Pediatrics

Clinicians must monitor children with sickle cell disease for eye complications as much as they do for adults, a new research review suggests.

Earlier research indicated that older patients were more at risk for eye complications from sickle cell disease, but the new study found that a full third of young people aged 10-25 years with sickle cell disease had retinopathy, including nonproliferative retinopathy (33%) and proliferative retinopathy (6%), which can progress to vision loss.

Two patients experienced retinal detachment, while two suffered retinal artery occlusion. One patient with retinal artery occlusion lost their vision and had a final best-corrected visual acuity of 20/60, according to the researchers, who presented their findings at the annual meeting of the American Academy of Ophthalmology.

“Our data underscores the need for patients – including pediatric patients – with sickle cell disease to get routine ophthalmic screenings along with appropriate systemic and ophthalmic treatment,” Mary Ellen Hoehn, MD, a professor of ophthalmology at the University of Tennessee Health Science Center, Memphis, who led the research, said in a press release.

The review covered records for 652 patients with sickle cell disease aged 10-25 years (median age, 14 years), who underwent eye exams over a 12-year period.

Besides looking at rates of retinopathy, Dr. Hoehn’s group studied which treatments were most effective. They found that hydroxyurea and chronic transfusions best lowered retinopathy rates among all genotypes.

“We hope that people will use this information to better care for patients with sickle cell disease, and that more timely ophthalmic screen exams will be performed so that vision-threatening complications from this disease are prevented,” Dr. Hoehn said.

The authors reported no relevant financial relationships.

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

Clinicians must monitor children with sickle cell disease for eye complications as much as they do for adults, a new research review suggests.

Earlier research indicated that older patients were more at risk for eye complications from sickle cell disease, but the new study found that a full third of young people aged 10-25 years with sickle cell disease had retinopathy, including nonproliferative retinopathy (33%) and proliferative retinopathy (6%), which can progress to vision loss.

Two patients experienced retinal detachment, while two suffered retinal artery occlusion. One patient with retinal artery occlusion lost their vision and had a final best-corrected visual acuity of 20/60, according to the researchers, who presented their findings at the annual meeting of the American Academy of Ophthalmology.

“Our data underscores the need for patients – including pediatric patients – with sickle cell disease to get routine ophthalmic screenings along with appropriate systemic and ophthalmic treatment,” Mary Ellen Hoehn, MD, a professor of ophthalmology at the University of Tennessee Health Science Center, Memphis, who led the research, said in a press release.

The review covered records for 652 patients with sickle cell disease aged 10-25 years (median age, 14 years), who underwent eye exams over a 12-year period.

Besides looking at rates of retinopathy, Dr. Hoehn’s group studied which treatments were most effective. They found that hydroxyurea and chronic transfusions best lowered retinopathy rates among all genotypes.

“We hope that people will use this information to better care for patients with sickle cell disease, and that more timely ophthalmic screen exams will be performed so that vision-threatening complications from this disease are prevented,” Dr. Hoehn said.

The authors reported no relevant financial relationships.

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

Clinicians must monitor children with sickle cell disease for eye complications as much as they do for adults, a new research review suggests.

Earlier research indicated that older patients were more at risk for eye complications from sickle cell disease, but the new study found that a full third of young people aged 10-25 years with sickle cell disease had retinopathy, including nonproliferative retinopathy (33%) and proliferative retinopathy (6%), which can progress to vision loss.

Two patients experienced retinal detachment, while two suffered retinal artery occlusion. One patient with retinal artery occlusion lost their vision and had a final best-corrected visual acuity of 20/60, according to the researchers, who presented their findings at the annual meeting of the American Academy of Ophthalmology.

“Our data underscores the need for patients – including pediatric patients – with sickle cell disease to get routine ophthalmic screenings along with appropriate systemic and ophthalmic treatment,” Mary Ellen Hoehn, MD, a professor of ophthalmology at the University of Tennessee Health Science Center, Memphis, who led the research, said in a press release.

The review covered records for 652 patients with sickle cell disease aged 10-25 years (median age, 14 years), who underwent eye exams over a 12-year period.

Besides looking at rates of retinopathy, Dr. Hoehn’s group studied which treatments were most effective. They found that hydroxyurea and chronic transfusions best lowered retinopathy rates among all genotypes.

“We hope that people will use this information to better care for patients with sickle cell disease, and that more timely ophthalmic screen exams will be performed so that vision-threatening complications from this disease are prevented,” Dr. Hoehn said.

The authors reported no relevant financial relationships.

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

Two sets of evidence reports address the primary care physicians’ role in children and adolescents’ oral health and the effectiveness of the fluoride gels and sealants offered at dental offices and schools.

Both were published online in JAMA.

In one report, the United States Preventive Services Task Force (USPSTF) concludes that there is not enough evidence to assess harms versus benefits of routine screening or interventions for oral health conditions, including dental caries, in primary care for asymptomatic children and adolescents aged 5-17 years.

The evidence report on administering fluoride supplements, fluoride gels, sealants and varnish finds evidence that they improve outcomes. The report was done to inform the USPSTF for a new recommendation on primary care screening, dental referral, behavioral counseling, and preventive interventions for oral health in children and adolescents aged 5-17.
 

Primary care physicians’ role

One problem the USPSTF identified in its report was limited evidence on available clinical screening tools or assessments to identify which children have oral health conditions in the primary care setting.

The USPSTF’s team, led by Michael J. Barry, MD, of Harvard Medical School in Boston, calls for more research to fill in the gaps before it can reassess.

Michael S. Reddy, DMD, DMSc, with University of California San Francisco School of Dentistry, Oral Health Affairs, said in an accompanying editorial that the current lack of data should not keep primary care physicians from considering oral health during routine medical exams or keep dentists from finding ways to collaborate with primary care physicians. “Medical primary care must partner with dentistry,” they wrote.

Until there is enough evidence for a USPSTF reevaluation on the topic, primary care clinicians should ask patients about their oral hygiene routines, whether they have any dental symptoms, and when they last saw a dentist, as well as referring to a dentist as necessary, the editorialists wrote.

That works both ways, the editorialists added. “Equally important, oral health professionals are encouraged to collaborate and be a resource for their primary care colleagues. Prevention is one of the best tools clinicians have, and it is promoted by integrated, whole-person health effort, “ wrote Dr. Reddy and colleagues.

When oral health stays separate from medical care, patients are left vulnerable, and referrals between medical and dental offices should be a stronger two-way system, the editorialists said.

“[N]ot every primary care patient has access to a dentist,” they wrote. “Oral health screening and referral by medical primary care clinicians can help ensure that individuals get to the dental chair to receive needed interventions that can benefit both oral and potentially overall health. Likewise, medical challenges and oral mucosal manifestations of chronic health conditions detected at a dental visit should result in medical referral, allowing prompt evaluation and treatment.”
 

Evidence that gels, varnish, sealants are effective

In a companion paper, done to inform the USPSTF, Roger Chou, MD, with Pacific Northwest Evidence-based Practice Center, Department of Medical Informatics and Clinical Epidemiology at Oregon Health & Science University in Portland, and colleagues found that when administered by a dental professional or in school settings, fluoride supplements, gels and varnish, and resin-based sealants improved health outcomes.

The findings were based on three systematic reviews (20,684 participants) and 19 randomized clinical trials; three nonrandomized trials; and one observational study (total 15,026 participants.)

With fluoride versus placebo or no intervention, researchers found a decrease from baseline in the number of decayed, missing, or filled permanent teeth (DMFT index) or decayed or filled permanent teeth (DFT index). The average difference was −0.73 [95% confidence interval [CI], −1.30 to −0.19]) at 1.5 to 3 years (six trials; n = 1,395).

Fluoride gels were associated with a DMFT- or DFT-prevented fraction of 0.18 (95% CI, 0.09-0.27) at outcomes closest to 3 years (four trials; n = 1,525).

Researchers found an association between fluoride varnish and a DMFT- or DFT-prevented fraction of 0.44 (95% CI, 0.11-0.76) at 1 to 4.5 years (five trials; n = 3,902). The sealants tested were associated with decreased risk of caries in first molars (odds ratio, 0.21 [95% CI, 0.16-0.28]) at 48-54 months (four trials; n = 440).

They noted that the feasibility of administering preventive measures in primary care is unknown; the effectiveness shown here was based on administration in dental and supervised school settings.

Barriers in primary care settings may include lack of training and equipment (particularly for sealants), uncertain reimbursement and lack of acceptance and uptake.
 

USPSTF working to close evidence gaps

Wanda Nicholson, MD, MPH, Prevention and Community Health, George Washington Milken Institute of Public Health in Washington, wrote in an accompanying editorial that to speed necessary research to facilitate recommendations, “the USPSTF and its stakeholders need a transparent, easily implementable communication tool that will systematically describe the research necessary to be directly responsive to the evidence gaps.”

The editorialists noted that the USPSTF in trying to update recommendations often has few, if any, high-quality additional studies to consider since its previous recommendation.

To address that, meetings were conducted in November of 2022 involving USPSTF members, Agency for Healthcare Research and Quality (AHRQ) staff, and leadership from the Office of Disease Prevention and the National Institutes of Health. Members formed a working group “to develop a standardized template for communicating research gaps” according to a framework developed by the National Academies of Sciences, Engineering, and Medicine.

Dr. Nicholson and colleagues wrote, “classifying evidence gaps and calling for specific research needs is a prudent, collaborative step in addressing missing evidence,” particularly for underserved populations.

The authors and editorialists declared no relevant conflicts of interest.

Two sets of evidence reports address the primary care physicians’ role in children and adolescents’ oral health and the effectiveness of the fluoride gels and sealants offered at dental offices and schools.

Both were published online in JAMA.

In one report, the United States Preventive Services Task Force (USPSTF) concludes that there is not enough evidence to assess harms versus benefits of routine screening or interventions for oral health conditions, including dental caries, in primary care for asymptomatic children and adolescents aged 5-17 years.

The evidence report on administering fluoride supplements, fluoride gels, sealants and varnish finds evidence that they improve outcomes. The report was done to inform the USPSTF for a new recommendation on primary care screening, dental referral, behavioral counseling, and preventive interventions for oral health in children and adolescents aged 5-17.
 

Primary care physicians’ role

One problem the USPSTF identified in its report was limited evidence on available clinical screening tools or assessments to identify which children have oral health conditions in the primary care setting.

The USPSTF’s team, led by Michael J. Barry, MD, of Harvard Medical School in Boston, calls for more research to fill in the gaps before it can reassess.

Michael S. Reddy, DMD, DMSc, with University of California San Francisco School of Dentistry, Oral Health Affairs, said in an accompanying editorial that the current lack of data should not keep primary care physicians from considering oral health during routine medical exams or keep dentists from finding ways to collaborate with primary care physicians. “Medical primary care must partner with dentistry,” they wrote.

Until there is enough evidence for a USPSTF reevaluation on the topic, primary care clinicians should ask patients about their oral hygiene routines, whether they have any dental symptoms, and when they last saw a dentist, as well as referring to a dentist as necessary, the editorialists wrote.

That works both ways, the editorialists added. “Equally important, oral health professionals are encouraged to collaborate and be a resource for their primary care colleagues. Prevention is one of the best tools clinicians have, and it is promoted by integrated, whole-person health effort, “ wrote Dr. Reddy and colleagues.

When oral health stays separate from medical care, patients are left vulnerable, and referrals between medical and dental offices should be a stronger two-way system, the editorialists said.

“[N]ot every primary care patient has access to a dentist,” they wrote. “Oral health screening and referral by medical primary care clinicians can help ensure that individuals get to the dental chair to receive needed interventions that can benefit both oral and potentially overall health. Likewise, medical challenges and oral mucosal manifestations of chronic health conditions detected at a dental visit should result in medical referral, allowing prompt evaluation and treatment.”
 

Evidence that gels, varnish, sealants are effective

In a companion paper, done to inform the USPSTF, Roger Chou, MD, with Pacific Northwest Evidence-based Practice Center, Department of Medical Informatics and Clinical Epidemiology at Oregon Health & Science University in Portland, and colleagues found that when administered by a dental professional or in school settings, fluoride supplements, gels and varnish, and resin-based sealants improved health outcomes.

The findings were based on three systematic reviews (20,684 participants) and 19 randomized clinical trials; three nonrandomized trials; and one observational study (total 15,026 participants.)

With fluoride versus placebo or no intervention, researchers found a decrease from baseline in the number of decayed, missing, or filled permanent teeth (DMFT index) or decayed or filled permanent teeth (DFT index). The average difference was −0.73 [95% confidence interval [CI], −1.30 to −0.19]) at 1.5 to 3 years (six trials; n = 1,395).

Fluoride gels were associated with a DMFT- or DFT-prevented fraction of 0.18 (95% CI, 0.09-0.27) at outcomes closest to 3 years (four trials; n = 1,525).

Researchers found an association between fluoride varnish and a DMFT- or DFT-prevented fraction of 0.44 (95% CI, 0.11-0.76) at 1 to 4.5 years (five trials; n = 3,902). The sealants tested were associated with decreased risk of caries in first molars (odds ratio, 0.21 [95% CI, 0.16-0.28]) at 48-54 months (four trials; n = 440).

They noted that the feasibility of administering preventive measures in primary care is unknown; the effectiveness shown here was based on administration in dental and supervised school settings.

Barriers in primary care settings may include lack of training and equipment (particularly for sealants), uncertain reimbursement and lack of acceptance and uptake.
 

USPSTF working to close evidence gaps

Wanda Nicholson, MD, MPH, Prevention and Community Health, George Washington Milken Institute of Public Health in Washington, wrote in an accompanying editorial that to speed necessary research to facilitate recommendations, “the USPSTF and its stakeholders need a transparent, easily implementable communication tool that will systematically describe the research necessary to be directly responsive to the evidence gaps.”

The editorialists noted that the USPSTF in trying to update recommendations often has few, if any, high-quality additional studies to consider since its previous recommendation.

To address that, meetings were conducted in November of 2022 involving USPSTF members, Agency for Healthcare Research and Quality (AHRQ) staff, and leadership from the Office of Disease Prevention and the National Institutes of Health. Members formed a working group “to develop a standardized template for communicating research gaps” according to a framework developed by the National Academies of Sciences, Engineering, and Medicine.

Dr. Nicholson and colleagues wrote, “classifying evidence gaps and calling for specific research needs is a prudent, collaborative step in addressing missing evidence,” particularly for underserved populations.

The authors and editorialists declared no relevant conflicts of interest.

Two sets of evidence reports address the primary care physicians’ role in children and adolescents’ oral health and the effectiveness of the fluoride gels and sealants offered at dental offices and schools.

Both were published online in JAMA.

In one report, the United States Preventive Services Task Force (USPSTF) concludes that there is not enough evidence to assess harms versus benefits of routine screening or interventions for oral health conditions, including dental caries, in primary care for asymptomatic children and adolescents aged 5-17 years.

The evidence report on administering fluoride supplements, fluoride gels, sealants and varnish finds evidence that they improve outcomes. The report was done to inform the USPSTF for a new recommendation on primary care screening, dental referral, behavioral counseling, and preventive interventions for oral health in children and adolescents aged 5-17.
 

Primary care physicians’ role

One problem the USPSTF identified in its report was limited evidence on available clinical screening tools or assessments to identify which children have oral health conditions in the primary care setting.

The USPSTF’s team, led by Michael J. Barry, MD, of Harvard Medical School in Boston, calls for more research to fill in the gaps before it can reassess.

Michael S. Reddy, DMD, DMSc, with University of California San Francisco School of Dentistry, Oral Health Affairs, said in an accompanying editorial that the current lack of data should not keep primary care physicians from considering oral health during routine medical exams or keep dentists from finding ways to collaborate with primary care physicians. “Medical primary care must partner with dentistry,” they wrote.

Until there is enough evidence for a USPSTF reevaluation on the topic, primary care clinicians should ask patients about their oral hygiene routines, whether they have any dental symptoms, and when they last saw a dentist, as well as referring to a dentist as necessary, the editorialists wrote.

That works both ways, the editorialists added. “Equally important, oral health professionals are encouraged to collaborate and be a resource for their primary care colleagues. Prevention is one of the best tools clinicians have, and it is promoted by integrated, whole-person health effort, “ wrote Dr. Reddy and colleagues.

When oral health stays separate from medical care, patients are left vulnerable, and referrals between medical and dental offices should be a stronger two-way system, the editorialists said.

“[N]ot every primary care patient has access to a dentist,” they wrote. “Oral health screening and referral by medical primary care clinicians can help ensure that individuals get to the dental chair to receive needed interventions that can benefit both oral and potentially overall health. Likewise, medical challenges and oral mucosal manifestations of chronic health conditions detected at a dental visit should result in medical referral, allowing prompt evaluation and treatment.”
 

Evidence that gels, varnish, sealants are effective

In a companion paper, done to inform the USPSTF, Roger Chou, MD, with Pacific Northwest Evidence-based Practice Center, Department of Medical Informatics and Clinical Epidemiology at Oregon Health & Science University in Portland, and colleagues found that when administered by a dental professional or in school settings, fluoride supplements, gels and varnish, and resin-based sealants improved health outcomes.

The findings were based on three systematic reviews (20,684 participants) and 19 randomized clinical trials; three nonrandomized trials; and one observational study (total 15,026 participants.)

With fluoride versus placebo or no intervention, researchers found a decrease from baseline in the number of decayed, missing, or filled permanent teeth (DMFT index) or decayed or filled permanent teeth (DFT index). The average difference was −0.73 [95% confidence interval [CI], −1.30 to −0.19]) at 1.5 to 3 years (six trials; n = 1,395).

Fluoride gels were associated with a DMFT- or DFT-prevented fraction of 0.18 (95% CI, 0.09-0.27) at outcomes closest to 3 years (four trials; n = 1,525).

Researchers found an association between fluoride varnish and a DMFT- or DFT-prevented fraction of 0.44 (95% CI, 0.11-0.76) at 1 to 4.5 years (five trials; n = 3,902). The sealants tested were associated with decreased risk of caries in first molars (odds ratio, 0.21 [95% CI, 0.16-0.28]) at 48-54 months (four trials; n = 440).

They noted that the feasibility of administering preventive measures in primary care is unknown; the effectiveness shown here was based on administration in dental and supervised school settings.

Barriers in primary care settings may include lack of training and equipment (particularly for sealants), uncertain reimbursement and lack of acceptance and uptake.
 

USPSTF working to close evidence gaps

Wanda Nicholson, MD, MPH, Prevention and Community Health, George Washington Milken Institute of Public Health in Washington, wrote in an accompanying editorial that to speed necessary research to facilitate recommendations, “the USPSTF and its stakeholders need a transparent, easily implementable communication tool that will systematically describe the research necessary to be directly responsive to the evidence gaps.”

The editorialists noted that the USPSTF in trying to update recommendations often has few, if any, high-quality additional studies to consider since its previous recommendation.

To address that, meetings were conducted in November of 2022 involving USPSTF members, Agency for Healthcare Research and Quality (AHRQ) staff, and leadership from the Office of Disease Prevention and the National Institutes of Health. Members formed a working group “to develop a standardized template for communicating research gaps” according to a framework developed by the National Academies of Sciences, Engineering, and Medicine.

Dr. Nicholson and colleagues wrote, “classifying evidence gaps and calling for specific research needs is a prudent, collaborative step in addressing missing evidence,” particularly for underserved populations.

The authors and editorialists declared no relevant conflicts of interest.

Suicide is among the top three causes of death for young people in the United States. According to the Centers for Disease Control and Prevention, the rate of suicide deaths has climbed from 4.4 per 100,000 American 12- to 17-year-olds in 2011 to 6.5 per 100,000 in 2021, an increase of almost 50%. As with accidents and homicides, we hope these are preventable deaths, although the factors contributing to them are complex.

We do know that more than half of the people who die by suicide visit a health care provider within 4 weeks of their death, highlighting an opportunity for screening and intervention.
 

Suicide screening

In 2022, the American Academy of Pediatrics (AAP) recommended that all adolescents get screened for suicide risk annually. Given that less than 1 in 10,000 adolescents commit suicide and that there is no definitive data on how to prevent suicide in any individual, the goal of suicide screening is much broader than preventing suicide. Beyond universal screening, we will review how being open and curious with all of your patients can be the most extraordinary screening instrument.

There is extensive data that tells us that far from causing suicide, asking about suicidal thoughts is protective. When you make suicidal thoughts discussable, you directly counteract the isolation, stigma, and shame that are strong predictors of actual suicide attempts. You model the value of bringing difficult or frightening thoughts to the attention of caring adults, and you model calm listening rather than emotional overreaction for their parents. The resulting connectedness can lower the risk for vulnerable patients and enhance resilience for all of your patients.
 

Who is at greater risk?

We have robust data to guide our understanding of which youth have suicidal ideation, which is distinct from those who attempt suicide, which also may be quite distinct from those who complete. The CDC reports that the rate of suicidal thoughts (“seriously considering suicide”) in high school students climbed from 16% in 2011 to 22% in 2021. In that decade, the number of high schoolers with a suicide plan climbed from 13% to 18%, and those with suicide attempts climbed from 8% to 10%. Girls are at higher risk for suicidal thoughts and attempts, but boys are at greater risk for suicide completion. Black youth were more likely to attempt suicide than were their Asian, Hispanic, or White peers and LGBTQ+ youth are at particular risk; in 2021, they were three times as likely as were their heterosexual peers to have suicidal thoughts and attempts. Youth with psychiatric illness (particularly PTSD, mood or thought disorders), a family history of suicide, a history of risk-taking behavior (including sexual activity, smoking, drinking, and drug use) and those with prior suicide attempts are at the highest risk for suicide. Adding all these risk factors together means that many, if not the majority, of teenagers have risk factors.

Focus on the patient

In your office, though, a public health approach should give way to curiosity about your individual patient. Suicidal thoughts usually follow a substantial stress. Pay attention to exceptional stresses, especially if they have a component of social stigma or isolation. Did your patient report another student for an assault? Are they now being bullied or ostracized by friends? Have they lost an especially important relationship? Some other stresses may seem minor, such as a poor grade on a test. But for a very driven, perfectionistic teenager who believes that a perfect 4.0 GPA is essential to college admission and future success and happiness, one poor grade may feel like a catastrophe.

When your patients tell you about a challenge or setback, slow down and be curious. Listen to the importance they give it. How have they managed it? Are they finding it hard to go to school or back to practice? Do they feel discouraged or even hopeless? Discouragement is a normal response to adversity, but it should be temporary. This approach can make it easy to ask if they have ever wished they were dead, or made a suicide plan or an attempt. When you calmly and supportively learn about their inner experience, it will be easy for young people to be honest with you.

There will be teenagers in your practice who are sensation-seeking and impulsive, and you should pay special attention to this group. They may not be classically depressed, but in the aftermath of a stressful experience that they find humiliating or shameful, they are at risk for an impulsive act that could still be lethal. Be curious with these patients after they feel they have let down their team or their family, or if they have been caught in a crime or cheating, or even if their girlfriend breaks up with them. Find out how they are managing, and where their support comes from. Ask them in a nonjudgmental manner about whether they are having thoughts about death or suicide, and if those thoughts are troubling, frequent, or feel like a relief. What has stopped them from acting on these thoughts? Offer your patient the perspective that such thoughts may be normal in the face of a large stress, but that the pain of stress always subsides, whereas suicide is irreversible.

There will also be patients in your practice who cut themselves. This is sometimes called “nonsuicidal self-injury,” and it often raises concern about suicide risk. While accelerating frequency of self-injury in a teenager who is suicidal can indicate growing risk, this behavior alone is usually a mechanism for regulating emotion. Ask your patient about when they cut themselves. What are the triggers? How do they feel afterward? Are their friends all doing it? Is it only after fighting with their parents? Or does it make their parents worry instead of getting angry? As you learn about the nature of the behavior, you will be able to offer thoughtful guidance about better strategies for stress management or to pursue further assessment and support.
 

Next steps

Speaking comfortably with your patients about suicidal thoughts and behaviors requires that you also feel comfortable with what comes next. As in the ASQ screening instrument recommended by the AAP, you should always follow affirmative answers about suicidal thoughts with more questions. Do they have a plan? Do they have access to lethal means including any guns in the home? Have they ever made an attempt? Are they thinking about killing themselves now? If the thoughts are current, they have access, and they have tried before, it is clear that they need an urgent assessment, probably in an emergency department. But when the thoughts were in the past or have never been connected to plans or intent, there is an opportunity to enhance their connectedness. You can diminish the potential for shame, stigma and isolation by reminding them that such thoughts and feelings are normal in the face of difficulty. They deserve support to help them face and manage their adversity, whether that stress comes from an internal or external source. How do they feel now that they have shared these thoughts with you? Most will describe feeling better, relieved, even hopeful once they are not facing intense thoughts and feelings alone.

You should tell them that you would like to bring their parents into the conversation. You want them to know they can turn to their parents if they are having these thoughts, so they are never alone in facing them. Parents can learn from your model of calm and supportive listening to fully understand the situation before turning together to talk about what might be helpful next steps. It is always prudent to create “speed bumps” between thought and action with impulsive teens, so recommend limiting access to any lethal means (firearms especially). But the strongest protective intervention is for the child to feel confident in and connected to their support network, trusting you and their parents to listen and understand before figuring out together what else is needed to address the situation.

Lastly, recognize that talking about difficult issues with teenagers is among the most stressful and demanding aspects of pediatric primary care. Talk to colleagues, never worry alone, and recognize and manage your own stress. This is among the best ways to model for your patients and their parents that every challenge can be met, but we often need support.
 

Dr. Swick is physician in chief at Ohana, Center for Child and Adolescent Behavioral Health, Community Hospital of the Monterey (Calif.) Peninsula. Dr. Jellinek is professor emeritus of psychiatry and pediatrics, Harvard Medical School, Boston. Email them at [email protected].

Suicide is among the top three causes of death for young people in the United States. According to the Centers for Disease Control and Prevention, the rate of suicide deaths has climbed from 4.4 per 100,000 American 12- to 17-year-olds in 2011 to 6.5 per 100,000 in 2021, an increase of almost 50%. As with accidents and homicides, we hope these are preventable deaths, although the factors contributing to them are complex.

We do know that more than half of the people who die by suicide visit a health care provider within 4 weeks of their death, highlighting an opportunity for screening and intervention.
 

Suicide screening

In 2022, the American Academy of Pediatrics (AAP) recommended that all adolescents get screened for suicide risk annually. Given that less than 1 in 10,000 adolescents commit suicide and that there is no definitive data on how to prevent suicide in any individual, the goal of suicide screening is much broader than preventing suicide. Beyond universal screening, we will review how being open and curious with all of your patients can be the most extraordinary screening instrument.

There is extensive data that tells us that far from causing suicide, asking about suicidal thoughts is protective. When you make suicidal thoughts discussable, you directly counteract the isolation, stigma, and shame that are strong predictors of actual suicide attempts. You model the value of bringing difficult or frightening thoughts to the attention of caring adults, and you model calm listening rather than emotional overreaction for their parents. The resulting connectedness can lower the risk for vulnerable patients and enhance resilience for all of your patients.
 

Who is at greater risk?

We have robust data to guide our understanding of which youth have suicidal ideation, which is distinct from those who attempt suicide, which also may be quite distinct from those who complete. The CDC reports that the rate of suicidal thoughts (“seriously considering suicide”) in high school students climbed from 16% in 2011 to 22% in 2021. In that decade, the number of high schoolers with a suicide plan climbed from 13% to 18%, and those with suicide attempts climbed from 8% to 10%. Girls are at higher risk for suicidal thoughts and attempts, but boys are at greater risk for suicide completion. Black youth were more likely to attempt suicide than were their Asian, Hispanic, or White peers and LGBTQ+ youth are at particular risk; in 2021, they were three times as likely as were their heterosexual peers to have suicidal thoughts and attempts. Youth with psychiatric illness (particularly PTSD, mood or thought disorders), a family history of suicide, a history of risk-taking behavior (including sexual activity, smoking, drinking, and drug use) and those with prior suicide attempts are at the highest risk for suicide. Adding all these risk factors together means that many, if not the majority, of teenagers have risk factors.

Focus on the patient

In your office, though, a public health approach should give way to curiosity about your individual patient. Suicidal thoughts usually follow a substantial stress. Pay attention to exceptional stresses, especially if they have a component of social stigma or isolation. Did your patient report another student for an assault? Are they now being bullied or ostracized by friends? Have they lost an especially important relationship? Some other stresses may seem minor, such as a poor grade on a test. But for a very driven, perfectionistic teenager who believes that a perfect 4.0 GPA is essential to college admission and future success and happiness, one poor grade may feel like a catastrophe.

When your patients tell you about a challenge or setback, slow down and be curious. Listen to the importance they give it. How have they managed it? Are they finding it hard to go to school or back to practice? Do they feel discouraged or even hopeless? Discouragement is a normal response to adversity, but it should be temporary. This approach can make it easy to ask if they have ever wished they were dead, or made a suicide plan or an attempt. When you calmly and supportively learn about their inner experience, it will be easy for young people to be honest with you.

There will be teenagers in your practice who are sensation-seeking and impulsive, and you should pay special attention to this group. They may not be classically depressed, but in the aftermath of a stressful experience that they find humiliating or shameful, they are at risk for an impulsive act that could still be lethal. Be curious with these patients after they feel they have let down their team or their family, or if they have been caught in a crime or cheating, or even if their girlfriend breaks up with them. Find out how they are managing, and where their support comes from. Ask them in a nonjudgmental manner about whether they are having thoughts about death or suicide, and if those thoughts are troubling, frequent, or feel like a relief. What has stopped them from acting on these thoughts? Offer your patient the perspective that such thoughts may be normal in the face of a large stress, but that the pain of stress always subsides, whereas suicide is irreversible.

There will also be patients in your practice who cut themselves. This is sometimes called “nonsuicidal self-injury,” and it often raises concern about suicide risk. While accelerating frequency of self-injury in a teenager who is suicidal can indicate growing risk, this behavior alone is usually a mechanism for regulating emotion. Ask your patient about when they cut themselves. What are the triggers? How do they feel afterward? Are their friends all doing it? Is it only after fighting with their parents? Or does it make their parents worry instead of getting angry? As you learn about the nature of the behavior, you will be able to offer thoughtful guidance about better strategies for stress management or to pursue further assessment and support.
 

Next steps

Speaking comfortably with your patients about suicidal thoughts and behaviors requires that you also feel comfortable with what comes next. As in the ASQ screening instrument recommended by the AAP, you should always follow affirmative answers about suicidal thoughts with more questions. Do they have a plan? Do they have access to lethal means including any guns in the home? Have they ever made an attempt? Are they thinking about killing themselves now? If the thoughts are current, they have access, and they have tried before, it is clear that they need an urgent assessment, probably in an emergency department. But when the thoughts were in the past or have never been connected to plans or intent, there is an opportunity to enhance their connectedness. You can diminish the potential for shame, stigma and isolation by reminding them that such thoughts and feelings are normal in the face of difficulty. They deserve support to help them face and manage their adversity, whether that stress comes from an internal or external source. How do they feel now that they have shared these thoughts with you? Most will describe feeling better, relieved, even hopeful once they are not facing intense thoughts and feelings alone.

You should tell them that you would like to bring their parents into the conversation. You want them to know they can turn to their parents if they are having these thoughts, so they are never alone in facing them. Parents can learn from your model of calm and supportive listening to fully understand the situation before turning together to talk about what might be helpful next steps. It is always prudent to create “speed bumps” between thought and action with impulsive teens, so recommend limiting access to any lethal means (firearms especially). But the strongest protective intervention is for the child to feel confident in and connected to their support network, trusting you and their parents to listen and understand before figuring out together what else is needed to address the situation.

Lastly, recognize that talking about difficult issues with teenagers is among the most stressful and demanding aspects of pediatric primary care. Talk to colleagues, never worry alone, and recognize and manage your own stress. This is among the best ways to model for your patients and their parents that every challenge can be met, but we often need support.
 

Dr. Swick is physician in chief at Ohana, Center for Child and Adolescent Behavioral Health, Community Hospital of the Monterey (Calif.) Peninsula. Dr. Jellinek is professor emeritus of psychiatry and pediatrics, Harvard Medical School, Boston. Email them at [email protected].

Suicide is among the top three causes of death for young people in the United States. According to the Centers for Disease Control and Prevention, the rate of suicide deaths has climbed from 4.4 per 100,000 American 12- to 17-year-olds in 2011 to 6.5 per 100,000 in 2021, an increase of almost 50%. As with accidents and homicides, we hope these are preventable deaths, although the factors contributing to them are complex.

We do know that more than half of the people who die by suicide visit a health care provider within 4 weeks of their death, highlighting an opportunity for screening and intervention.
 

Suicide screening

In 2022, the American Academy of Pediatrics (AAP) recommended that all adolescents get screened for suicide risk annually. Given that less than 1 in 10,000 adolescents commit suicide and that there is no definitive data on how to prevent suicide in any individual, the goal of suicide screening is much broader than preventing suicide. Beyond universal screening, we will review how being open and curious with all of your patients can be the most extraordinary screening instrument.

There is extensive data that tells us that far from causing suicide, asking about suicidal thoughts is protective. When you make suicidal thoughts discussable, you directly counteract the isolation, stigma, and shame that are strong predictors of actual suicide attempts. You model the value of bringing difficult or frightening thoughts to the attention of caring adults, and you model calm listening rather than emotional overreaction for their parents. The resulting connectedness can lower the risk for vulnerable patients and enhance resilience for all of your patients.
 

Who is at greater risk?

We have robust data to guide our understanding of which youth have suicidal ideation, which is distinct from those who attempt suicide, which also may be quite distinct from those who complete. The CDC reports that the rate of suicidal thoughts (“seriously considering suicide”) in high school students climbed from 16% in 2011 to 22% in 2021. In that decade, the number of high schoolers with a suicide plan climbed from 13% to 18%, and those with suicide attempts climbed from 8% to 10%. Girls are at higher risk for suicidal thoughts and attempts, but boys are at greater risk for suicide completion. Black youth were more likely to attempt suicide than were their Asian, Hispanic, or White peers and LGBTQ+ youth are at particular risk; in 2021, they were three times as likely as were their heterosexual peers to have suicidal thoughts and attempts. Youth with psychiatric illness (particularly PTSD, mood or thought disorders), a family history of suicide, a history of risk-taking behavior (including sexual activity, smoking, drinking, and drug use) and those with prior suicide attempts are at the highest risk for suicide. Adding all these risk factors together means that many, if not the majority, of teenagers have risk factors.

Focus on the patient

In your office, though, a public health approach should give way to curiosity about your individual patient. Suicidal thoughts usually follow a substantial stress. Pay attention to exceptional stresses, especially if they have a component of social stigma or isolation. Did your patient report another student for an assault? Are they now being bullied or ostracized by friends? Have they lost an especially important relationship? Some other stresses may seem minor, such as a poor grade on a test. But for a very driven, perfectionistic teenager who believes that a perfect 4.0 GPA is essential to college admission and future success and happiness, one poor grade may feel like a catastrophe.

When your patients tell you about a challenge or setback, slow down and be curious. Listen to the importance they give it. How have they managed it? Are they finding it hard to go to school or back to practice? Do they feel discouraged or even hopeless? Discouragement is a normal response to adversity, but it should be temporary. This approach can make it easy to ask if they have ever wished they were dead, or made a suicide plan or an attempt. When you calmly and supportively learn about their inner experience, it will be easy for young people to be honest with you.

There will be teenagers in your practice who are sensation-seeking and impulsive, and you should pay special attention to this group. They may not be classically depressed, but in the aftermath of a stressful experience that they find humiliating or shameful, they are at risk for an impulsive act that could still be lethal. Be curious with these patients after they feel they have let down their team or their family, or if they have been caught in a crime or cheating, or even if their girlfriend breaks up with them. Find out how they are managing, and where their support comes from. Ask them in a nonjudgmental manner about whether they are having thoughts about death or suicide, and if those thoughts are troubling, frequent, or feel like a relief. What has stopped them from acting on these thoughts? Offer your patient the perspective that such thoughts may be normal in the face of a large stress, but that the pain of stress always subsides, whereas suicide is irreversible.

There will also be patients in your practice who cut themselves. This is sometimes called “nonsuicidal self-injury,” and it often raises concern about suicide risk. While accelerating frequency of self-injury in a teenager who is suicidal can indicate growing risk, this behavior alone is usually a mechanism for regulating emotion. Ask your patient about when they cut themselves. What are the triggers? How do they feel afterward? Are their friends all doing it? Is it only after fighting with their parents? Or does it make their parents worry instead of getting angry? As you learn about the nature of the behavior, you will be able to offer thoughtful guidance about better strategies for stress management or to pursue further assessment and support.
 

Next steps

Speaking comfortably with your patients about suicidal thoughts and behaviors requires that you also feel comfortable with what comes next. As in the ASQ screening instrument recommended by the AAP, you should always follow affirmative answers about suicidal thoughts with more questions. Do they have a plan? Do they have access to lethal means including any guns in the home? Have they ever made an attempt? Are they thinking about killing themselves now? If the thoughts are current, they have access, and they have tried before, it is clear that they need an urgent assessment, probably in an emergency department. But when the thoughts were in the past or have never been connected to plans or intent, there is an opportunity to enhance their connectedness. You can diminish the potential for shame, stigma and isolation by reminding them that such thoughts and feelings are normal in the face of difficulty. They deserve support to help them face and manage their adversity, whether that stress comes from an internal or external source. How do they feel now that they have shared these thoughts with you? Most will describe feeling better, relieved, even hopeful once they are not facing intense thoughts and feelings alone.

You should tell them that you would like to bring their parents into the conversation. You want them to know they can turn to their parents if they are having these thoughts, so they are never alone in facing them. Parents can learn from your model of calm and supportive listening to fully understand the situation before turning together to talk about what might be helpful next steps. It is always prudent to create “speed bumps” between thought and action with impulsive teens, so recommend limiting access to any lethal means (firearms especially). But the strongest protective intervention is for the child to feel confident in and connected to their support network, trusting you and their parents to listen and understand before figuring out together what else is needed to address the situation.

Lastly, recognize that talking about difficult issues with teenagers is among the most stressful and demanding aspects of pediatric primary care. Talk to colleagues, never worry alone, and recognize and manage your own stress. This is among the best ways to model for your patients and their parents that every challenge can be met, but we often need support.
 

Dr. Swick is physician in chief at Ohana, Center for Child and Adolescent Behavioral Health, Community Hospital of the Monterey (Calif.) Peninsula. Dr. Jellinek is professor emeritus of psychiatry and pediatrics, Harvard Medical School, Boston. Email them at [email protected].

PHOENIX – Children with Duchenne muscular dystrophy (DMD) treated with the only gene therapy to date to be approved for treatment of disease in the United States show sustained maintenance of motor function after 4 years, compared with untreated patients who showed significant decline over the same time period, new research shows.

“Functional assessments demonstrated long-term sustained stabilization of motor function that was clinically meaningful, at ages where functional decline would be expected based on natural history,” the investigators noted in their abstract. Furthermore, the treatment, known as delandistrogene moxeparvovec-rokl (SRP-9001), was well tolerated 4 years post treatment.

The study was presented at the annual meeting of the American Association of Neuromuscular Electrodiagnostic Medicine.
 

Severe type of DMD

Considered one of the most severe forms of muscular dystrophy, DMD causes progressive muscle wasting stemming from the root genetic cause of missing dystrophin in muscle cells. Often referred to as a molecular “shock absorber,” dystrophin stabilizes the sarcolemma during muscle contractions to prevent degeneration.

SRP-9001, a single-dose recombinant gene therapy administered as an intravenous infusion, was designed to deliver a trimmed down form of dystrophin to compensate for the deficit.

In July, the adeno-associated virus vector (AAV)–based SRP-9001 gene therapy was granted accelerated approval by the Food and Drug Administration for the treatment of ambulatory pediatric patients aged 4-5 years with DMD with a confirmed mutation in the DMD gene.

The therapy is administered over 1-2 hours at a dose of 133 trillion vector genomes per kilogram of body weight.

For Study 101, one of several evaluating the novel therapy, a research team led by senior investigator Jerry Mendell, MD, an attending neurologist at Nationwide Children’s Hospital and professor of pediatrics and neurology at Ohio State University, both in Columbus,evaluated data on four ambulatory male patients aged 4-8 years who received a single IV infusion of the therapy.

All patients also received prednisone 1 mg/kg, 1 day preinfusion and 30 days post infusion.

At 4 years post treatment, there were no new safety events. All treatment-related adverse events occurred mainly within the first 70 days, and all resolved.

The most commonly reported adverse reactions of the gene therapy include vomiting, nausea, increases in liver enzymes, pyrexia (fever), and thrombocytopenia, all of which occurred within 90 days of infusion and been manageable.

Risk mitigation strategies for hepatotoxicity or acute liver injury include pre- and postinfusion monitoring of liver enzymes, the authors noted.

No serious abnormalities were observed in hematologic or chemistry panels, and while three patients had elevated gamma-glutamyl transpeptidase in the first 3 months post treatment, those cases resolved with oral steroid treatment.

Significant improvements in function were observed, with a mean improvement in North Star Ambulatory Assessment (NSAA) scores from baseline of 7.0 points (range, 4-11).

Exploratory analyses further showed that, compared with a propensity score–weighted external control cohort of 21 patients with DMD who did not receive the therapy, those receiving SRP-9001 had a statistically significant difference of 9.4 points in least-squares mean change from baseline to 4 years on the NSAA score (P = .0125).

Similar trends were observed in improvement from baseline in key measures of time to rise, 4-stair climb, and 10- and 100-meter walk/run function tests.

Other reported adverse events include acute serious liver injury, immune-mediated myositis, and myocarditis. Because of the latter risk, the therapy is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

The current 4-year update on SRP-9001 adds to clinical trial results that have been reported on more than 80 patients treated to date, with favorable results and consistent safety profiles reported at other time points.

Continued FDA approval for the therapy will be contingent upon verification of a clinical benefit in the confirmatory trials, including the EMBARK trial.
 

Increased function, long-term stability

Discussing the research at the meeting, Craig McDonald, MD, professor and chair of physical medicine & rehabilitation, a professor of pediatrics and study chair of the CINRG Duchenne Natural History Study at University of California Davis Health, noted that top-line results from the ongoing, confirmatory phase 3 EMBARK trial show functional benefits of SRP-9001 not only in 4- to 5-year-olds but also in other older age groups.

“What’s really striking, and in my mind the most impressive, is that when you follow these patients out 3 or 4 years ... you see there is this bump in function followed by long-term stability, whereas the external control cohort predictably shows really quite significant declines in their [NSAA] functional values,” he said in his presentation.

“When you look at each individually treated patient versus their own predicted trajectory using their baseline values on the time function test, each of the patients actually has a really quite impressive stabilization of function over their predicted disease trajectory,” he added.

A caveat that SRP-9001 shares with other gene therapies is the issue of cost – reported in the range of $2 million–$3 million.

In the context of racial and socioeconomic disparities in access to diagnosis and care reported in DMD, Emma Ciafaloni, MD, a professor of neurology and pediatrics at the University of Rochester (N.Y.) Medical Center, underscored the need to consider approval versus access to gene therapies and how to optimize access to the novel treatments. 

“We need to consider what the cost is, how it’s going to be accessed, and whether there is a sustainable model,” said Ciafaloni, who was not associated with the study. “There will need to be institutional readiness and support for specialized multidisciplinary clinics for gene therapy.”

She also noted “we need to consider how we can do better on a broader level, because this is not a provider problem or a manufacturer problem — it’s a society problem.”

The study was funded by Sarepta Therapeutics. McDonald reported consulting work for Sarepta Therapeutics and has been an investigator in SRP-9001 research. Ciafaloni reported serving on advisory boards or other relationships with Alexion, Argenx, Biogen, Amicus, Momenta, Medscape, Pfizer, Sanofi/Genzyme, Sarepta, Jansen, NS Pharma, CureSMA, Orphazyme, the Patient-Centered Outcomes Research Institute, PPMD, PTC Therapeutics, and Santhera.

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

PHOENIX – Children with Duchenne muscular dystrophy (DMD) treated with the only gene therapy to date to be approved for treatment of disease in the United States show sustained maintenance of motor function after 4 years, compared with untreated patients who showed significant decline over the same time period, new research shows.

“Functional assessments demonstrated long-term sustained stabilization of motor function that was clinically meaningful, at ages where functional decline would be expected based on natural history,” the investigators noted in their abstract. Furthermore, the treatment, known as delandistrogene moxeparvovec-rokl (SRP-9001), was well tolerated 4 years post treatment.

The study was presented at the annual meeting of the American Association of Neuromuscular Electrodiagnostic Medicine.
 

Severe type of DMD

Considered one of the most severe forms of muscular dystrophy, DMD causes progressive muscle wasting stemming from the root genetic cause of missing dystrophin in muscle cells. Often referred to as a molecular “shock absorber,” dystrophin stabilizes the sarcolemma during muscle contractions to prevent degeneration.

SRP-9001, a single-dose recombinant gene therapy administered as an intravenous infusion, was designed to deliver a trimmed down form of dystrophin to compensate for the deficit.

In July, the adeno-associated virus vector (AAV)–based SRP-9001 gene therapy was granted accelerated approval by the Food and Drug Administration for the treatment of ambulatory pediatric patients aged 4-5 years with DMD with a confirmed mutation in the DMD gene.

The therapy is administered over 1-2 hours at a dose of 133 trillion vector genomes per kilogram of body weight.

For Study 101, one of several evaluating the novel therapy, a research team led by senior investigator Jerry Mendell, MD, an attending neurologist at Nationwide Children’s Hospital and professor of pediatrics and neurology at Ohio State University, both in Columbus,evaluated data on four ambulatory male patients aged 4-8 years who received a single IV infusion of the therapy.

All patients also received prednisone 1 mg/kg, 1 day preinfusion and 30 days post infusion.

At 4 years post treatment, there were no new safety events. All treatment-related adverse events occurred mainly within the first 70 days, and all resolved.

The most commonly reported adverse reactions of the gene therapy include vomiting, nausea, increases in liver enzymes, pyrexia (fever), and thrombocytopenia, all of which occurred within 90 days of infusion and been manageable.

Risk mitigation strategies for hepatotoxicity or acute liver injury include pre- and postinfusion monitoring of liver enzymes, the authors noted.

No serious abnormalities were observed in hematologic or chemistry panels, and while three patients had elevated gamma-glutamyl transpeptidase in the first 3 months post treatment, those cases resolved with oral steroid treatment.

Significant improvements in function were observed, with a mean improvement in North Star Ambulatory Assessment (NSAA) scores from baseline of 7.0 points (range, 4-11).

Exploratory analyses further showed that, compared with a propensity score–weighted external control cohort of 21 patients with DMD who did not receive the therapy, those receiving SRP-9001 had a statistically significant difference of 9.4 points in least-squares mean change from baseline to 4 years on the NSAA score (P = .0125).

Similar trends were observed in improvement from baseline in key measures of time to rise, 4-stair climb, and 10- and 100-meter walk/run function tests.

Other reported adverse events include acute serious liver injury, immune-mediated myositis, and myocarditis. Because of the latter risk, the therapy is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

The current 4-year update on SRP-9001 adds to clinical trial results that have been reported on more than 80 patients treated to date, with favorable results and consistent safety profiles reported at other time points.

Continued FDA approval for the therapy will be contingent upon verification of a clinical benefit in the confirmatory trials, including the EMBARK trial.
 

Increased function, long-term stability

Discussing the research at the meeting, Craig McDonald, MD, professor and chair of physical medicine & rehabilitation, a professor of pediatrics and study chair of the CINRG Duchenne Natural History Study at University of California Davis Health, noted that top-line results from the ongoing, confirmatory phase 3 EMBARK trial show functional benefits of SRP-9001 not only in 4- to 5-year-olds but also in other older age groups.

“What’s really striking, and in my mind the most impressive, is that when you follow these patients out 3 or 4 years ... you see there is this bump in function followed by long-term stability, whereas the external control cohort predictably shows really quite significant declines in their [NSAA] functional values,” he said in his presentation.

“When you look at each individually treated patient versus their own predicted trajectory using their baseline values on the time function test, each of the patients actually has a really quite impressive stabilization of function over their predicted disease trajectory,” he added.

A caveat that SRP-9001 shares with other gene therapies is the issue of cost – reported in the range of $2 million–$3 million.

In the context of racial and socioeconomic disparities in access to diagnosis and care reported in DMD, Emma Ciafaloni, MD, a professor of neurology and pediatrics at the University of Rochester (N.Y.) Medical Center, underscored the need to consider approval versus access to gene therapies and how to optimize access to the novel treatments. 

“We need to consider what the cost is, how it’s going to be accessed, and whether there is a sustainable model,” said Ciafaloni, who was not associated with the study. “There will need to be institutional readiness and support for specialized multidisciplinary clinics for gene therapy.”

She also noted “we need to consider how we can do better on a broader level, because this is not a provider problem or a manufacturer problem — it’s a society problem.”

The study was funded by Sarepta Therapeutics. McDonald reported consulting work for Sarepta Therapeutics and has been an investigator in SRP-9001 research. Ciafaloni reported serving on advisory boards or other relationships with Alexion, Argenx, Biogen, Amicus, Momenta, Medscape, Pfizer, Sanofi/Genzyme, Sarepta, Jansen, NS Pharma, CureSMA, Orphazyme, the Patient-Centered Outcomes Research Institute, PPMD, PTC Therapeutics, and Santhera.

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

PHOENIX – Children with Duchenne muscular dystrophy (DMD) treated with the only gene therapy to date to be approved for treatment of disease in the United States show sustained maintenance of motor function after 4 years, compared with untreated patients who showed significant decline over the same time period, new research shows.

“Functional assessments demonstrated long-term sustained stabilization of motor function that was clinically meaningful, at ages where functional decline would be expected based on natural history,” the investigators noted in their abstract. Furthermore, the treatment, known as delandistrogene moxeparvovec-rokl (SRP-9001), was well tolerated 4 years post treatment.

The study was presented at the annual meeting of the American Association of Neuromuscular Electrodiagnostic Medicine.
 

Severe type of DMD

Considered one of the most severe forms of muscular dystrophy, DMD causes progressive muscle wasting stemming from the root genetic cause of missing dystrophin in muscle cells. Often referred to as a molecular “shock absorber,” dystrophin stabilizes the sarcolemma during muscle contractions to prevent degeneration.

SRP-9001, a single-dose recombinant gene therapy administered as an intravenous infusion, was designed to deliver a trimmed down form of dystrophin to compensate for the deficit.

In July, the adeno-associated virus vector (AAV)–based SRP-9001 gene therapy was granted accelerated approval by the Food and Drug Administration for the treatment of ambulatory pediatric patients aged 4-5 years with DMD with a confirmed mutation in the DMD gene.

The therapy is administered over 1-2 hours at a dose of 133 trillion vector genomes per kilogram of body weight.

For Study 101, one of several evaluating the novel therapy, a research team led by senior investigator Jerry Mendell, MD, an attending neurologist at Nationwide Children’s Hospital and professor of pediatrics and neurology at Ohio State University, both in Columbus,evaluated data on four ambulatory male patients aged 4-8 years who received a single IV infusion of the therapy.

All patients also received prednisone 1 mg/kg, 1 day preinfusion and 30 days post infusion.

At 4 years post treatment, there were no new safety events. All treatment-related adverse events occurred mainly within the first 70 days, and all resolved.

The most commonly reported adverse reactions of the gene therapy include vomiting, nausea, increases in liver enzymes, pyrexia (fever), and thrombocytopenia, all of which occurred within 90 days of infusion and been manageable.

Risk mitigation strategies for hepatotoxicity or acute liver injury include pre- and postinfusion monitoring of liver enzymes, the authors noted.

No serious abnormalities were observed in hematologic or chemistry panels, and while three patients had elevated gamma-glutamyl transpeptidase in the first 3 months post treatment, those cases resolved with oral steroid treatment.

Significant improvements in function were observed, with a mean improvement in North Star Ambulatory Assessment (NSAA) scores from baseline of 7.0 points (range, 4-11).

Exploratory analyses further showed that, compared with a propensity score–weighted external control cohort of 21 patients with DMD who did not receive the therapy, those receiving SRP-9001 had a statistically significant difference of 9.4 points in least-squares mean change from baseline to 4 years on the NSAA score (P = .0125).

Similar trends were observed in improvement from baseline in key measures of time to rise, 4-stair climb, and 10- and 100-meter walk/run function tests.

Other reported adverse events include acute serious liver injury, immune-mediated myositis, and myocarditis. Because of the latter risk, the therapy is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

The current 4-year update on SRP-9001 adds to clinical trial results that have been reported on more than 80 patients treated to date, with favorable results and consistent safety profiles reported at other time points.

Continued FDA approval for the therapy will be contingent upon verification of a clinical benefit in the confirmatory trials, including the EMBARK trial.
 

Increased function, long-term stability

Discussing the research at the meeting, Craig McDonald, MD, professor and chair of physical medicine & rehabilitation, a professor of pediatrics and study chair of the CINRG Duchenne Natural History Study at University of California Davis Health, noted that top-line results from the ongoing, confirmatory phase 3 EMBARK trial show functional benefits of SRP-9001 not only in 4- to 5-year-olds but also in other older age groups.

“What’s really striking, and in my mind the most impressive, is that when you follow these patients out 3 or 4 years ... you see there is this bump in function followed by long-term stability, whereas the external control cohort predictably shows really quite significant declines in their [NSAA] functional values,” he said in his presentation.

“When you look at each individually treated patient versus their own predicted trajectory using their baseline values on the time function test, each of the patients actually has a really quite impressive stabilization of function over their predicted disease trajectory,” he added.

A caveat that SRP-9001 shares with other gene therapies is the issue of cost – reported in the range of $2 million–$3 million.

In the context of racial and socioeconomic disparities in access to diagnosis and care reported in DMD, Emma Ciafaloni, MD, a professor of neurology and pediatrics at the University of Rochester (N.Y.) Medical Center, underscored the need to consider approval versus access to gene therapies and how to optimize access to the novel treatments. 

“We need to consider what the cost is, how it’s going to be accessed, and whether there is a sustainable model,” said Ciafaloni, who was not associated with the study. “There will need to be institutional readiness and support for specialized multidisciplinary clinics for gene therapy.”

She also noted “we need to consider how we can do better on a broader level, because this is not a provider problem or a manufacturer problem — it’s a society problem.”

The study was funded by Sarepta Therapeutics. McDonald reported consulting work for Sarepta Therapeutics and has been an investigator in SRP-9001 research. Ciafaloni reported serving on advisory boards or other relationships with Alexion, Argenx, Biogen, Amicus, Momenta, Medscape, Pfizer, Sanofi/Genzyme, Sarepta, Jansen, NS Pharma, CureSMA, Orphazyme, the Patient-Centered Outcomes Research Institute, PPMD, PTC Therapeutics, and Santhera.

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

Children with obstructive sleep apnea syndrome showed significantly reduced nasal ventilation function, compared with healthy controls, based on data from more than 200 individuals.

Previous research has shown an increased risk of obstructive sleep apnea syndrome (OSAS) in patients with compromised nasal respiration, but the association between increased nasal resistance (NR) and OSAS in children is controversial and remains unclear, wrote Ying Pang, MD, of Children’s Hospital of Chongqing Medical University, China, and colleagues.

In a study published in the Ear, Nose & Throat Journal, the researchers enrolled 109 children aged 6-12 years with OSAS and 116 healthy control children, with the goal of examining the role of nasal ventilation function on OSAS. Participants underwent acoustic rhinometry (AR) following polysomnography, and measurements of the nasal minimal cross-sectional area (NMCA) were taken in 3 segments, as were nasal cavity volume (NCV) from 0 cm to 5 cm, nasopharyngeal volume (NPV) from 6 cm to 8 cm, and distance of the minimal cross-sectional area to the nostril (DCAN). The children also underwent NR testing in both nostrils while awake and lying in a supine position.

Overall, the NR of children with OSAS were significantly higher than that of controls (P < .05). For AR, children with OSAS had significantly lower measures of NMCA, NCV, and NPV, but DCAN values were between the groups. Both AR and NR measures were similar among children with mild, moderate, or severe OSAS.

A subset of 90 children with mild or moderate OSAS were treated with intranasal corticosteroids (ICS) and oral montelukast for 12 weeks. Of these, 69 completed the study and were divided into three groups: effectively cured (group A), successfully treated (group B), and treatment failure (group C). The researchers compared the size of the tonsil adenoids, the polysomnography, NR, and AR before and after treatment and found significant differences in NR, NMCA, and NCV for the A and B groups but no significant changes in DCAN following treatment.

For group A, treatment was associated with a significant reduction in adenoid size and increase in NPV, but these changes did not occur in group B.

The findings were limited by several factors, including the small sample size and measurement of NR when patients were awake and sitting upright, and larger studies are needed to confirm the results, the researchers noted.

However, the results suggest that NVF plays a role in the pathogenesis of OSAS in children and suggest a need to improve NVF in treating these patients they concluded.

This study was supported by the Medical Project of Chongqing Municipal Science and Health Bureau of China. The researchers had no financial conflicts to disclose.

Children with obstructive sleep apnea syndrome showed significantly reduced nasal ventilation function, compared with healthy controls, based on data from more than 200 individuals.

Previous research has shown an increased risk of obstructive sleep apnea syndrome (OSAS) in patients with compromised nasal respiration, but the association between increased nasal resistance (NR) and OSAS in children is controversial and remains unclear, wrote Ying Pang, MD, of Children’s Hospital of Chongqing Medical University, China, and colleagues.

In a study published in the Ear, Nose & Throat Journal, the researchers enrolled 109 children aged 6-12 years with OSAS and 116 healthy control children, with the goal of examining the role of nasal ventilation function on OSAS. Participants underwent acoustic rhinometry (AR) following polysomnography, and measurements of the nasal minimal cross-sectional area (NMCA) were taken in 3 segments, as were nasal cavity volume (NCV) from 0 cm to 5 cm, nasopharyngeal volume (NPV) from 6 cm to 8 cm, and distance of the minimal cross-sectional area to the nostril (DCAN). The children also underwent NR testing in both nostrils while awake and lying in a supine position.

Overall, the NR of children with OSAS were significantly higher than that of controls (P < .05). For AR, children with OSAS had significantly lower measures of NMCA, NCV, and NPV, but DCAN values were between the groups. Both AR and NR measures were similar among children with mild, moderate, or severe OSAS.

A subset of 90 children with mild or moderate OSAS were treated with intranasal corticosteroids (ICS) and oral montelukast for 12 weeks. Of these, 69 completed the study and were divided into three groups: effectively cured (group A), successfully treated (group B), and treatment failure (group C). The researchers compared the size of the tonsil adenoids, the polysomnography, NR, and AR before and after treatment and found significant differences in NR, NMCA, and NCV for the A and B groups but no significant changes in DCAN following treatment.

For group A, treatment was associated with a significant reduction in adenoid size and increase in NPV, but these changes did not occur in group B.

The findings were limited by several factors, including the small sample size and measurement of NR when patients were awake and sitting upright, and larger studies are needed to confirm the results, the researchers noted.

However, the results suggest that NVF plays a role in the pathogenesis of OSAS in children and suggest a need to improve NVF in treating these patients they concluded.

This study was supported by the Medical Project of Chongqing Municipal Science and Health Bureau of China. The researchers had no financial conflicts to disclose.

Children with obstructive sleep apnea syndrome showed significantly reduced nasal ventilation function, compared with healthy controls, based on data from more than 200 individuals.

Previous research has shown an increased risk of obstructive sleep apnea syndrome (OSAS) in patients with compromised nasal respiration, but the association between increased nasal resistance (NR) and OSAS in children is controversial and remains unclear, wrote Ying Pang, MD, of Children’s Hospital of Chongqing Medical University, China, and colleagues.

In a study published in the Ear, Nose & Throat Journal, the researchers enrolled 109 children aged 6-12 years with OSAS and 116 healthy control children, with the goal of examining the role of nasal ventilation function on OSAS. Participants underwent acoustic rhinometry (AR) following polysomnography, and measurements of the nasal minimal cross-sectional area (NMCA) were taken in 3 segments, as were nasal cavity volume (NCV) from 0 cm to 5 cm, nasopharyngeal volume (NPV) from 6 cm to 8 cm, and distance of the minimal cross-sectional area to the nostril (DCAN). The children also underwent NR testing in both nostrils while awake and lying in a supine position.

Overall, the NR of children with OSAS were significantly higher than that of controls (P < .05). For AR, children with OSAS had significantly lower measures of NMCA, NCV, and NPV, but DCAN values were between the groups. Both AR and NR measures were similar among children with mild, moderate, or severe OSAS.

A subset of 90 children with mild or moderate OSAS were treated with intranasal corticosteroids (ICS) and oral montelukast for 12 weeks. Of these, 69 completed the study and were divided into three groups: effectively cured (group A), successfully treated (group B), and treatment failure (group C). The researchers compared the size of the tonsil adenoids, the polysomnography, NR, and AR before and after treatment and found significant differences in NR, NMCA, and NCV for the A and B groups but no significant changes in DCAN following treatment.

For group A, treatment was associated with a significant reduction in adenoid size and increase in NPV, but these changes did not occur in group B.

The findings were limited by several factors, including the small sample size and measurement of NR when patients were awake and sitting upright, and larger studies are needed to confirm the results, the researchers noted.

However, the results suggest that NVF plays a role in the pathogenesis of OSAS in children and suggest a need to improve NVF in treating these patients they concluded.

This study was supported by the Medical Project of Chongqing Municipal Science and Health Bureau of China. The researchers had no financial conflicts to disclose.

The Advisory Committee on Immunization Practices (ACIP) recently issued updated recommendations on the use of vaccines to protect against COVID-19.¹ In addition, 3 new COVID-19 vaccine products have been approved for use in the United States since September. Before we discuss both of these items, it’s important to understand why we’re still talking about COVID-19 vaccines.

The impact of vaccination can’t be understated. Vaccines to protect against COVID-19 have been hugely successful in preventing mortality and morbidity from illness caused by SARS-CoV-2. It is estimated that in the first year alone, after vaccines became widely available, they saved more than 14 million lives globally.² By the end of 2022, they had prevented 18.5 million hospitalizations and 3.2 million deaths in the United States.³ However, waning levels of vaccine-induced immunity and the continuous mutation of the virus have prompted the need for booster doses of vaccine and development of new vaccines.

Enter this year’s vaccines. The new products include updated (2023-2024 formula) COVID-19 mRNA vaccines from Moderna and Pfizer-BioNTech, for use in those ages 6 months and older, and Novavax COVID-19 vaccine for use in those ages 12 years and older. All 3 provide protection against the currently circulating XBB variants, which by September 2023 accounted for > 99% of circulating SARS-CoV-2 strains in the United States.¹

Novavax is an option for those who are hesitant to use an mRNA-based vaccine, although the exact recommendations for its use are still pending. Of note, the previously approved bivalent vaccines and the previous Novavax monovalent vaccine are no longer approved for use in the United States.

Current recommendations. For those ages 5 years and older, the recommendation is for a single dose of the 2023-2024 COVID-19 vaccine regardless of previous vaccination history—except for those who were previously unvaccinated and choose Novavax. (Those individuals should receive 2 doses, 3 to 8 weeks apart.) For those ages 6 months through 4 years, the recommended number of doses varies by vaccine and previous vaccination history¹; a table can be found at www.cdc.gov/mmwr/volumes/72/wr/mm7242e1.htm.

Those who are moderately to severely immunocompromised should receive a 3-dose series with one of the 2023-2024 COVID-19 vaccines and may receive 1 or more additional updated doses.¹ These recommendations are more nuanced, and a full description of them can be found at www.cdc.gov/vaccines/covid-19/clinical-considerations/covid-19-vaccines-us.html.

Major changes in this year’s recommendations,⁴ compared to those previously made on the use of the bivalent vaccines, include:

• Eliminating complex recommendations for 5-year-olds, who are now included in the standard recommendation
• Reducing the number of COVID-19 vaccine products in use by standardizing the dose (25 mcg) for those ages 6 months to 11 years
• Choosing to monitor epidemiology and vaccine effectiveness data to determine whether an additional dose of this year’s vaccine will be needed for those ages 65 years and older, rather than making a recommendation now.

Who’s paying? Another change is how COVID-19 vaccines are paid for. The United States is moving from a system of federal procurement and distribution to the commercial marketplace. This may lead to some disruption and confusion.

All commercial health plans, as well as Medicare and Medicaid, must cover vaccines recommend by the ACIP with no out-of-pocket cost. The Vaccines for Children program provides free vaccine for uninsured and underinsured children up to age 19 years.

However, that leaves no payer for uninsured adults. In response, the CDC has announced the establishment of the Bridge Access Program, which is a private/government partnership to provide the vaccine to this age group. Details about where an adult can obtain a free COVID-19 vaccine through this program can be found by visiting www.cdc.gov/vaccines/programs/bridge/index.html or by calling 800-CDC-INFO.

A dynamic situation. COVID-19 vaccines and associated recommendations are likely to change with time, as we learn how best to formulate them to adjust to virus mutations and determine the optimal intervals to adjust and administer these vaccines. The result may (or may not) eventually resemble the approach recommended for influenza vaccines, which is annual assessment and adjustment of the targeted antigens, when needed, and annual universal vaccination.

The Advisory Committee on Immunization Practices (ACIP) recently issued updated recommendations on the use of vaccines to protect against COVID-19.¹ In addition, 3 new COVID-19 vaccine products have been approved for use in the United States since September. Before we discuss both of these items, it’s important to understand why we’re still talking about COVID-19 vaccines.

The impact of vaccination can’t be understated. Vaccines to protect against COVID-19 have been hugely successful in preventing mortality and morbidity from illness caused by SARS-CoV-2. It is estimated that in the first year alone, after vaccines became widely available, they saved more than 14 million lives globally.² By the end of 2022, they had prevented 18.5 million hospitalizations and 3.2 million deaths in the United States.³ However, waning levels of vaccine-induced immunity and the continuous mutation of the virus have prompted the need for booster doses of vaccine and development of new vaccines.

Enter this year’s vaccines. The new products include updated (2023-2024 formula) COVID-19 mRNA vaccines from Moderna and Pfizer-BioNTech, for use in those ages 6 months and older, and Novavax COVID-19 vaccine for use in those ages 12 years and older. All 3 provide protection against the currently circulating XBB variants, which by September 2023 accounted for > 99% of circulating SARS-CoV-2 strains in the United States.¹

Novavax is an option for those who are hesitant to use an mRNA-based vaccine, although the exact recommendations for its use are still pending. Of note, the previously approved bivalent vaccines and the previous Novavax monovalent vaccine are no longer approved for use in the United States.

Current recommendations. For those ages 5 years and older, the recommendation is for a single dose of the 2023-2024 COVID-19 vaccine regardless of previous vaccination history—except for those who were previously unvaccinated and choose Novavax. (Those individuals should receive 2 doses, 3 to 8 weeks apart.) For those ages 6 months through 4 years, the recommended number of doses varies by vaccine and previous vaccination history¹; a table can be found at www.cdc.gov/mmwr/volumes/72/wr/mm7242e1.htm.

Those who are moderately to severely immunocompromised should receive a 3-dose series with one of the 2023-2024 COVID-19 vaccines and may receive 1 or more additional updated doses.¹ These recommendations are more nuanced, and a full description of them can be found at www.cdc.gov/vaccines/covid-19/clinical-considerations/covid-19-vaccines-us.html.

Major changes in this year’s recommendations,⁴ compared to those previously made on the use of the bivalent vaccines, include:

• Eliminating complex recommendations for 5-year-olds, who are now included in the standard recommendation
• Reducing the number of COVID-19 vaccine products in use by standardizing the dose (25 mcg) for those ages 6 months to 11 years
• Choosing to monitor epidemiology and vaccine effectiveness data to determine whether an additional dose of this year’s vaccine will be needed for those ages 65 years and older, rather than making a recommendation now.

Who’s paying? Another change is how COVID-19 vaccines are paid for. The United States is moving from a system of federal procurement and distribution to the commercial marketplace. This may lead to some disruption and confusion.

All commercial health plans, as well as Medicare and Medicaid, must cover vaccines recommend by the ACIP with no out-of-pocket cost. The Vaccines for Children program provides free vaccine for uninsured and underinsured children up to age 19 years.

However, that leaves no payer for uninsured adults. In response, the CDC has announced the establishment of the Bridge Access Program, which is a private/government partnership to provide the vaccine to this age group. Details about where an adult can obtain a free COVID-19 vaccine through this program can be found by visiting www.cdc.gov/vaccines/programs/bridge/index.html or by calling 800-CDC-INFO.

A dynamic situation. COVID-19 vaccines and associated recommendations are likely to change with time, as we learn how best to formulate them to adjust to virus mutations and determine the optimal intervals to adjust and administer these vaccines. The result may (or may not) eventually resemble the approach recommended for influenza vaccines, which is annual assessment and adjustment of the targeted antigens, when needed, and annual universal vaccination.

The Advisory Committee on Immunization Practices (ACIP) recently issued updated recommendations on the use of vaccines to protect against COVID-19.¹ In addition, 3 new COVID-19 vaccine products have been approved for use in the United States since September. Before we discuss both of these items, it’s important to understand why we’re still talking about COVID-19 vaccines.

The impact of vaccination can’t be understated. Vaccines to protect against COVID-19 have been hugely successful in preventing mortality and morbidity from illness caused by SARS-CoV-2. It is estimated that in the first year alone, after vaccines became widely available, they saved more than 14 million lives globally.² By the end of 2022, they had prevented 18.5 million hospitalizations and 3.2 million deaths in the United States.³ However, waning levels of vaccine-induced immunity and the continuous mutation of the virus have prompted the need for booster doses of vaccine and development of new vaccines.

Enter this year’s vaccines. The new products include updated (2023-2024 formula) COVID-19 mRNA vaccines from Moderna and Pfizer-BioNTech, for use in those ages 6 months and older, and Novavax COVID-19 vaccine for use in those ages 12 years and older. All 3 provide protection against the currently circulating XBB variants, which by September 2023 accounted for > 99% of circulating SARS-CoV-2 strains in the United States.¹

Novavax is an option for those who are hesitant to use an mRNA-based vaccine, although the exact recommendations for its use are still pending. Of note, the previously approved bivalent vaccines and the previous Novavax monovalent vaccine are no longer approved for use in the United States.

Current recommendations. For those ages 5 years and older, the recommendation is for a single dose of the 2023-2024 COVID-19 vaccine regardless of previous vaccination history—except for those who were previously unvaccinated and choose Novavax. (Those individuals should receive 2 doses, 3 to 8 weeks apart.) For those ages 6 months through 4 years, the recommended number of doses varies by vaccine and previous vaccination history¹; a table can be found at www.cdc.gov/mmwr/volumes/72/wr/mm7242e1.htm.

Those who are moderately to severely immunocompromised should receive a 3-dose series with one of the 2023-2024 COVID-19 vaccines and may receive 1 or more additional updated doses.¹ These recommendations are more nuanced, and a full description of them can be found at www.cdc.gov/vaccines/covid-19/clinical-considerations/covid-19-vaccines-us.html.

Major changes in this year’s recommendations,⁴ compared to those previously made on the use of the bivalent vaccines, include:

• Eliminating complex recommendations for 5-year-olds, who are now included in the standard recommendation
• Reducing the number of COVID-19 vaccine products in use by standardizing the dose (25 mcg) for those ages 6 months to 11 years
• Choosing to monitor epidemiology and vaccine effectiveness data to determine whether an additional dose of this year’s vaccine will be needed for those ages 65 years and older, rather than making a recommendation now.

Who’s paying? Another change is how COVID-19 vaccines are paid for. The United States is moving from a system of federal procurement and distribution to the commercial marketplace. This may lead to some disruption and confusion.

All commercial health plans, as well as Medicare and Medicaid, must cover vaccines recommend by the ACIP with no out-of-pocket cost. The Vaccines for Children program provides free vaccine for uninsured and underinsured children up to age 19 years.

However, that leaves no payer for uninsured adults. In response, the CDC has announced the establishment of the Bridge Access Program, which is a private/government partnership to provide the vaccine to this age group. Details about where an adult can obtain a free COVID-19 vaccine through this program can be found by visiting www.cdc.gov/vaccines/programs/bridge/index.html or by calling 800-CDC-INFO.

A dynamic situation. COVID-19 vaccines and associated recommendations are likely to change with time, as we learn how best to formulate them to adjust to virus mutations and determine the optimal intervals to adjust and administer these vaccines. The result may (or may not) eventually resemble the approach recommended for influenza vaccines, which is annual assessment and adjustment of the targeted antigens, when needed, and annual universal vaccination.

I read a few articles recently that raised my concern about a laissez faire attitude regarding treatment and prevention of infectious disease and lack of a broader understanding of why we treat our patients.
 

Strep throat

Let’s start with group A streptococcal pharyngitis – strep throat. There are at least five reasons to treat strep throat with antibiotics.

Lest we forget, there is the prevention of acute rheumatic fever! Of course, acute rheumatic fever is rare in high-income countries like the United States, but we have had outbreaks in the past and we will have outbreaks in the future. All it takes is circulation of rheumatogenic strains and susceptible hosts.

Also, antibiotic treatment may prevent acute post-streptococcal glomerulonephritis, although that benefit is somewhat controversial.

Antibiotic treatment may prevent development of another controversial, nonsuppurative streptococcal complication, namely, pediatric autoimmune neuropsychiatric disorders associated with streptococcal infections (PANDAS).

Second, group A strep causes suppurative complications such as acute otitis media, peritonsillar abscess, mastoiditis, and sepsis, among others, and antibiotic treatment reduces those risks. Group A strep can cause impetigo, cellulitis, necrotizing fasciitis (flesh-eating disease), and toxic shock syndrome; antibiotics reduce those risks.

Third, while strep throat is a self-limited infection in terms of symptoms, it has been clearly shown that antibiotics cause symptoms to resolve more quickly. I must confess that it galls me when pundits suggest that reducing symptoms of any infectious disease by a day or 2 doesn’t matter for children, when adults with even mild symptoms rush to a clinician with hopes of treatment to shorten illness by a day.

Fourth, antibiotics shorten contagion. In fact, treatment in the morning of an office visit can allow a child to return to school the next day.¹

Lastly on this topic, if a clinician had a positive strep culture or rapid test on a patient and did not treat with antibiotics, which is not the standard of care, and that patient went on to a nonsuppurative or suppurative complication, then what?

I am not advocating wholesale antibiotic treatment of all sore throats because antibiotics carry risks from use. Most sore throats are not strep throats. The first step is the examination to decide if a strep test is warranted. There are clinical scoring systems available. But the essence of the clinical criteria relies on age of child (strep is mostly seen in 5- to 15-year-olds), season (not summer), known exposure to strep, absence of rhinorrhea, absence of cough, presence of rapid onset of symptoms, usually with fever, and moderate to severe redness, often with exudates. Gratefully, in the United States, we have rapid strep tests that are covered by insurance. This is not the case even in many other high-income countries and certainly, generally, not available at all in moderate to low income countries. With a rapid test, a point-of-care microbiologic diagnosis can be made with reasonable accuracy. Antibiotic treatment should be reserved for patients with positive laboratory confirmation of Group A streptococci, either by rapid test or culture.
 

Ear infections

Next, let’s address treatment of acute otitis media – ear infections. There are at least six reasons to treat ear infections with antibiotics. Worldwide, the No. 1 cause of acquired deafness in children today is ear infections. This is rarely seen in the United States because we rarely have patients with chronic suppurative otitis media since antibiotics are typically prescribed.

Second, ear infections have suppurative complications such as mastoiditis, labyrinthitis, malignant otitis, brain abscess, sepsis, and meningitis. The World Health Organization attributes 20,000 deaths per year to complications from ear infections.

Third, ear infections can lead to eardrum rupture and subsequent chronic middle ear drainage.

Fourth, untreated otitis more often progresses to a nonsuppurative complication – a cholesteatoma.

Fifth, while earache is a self-limited illness, antibiotics shorten the acute symptoms by a day or 2 and lessen the duration of middle ear effusion after infection that can cause temporary hearing loss. Once again, as a child advocate, I would point out that pain from an ear infection is often severe and the lingering effects of a middle ear effusion are annoying to say the least.

Lastly on this topic, if a clinician makes the diagnosis of an ear infection in a patient and does not treat with antibiotics, the decision should be within the guidelines of the standard of care as described by the American Academy of Pediatrics² with decision-making based on patient age and severity of symptoms.

I am not advocating wholesale antibiotic treatment of all ear pain or presumed ear pain. With this clinical condition we currently do not have a diagnostic test, and therein lies the conundrum. Most acute otitis media occurs among children age 6-24 months old, and this leads most clinicians to overdiagnose the infection. A child in that age group is nonverbal and in the context of a viral upper respiratory illness the symptoms of acute otitis media overlap completely with those of a viral URI. Therefore, an adequate examination is necessary. Confronted with an irritable child who is uncooperative with a challenging otoscopic examination, an ear canal with wax blocking an adequate view of the tympanic membrane, and a parent in a hurry to get back to work or home, the inclination is to observe a “little bit of redness” and prescribe unnecessary antibiotics. Even though redness is not a good diagnostic indicator, whereas a full or bulging eardrum is for the diagnosis of acute otitis media, I shudder at how often I see in a medical record a description of redness of the eardrum and no comment on the fullness that occurs when an authentic infection is most likely.

I could extend this column discussing acute sinusitis and cough illnesses as they are two other conditions associated with infection where antibiotics have their important place and where antibiotics are also overused. Instead, I will end by summarizing my viewpoint that judicious antibiotic use is of high importance for prevention of antibiotic resistance at the individual patient level and the community level. However, we should not become complacent about the risks to untreated children experiencing common respiratory infections because there are many justifiable reasons to treat children as discussed here.

Dr. Pichichero is a specialist in pediatric infectious diseases, Center for Infectious Diseases and Immunology, and director of the Research Institute at Rochester (N.Y.) General Hospital. He has no conflicts of interest to disclose.

References

1. Schwartz RH et al. A reappraisal of the minimum duration of antibiotic treatment before approval of return to school for children with streptococcal pharyngitis. Pediatr Infect Dis J. 2015 Dec. doi: 10.1097/INF.0000000000000883.

2. Lieberthal AS et al. The diagnosis and management of acute otitis media. Pediatrics. 2013 Mar. doi: 10.1542/peds.2012-3488.

I read a few articles recently that raised my concern about a laissez faire attitude regarding treatment and prevention of infectious disease and lack of a broader understanding of why we treat our patients.
 

Strep throat

Let’s start with group A streptococcal pharyngitis – strep throat. There are at least five reasons to treat strep throat with antibiotics.

Lest we forget, there is the prevention of acute rheumatic fever! Of course, acute rheumatic fever is rare in high-income countries like the United States, but we have had outbreaks in the past and we will have outbreaks in the future. All it takes is circulation of rheumatogenic strains and susceptible hosts.

Also, antibiotic treatment may prevent acute post-streptococcal glomerulonephritis, although that benefit is somewhat controversial.

Antibiotic treatment may prevent development of another controversial, nonsuppurative streptococcal complication, namely, pediatric autoimmune neuropsychiatric disorders associated with streptococcal infections (PANDAS).

Second, group A strep causes suppurative complications such as acute otitis media, peritonsillar abscess, mastoiditis, and sepsis, among others, and antibiotic treatment reduces those risks. Group A strep can cause impetigo, cellulitis, necrotizing fasciitis (flesh-eating disease), and toxic shock syndrome; antibiotics reduce those risks.

Third, while strep throat is a self-limited infection in terms of symptoms, it has been clearly shown that antibiotics cause symptoms to resolve more quickly. I must confess that it galls me when pundits suggest that reducing symptoms of any infectious disease by a day or 2 doesn’t matter for children, when adults with even mild symptoms rush to a clinician with hopes of treatment to shorten illness by a day.

Fourth, antibiotics shorten contagion. In fact, treatment in the morning of an office visit can allow a child to return to school the next day.¹

Lastly on this topic, if a clinician had a positive strep culture or rapid test on a patient and did not treat with antibiotics, which is not the standard of care, and that patient went on to a nonsuppurative or suppurative complication, then what?

I am not advocating wholesale antibiotic treatment of all sore throats because antibiotics carry risks from use. Most sore throats are not strep throats. The first step is the examination to decide if a strep test is warranted. There are clinical scoring systems available. But the essence of the clinical criteria relies on age of child (strep is mostly seen in 5- to 15-year-olds), season (not summer), known exposure to strep, absence of rhinorrhea, absence of cough, presence of rapid onset of symptoms, usually with fever, and moderate to severe redness, often with exudates. Gratefully, in the United States, we have rapid strep tests that are covered by insurance. This is not the case even in many other high-income countries and certainly, generally, not available at all in moderate to low income countries. With a rapid test, a point-of-care microbiologic diagnosis can be made with reasonable accuracy. Antibiotic treatment should be reserved for patients with positive laboratory confirmation of Group A streptococci, either by rapid test or culture.
 

Ear infections

Next, let’s address treatment of acute otitis media – ear infections. There are at least six reasons to treat ear infections with antibiotics. Worldwide, the No. 1 cause of acquired deafness in children today is ear infections. This is rarely seen in the United States because we rarely have patients with chronic suppurative otitis media since antibiotics are typically prescribed.

Second, ear infections have suppurative complications such as mastoiditis, labyrinthitis, malignant otitis, brain abscess, sepsis, and meningitis. The World Health Organization attributes 20,000 deaths per year to complications from ear infections.

Third, ear infections can lead to eardrum rupture and subsequent chronic middle ear drainage.

Fourth, untreated otitis more often progresses to a nonsuppurative complication – a cholesteatoma.

Fifth, while earache is a self-limited illness, antibiotics shorten the acute symptoms by a day or 2 and lessen the duration of middle ear effusion after infection that can cause temporary hearing loss. Once again, as a child advocate, I would point out that pain from an ear infection is often severe and the lingering effects of a middle ear effusion are annoying to say the least.

Lastly on this topic, if a clinician makes the diagnosis of an ear infection in a patient and does not treat with antibiotics, the decision should be within the guidelines of the standard of care as described by the American Academy of Pediatrics² with decision-making based on patient age and severity of symptoms.

I am not advocating wholesale antibiotic treatment of all ear pain or presumed ear pain. With this clinical condition we currently do not have a diagnostic test, and therein lies the conundrum. Most acute otitis media occurs among children age 6-24 months old, and this leads most clinicians to overdiagnose the infection. A child in that age group is nonverbal and in the context of a viral upper respiratory illness the symptoms of acute otitis media overlap completely with those of a viral URI. Therefore, an adequate examination is necessary. Confronted with an irritable child who is uncooperative with a challenging otoscopic examination, an ear canal with wax blocking an adequate view of the tympanic membrane, and a parent in a hurry to get back to work or home, the inclination is to observe a “little bit of redness” and prescribe unnecessary antibiotics. Even though redness is not a good diagnostic indicator, whereas a full or bulging eardrum is for the diagnosis of acute otitis media, I shudder at how often I see in a medical record a description of redness of the eardrum and no comment on the fullness that occurs when an authentic infection is most likely.

I could extend this column discussing acute sinusitis and cough illnesses as they are two other conditions associated with infection where antibiotics have their important place and where antibiotics are also overused. Instead, I will end by summarizing my viewpoint that judicious antibiotic use is of high importance for prevention of antibiotic resistance at the individual patient level and the community level. However, we should not become complacent about the risks to untreated children experiencing common respiratory infections because there are many justifiable reasons to treat children as discussed here.

Dr. Pichichero is a specialist in pediatric infectious diseases, Center for Infectious Diseases and Immunology, and director of the Research Institute at Rochester (N.Y.) General Hospital. He has no conflicts of interest to disclose.

References

1. Schwartz RH et al. A reappraisal of the minimum duration of antibiotic treatment before approval of return to school for children with streptococcal pharyngitis. Pediatr Infect Dis J. 2015 Dec. doi: 10.1097/INF.0000000000000883.

2. Lieberthal AS et al. The diagnosis and management of acute otitis media. Pediatrics. 2013 Mar. doi: 10.1542/peds.2012-3488.

I read a few articles recently that raised my concern about a laissez faire attitude regarding treatment and prevention of infectious disease and lack of a broader understanding of why we treat our patients.
 

Strep throat

Let’s start with group A streptococcal pharyngitis – strep throat. There are at least five reasons to treat strep throat with antibiotics.

Lest we forget, there is the prevention of acute rheumatic fever! Of course, acute rheumatic fever is rare in high-income countries like the United States, but we have had outbreaks in the past and we will have outbreaks in the future. All it takes is circulation of rheumatogenic strains and susceptible hosts.

Also, antibiotic treatment may prevent acute post-streptococcal glomerulonephritis, although that benefit is somewhat controversial.

Antibiotic treatment may prevent development of another controversial, nonsuppurative streptococcal complication, namely, pediatric autoimmune neuropsychiatric disorders associated with streptococcal infections (PANDAS).

Second, group A strep causes suppurative complications such as acute otitis media, peritonsillar abscess, mastoiditis, and sepsis, among others, and antibiotic treatment reduces those risks. Group A strep can cause impetigo, cellulitis, necrotizing fasciitis (flesh-eating disease), and toxic shock syndrome; antibiotics reduce those risks.

Third, while strep throat is a self-limited infection in terms of symptoms, it has been clearly shown that antibiotics cause symptoms to resolve more quickly. I must confess that it galls me when pundits suggest that reducing symptoms of any infectious disease by a day or 2 doesn’t matter for children, when adults with even mild symptoms rush to a clinician with hopes of treatment to shorten illness by a day.

Fourth, antibiotics shorten contagion. In fact, treatment in the morning of an office visit can allow a child to return to school the next day.¹

Lastly on this topic, if a clinician had a positive strep culture or rapid test on a patient and did not treat with antibiotics, which is not the standard of care, and that patient went on to a nonsuppurative or suppurative complication, then what?

I am not advocating wholesale antibiotic treatment of all sore throats because antibiotics carry risks from use. Most sore throats are not strep throats. The first step is the examination to decide if a strep test is warranted. There are clinical scoring systems available. But the essence of the clinical criteria relies on age of child (strep is mostly seen in 5- to 15-year-olds), season (not summer), known exposure to strep, absence of rhinorrhea, absence of cough, presence of rapid onset of symptoms, usually with fever, and moderate to severe redness, often with exudates. Gratefully, in the United States, we have rapid strep tests that are covered by insurance. This is not the case even in many other high-income countries and certainly, generally, not available at all in moderate to low income countries. With a rapid test, a point-of-care microbiologic diagnosis can be made with reasonable accuracy. Antibiotic treatment should be reserved for patients with positive laboratory confirmation of Group A streptococci, either by rapid test or culture.
 

Ear infections

Next, let’s address treatment of acute otitis media – ear infections. There are at least six reasons to treat ear infections with antibiotics. Worldwide, the No. 1 cause of acquired deafness in children today is ear infections. This is rarely seen in the United States because we rarely have patients with chronic suppurative otitis media since antibiotics are typically prescribed.

Second, ear infections have suppurative complications such as mastoiditis, labyrinthitis, malignant otitis, brain abscess, sepsis, and meningitis. The World Health Organization attributes 20,000 deaths per year to complications from ear infections.

Third, ear infections can lead to eardrum rupture and subsequent chronic middle ear drainage.

Fourth, untreated otitis more often progresses to a nonsuppurative complication – a cholesteatoma.

Fifth, while earache is a self-limited illness, antibiotics shorten the acute symptoms by a day or 2 and lessen the duration of middle ear effusion after infection that can cause temporary hearing loss. Once again, as a child advocate, I would point out that pain from an ear infection is often severe and the lingering effects of a middle ear effusion are annoying to say the least.

Lastly on this topic, if a clinician makes the diagnosis of an ear infection in a patient and does not treat with antibiotics, the decision should be within the guidelines of the standard of care as described by the American Academy of Pediatrics² with decision-making based on patient age and severity of symptoms.

I am not advocating wholesale antibiotic treatment of all ear pain or presumed ear pain. With this clinical condition we currently do not have a diagnostic test, and therein lies the conundrum. Most acute otitis media occurs among children age 6-24 months old, and this leads most clinicians to overdiagnose the infection. A child in that age group is nonverbal and in the context of a viral upper respiratory illness the symptoms of acute otitis media overlap completely with those of a viral URI. Therefore, an adequate examination is necessary. Confronted with an irritable child who is uncooperative with a challenging otoscopic examination, an ear canal with wax blocking an adequate view of the tympanic membrane, and a parent in a hurry to get back to work or home, the inclination is to observe a “little bit of redness” and prescribe unnecessary antibiotics. Even though redness is not a good diagnostic indicator, whereas a full or bulging eardrum is for the diagnosis of acute otitis media, I shudder at how often I see in a medical record a description of redness of the eardrum and no comment on the fullness that occurs when an authentic infection is most likely.

I could extend this column discussing acute sinusitis and cough illnesses as they are two other conditions associated with infection where antibiotics have their important place and where antibiotics are also overused. Instead, I will end by summarizing my viewpoint that judicious antibiotic use is of high importance for prevention of antibiotic resistance at the individual patient level and the community level. However, we should not become complacent about the risks to untreated children experiencing common respiratory infections because there are many justifiable reasons to treat children as discussed here.

Dr. Pichichero is a specialist in pediatric infectious diseases, Center for Infectious Diseases and Immunology, and director of the Research Institute at Rochester (N.Y.) General Hospital. He has no conflicts of interest to disclose.

References

1. Schwartz RH et al. A reappraisal of the minimum duration of antibiotic treatment before approval of return to school for children with streptococcal pharyngitis. Pediatr Infect Dis J. 2015 Dec. doi: 10.1097/INF.0000000000000883.

2. Lieberthal AS et al. The diagnosis and management of acute otitis media. Pediatrics. 2013 Mar. doi: 10.1542/peds.2012-3488.

All infants and children perinatally exposed to the hepatitis C virus (HCV) should be tested and, if necessary, treated, according to new guidance from the Centers for Disease Control and Prevention.

In utero–exposed infants should be tested at 2-6 months of life, much earlier than the current strategy of testing at 18 months.

HCV infection, which can lead to liver fibrosis and cirrhosis, liver failure, hepatic cancer, and transplant, will develop in 6%-7% of all perinatally exposed infants and children. Curative therapy with direct-acting antivirals can be administered starting at age 3, the CDC noted in Morbidity and Mortality Week Report (MMWR).

About 70% of children 18 months and older are not being tested with the current strategy of anti-HCV testing.

This current MMWR report supplements the 2020 CDC recommendations for adult HCV screening, which includes universal screening among pregnant persons during each pregnancy.
 

The new recommendations

• Perinatally exposed infants should receive a nucleic acid amplification test for HCV RNA at 2-6 months of age to identify those who might develop chronic HCV infection if not treated.
• Those with detectable HCV RNA should be managed in consultation with an expert in pediatric HCV.
• Infants with undetectable HCV RNA do not require further follow-up unless clinically warranted.

“Testing perinatally exposed infants beginning at age 2 months with a NAT for HCV RNA is cost-effective and allows for earlier linkage to care, appropriate evaluation, and the opportunity to provide curative, life-saving therapy,” the MMWR report said.
 

A growing problem

The CDC noted that rates of HCV infections during pregnancy are on the rise, corresponding with the ongoing opioid crisis and intravenous drug use.

Yet most perinatally exposed children are not tested for HCV infection and are not referred for hepatitis C care. Reasons might include lack of awareness of perinatal exposure by pediatric providers, lack of regular pediatric care among exposed children, and switching of health care providers before the former recommended testing age of 18 months.

The CDC’s testing recommendation is welcome news to Dawnette A. Lewis, MD, a maternal fetal medicine specialist at Northwell Health in New Hyde Park, N.Y. “As opposed to data for hep B and HIV, we have traditionally had little information and experience regarding the transmission and impact of hep C in pregnant women and their babies. We’ve been having that conversation about the lack of information for some time, and now there’s an opportunity to get evolving data on hep C and how it affects the baby, ” she said.

Northwell Health

Northwestern Medicine

Northwestern Medicine

All infants and children perinatally exposed to the hepatitis C virus (HCV) should be tested and, if necessary, treated, according to new guidance from the Centers for Disease Control and Prevention.

In utero–exposed infants should be tested at 2-6 months of life, much earlier than the current strategy of testing at 18 months.

HCV infection, which can lead to liver fibrosis and cirrhosis, liver failure, hepatic cancer, and transplant, will develop in 6%-7% of all perinatally exposed infants and children. Curative therapy with direct-acting antivirals can be administered starting at age 3, the CDC noted in Morbidity and Mortality Week Report (MMWR).

About 70% of children 18 months and older are not being tested with the current strategy of anti-HCV testing.

This current MMWR report supplements the 2020 CDC recommendations for adult HCV screening, which includes universal screening among pregnant persons during each pregnancy.
 

The new recommendations

• Perinatally exposed infants should receive a nucleic acid amplification test for HCV RNA at 2-6 months of age to identify those who might develop chronic HCV infection if not treated.
• Those with detectable HCV RNA should be managed in consultation with an expert in pediatric HCV.
• Infants with undetectable HCV RNA do not require further follow-up unless clinically warranted.

“Testing perinatally exposed infants beginning at age 2 months with a NAT for HCV RNA is cost-effective and allows for earlier linkage to care, appropriate evaluation, and the opportunity to provide curative, life-saving therapy,” the MMWR report said.
 

A growing problem

The CDC noted that rates of HCV infections during pregnancy are on the rise, corresponding with the ongoing opioid crisis and intravenous drug use.

Yet most perinatally exposed children are not tested for HCV infection and are not referred for hepatitis C care. Reasons might include lack of awareness of perinatal exposure by pediatric providers, lack of regular pediatric care among exposed children, and switching of health care providers before the former recommended testing age of 18 months.

The CDC’s testing recommendation is welcome news to Dawnette A. Lewis, MD, a maternal fetal medicine specialist at Northwell Health in New Hyde Park, N.Y. “As opposed to data for hep B and HIV, we have traditionally had little information and experience regarding the transmission and impact of hep C in pregnant women and their babies. We’ve been having that conversation about the lack of information for some time, and now there’s an opportunity to get evolving data on hep C and how it affects the baby, ” she said.

Northwell Health

Northwestern Medicine

Northwestern Medicine

All infants and children perinatally exposed to the hepatitis C virus (HCV) should be tested and, if necessary, treated, according to new guidance from the Centers for Disease Control and Prevention.

In utero–exposed infants should be tested at 2-6 months of life, much earlier than the current strategy of testing at 18 months.

HCV infection, which can lead to liver fibrosis and cirrhosis, liver failure, hepatic cancer, and transplant, will develop in 6%-7% of all perinatally exposed infants and children. Curative therapy with direct-acting antivirals can be administered starting at age 3, the CDC noted in Morbidity and Mortality Week Report (MMWR).

About 70% of children 18 months and older are not being tested with the current strategy of anti-HCV testing.

This current MMWR report supplements the 2020 CDC recommendations for adult HCV screening, which includes universal screening among pregnant persons during each pregnancy.
 

The new recommendations

• Perinatally exposed infants should receive a nucleic acid amplification test for HCV RNA at 2-6 months of age to identify those who might develop chronic HCV infection if not treated.
• Those with detectable HCV RNA should be managed in consultation with an expert in pediatric HCV.
• Infants with undetectable HCV RNA do not require further follow-up unless clinically warranted.

“Testing perinatally exposed infants beginning at age 2 months with a NAT for HCV RNA is cost-effective and allows for earlier linkage to care, appropriate evaluation, and the opportunity to provide curative, life-saving therapy,” the MMWR report said.
 

A growing problem

The CDC noted that rates of HCV infections during pregnancy are on the rise, corresponding with the ongoing opioid crisis and intravenous drug use.

Yet most perinatally exposed children are not tested for HCV infection and are not referred for hepatitis C care. Reasons might include lack of awareness of perinatal exposure by pediatric providers, lack of regular pediatric care among exposed children, and switching of health care providers before the former recommended testing age of 18 months.

The CDC’s testing recommendation is welcome news to Dawnette A. Lewis, MD, a maternal fetal medicine specialist at Northwell Health in New Hyde Park, N.Y. “As opposed to data for hep B and HIV, we have traditionally had little information and experience regarding the transmission and impact of hep C in pregnant women and their babies. We’ve been having that conversation about the lack of information for some time, and now there’s an opportunity to get evolving data on hep C and how it affects the baby, ” she said.

Northwell Health

Northwestern Medicine

Northwestern Medicine

Pulmonologist Evan Stepp, MD, FCCP, has a teenage daughter who doesn’t smoke or vape – as far as he knows, Stepp will admit – but the statistics on youth smoking are alarming enough to have him worried.

On one hand, fewer Americans are smoking today than ever before. Since 1992, the percentage of people who told Gallup that they’d had a cigarette in the past week has dropped from 28% to 11%. Meanwhile, the rate of new lung cancer cases declined from 65 per every 100,000 people in 1992 to 34 per 100,000 in 2020, according to the National Cancer Institute.

While those statistics are worth celebrating, they hide an alarming reality: A disproportionate number of teens and young adults today are addicted to nicotine.

According to a November 2022 report from the Centers for Disease Control and Prevention (CDC), 1 in 6 high school students and 1 in 20 middle schoolers are using a nicotine product at least once every day.

“It’s a completely different picture for nicotine cessation in youth,” Dr. Stepp, who is an associate professor at National Jewish Health in Denver, said. “Because of the fact that the nicotine addiction is occurring in a developing brain, which raises many other nicotine-related harms.”
 

Why teens vape

Today’s teens are smoking less actual tobacco, and, instead, overwhelmingly prefer e-cigarettes or vaping. In fact, 85% of high school–aged smokers and 72% of middle school smokers reach for a vape over regular cigarettes or smokeless tobacco, according to the CDC.

It’s not hard to understand why: e-cigarettes use a heating element to turn a nicotine-infused liquid into an aerosol, with no open flame, ash, or lingering smoke. The vapes themselves are easy to conceal, and if someone needed to hide an e-cigarette from particularly perceptive parents or teachers, they can find vapes built into hoodies, fake smartwatches, and USB drives.

Plus, the liquids often come in flavors like fruit, bubble gum, mint, and vanilla, because unflavored nicotine isn’t exactly appealing. “Huge concentrations of nicotine salts are just miserable to breathe in,” Dr. Stepp said. “Flavors are necessary to make these products palatable, and those flavors end up being a huge draw for youth users to get exposed to nicotine addiction.”
 

Challenges surrounding smoking cessation in youth

The powerful effect of nicotine in youth means the need for effective cessation strategies is both more urgent and more difficult. But while physicians can prescribe to adults the antidepressants varenicline and bupropion, along with nicotine replacement therapy, to help ease withdrawal symptoms, the US Food and Drug Administration (FDA) has not approved those medications for anyone under the age of 18.

Research on cessation medications in young people is limited: A recent meta-analysis found only four studies on people between the ages of 12 and 21. In teens, antidepressants seem to help quitting for the first few weeks but are unproven as a long-term solution.

“That really has been a challenge for the 1 in 6 high school students who are current users of tobacco products,” said pediatrician, Susan Walley, MD, a co-author of the American Academy of Pediatrics’ recent position papers on children and smoking.

“One of the things that is important to keep at the forefront of the conversation is that nicotine addiction is a chronic medical disease, and it’s a form of substance abuse,” Dr. Walley said. “We know that we need more research in adolescent tobacco cessation, and it really is about the funding, about research dollars.”

Without medications, smoking cessation in teens relies largely on counseling strategies. A 2017 review published by Cochrane Library found that group counseling was the most effective quitting method, with teens participating in group sessions 35% more likely to stop using nicotine products up to a year later, compared with teens who did not receive any counseling.

Counseling can help educate teens (and parents) on some of the realities of e-cigarettes, bridging the gap between well-established anti-smoking campaigns and the anti-vape campaigns that have yet to catch up.

“We have done a great job promoting cigarette use as dangerous,” Dr. Walley said. “[But] many teens who would never pick up a cigarette –because they know the health risks – are vaping.”
 

How to get a teen to quit

Cessation and prevention strategies are closely linked, and interventions can start in middle school-aged children up through high school and young adults. Simply asking a 12-year-old, “Do you know anyone who smokes?” can help start a conversation that leads to an attempt to quit.

Teens may be compelled to smoke through digital advertising and influencer endorsements on social media platforms, but Gen Z is turned off by the idea that it’s being manipulated by the tobacco industry. Juul, for example, is partially owned by Altria, which makes Marlboros, and Vuse is wholly owned by R.J. Reynolds, which makes Camel cigarettes.

“If you can get somebody to understand that Big Tobacco is trying to manipulate you as a young person to want to illegally obtain and use their products, which are incredibly addictive, thus ensuring you will remain a loyal customer, that could be the thing that pushes them over the hump,” Dr. Stepp said. “You push it away like you would push away a parent trying to tell you how to park a car in the driveway.”

And just because a smoker relapses, it doesn’t mean the cessation was a complete failure. The younger someone is when they stop smoking, the less likely they are to suffer from the long-term health consequences of smoking, according to a 2021 study in the Journal of the American Medical Association. “With the right counseling,” Dr. Walley said, “each relapse is an opportunity for losing the habit permanently.”

This article was adapted from the Summer 2023 online issue of CHEST Advocates. For the full article – and to engage with the other content from this issue – visit https://chestnet.org/chest-­advocates.

Pulmonologist Evan Stepp, MD, FCCP, has a teenage daughter who doesn’t smoke or vape – as far as he knows, Stepp will admit – but the statistics on youth smoking are alarming enough to have him worried.

On one hand, fewer Americans are smoking today than ever before. Since 1992, the percentage of people who told Gallup that they’d had a cigarette in the past week has dropped from 28% to 11%. Meanwhile, the rate of new lung cancer cases declined from 65 per every 100,000 people in 1992 to 34 per 100,000 in 2020, according to the National Cancer Institute.

While those statistics are worth celebrating, they hide an alarming reality: A disproportionate number of teens and young adults today are addicted to nicotine.

According to a November 2022 report from the Centers for Disease Control and Prevention (CDC), 1 in 6 high school students and 1 in 20 middle schoolers are using a nicotine product at least once every day.

“It’s a completely different picture for nicotine cessation in youth,” Dr. Stepp, who is an associate professor at National Jewish Health in Denver, said. “Because of the fact that the nicotine addiction is occurring in a developing brain, which raises many other nicotine-related harms.”
 

Why teens vape

Today’s teens are smoking less actual tobacco, and, instead, overwhelmingly prefer e-cigarettes or vaping. In fact, 85% of high school–aged smokers and 72% of middle school smokers reach for a vape over regular cigarettes or smokeless tobacco, according to the CDC.

It’s not hard to understand why: e-cigarettes use a heating element to turn a nicotine-infused liquid into an aerosol, with no open flame, ash, or lingering smoke. The vapes themselves are easy to conceal, and if someone needed to hide an e-cigarette from particularly perceptive parents or teachers, they can find vapes built into hoodies, fake smartwatches, and USB drives.

Plus, the liquids often come in flavors like fruit, bubble gum, mint, and vanilla, because unflavored nicotine isn’t exactly appealing. “Huge concentrations of nicotine salts are just miserable to breathe in,” Dr. Stepp said. “Flavors are necessary to make these products palatable, and those flavors end up being a huge draw for youth users to get exposed to nicotine addiction.”
 

Challenges surrounding smoking cessation in youth

The powerful effect of nicotine in youth means the need for effective cessation strategies is both more urgent and more difficult. But while physicians can prescribe to adults the antidepressants varenicline and bupropion, along with nicotine replacement therapy, to help ease withdrawal symptoms, the US Food and Drug Administration (FDA) has not approved those medications for anyone under the age of 18.

Research on cessation medications in young people is limited: A recent meta-analysis found only four studies on people between the ages of 12 and 21. In teens, antidepressants seem to help quitting for the first few weeks but are unproven as a long-term solution.

“That really has been a challenge for the 1 in 6 high school students who are current users of tobacco products,” said pediatrician, Susan Walley, MD, a co-author of the American Academy of Pediatrics’ recent position papers on children and smoking.

“One of the things that is important to keep at the forefront of the conversation is that nicotine addiction is a chronic medical disease, and it’s a form of substance abuse,” Dr. Walley said. “We know that we need more research in adolescent tobacco cessation, and it really is about the funding, about research dollars.”

Without medications, smoking cessation in teens relies largely on counseling strategies. A 2017 review published by Cochrane Library found that group counseling was the most effective quitting method, with teens participating in group sessions 35% more likely to stop using nicotine products up to a year later, compared with teens who did not receive any counseling.

Counseling can help educate teens (and parents) on some of the realities of e-cigarettes, bridging the gap between well-established anti-smoking campaigns and the anti-vape campaigns that have yet to catch up.

“We have done a great job promoting cigarette use as dangerous,” Dr. Walley said. “[But] many teens who would never pick up a cigarette –because they know the health risks – are vaping.”
 

How to get a teen to quit

Cessation and prevention strategies are closely linked, and interventions can start in middle school-aged children up through high school and young adults. Simply asking a 12-year-old, “Do you know anyone who smokes?” can help start a conversation that leads to an attempt to quit.

Teens may be compelled to smoke through digital advertising and influencer endorsements on social media platforms, but Gen Z is turned off by the idea that it’s being manipulated by the tobacco industry. Juul, for example, is partially owned by Altria, which makes Marlboros, and Vuse is wholly owned by R.J. Reynolds, which makes Camel cigarettes.

“If you can get somebody to understand that Big Tobacco is trying to manipulate you as a young person to want to illegally obtain and use their products, which are incredibly addictive, thus ensuring you will remain a loyal customer, that could be the thing that pushes them over the hump,” Dr. Stepp said. “You push it away like you would push away a parent trying to tell you how to park a car in the driveway.”

And just because a smoker relapses, it doesn’t mean the cessation was a complete failure. The younger someone is when they stop smoking, the less likely they are to suffer from the long-term health consequences of smoking, according to a 2021 study in the Journal of the American Medical Association. “With the right counseling,” Dr. Walley said, “each relapse is an opportunity for losing the habit permanently.”

This article was adapted from the Summer 2023 online issue of CHEST Advocates. For the full article – and to engage with the other content from this issue – visit https://chestnet.org/chest-­advocates.

Pulmonologist Evan Stepp, MD, FCCP, has a teenage daughter who doesn’t smoke or vape – as far as he knows, Stepp will admit – but the statistics on youth smoking are alarming enough to have him worried.

On one hand, fewer Americans are smoking today than ever before. Since 1992, the percentage of people who told Gallup that they’d had a cigarette in the past week has dropped from 28% to 11%. Meanwhile, the rate of new lung cancer cases declined from 65 per every 100,000 people in 1992 to 34 per 100,000 in 2020, according to the National Cancer Institute.

While those statistics are worth celebrating, they hide an alarming reality: A disproportionate number of teens and young adults today are addicted to nicotine.

According to a November 2022 report from the Centers for Disease Control and Prevention (CDC), 1 in 6 high school students and 1 in 20 middle schoolers are using a nicotine product at least once every day.

“It’s a completely different picture for nicotine cessation in youth,” Dr. Stepp, who is an associate professor at National Jewish Health in Denver, said. “Because of the fact that the nicotine addiction is occurring in a developing brain, which raises many other nicotine-related harms.”
 

Why teens vape

Today’s teens are smoking less actual tobacco, and, instead, overwhelmingly prefer e-cigarettes or vaping. In fact, 85% of high school–aged smokers and 72% of middle school smokers reach for a vape over regular cigarettes or smokeless tobacco, according to the CDC.

It’s not hard to understand why: e-cigarettes use a heating element to turn a nicotine-infused liquid into an aerosol, with no open flame, ash, or lingering smoke. The vapes themselves are easy to conceal, and if someone needed to hide an e-cigarette from particularly perceptive parents or teachers, they can find vapes built into hoodies, fake smartwatches, and USB drives.

Plus, the liquids often come in flavors like fruit, bubble gum, mint, and vanilla, because unflavored nicotine isn’t exactly appealing. “Huge concentrations of nicotine salts are just miserable to breathe in,” Dr. Stepp said. “Flavors are necessary to make these products palatable, and those flavors end up being a huge draw for youth users to get exposed to nicotine addiction.”
 

Challenges surrounding smoking cessation in youth

The powerful effect of nicotine in youth means the need for effective cessation strategies is both more urgent and more difficult. But while physicians can prescribe to adults the antidepressants varenicline and bupropion, along with nicotine replacement therapy, to help ease withdrawal symptoms, the US Food and Drug Administration (FDA) has not approved those medications for anyone under the age of 18.

Research on cessation medications in young people is limited: A recent meta-analysis found only four studies on people between the ages of 12 and 21. In teens, antidepressants seem to help quitting for the first few weeks but are unproven as a long-term solution.

“That really has been a challenge for the 1 in 6 high school students who are current users of tobacco products,” said pediatrician, Susan Walley, MD, a co-author of the American Academy of Pediatrics’ recent position papers on children and smoking.

“One of the things that is important to keep at the forefront of the conversation is that nicotine addiction is a chronic medical disease, and it’s a form of substance abuse,” Dr. Walley said. “We know that we need more research in adolescent tobacco cessation, and it really is about the funding, about research dollars.”

Without medications, smoking cessation in teens relies largely on counseling strategies. A 2017 review published by Cochrane Library found that group counseling was the most effective quitting method, with teens participating in group sessions 35% more likely to stop using nicotine products up to a year later, compared with teens who did not receive any counseling.

Counseling can help educate teens (and parents) on some of the realities of e-cigarettes, bridging the gap between well-established anti-smoking campaigns and the anti-vape campaigns that have yet to catch up.

“We have done a great job promoting cigarette use as dangerous,” Dr. Walley said. “[But] many teens who would never pick up a cigarette –because they know the health risks – are vaping.”
 

How to get a teen to quit

Cessation and prevention strategies are closely linked, and interventions can start in middle school-aged children up through high school and young adults. Simply asking a 12-year-old, “Do you know anyone who smokes?” can help start a conversation that leads to an attempt to quit.

Teens may be compelled to smoke through digital advertising and influencer endorsements on social media platforms, but Gen Z is turned off by the idea that it’s being manipulated by the tobacco industry. Juul, for example, is partially owned by Altria, which makes Marlboros, and Vuse is wholly owned by R.J. Reynolds, which makes Camel cigarettes.

“If you can get somebody to understand that Big Tobacco is trying to manipulate you as a young person to want to illegally obtain and use their products, which are incredibly addictive, thus ensuring you will remain a loyal customer, that could be the thing that pushes them over the hump,” Dr. Stepp said. “You push it away like you would push away a parent trying to tell you how to park a car in the driveway.”

And just because a smoker relapses, it doesn’t mean the cessation was a complete failure. The younger someone is when they stop smoking, the less likely they are to suffer from the long-term health consequences of smoking, according to a 2021 study in the Journal of the American Medical Association. “With the right counseling,” Dr. Walley said, “each relapse is an opportunity for losing the habit permanently.”

This article was adapted from the Summer 2023 online issue of CHEST Advocates. For the full article – and to engage with the other content from this issue – visit https://chestnet.org/chest-­advocates.

WASHINGTON – Transgender and other gender-diverse youth who visited the emergency department (ED) at a single institution had more than five times greater odds of a positive suicide screening compared with their cisgender peers, according to a study presented at the annual meeting of the American Academy of Pediatrics.

“The take-home message here is this study emphasizes the importance of universal screening to identify gender-diverse youth at risk,” Amanda Burnside, PhD, assistant professor of psychiatry and behavioral sciences at Ann and Robert H. Lurie Children’s Hospital of Chicago and Northwestern University, told attendees. “We really need to develop robust strategies and systems to link better mental health services.”

Ann and Robert H. Lurie Children’s Hospital of Chicago

Suicide rates in transgender and gender-diverse youth are exceptionally high among youth in the U.S., Dr. Burnside said during her presentation. For example, the 2022 LGBTQ health survey from the Trevor Project found that much higher percentages of transgender and gender nonconforming youth had considered suicide in the past year compared with cisgender youth, even within the LGBTQ umbrella. Among nearly 34,000 LGBTQ youth aged 13-24, nearly half of trans females (48%) and more than half of trans males (59%) had considered suicide, compared with 28% of cisgender males and 37% of cisgender females. The rate among nonbinary/genderqueer individuals was 53%, and it was 48% for those questioning their gender.

Current methods of identifying trans and gender-diverse (TGD) youth in the hospital, however, may not actually be capturing the entire population.

“In health care settings, research involving TGD individuals has historically been limited to specialized clinic populations or youth with gender-specific diagnostic codes documented in the electronic medical record,” an approach that “likely significantly underestimates the prevalence of TGD youth in health care settings.” While at least one study has attempted to bridge this gap by searching the EMR for keywords, that study only tried to identify trans youth and not other youth on the gender diversity spectrum, such as nonbinary youth or those questioning their gender identity. Dr. Burnside and her colleagues therefore designed a study that used keywords to identify both trans youth and other gender-diverse youth who visited the ED so they could assess the rate of positive suicide screens in this population.
 

Underestimating the population at risk?

The researchers conducted a retrospective cross-sectional study of EMR data for all ED visits during which the patient underwent suicide screening. For the period of November 2019 to August 2022, they collected data on the screening results and the patient’s gender identity, age, race/ethnicity, insurance status, chief complaint in the ED and child opportunity index, which assess a youth’s access to resources based on geography. The suicide screener used was the Ask Suicide–Screening Questions (ASQ) tool.

The keywords they looked for in the EMR to identify trans and gender-diverse youth included transgender, pronouns, agender, gender dysphoria, male-to-female, female-to-male, nonbinary, preferred name, and they/them (captured as a complete term, not as “they” and “them” separately).

“If a keyword was present, the surrounding text was extracted and reviewed by two members of our team,” Dr. Burnside explained in her presentation. “We categorized keywords into either indicative of gender-diverse identity or not, and if it wasn’t clear based on the text extracted, we would conduct a manual chart review,” though that only occurred in about 3% of cases, she added.

Among 15,413 ED encounters with a suicide screen, the researchers identified 1,126 of these keywords in the EMR, among which 91.2% were classified as referring to a gender-diverse patient. Nearly all of the words were at least 90% effective in identify a gender-diverse youth, Dr. Burnside said, and all of the 197 instances of “they/them” were classified as gender diverse.

The accuracy was a little lower for the two keywords that appeared most frequently: For “pronouns,” 86.3% of 306 instances were classified as gender diverse, and for “transgender,” 83.1% of 207 instances were classified as gender diverse. Since some providers ask all patients their pronouns, the presence of “pronouns” in the EMR alone did not necessarily indicate the patient was gender diverse, Dr. Burnside said. A common reason the term “transgender” occurred in the EMR of non–gender diverse patients is that the department’s list of crisis resources includes transgender hotlines.

After identifying all the keywords, the researchers determined how many of these occurred in unique ED encounters and removed those with incomplete screening. Overall, they found 565 encounters by 399 gender-diverse individuals who had a suicide screening, representing 4.6% of total visits. This percentage is slightly lower than recent population-based estimates of gender-diverse youth, the researchers noted.

This population ranged from 8 to 23 years old, and 43% were publicly insured. The chief complaint for most of the patients (77.5%) was a mental health one. They were predominantly White (43%) or Hispanic (35%), with 10% Black youth, 4% Asian youth, and 8% youth who were “other” or two or more races. About half (52%) lived in a neighborhood with a “low” or “very low” child opportunity index.

Within this population, 81% of the patients screened positive on the suicide screening, compared with 23% positive screens across all ED visits. One in ten (10%) gender-diverse youth had active suicidal ideation, compared with 3.4% of the rest of the ED patient population. The researchers calculated that gender-diverse youth had 5.35 times greater odds of screening positive than cisgender youth in the ED (95% confidence interval [CI] 8.7-15.92). Further, a quarter (25%) of the trans and gender-diverse youth who screened positive for suicide risk had come to the ED for a primary complaint unrelated to mental health.

“We had a kid who came in because he broke his arm who had active suicidal ideation,” study coauthor Jennifer A. Hoffmann, MD, assistant professor of pediatrics at the Ann & Robert H. Lurie Children’s Hospital of Chicago and Northwestern University, mentioned after the presentation. That particular patient even had a suicide plan, but was identified as actively suicidal only because of the screening. In other cases, she said, a youth may come in with self-inflicted injuries, and while those are the primary complaint, they are linked with suicidal ideation.

Ann &amp; Robert H. Lurie Children&#039;s Hospital of Chicago

Uncovering valuable information

Jason Rafferty, MD, MPH, EdM, clinical assistant professor of pediatrics and of psychiatry and human behavior at Brown University, Providence, R.I., who attended the presentation, noted that the study provides information on a population that’s often difficult to get through traditional EMR research methods.

Brown University

“A lot of medical record systems don’t have uniform ways of capturing [gender diversity], but what we know as providers is that kids are really struggling and that it’s not a surprise that we’re seeing these disparities with suicidality,” Dr. Rafferty said.

The study also provides more discrete estimates by age than what most other current research measures, which tends to be lifetime suicidality as opposed to suicidal thoughts or attempts within the past year, Dr. Rafferty added.

”What this shows is, for adolescents, the risk of suicide is something we need to be paying attention to. Because it’s not that it’s something that only happens in adults, this really dispels a lot of the misquoting of the data that’s out there.” That kind of information is valuable for determining resource allocation, he said. “A disparity like this really underlies the importance of mental health resources in this field,” he said.

Dr. Burnside, Dr. Hoffmann, and Dr. Rafferty had no disclosures, and no external funding sources were noted.

WASHINGTON – Transgender and other gender-diverse youth who visited the emergency department (ED) at a single institution had more than five times greater odds of a positive suicide screening compared with their cisgender peers, according to a study presented at the annual meeting of the American Academy of Pediatrics.

“The take-home message here is this study emphasizes the importance of universal screening to identify gender-diverse youth at risk,” Amanda Burnside, PhD, assistant professor of psychiatry and behavioral sciences at Ann and Robert H. Lurie Children’s Hospital of Chicago and Northwestern University, told attendees. “We really need to develop robust strategies and systems to link better mental health services.”

Ann and Robert H. Lurie Children’s Hospital of Chicago

Suicide rates in transgender and gender-diverse youth are exceptionally high among youth in the U.S., Dr. Burnside said during her presentation. For example, the 2022 LGBTQ health survey from the Trevor Project found that much higher percentages of transgender and gender nonconforming youth had considered suicide in the past year compared with cisgender youth, even within the LGBTQ umbrella. Among nearly 34,000 LGBTQ youth aged 13-24, nearly half of trans females (48%) and more than half of trans males (59%) had considered suicide, compared with 28% of cisgender males and 37% of cisgender females. The rate among nonbinary/genderqueer individuals was 53%, and it was 48% for those questioning their gender.

Current methods of identifying trans and gender-diverse (TGD) youth in the hospital, however, may not actually be capturing the entire population.

“In health care settings, research involving TGD individuals has historically been limited to specialized clinic populations or youth with gender-specific diagnostic codes documented in the electronic medical record,” an approach that “likely significantly underestimates the prevalence of TGD youth in health care settings.” While at least one study has attempted to bridge this gap by searching the EMR for keywords, that study only tried to identify trans youth and not other youth on the gender diversity spectrum, such as nonbinary youth or those questioning their gender identity. Dr. Burnside and her colleagues therefore designed a study that used keywords to identify both trans youth and other gender-diverse youth who visited the ED so they could assess the rate of positive suicide screens in this population.
 

Underestimating the population at risk?

The researchers conducted a retrospective cross-sectional study of EMR data for all ED visits during which the patient underwent suicide screening. For the period of November 2019 to August 2022, they collected data on the screening results and the patient’s gender identity, age, race/ethnicity, insurance status, chief complaint in the ED and child opportunity index, which assess a youth’s access to resources based on geography. The suicide screener used was the Ask Suicide–Screening Questions (ASQ) tool.

The keywords they looked for in the EMR to identify trans and gender-diverse youth included transgender, pronouns, agender, gender dysphoria, male-to-female, female-to-male, nonbinary, preferred name, and they/them (captured as a complete term, not as “they” and “them” separately).

“If a keyword was present, the surrounding text was extracted and reviewed by two members of our team,” Dr. Burnside explained in her presentation. “We categorized keywords into either indicative of gender-diverse identity or not, and if it wasn’t clear based on the text extracted, we would conduct a manual chart review,” though that only occurred in about 3% of cases, she added.

Among 15,413 ED encounters with a suicide screen, the researchers identified 1,126 of these keywords in the EMR, among which 91.2% were classified as referring to a gender-diverse patient. Nearly all of the words were at least 90% effective in identify a gender-diverse youth, Dr. Burnside said, and all of the 197 instances of “they/them” were classified as gender diverse.

The accuracy was a little lower for the two keywords that appeared most frequently: For “pronouns,” 86.3% of 306 instances were classified as gender diverse, and for “transgender,” 83.1% of 207 instances were classified as gender diverse. Since some providers ask all patients their pronouns, the presence of “pronouns” in the EMR alone did not necessarily indicate the patient was gender diverse, Dr. Burnside said. A common reason the term “transgender” occurred in the EMR of non–gender diverse patients is that the department’s list of crisis resources includes transgender hotlines.

After identifying all the keywords, the researchers determined how many of these occurred in unique ED encounters and removed those with incomplete screening. Overall, they found 565 encounters by 399 gender-diverse individuals who had a suicide screening, representing 4.6% of total visits. This percentage is slightly lower than recent population-based estimates of gender-diverse youth, the researchers noted.

This population ranged from 8 to 23 years old, and 43% were publicly insured. The chief complaint for most of the patients (77.5%) was a mental health one. They were predominantly White (43%) or Hispanic (35%), with 10% Black youth, 4% Asian youth, and 8% youth who were “other” or two or more races. About half (52%) lived in a neighborhood with a “low” or “very low” child opportunity index.

Within this population, 81% of the patients screened positive on the suicide screening, compared with 23% positive screens across all ED visits. One in ten (10%) gender-diverse youth had active suicidal ideation, compared with 3.4% of the rest of the ED patient population. The researchers calculated that gender-diverse youth had 5.35 times greater odds of screening positive than cisgender youth in the ED (95% confidence interval [CI] 8.7-15.92). Further, a quarter (25%) of the trans and gender-diverse youth who screened positive for suicide risk had come to the ED for a primary complaint unrelated to mental health.

“We had a kid who came in because he broke his arm who had active suicidal ideation,” study coauthor Jennifer A. Hoffmann, MD, assistant professor of pediatrics at the Ann & Robert H. Lurie Children’s Hospital of Chicago and Northwestern University, mentioned after the presentation. That particular patient even had a suicide plan, but was identified as actively suicidal only because of the screening. In other cases, she said, a youth may come in with self-inflicted injuries, and while those are the primary complaint, they are linked with suicidal ideation.

Ann &amp; Robert H. Lurie Children&#039;s Hospital of Chicago

Uncovering valuable information

Jason Rafferty, MD, MPH, EdM, clinical assistant professor of pediatrics and of psychiatry and human behavior at Brown University, Providence, R.I., who attended the presentation, noted that the study provides information on a population that’s often difficult to get through traditional EMR research methods.

Brown University

“A lot of medical record systems don’t have uniform ways of capturing [gender diversity], but what we know as providers is that kids are really struggling and that it’s not a surprise that we’re seeing these disparities with suicidality,” Dr. Rafferty said.

The study also provides more discrete estimates by age than what most other current research measures, which tends to be lifetime suicidality as opposed to suicidal thoughts or attempts within the past year, Dr. Rafferty added.

”What this shows is, for adolescents, the risk of suicide is something we need to be paying attention to. Because it’s not that it’s something that only happens in adults, this really dispels a lot of the misquoting of the data that’s out there.” That kind of information is valuable for determining resource allocation, he said. “A disparity like this really underlies the importance of mental health resources in this field,” he said.

Dr. Burnside, Dr. Hoffmann, and Dr. Rafferty had no disclosures, and no external funding sources were noted.

WASHINGTON – Transgender and other gender-diverse youth who visited the emergency department (ED) at a single institution had more than five times greater odds of a positive suicide screening compared with their cisgender peers, according to a study presented at the annual meeting of the American Academy of Pediatrics.

“The take-home message here is this study emphasizes the importance of universal screening to identify gender-diverse youth at risk,” Amanda Burnside, PhD, assistant professor of psychiatry and behavioral sciences at Ann and Robert H. Lurie Children’s Hospital of Chicago and Northwestern University, told attendees. “We really need to develop robust strategies and systems to link better mental health services.”

Ann and Robert H. Lurie Children’s Hospital of Chicago

Suicide rates in transgender and gender-diverse youth are exceptionally high among youth in the U.S., Dr. Burnside said during her presentation. For example, the 2022 LGBTQ health survey from the Trevor Project found that much higher percentages of transgender and gender nonconforming youth had considered suicide in the past year compared with cisgender youth, even within the LGBTQ umbrella. Among nearly 34,000 LGBTQ youth aged 13-24, nearly half of trans females (48%) and more than half of trans males (59%) had considered suicide, compared with 28% of cisgender males and 37% of cisgender females. The rate among nonbinary/genderqueer individuals was 53%, and it was 48% for those questioning their gender.

Current methods of identifying trans and gender-diverse (TGD) youth in the hospital, however, may not actually be capturing the entire population.

“In health care settings, research involving TGD individuals has historically been limited to specialized clinic populations or youth with gender-specific diagnostic codes documented in the electronic medical record,” an approach that “likely significantly underestimates the prevalence of TGD youth in health care settings.” While at least one study has attempted to bridge this gap by searching the EMR for keywords, that study only tried to identify trans youth and not other youth on the gender diversity spectrum, such as nonbinary youth or those questioning their gender identity. Dr. Burnside and her colleagues therefore designed a study that used keywords to identify both trans youth and other gender-diverse youth who visited the ED so they could assess the rate of positive suicide screens in this population.
 

Underestimating the population at risk?

The researchers conducted a retrospective cross-sectional study of EMR data for all ED visits during which the patient underwent suicide screening. For the period of November 2019 to August 2022, they collected data on the screening results and the patient’s gender identity, age, race/ethnicity, insurance status, chief complaint in the ED and child opportunity index, which assess a youth’s access to resources based on geography. The suicide screener used was the Ask Suicide–Screening Questions (ASQ) tool.

The keywords they looked for in the EMR to identify trans and gender-diverse youth included transgender, pronouns, agender, gender dysphoria, male-to-female, female-to-male, nonbinary, preferred name, and they/them (captured as a complete term, not as “they” and “them” separately).

“If a keyword was present, the surrounding text was extracted and reviewed by two members of our team,” Dr. Burnside explained in her presentation. “We categorized keywords into either indicative of gender-diverse identity or not, and if it wasn’t clear based on the text extracted, we would conduct a manual chart review,” though that only occurred in about 3% of cases, she added.

Among 15,413 ED encounters with a suicide screen, the researchers identified 1,126 of these keywords in the EMR, among which 91.2% were classified as referring to a gender-diverse patient. Nearly all of the words were at least 90% effective in identify a gender-diverse youth, Dr. Burnside said, and all of the 197 instances of “they/them” were classified as gender diverse.

The accuracy was a little lower for the two keywords that appeared most frequently: For “pronouns,” 86.3% of 306 instances were classified as gender diverse, and for “transgender,” 83.1% of 207 instances were classified as gender diverse. Since some providers ask all patients their pronouns, the presence of “pronouns” in the EMR alone did not necessarily indicate the patient was gender diverse, Dr. Burnside said. A common reason the term “transgender” occurred in the EMR of non–gender diverse patients is that the department’s list of crisis resources includes transgender hotlines.

After identifying all the keywords, the researchers determined how many of these occurred in unique ED encounters and removed those with incomplete screening. Overall, they found 565 encounters by 399 gender-diverse individuals who had a suicide screening, representing 4.6% of total visits. This percentage is slightly lower than recent population-based estimates of gender-diverse youth, the researchers noted.

This population ranged from 8 to 23 years old, and 43% were publicly insured. The chief complaint for most of the patients (77.5%) was a mental health one. They were predominantly White (43%) or Hispanic (35%), with 10% Black youth, 4% Asian youth, and 8% youth who were “other” or two or more races. About half (52%) lived in a neighborhood with a “low” or “very low” child opportunity index.

Within this population, 81% of the patients screened positive on the suicide screening, compared with 23% positive screens across all ED visits. One in ten (10%) gender-diverse youth had active suicidal ideation, compared with 3.4% of the rest of the ED patient population. The researchers calculated that gender-diverse youth had 5.35 times greater odds of screening positive than cisgender youth in the ED (95% confidence interval [CI] 8.7-15.92). Further, a quarter (25%) of the trans and gender-diverse youth who screened positive for suicide risk had come to the ED for a primary complaint unrelated to mental health.

“We had a kid who came in because he broke his arm who had active suicidal ideation,” study coauthor Jennifer A. Hoffmann, MD, assistant professor of pediatrics at the Ann & Robert H. Lurie Children’s Hospital of Chicago and Northwestern University, mentioned after the presentation. That particular patient even had a suicide plan, but was identified as actively suicidal only because of the screening. In other cases, she said, a youth may come in with self-inflicted injuries, and while those are the primary complaint, they are linked with suicidal ideation.

Ann &amp; Robert H. Lurie Children&#039;s Hospital of Chicago

Uncovering valuable information

Jason Rafferty, MD, MPH, EdM, clinical assistant professor of pediatrics and of psychiatry and human behavior at Brown University, Providence, R.I., who attended the presentation, noted that the study provides information on a population that’s often difficult to get through traditional EMR research methods.

Brown University

“A lot of medical record systems don’t have uniform ways of capturing [gender diversity], but what we know as providers is that kids are really struggling and that it’s not a surprise that we’re seeing these disparities with suicidality,” Dr. Rafferty said.

The study also provides more discrete estimates by age than what most other current research measures, which tends to be lifetime suicidality as opposed to suicidal thoughts or attempts within the past year, Dr. Rafferty added.

”What this shows is, for adolescents, the risk of suicide is something we need to be paying attention to. Because it’s not that it’s something that only happens in adults, this really dispels a lot of the misquoting of the data that’s out there.” That kind of information is valuable for determining resource allocation, he said. “A disparity like this really underlies the importance of mental health resources in this field,” he said.

Dr. Burnside, Dr. Hoffmann, and Dr. Rafferty had no disclosures, and no external funding sources were noted.