious to get back to your normal way of doing business.”

The authors and Dr. Cheng have no relevant financial relationships to disclose.

A telehealth primary care visit more than doubled the well-visit show rate for a cohort of hard-to-reach adolescents, results of a small pilot study show.

Brian P. Jenssen, MD, MSHP, department of pediatrics, University of Pennsylvania, Philadelphia, led the pilot study and the project team, which included physicians, researchers, and experts in innovation, quality improvement, and data analytics.

Findings were published online in Annals of Family Medicine.

Keeping adolescents in consistent primary care can be challenging for many reasons. The study authors note, “Only 50% of adolescents have had a health supervision visit in the past year, missing a critical opportunity for clinicians to influence health, development, screening, and counseling.”

Interest high in hard-to-reach group

This study included a particularly hard-to-reach group of 18-year-old patients at an urban primary care clinic who were lost to follow-up and had Medicaid insurance. They had not completed a well visit in more than 2 years and had a history of no-show visits.

Interest in the pilot program was high. The authors write: “We contacted patients (or their caregivers) to gauge interest in a virtual well visit with a goal to fill five telehealth slots in one evening block with one clinician. Due to high patient interest and demand, we expanded to 15 slots over three evenings, filling the slots after contacting just 24 patients.”

Professional organizations have recommended a telehealth/in-person hybrid care model to meet hard-to-reach adolescents “wherever they are,” the authors note, but the concept has not been well studied.

Under the hybrid model, the first visit is through telehealth and in-person follow-up is scheduled as needed.

Navigators contacted patients to remind them of the appointment, and helped activate the patient portal and complete previsit screening questions for depression and other health risks.

Telehealth visits were billed as preventive visits and in-person follow-up visits as no-charge nurse visits, and these payments were supported by Medicaid.
 

Sharp increase in show rate

In the pilot study, of the 15 patients scheduled for the telehealth visit, 11 connected virtually (73% show rate). Of those, nine needed in-person follow-up, and five completed the follow-up.

Before the intervention, the average well-visit show rate for this patient group was 33%.

Clinicians counseled all the patients about substance use and safe sex. One patient screened positive for depression and was then connected to services. Two patients were started on birth control.

During the in-person follow-up, all patients received vaccinations (influenza, meningococcal, and/or COVID-19) and were screened for sexually transmitted infection. Eight patients completed the satisfaction survey and all said they liked the convenience of the telehealth visit.
 

Telehealth may reduce barriers for teens

Anthony Cheng, MD, a family medicine physician at Oregon Health & Science University in Portland, who was not part of the study, said he found the hybrid model promising.

One reason is that telehealth eliminates the need for transportation to medical appointments, which can be a barrier for adolescents.

Among the top causes of death for young people are mental health issues and addressing those, Dr. Cheng noted, is well-suited to a telehealth visit.

“There’s so much we can do if we can establish a relationship and maintain a relationship with our patients as young adults,” he said. “People do better when they have a regular source of care.”

He added that adolescents also have grown up communicating via screens so it’s often more comfortable for them to communicate with health care providers this way.

Dr. Cheng said adopting such a model may be difficult for providers reluctant to switch from the practice model with which they are most comfortable.

“We prefer to do things we have the most confidence in,” he said. “It does take an investment to train staff and build your own clinical comfort. If that experience wasn’t good over the past 3 years, you may be anxious to get back to your normal way of doing business.”

The authors and Dr. Cheng have no relevant financial relationships to disclose.

At the new Walnut Creek Clinic in the East Bay of the San Francisco Bay area, kids get a “Comfort Promise.”

The clinic extends the work of the Stad Center for Pediatric Pain, Palliative & Integrative Medicine beyond the locations in University of California San Francisco Benioff Children’s Hospitals in San Francisco and Oakland.

At Walnut Creek, clinical acupuncturists, massage therapists, and specialists in hypnosis complement advanced medical care with integrative techniques.

The “Comfort Promise” program, which is being rolled out at that clinic and other UCSF pediatric clinics through the end of 2024, is the clinicians’ pledge to do everything in their power to make tests, infusions, and vaccinations “practically pain free.”

Needle sticks, for example, can be a common source of pain and anxiety for kids. Techniques to minimize pain vary by age. Among the ways the clinicians minimize needle pain for a child 6- to 12-years-old are:

• Giving the child control options to pick which arm; and watch the injection, pause it, or stop it with a communication sign.
• Introducing memory shaping by asking the child about the experience afterward and presenting it in a positive way by praising the acts of sitting still, breathing deeply, or being brave.
• Using distractors such as asking the child to hold a favorite item from home, storytelling, coloring, singing, or using breathing exercises.

Stefan Friedrichsdorf, MD, chief of the UCSF division of pediatric pain, palliative & integrative medicine, said in a statement: “For kids with chronic pain, complex pain medications can cause more harm than benefit. Our goal is to combine exercise and physical therapy with integrative medicine and skills-based psychotherapy to help them become pain free in their everyday life.”
 

Bundling appointments for early impact

At Lurie Children’s Hospital of Chicago, the chronic pain treatment program bundles visits with experts in several disciplines, include social workers, psychologists, and physical therapists, in addition to the medical team, so that patients can complete a first round of visits with multiple specialists in a short period, as opposed to several months.

Natalie Weatherred, APRN-NP, CPNP-PC, a pediatric nurse practitioner in anesthesiology and the pain clinic coordinator, said in an interview that the up-front visits involve between four and eight follow-up sessions in a short period with everybody in the multidisciplinary team “to really help jump-start their pain treatment.”

She pointed out that many families come from distant parts of the state or beyond so the bundled appointments are also important for easing burden on families.

Sarah Duggan, APRN-NP, CPNP-PC, also a pediatric nurse practitioner in anesthesiology at Lurie’s, pointed out that patients at their clinic often have other chronic conditions as well, such as such as postural orthostatic tachycardia syndrome so the care integration is particularly important.

“We can get them the appropriate care that they need and the resources they need, much sooner than we would have been able to do 5 or 10 years ago,” Ms. Duggan said.
 

Virtual reality distraction instead of sedation

Henry Huang, MD, anesthesiologist and pain physician at Texas Children’s Hospital, Houston, said a special team there collaborates with the Chariot Program at Stanford (Calif.) University and incorporates virtual reality to distract children from pain and anxiety and harness their imaginations during induction for anesthesia, intravenous placement, and vaccinations.

“At our institution we’ve been recruiting patients to do a proof of concept to do virtual reality distraction for pain procedures, such as nerve blocks or steroid injections,” Dr. Huang said.

Traditionally, kids would have received oral or intravenous sedation to help them cope with the fear and pain.

“We’ve been successful in several cases without relying on any sedation,” he said. “The next target is to expand that to the chronic pain population.”

The distraction techniques are promising for a wide range of ages, he said, and the programming is tailored to the child’s ability to interact with the technology.

He said he is also part of a group promoting use of ultrasound instead of x-rays to guide injections to the spine and chest to reduce children’s exposure to radiation. His group is helping teach these methods to other clinicians nationally.

Dr. Huang said the most important development in chronic pediatric pain has been the growth of rehab centers that include the medical team, and practitioners from psychology as well as occupational and physical therapy.

“More and more hospitals are recognizing the importance of these pain rehab centers,” he said.

The problem, Dr. Huang said, is that these programs have always been resource intensive and involve highly specialized clinicians. The cost and the limited number of specialists make it difficult for widespread rollout.

“That’s always been the challenge from the pediatric pain world,” he said.
 

Recognizing the complexity of kids’ chronic pain

Angela Garcia, MD, a consulting physician for pediatric rehabilitation medicine at UPMC Children’s Hospital of Pittsburgh said recognizing the validity and complexity of pediatric pain has led to multidisciplinary approaches and specialty clinics for chronic pain instead of primarily pharmaceutical solutions.

Techniques such as biofeedback and acupuncture are becoming more mainstream in pediatric chronic care, she said.

At the UPMC clinic, children and their families talk with a care team about their values and what they want to accomplish in managing the child’s pain. They ask what the pain is preventing the child from doing.

“Their goals really are our goals,” she said.

She said she also refers almost all patients to one of the center’s pain psychologists.

“Pain is biopsychosocial,” she said. “We want to make sure we’re addressing how to cope with pain.”

Dr. Garcia said she hopes nutritional therapy is one of the next approaches the clinic will incorporate, particularly surrounding how dietary changes can reduce inflammation “and heal the body from the inside out.”

She said the hospital is also looking at developing an inpatient pain program for kids whose functioning has changed so drastically that they need more intensive therapies.

Whatever the treatment approach, she said, addressing the pain early is critical.

“There is an increased risk of a child with chronic pain becoming an adult with chronic pain,” Dr. Garcia pointed out, “and that can lead to a decrease in the ability to participate in society.”

Ms. Weatherred, Ms. Duggan, Dr. Huang, and Dr. Garcia reported no relevant financial relationships.

At the new Walnut Creek Clinic in the East Bay of the San Francisco Bay area, kids get a “Comfort Promise.”

The clinic extends the work of the Stad Center for Pediatric Pain, Palliative & Integrative Medicine beyond the locations in University of California San Francisco Benioff Children’s Hospitals in San Francisco and Oakland.

At Walnut Creek, clinical acupuncturists, massage therapists, and specialists in hypnosis complement advanced medical care with integrative techniques.

The “Comfort Promise” program, which is being rolled out at that clinic and other UCSF pediatric clinics through the end of 2024, is the clinicians’ pledge to do everything in their power to make tests, infusions, and vaccinations “practically pain free.”

Needle sticks, for example, can be a common source of pain and anxiety for kids. Techniques to minimize pain vary by age. Among the ways the clinicians minimize needle pain for a child 6- to 12-years-old are:

• Giving the child control options to pick which arm; and watch the injection, pause it, or stop it with a communication sign.
• Introducing memory shaping by asking the child about the experience afterward and presenting it in a positive way by praising the acts of sitting still, breathing deeply, or being brave.
• Using distractors such as asking the child to hold a favorite item from home, storytelling, coloring, singing, or using breathing exercises.

Stefan Friedrichsdorf, MD, chief of the UCSF division of pediatric pain, palliative & integrative medicine, said in a statement: “For kids with chronic pain, complex pain medications can cause more harm than benefit. Our goal is to combine exercise and physical therapy with integrative medicine and skills-based psychotherapy to help them become pain free in their everyday life.”
 

Bundling appointments for early impact

At Lurie Children’s Hospital of Chicago, the chronic pain treatment program bundles visits with experts in several disciplines, include social workers, psychologists, and physical therapists, in addition to the medical team, so that patients can complete a first round of visits with multiple specialists in a short period, as opposed to several months.

Natalie Weatherred, APRN-NP, CPNP-PC, a pediatric nurse practitioner in anesthesiology and the pain clinic coordinator, said in an interview that the up-front visits involve between four and eight follow-up sessions in a short period with everybody in the multidisciplinary team “to really help jump-start their pain treatment.”

She pointed out that many families come from distant parts of the state or beyond so the bundled appointments are also important for easing burden on families.

Sarah Duggan, APRN-NP, CPNP-PC, also a pediatric nurse practitioner in anesthesiology at Lurie’s, pointed out that patients at their clinic often have other chronic conditions as well, such as such as postural orthostatic tachycardia syndrome so the care integration is particularly important.

“We can get them the appropriate care that they need and the resources they need, much sooner than we would have been able to do 5 or 10 years ago,” Ms. Duggan said.
 

Virtual reality distraction instead of sedation

Henry Huang, MD, anesthesiologist and pain physician at Texas Children’s Hospital, Houston, said a special team there collaborates with the Chariot Program at Stanford (Calif.) University and incorporates virtual reality to distract children from pain and anxiety and harness their imaginations during induction for anesthesia, intravenous placement, and vaccinations.

“At our institution we’ve been recruiting patients to do a proof of concept to do virtual reality distraction for pain procedures, such as nerve blocks or steroid injections,” Dr. Huang said.

Traditionally, kids would have received oral or intravenous sedation to help them cope with the fear and pain.

“We’ve been successful in several cases without relying on any sedation,” he said. “The next target is to expand that to the chronic pain population.”

The distraction techniques are promising for a wide range of ages, he said, and the programming is tailored to the child’s ability to interact with the technology.

He said he is also part of a group promoting use of ultrasound instead of x-rays to guide injections to the spine and chest to reduce children’s exposure to radiation. His group is helping teach these methods to other clinicians nationally.

Dr. Huang said the most important development in chronic pediatric pain has been the growth of rehab centers that include the medical team, and practitioners from psychology as well as occupational and physical therapy.

“More and more hospitals are recognizing the importance of these pain rehab centers,” he said.

The problem, Dr. Huang said, is that these programs have always been resource intensive and involve highly specialized clinicians. The cost and the limited number of specialists make it difficult for widespread rollout.

“That’s always been the challenge from the pediatric pain world,” he said.
 

Recognizing the complexity of kids’ chronic pain

Angela Garcia, MD, a consulting physician for pediatric rehabilitation medicine at UPMC Children’s Hospital of Pittsburgh said recognizing the validity and complexity of pediatric pain has led to multidisciplinary approaches and specialty clinics for chronic pain instead of primarily pharmaceutical solutions.

Techniques such as biofeedback and acupuncture are becoming more mainstream in pediatric chronic care, she said.

At the UPMC clinic, children and their families talk with a care team about their values and what they want to accomplish in managing the child’s pain. They ask what the pain is preventing the child from doing.

“Their goals really are our goals,” she said.

She said she also refers almost all patients to one of the center’s pain psychologists.

“Pain is biopsychosocial,” she said. “We want to make sure we’re addressing how to cope with pain.”

Dr. Garcia said she hopes nutritional therapy is one of the next approaches the clinic will incorporate, particularly surrounding how dietary changes can reduce inflammation “and heal the body from the inside out.”

She said the hospital is also looking at developing an inpatient pain program for kids whose functioning has changed so drastically that they need more intensive therapies.

Whatever the treatment approach, she said, addressing the pain early is critical.

“There is an increased risk of a child with chronic pain becoming an adult with chronic pain,” Dr. Garcia pointed out, “and that can lead to a decrease in the ability to participate in society.”

Ms. Weatherred, Ms. Duggan, Dr. Huang, and Dr. Garcia reported no relevant financial relationships.

At the new Walnut Creek Clinic in the East Bay of the San Francisco Bay area, kids get a “Comfort Promise.”

The clinic extends the work of the Stad Center for Pediatric Pain, Palliative & Integrative Medicine beyond the locations in University of California San Francisco Benioff Children’s Hospitals in San Francisco and Oakland.

At Walnut Creek, clinical acupuncturists, massage therapists, and specialists in hypnosis complement advanced medical care with integrative techniques.

The “Comfort Promise” program, which is being rolled out at that clinic and other UCSF pediatric clinics through the end of 2024, is the clinicians’ pledge to do everything in their power to make tests, infusions, and vaccinations “practically pain free.”

Needle sticks, for example, can be a common source of pain and anxiety for kids. Techniques to minimize pain vary by age. Among the ways the clinicians minimize needle pain for a child 6- to 12-years-old are:

• Giving the child control options to pick which arm; and watch the injection, pause it, or stop it with a communication sign.
• Introducing memory shaping by asking the child about the experience afterward and presenting it in a positive way by praising the acts of sitting still, breathing deeply, or being brave.
• Using distractors such as asking the child to hold a favorite item from home, storytelling, coloring, singing, or using breathing exercises.

Stefan Friedrichsdorf, MD, chief of the UCSF division of pediatric pain, palliative & integrative medicine, said in a statement: “For kids with chronic pain, complex pain medications can cause more harm than benefit. Our goal is to combine exercise and physical therapy with integrative medicine and skills-based psychotherapy to help them become pain free in their everyday life.”
 

Bundling appointments for early impact

At Lurie Children’s Hospital of Chicago, the chronic pain treatment program bundles visits with experts in several disciplines, include social workers, psychologists, and physical therapists, in addition to the medical team, so that patients can complete a first round of visits with multiple specialists in a short period, as opposed to several months.

Natalie Weatherred, APRN-NP, CPNP-PC, a pediatric nurse practitioner in anesthesiology and the pain clinic coordinator, said in an interview that the up-front visits involve between four and eight follow-up sessions in a short period with everybody in the multidisciplinary team “to really help jump-start their pain treatment.”

She pointed out that many families come from distant parts of the state or beyond so the bundled appointments are also important for easing burden on families.

Sarah Duggan, APRN-NP, CPNP-PC, also a pediatric nurse practitioner in anesthesiology at Lurie’s, pointed out that patients at their clinic often have other chronic conditions as well, such as such as postural orthostatic tachycardia syndrome so the care integration is particularly important.

“We can get them the appropriate care that they need and the resources they need, much sooner than we would have been able to do 5 or 10 years ago,” Ms. Duggan said.
 

Virtual reality distraction instead of sedation

Henry Huang, MD, anesthesiologist and pain physician at Texas Children’s Hospital, Houston, said a special team there collaborates with the Chariot Program at Stanford (Calif.) University and incorporates virtual reality to distract children from pain and anxiety and harness their imaginations during induction for anesthesia, intravenous placement, and vaccinations.

“At our institution we’ve been recruiting patients to do a proof of concept to do virtual reality distraction for pain procedures, such as nerve blocks or steroid injections,” Dr. Huang said.

Traditionally, kids would have received oral or intravenous sedation to help them cope with the fear and pain.

“We’ve been successful in several cases without relying on any sedation,” he said. “The next target is to expand that to the chronic pain population.”

The distraction techniques are promising for a wide range of ages, he said, and the programming is tailored to the child’s ability to interact with the technology.

He said he is also part of a group promoting use of ultrasound instead of x-rays to guide injections to the spine and chest to reduce children’s exposure to radiation. His group is helping teach these methods to other clinicians nationally.

Dr. Huang said the most important development in chronic pediatric pain has been the growth of rehab centers that include the medical team, and practitioners from psychology as well as occupational and physical therapy.

“More and more hospitals are recognizing the importance of these pain rehab centers,” he said.

The problem, Dr. Huang said, is that these programs have always been resource intensive and involve highly specialized clinicians. The cost and the limited number of specialists make it difficult for widespread rollout.

“That’s always been the challenge from the pediatric pain world,” he said.
 

Recognizing the complexity of kids’ chronic pain

Angela Garcia, MD, a consulting physician for pediatric rehabilitation medicine at UPMC Children’s Hospital of Pittsburgh said recognizing the validity and complexity of pediatric pain has led to multidisciplinary approaches and specialty clinics for chronic pain instead of primarily pharmaceutical solutions.

Techniques such as biofeedback and acupuncture are becoming more mainstream in pediatric chronic care, she said.

At the UPMC clinic, children and their families talk with a care team about their values and what they want to accomplish in managing the child’s pain. They ask what the pain is preventing the child from doing.

“Their goals really are our goals,” she said.

She said she also refers almost all patients to one of the center’s pain psychologists.

“Pain is biopsychosocial,” she said. “We want to make sure we’re addressing how to cope with pain.”

Dr. Garcia said she hopes nutritional therapy is one of the next approaches the clinic will incorporate, particularly surrounding how dietary changes can reduce inflammation “and heal the body from the inside out.”

She said the hospital is also looking at developing an inpatient pain program for kids whose functioning has changed so drastically that they need more intensive therapies.

Whatever the treatment approach, she said, addressing the pain early is critical.

“There is an increased risk of a child with chronic pain becoming an adult with chronic pain,” Dr. Garcia pointed out, “and that can lead to a decrease in the ability to participate in society.”

Ms. Weatherred, Ms. Duggan, Dr. Huang, and Dr. Garcia reported no relevant financial relationships.

There is no increased risk of substance abuse later in life among children treated with stimulants for attention-deficit/hyperactivity disorder (ADHD), results of a large study show.

University of Pittsburgh

“Throughout rigorous analyses, and after accounting for more than 70 variables in this longitudinal sample of children with ADHD taking stimulants, we did not find an association with later substance use,” lead investigator Brooke Molina, PhD, director of the youth and family research program at the University of Pittsburgh, said in an interview.

The findings were published online in JAMA Psychiatry.
 

Protective effect?

Owing to symptoms of impulsivity inherent to ADHD, the disorder itself carries a risk for elevated substance use, the investigators note.

They speculate that this may be why some previous research suggests prescription stimulants reduce the risk of subsequent substance use disorder. However, other studies have found no such protective link.

To shed more light on the issue, the investigators used data from the Multimodal Treatment Study of ADHD, a multicenter, 14-month randomized clinical trial of medication and behavioral therapy for children with ADHD. However, for the purposes of the present study, investigators focused only on stimulant use in children.

At the time of recruitment, the children were aged 7-9 and had been diagnosed with ADHD between 1994 and 1996.

Investigators assessed the participants prior to randomization, at months 3 and 9, and at the end of treatment. They were then followed for 16 years and were assessed at years 2, 3, 6, 8, 10, 12, 14, and 16 until a mean age of 25.

During 12-, 14-, and 16-year follow-up, participants completed a questionnaire on their use of alcohol, marijuana, cigarettes, and several illicit and prescription drugs.

Investigators collected information on participants’ stimulant treatment via the Services for Children and Adolescents Parent Interview until they reached age 18. After that, participants reported their own stimulant treatment.

A total of 579 participants were included in the analysis. Of these, 61% were White, 20% were Black, and 8% were Hispanic.
 

Decline in stimulant use over time

The analysis showed that stimulant use declined “precipitously” over time – from 60% at the 2- and 3-year assessments to an average of 7% during early adulthood.

The investigators also found that for some participants, substance use increased steadily through adolescence and remained stable through early adulthood. For instance, 36.5% of the adolescents in the total cohort reported smoking tobacco daily, and 29.6% reported using marijuana every week.

In addition, approximately 21% of the participants indulged in heavy drinking at least once a week, and 6% reported “other” substance use, which included sedative misuse, heroin, inhalants, hallucinogens, or other substances taken to “get high.”

After accounting for developmental trends in substance use in the sample through adolescence into early adulthood with several rigorous statistical models, the researchers found no association between current or prior stimulant treatment and cigarette, marijuana, alcohol, or other substance use, with one exception.

While cumulative stimulant treatment was associated with increased heavy drinking, the effect size of this association was small. Each additional year of cumulative stimulant use was estimated to increase participants’ likelihood of any binge drinking/drunkenness vs. none in the past year by 4% (95% confidence interval, 0.01-0.08; P =.03).

When the investigators used a causal analytic method to account for age and other time-varying characteristics, including household income, behavior problems, and parental support, there was no evidence that current (B range, –0.62-0.34) or prior stimulant treatment (B range, –0.06-0.70) or their interaction (B range, –0.49-0.86) was associated with substance use in adulthood.

Dr. Molina noted that although participants were recruited from multiple sites, the sample may not be generalizable because children and parents who present for an intensive treatment study such as this are not necessarily representative of the general ADHD population.
 

Reassuring findings

In a comment, Julie Schweitzer, PhD, professor of psychiatry and behavioral sciences at the University of California, Davis, said she hopes the study findings will quell the stigma surrounding stimulant use by children with ADHD.

“Parents’ fears that stimulant use will lead to a substance use disorder inhibits them from bringing their children for an ADHD evaluation, thus reducing the likelihood that they will receive timely treatment,” Dr. Schweitzer said.

“While stimulant medication is the first-line treatment most often recommended for most persons with ADHD, by not following through on evaluations, parents also miss the opportunity to learn about nonpharmacological strategies that might also be helpful to help cope with ADHD symptoms and its potential co-occurring challenges,” she added.

Dr. Schweitzer also noted that many parents hope their children will outgrow the symptoms without realizing that by not obtaining an evaluation and treatment for their child, there is an associated cost, including less than optimal academic performance, social relationships, and emotional health.

The Multimodal Treatment Study of Children with ADHD was a National Institute of Mental Health cooperative agreement randomized clinical trial, continued under an NIMH contract as a follow-up study and under a National Institute on Drug Abuse contract followed by a data analysis grant. Dr. Molina reported grants from the NIMH and the National Institute on Drug Abuse during the conduct of the study.

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

There is no increased risk of substance abuse later in life among children treated with stimulants for attention-deficit/hyperactivity disorder (ADHD), results of a large study show.

University of Pittsburgh

“Throughout rigorous analyses, and after accounting for more than 70 variables in this longitudinal sample of children with ADHD taking stimulants, we did not find an association with later substance use,” lead investigator Brooke Molina, PhD, director of the youth and family research program at the University of Pittsburgh, said in an interview.

The findings were published online in JAMA Psychiatry.
 

Protective effect?

Owing to symptoms of impulsivity inherent to ADHD, the disorder itself carries a risk for elevated substance use, the investigators note.

They speculate that this may be why some previous research suggests prescription stimulants reduce the risk of subsequent substance use disorder. However, other studies have found no such protective link.

To shed more light on the issue, the investigators used data from the Multimodal Treatment Study of ADHD, a multicenter, 14-month randomized clinical trial of medication and behavioral therapy for children with ADHD. However, for the purposes of the present study, investigators focused only on stimulant use in children.

At the time of recruitment, the children were aged 7-9 and had been diagnosed with ADHD between 1994 and 1996.

Investigators assessed the participants prior to randomization, at months 3 and 9, and at the end of treatment. They were then followed for 16 years and were assessed at years 2, 3, 6, 8, 10, 12, 14, and 16 until a mean age of 25.

During 12-, 14-, and 16-year follow-up, participants completed a questionnaire on their use of alcohol, marijuana, cigarettes, and several illicit and prescription drugs.

Investigators collected information on participants’ stimulant treatment via the Services for Children and Adolescents Parent Interview until they reached age 18. After that, participants reported their own stimulant treatment.

A total of 579 participants were included in the analysis. Of these, 61% were White, 20% were Black, and 8% were Hispanic.
 

Decline in stimulant use over time

The analysis showed that stimulant use declined “precipitously” over time – from 60% at the 2- and 3-year assessments to an average of 7% during early adulthood.

The investigators also found that for some participants, substance use increased steadily through adolescence and remained stable through early adulthood. For instance, 36.5% of the adolescents in the total cohort reported smoking tobacco daily, and 29.6% reported using marijuana every week.

In addition, approximately 21% of the participants indulged in heavy drinking at least once a week, and 6% reported “other” substance use, which included sedative misuse, heroin, inhalants, hallucinogens, or other substances taken to “get high.”

After accounting for developmental trends in substance use in the sample through adolescence into early adulthood with several rigorous statistical models, the researchers found no association between current or prior stimulant treatment and cigarette, marijuana, alcohol, or other substance use, with one exception.

While cumulative stimulant treatment was associated with increased heavy drinking, the effect size of this association was small. Each additional year of cumulative stimulant use was estimated to increase participants’ likelihood of any binge drinking/drunkenness vs. none in the past year by 4% (95% confidence interval, 0.01-0.08; P =.03).

When the investigators used a causal analytic method to account for age and other time-varying characteristics, including household income, behavior problems, and parental support, there was no evidence that current (B range, –0.62-0.34) or prior stimulant treatment (B range, –0.06-0.70) or their interaction (B range, –0.49-0.86) was associated with substance use in adulthood.

Dr. Molina noted that although participants were recruited from multiple sites, the sample may not be generalizable because children and parents who present for an intensive treatment study such as this are not necessarily representative of the general ADHD population.
 

Reassuring findings

In a comment, Julie Schweitzer, PhD, professor of psychiatry and behavioral sciences at the University of California, Davis, said she hopes the study findings will quell the stigma surrounding stimulant use by children with ADHD.

“Parents’ fears that stimulant use will lead to a substance use disorder inhibits them from bringing their children for an ADHD evaluation, thus reducing the likelihood that they will receive timely treatment,” Dr. Schweitzer said.

“While stimulant medication is the first-line treatment most often recommended for most persons with ADHD, by not following through on evaluations, parents also miss the opportunity to learn about nonpharmacological strategies that might also be helpful to help cope with ADHD symptoms and its potential co-occurring challenges,” she added.

Dr. Schweitzer also noted that many parents hope their children will outgrow the symptoms without realizing that by not obtaining an evaluation and treatment for their child, there is an associated cost, including less than optimal academic performance, social relationships, and emotional health.

The Multimodal Treatment Study of Children with ADHD was a National Institute of Mental Health cooperative agreement randomized clinical trial, continued under an NIMH contract as a follow-up study and under a National Institute on Drug Abuse contract followed by a data analysis grant. Dr. Molina reported grants from the NIMH and the National Institute on Drug Abuse during the conduct of the study.

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

There is no increased risk of substance abuse later in life among children treated with stimulants for attention-deficit/hyperactivity disorder (ADHD), results of a large study show.

University of Pittsburgh

“Throughout rigorous analyses, and after accounting for more than 70 variables in this longitudinal sample of children with ADHD taking stimulants, we did not find an association with later substance use,” lead investigator Brooke Molina, PhD, director of the youth and family research program at the University of Pittsburgh, said in an interview.

The findings were published online in JAMA Psychiatry.
 

Protective effect?

Owing to symptoms of impulsivity inherent to ADHD, the disorder itself carries a risk for elevated substance use, the investigators note.

They speculate that this may be why some previous research suggests prescription stimulants reduce the risk of subsequent substance use disorder. However, other studies have found no such protective link.

To shed more light on the issue, the investigators used data from the Multimodal Treatment Study of ADHD, a multicenter, 14-month randomized clinical trial of medication and behavioral therapy for children with ADHD. However, for the purposes of the present study, investigators focused only on stimulant use in children.

At the time of recruitment, the children were aged 7-9 and had been diagnosed with ADHD between 1994 and 1996.

Investigators assessed the participants prior to randomization, at months 3 and 9, and at the end of treatment. They were then followed for 16 years and were assessed at years 2, 3, 6, 8, 10, 12, 14, and 16 until a mean age of 25.

During 12-, 14-, and 16-year follow-up, participants completed a questionnaire on their use of alcohol, marijuana, cigarettes, and several illicit and prescription drugs.

Investigators collected information on participants’ stimulant treatment via the Services for Children and Adolescents Parent Interview until they reached age 18. After that, participants reported their own stimulant treatment.

A total of 579 participants were included in the analysis. Of these, 61% were White, 20% were Black, and 8% were Hispanic.
 

Decline in stimulant use over time

The analysis showed that stimulant use declined “precipitously” over time – from 60% at the 2- and 3-year assessments to an average of 7% during early adulthood.

The investigators also found that for some participants, substance use increased steadily through adolescence and remained stable through early adulthood. For instance, 36.5% of the adolescents in the total cohort reported smoking tobacco daily, and 29.6% reported using marijuana every week.

In addition, approximately 21% of the participants indulged in heavy drinking at least once a week, and 6% reported “other” substance use, which included sedative misuse, heroin, inhalants, hallucinogens, or other substances taken to “get high.”

After accounting for developmental trends in substance use in the sample through adolescence into early adulthood with several rigorous statistical models, the researchers found no association between current or prior stimulant treatment and cigarette, marijuana, alcohol, or other substance use, with one exception.

While cumulative stimulant treatment was associated with increased heavy drinking, the effect size of this association was small. Each additional year of cumulative stimulant use was estimated to increase participants’ likelihood of any binge drinking/drunkenness vs. none in the past year by 4% (95% confidence interval, 0.01-0.08; P =.03).

When the investigators used a causal analytic method to account for age and other time-varying characteristics, including household income, behavior problems, and parental support, there was no evidence that current (B range, –0.62-0.34) or prior stimulant treatment (B range, –0.06-0.70) or their interaction (B range, –0.49-0.86) was associated with substance use in adulthood.

Dr. Molina noted that although participants were recruited from multiple sites, the sample may not be generalizable because children and parents who present for an intensive treatment study such as this are not necessarily representative of the general ADHD population.
 

Reassuring findings

In a comment, Julie Schweitzer, PhD, professor of psychiatry and behavioral sciences at the University of California, Davis, said she hopes the study findings will quell the stigma surrounding stimulant use by children with ADHD.

“Parents’ fears that stimulant use will lead to a substance use disorder inhibits them from bringing their children for an ADHD evaluation, thus reducing the likelihood that they will receive timely treatment,” Dr. Schweitzer said.

“While stimulant medication is the first-line treatment most often recommended for most persons with ADHD, by not following through on evaluations, parents also miss the opportunity to learn about nonpharmacological strategies that might also be helpful to help cope with ADHD symptoms and its potential co-occurring challenges,” she added.

Dr. Schweitzer also noted that many parents hope their children will outgrow the symptoms without realizing that by not obtaining an evaluation and treatment for their child, there is an associated cost, including less than optimal academic performance, social relationships, and emotional health.

The Multimodal Treatment Study of Children with ADHD was a National Institute of Mental Health cooperative agreement randomized clinical trial, continued under an NIMH contract as a follow-up study and under a National Institute on Drug Abuse contract followed by a data analysis grant. Dr. Molina reported grants from the NIMH and the National Institute on Drug Abuse during the conduct of the study.

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