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EU agency issues positive opinion on ritlecitinib
, paving the way for possible marketing authorization of the drug in the European Union for individuals 12 years of age and older. A final decision is expected in the coming months.
The development, which was announced by the manufacturer, Pfizer, on July 21, 2023, follows approval of ritlecitinib (Litfulo) for the treatment of severe alopecia areata in adults and adolescents 12 years and older by the Food and Drug Administration and the Japanese Ministry of Health, Labour, and Welfare in June 2023. According to a press release from Pfizer, submissions to other regulatory agencies for the use of ritlecitinib in alopecia areata are ongoing.
The Marketing Authorization Application for ritlecitinib was based on results from a randomized, placebo-controlled, double-blind ALLEGRO Phase 2b/3 study.
, paving the way for possible marketing authorization of the drug in the European Union for individuals 12 years of age and older. A final decision is expected in the coming months.
The development, which was announced by the manufacturer, Pfizer, on July 21, 2023, follows approval of ritlecitinib (Litfulo) for the treatment of severe alopecia areata in adults and adolescents 12 years and older by the Food and Drug Administration and the Japanese Ministry of Health, Labour, and Welfare in June 2023. According to a press release from Pfizer, submissions to other regulatory agencies for the use of ritlecitinib in alopecia areata are ongoing.
The Marketing Authorization Application for ritlecitinib was based on results from a randomized, placebo-controlled, double-blind ALLEGRO Phase 2b/3 study.
, paving the way for possible marketing authorization of the drug in the European Union for individuals 12 years of age and older. A final decision is expected in the coming months.
The development, which was announced by the manufacturer, Pfizer, on July 21, 2023, follows approval of ritlecitinib (Litfulo) for the treatment of severe alopecia areata in adults and adolescents 12 years and older by the Food and Drug Administration and the Japanese Ministry of Health, Labour, and Welfare in June 2023. According to a press release from Pfizer, submissions to other regulatory agencies for the use of ritlecitinib in alopecia areata are ongoing.
The Marketing Authorization Application for ritlecitinib was based on results from a randomized, placebo-controlled, double-blind ALLEGRO Phase 2b/3 study.
What makes teens choose to use sunscreen?
a cornerstone of skin cancer prevention, according to results from a systematic review.
“We know that skin cancer is one of the most common malignancies in the world, and sun protection methods such as sunscreen make it highly preventable,” first author Carly R. Stevens, a student at Tulane University, New Orleans, said in an interview. “This study demonstrates the adolescent populations that are most vulnerable to sun damage and how we can help mitigate their risk of developing skin cancer through education methods, such as Sun Protection Outreach Teaching by Students.” 
Ms. Stevens and coauthors presented the findings during a poster session at the annual meeting of the Society for Pediatric Dermatology.
To investigate predictors of sunscreen use among high school students, they searched PubMed, Embase, and Web of Science using the terms (“sunscreen” or “SPF” or “sun protection”) and (“high school” or “teen” or “teenager” or “adolescent”) and limited the analysis to English studies reporting data on sunscreen use in U.S. high school students up to November 2021.
A total of 20 studies were included in the final review. The study populations ranged in number from 208 to 24,645. Of 11 studies that examined gender, all showed increased sunscreen use in females compared with males. Of five studies that examined age, all showed increased sunscreen use in younger adolescents, compared with their older counterparts.
Of four studies that examined the role of ethnicity on sunscreen use, White students were more likely to use sunscreen, compared with their peers of other ethnicities. “This may be due to perceived sun sensitivity, as [these four studies] also showed increased sunscreen use in populations that believed were more susceptible to sun damage,” the researchers wrote in their abstract.
In other findings, two studies that examined perceived self-efficacy concluded that higher levels of sunscreen use correlated with higher self-efficacy, while four studies concluded that high school students were more likely to use sunscreen if their parents encouraged them the wear it or if the parent used it themselves.
“With 40%-50% of ultraviolet damage being done before the age of 20, it’s crucial that we find ways to educate adolescents on the importance of sunscreen use and target those populations who were found to rarely use sunscreen in our study,” Ms. Stevens said.
In one outreach program, Sun Protection Outreach Teaching by Students (SPOTS), medical students visit middle and high schools to educate them about the importance of practicing sun protection. The program began as a collaboration between Saint Louis University and Washington University in St. Louis, but has expanded nationwide. Ms. Stevens described SPOTS as “a great way for medical students to present the information to middle and high school students in a way that is engaging and interactive.”
The researchers reported having no disclosures.
a cornerstone of skin cancer prevention, according to results from a systematic review.
“We know that skin cancer is one of the most common malignancies in the world, and sun protection methods such as sunscreen make it highly preventable,” first author Carly R. Stevens, a student at Tulane University, New Orleans, said in an interview. “This study demonstrates the adolescent populations that are most vulnerable to sun damage and how we can help mitigate their risk of developing skin cancer through education methods, such as Sun Protection Outreach Teaching by Students.”
Ms. Stevens and coauthors presented the findings during a poster session at the annual meeting of the Society for Pediatric Dermatology.
To investigate predictors of sunscreen use among high school students, they searched PubMed, Embase, and Web of Science using the terms (“sunscreen” or “SPF” or “sun protection”) and (“high school” or “teen” or “teenager” or “adolescent”) and limited the analysis to English studies reporting data on sunscreen use in U.S. high school students up to November 2021.
A total of 20 studies were included in the final review. The study populations ranged in number from 208 to 24,645. Of 11 studies that examined gender, all showed increased sunscreen use in females compared with males. Of five studies that examined age, all showed increased sunscreen use in younger adolescents, compared with their older counterparts.
Of four studies that examined the role of ethnicity on sunscreen use, White students were more likely to use sunscreen, compared with their peers of other ethnicities. “This may be due to perceived sun sensitivity, as [these four studies] also showed increased sunscreen use in populations that believed were more susceptible to sun damage,” the researchers wrote in their abstract.
In other findings, two studies that examined perceived self-efficacy concluded that higher levels of sunscreen use correlated with higher self-efficacy, while four studies concluded that high school students were more likely to use sunscreen if their parents encouraged them the wear it or if the parent used it themselves.
“With 40%-50% of ultraviolet damage being done before the age of 20, it’s crucial that we find ways to educate adolescents on the importance of sunscreen use and target those populations who were found to rarely use sunscreen in our study,” Ms. Stevens said.
In one outreach program, Sun Protection Outreach Teaching by Students (SPOTS), medical students visit middle and high schools to educate them about the importance of practicing sun protection. The program began as a collaboration between Saint Louis University and Washington University in St. Louis, but has expanded nationwide. Ms. Stevens described SPOTS as “a great way for medical students to present the information to middle and high school students in a way that is engaging and interactive.”
The researchers reported having no disclosures.
a cornerstone of skin cancer prevention, according to results from a systematic review.
“We know that skin cancer is one of the most common malignancies in the world, and sun protection methods such as sunscreen make it highly preventable,” first author Carly R. Stevens, a student at Tulane University, New Orleans, said in an interview. “This study demonstrates the adolescent populations that are most vulnerable to sun damage and how we can help mitigate their risk of developing skin cancer through education methods, such as Sun Protection Outreach Teaching by Students.”
Ms. Stevens and coauthors presented the findings during a poster session at the annual meeting of the Society for Pediatric Dermatology.
To investigate predictors of sunscreen use among high school students, they searched PubMed, Embase, and Web of Science using the terms (“sunscreen” or “SPF” or “sun protection”) and (“high school” or “teen” or “teenager” or “adolescent”) and limited the analysis to English studies reporting data on sunscreen use in U.S. high school students up to November 2021.
A total of 20 studies were included in the final review. The study populations ranged in number from 208 to 24,645. Of 11 studies that examined gender, all showed increased sunscreen use in females compared with males. Of five studies that examined age, all showed increased sunscreen use in younger adolescents, compared with their older counterparts.
Of four studies that examined the role of ethnicity on sunscreen use, White students were more likely to use sunscreen, compared with their peers of other ethnicities. “This may be due to perceived sun sensitivity, as [these four studies] also showed increased sunscreen use in populations that believed were more susceptible to sun damage,” the researchers wrote in their abstract.
In other findings, two studies that examined perceived self-efficacy concluded that higher levels of sunscreen use correlated with higher self-efficacy, while four studies concluded that high school students were more likely to use sunscreen if their parents encouraged them the wear it or if the parent used it themselves.
“With 40%-50% of ultraviolet damage being done before the age of 20, it’s crucial that we find ways to educate adolescents on the importance of sunscreen use and target those populations who were found to rarely use sunscreen in our study,” Ms. Stevens said.
In one outreach program, Sun Protection Outreach Teaching by Students (SPOTS), medical students visit middle and high schools to educate them about the importance of practicing sun protection. The program began as a collaboration between Saint Louis University and Washington University in St. Louis, but has expanded nationwide. Ms. Stevens described SPOTS as “a great way for medical students to present the information to middle and high school students in a way that is engaging and interactive.”
The researchers reported having no disclosures.
FROM SPD 2023
When treating AD in children, experts consider adherence, other aspects of treatment
ASHEVILLE, N.C. – according to a three-member expert panel mulling over strategies at the annual meeting of the Society for Pediatric Dermatology.
In introductory remarks, the three panelists briefly addressed different aspects for controlling AD, including drugs in the pipeline, the potential value of alternative therapies, and whom to blame when compliance is poor.
But panel discussion following these presentations provided an opportunity for audience engagement on practical strategies for improving AD control.
In her formal remarks prior to the panel discussion, Amy S. Paller, MD, professor of dermatology and pediatrics and chair of dermatology, Northwestern University, Chicago, and a pediatric dermatologist at the Lurie Children’s Hospital of Chicago, described emerging AD treatments. This included an update on the status of the interleukin-13 (IL-13) inhibitors tralokinumab (Adbry), which was approved by the FDA for treating AD in adults in December 2021, and lebrikizumab, which is thought likely to be soon approved in the United States on the basis of two recently published phase 3 trials.
Along with dupilumab (Dupixent) for moderate-to-severe AD in children who do not respond to optimized use of topical therapies, these new biologics appear likely to further expand choices for AD control for adults (and for kids with AD too, if eventually licensed in children), according to the data from the phase 3 studies.
During a panel discussion that followed, Stephen Gellis, MD, pediatric dermatologist and former chief of pediatric dermatology at Boston Children’s Hospital and Harvard Medical School, raised the point of optimizing tried and true topical therapies before using systemic agents. He noted that parents sometimes pressure clinicians to use a biologic – and that moving too quickly to the latest and most expensive drugs may not be necessary.
Dr. Paller acknowledged that she, like many pediatric dermatologists, employed immunosuppressants as her drugs of choice for many years – commonly starting with a few months of cyclosporine before transitioning to methotrexate, which has a delayed onset of action. In fact, she still uses this regimen in some children.
However, she now prefers dupilumab, which is the first biologic available for children in the United States with an AD indication in children as young as 6 months. She said dupilumab has fewer potential risks than cyclosporine, and it offers clinically meaningful improvement in most children. She noted that current guidelines discourage the use of systemic corticosteroids for AD in children, given their potential toxicity.
She strongly agreed with Dr. Gellis that clinicians should resist pressure to use any systemic agent if children are responding well to topical medications. In her own practice, Dr. Paller moves to systemic medications only after ensuring that there has been adherence to appropriate therapy and that there is not another diagnosis that might explain the recalcitrance to topical agents.
When a systemic medication is considered the next step, Dr. Paller reminded the audience of the importance of presenting the benefits and risks of all the options for AD control, which could include dupilumab and immunosuppressants as initial systemic therapy.
“Many parents choose biologic treatment first, given its lack of requirement for blood monitoring and faster action than methotrexate,” Dr. Paller noted.
Nevertheless, “biologics are much more costly than immunosuppressants, require an injection – which is stressful for the child and the parents – and may not be accessible for our patients,” Dr. Paller said. Cyclosporine and methotrexate are effective and are often the best options for moderate to severe disease in areas of the world where dupilumab is not available, but Dr. Paller most commonly uses these therapies only when reimbursement for dupilumab cannot be secured, injection is not an option, or when dupilumab is not sufficiently effective and tolerated.
Providing different perspectives, the two other panelists discussing the treatment of pediatric AD also saw a role for ensuring that topical agents are not offering adequate AD control before turning to the latest and most sophisticated therapies for AD.
For meeting parent expectations when children are improving slowly on topical therapies, Peter A. Lio, MD, director of the Chicago Integrative Eczema Center and clinical assistant professor of dermatology and pediatrics at Northwestern University, suggested that integrative medicine might be helpful.
For parents not fully comfortable with standard pharmacologic agents, Dr. Lio said there is evidence to support some of the complementary approaches, and these can be reassuring to parents with an interest in alternative medicines.
In Western medicine, it is common to hear terms like “attack,” “kill,” and “suppress,” disease, but alternative therapies are generally coupled with terms like “restore,” “strengthen,” and “tonify,” he said. “Who doesn’t want to be tonified?” he asked, noting that there are many sources of data suggesting that the number of patients seeking alternative medicine is “huge.” The alternative medicines are not generally taught in medical school and remain widely ignored in typical practice, but “our patients are interested even if we are not.”
Yet, there are data to support benefit from some of these alternative therapies, providing a win-win situation for patients who derive satisfaction from nontraditional therapies alone or combined with established pharmaceutical treatments.
Of these, Dr. Lio said there is support for the use of hempseed oil as a moisturizing agent and a strategy for improving barrier function in the skin of patients with AD. In a controlled crossover study, 2 teaspoons per day of dietary hempseed oil, a product that can be purchased in some grocery stores, was associated with significant reductions in skin dryness, itchiness, and use of topical medications relative to the same amount of olive oil, he noted.
Other examples include a compress made with black tea that was associated with an anti-inflammatory effect when followed by a moisturizer, a published study asserts. Although this was a trial in adults with facial dermatitis, Dr. Lio suggested that the same anti-inflammatory effect would be anticipated for other skin conditions, including AD in children.
As a third example, Dr. Lio said topical indigo, a traditional Chinese medicine used for a variety of dermatologic conditions, including psoriasis, has also demonstrated efficacy in a randomized trial, compared with vehicle for mild to severe AD.
Complementary medicines are not for everyone, but they may have a role when managing the expectations of parents who are not fully satisfied or express concern about regimens limited to mainstream therapies alone, according to Dr. Lio. In diseases that are not curable, such as AD, he thinks this is a strategy with potential for benefit and is reassuring to patients.
Another way to avoid moving to riskier or more expensive drugs quickly is to assure patients use the drugs that were prescribed first, according to Steven R. Feldman, MD, PhD, professor of dermatology, Wake Forest University, Winston-Salem, N.C.
Dr. Feldman believes that failure to adhere to therapy is basically the fault of the medical care system, not the patient. He made an analogy to a successful piano teacher, who provides a child with sheet music and then sees the child once a week to track progress. He juxtaposed this piano teacher to one who gives the child sheet music and tells the child to come back in 10 weeks for the recital. It is not hard to guess which approach would be more effective.
“Typically, doctors are worse than that second teacher,” he said. “Doctors are like a piano teacher that does not give you the sheet music but says, ‘Here is a prescription for some sheet music. Take this prescription to the sheet music store. I have no idea how much it will cost or whether your insurance will pay for it. But once you fill this prescription for sheet music, I want you to practice this every day,’ ” he said, adding, “Practicing this sheet music may cause rashes, diarrhea, or serious infection. When the patient next comes in 10-12 weeks later and is not better, the doctor says, ‘I will give you a harder piece of sheet music and maybe two or three other instruments to practice at the same time,’ ” said Dr. Feldman, expressing why the way clinicians practice might explain much of the poor adherence problem.
This largely explains why patients with AD do not immediately respond to the therapies doctors prescribe, Dr. Feldman implied, reiterating the theme that emerged from the AD panel: Better and more options are needed for AD of the most severe types, but better management, not better drugs, is typically what is needed for most patients.
Dr. Feldman, Dr. Lio, and Dr. Paller have financial relationships with more than 30 pharmaceutical and cosmetic companies, some of which manufacture therapies for atopic dermatitis.
This article was updated July 28, 2023, to clarify the comments and viewpoints of Dr. Amy Paller.
A version of this article first appeared on Medscape.com.
ASHEVILLE, N.C. – according to a three-member expert panel mulling over strategies at the annual meeting of the Society for Pediatric Dermatology.
In introductory remarks, the three panelists briefly addressed different aspects for controlling AD, including drugs in the pipeline, the potential value of alternative therapies, and whom to blame when compliance is poor.
But panel discussion following these presentations provided an opportunity for audience engagement on practical strategies for improving AD control.
In her formal remarks prior to the panel discussion, Amy S. Paller, MD, professor of dermatology and pediatrics and chair of dermatology, Northwestern University, Chicago, and a pediatric dermatologist at the Lurie Children’s Hospital of Chicago, described emerging AD treatments. This included an update on the status of the interleukin-13 (IL-13) inhibitors tralokinumab (Adbry), which was approved by the FDA for treating AD in adults in December 2021, and lebrikizumab, which is thought likely to be soon approved in the United States on the basis of two recently published phase 3 trials.
Along with dupilumab (Dupixent) for moderate-to-severe AD in children who do not respond to optimized use of topical therapies, these new biologics appear likely to further expand choices for AD control for adults (and for kids with AD too, if eventually licensed in children), according to the data from the phase 3 studies.
During a panel discussion that followed, Stephen Gellis, MD, pediatric dermatologist and former chief of pediatric dermatology at Boston Children’s Hospital and Harvard Medical School, raised the point of optimizing tried and true topical therapies before using systemic agents. He noted that parents sometimes pressure clinicians to use a biologic – and that moving too quickly to the latest and most expensive drugs may not be necessary.
Dr. Paller acknowledged that she, like many pediatric dermatologists, employed immunosuppressants as her drugs of choice for many years – commonly starting with a few months of cyclosporine before transitioning to methotrexate, which has a delayed onset of action. In fact, she still uses this regimen in some children.
However, she now prefers dupilumab, which is the first biologic available for children in the United States with an AD indication in children as young as 6 months. She said dupilumab has fewer potential risks than cyclosporine, and it offers clinically meaningful improvement in most children. She noted that current guidelines discourage the use of systemic corticosteroids for AD in children, given their potential toxicity.
She strongly agreed with Dr. Gellis that clinicians should resist pressure to use any systemic agent if children are responding well to topical medications. In her own practice, Dr. Paller moves to systemic medications only after ensuring that there has been adherence to appropriate therapy and that there is not another diagnosis that might explain the recalcitrance to topical agents.
When a systemic medication is considered the next step, Dr. Paller reminded the audience of the importance of presenting the benefits and risks of all the options for AD control, which could include dupilumab and immunosuppressants as initial systemic therapy.
“Many parents choose biologic treatment first, given its lack of requirement for blood monitoring and faster action than methotrexate,” Dr. Paller noted.
Nevertheless, “biologics are much more costly than immunosuppressants, require an injection – which is stressful for the child and the parents – and may not be accessible for our patients,” Dr. Paller said. Cyclosporine and methotrexate are effective and are often the best options for moderate to severe disease in areas of the world where dupilumab is not available, but Dr. Paller most commonly uses these therapies only when reimbursement for dupilumab cannot be secured, injection is not an option, or when dupilumab is not sufficiently effective and tolerated.
Providing different perspectives, the two other panelists discussing the treatment of pediatric AD also saw a role for ensuring that topical agents are not offering adequate AD control before turning to the latest and most sophisticated therapies for AD.
For meeting parent expectations when children are improving slowly on topical therapies, Peter A. Lio, MD, director of the Chicago Integrative Eczema Center and clinical assistant professor of dermatology and pediatrics at Northwestern University, suggested that integrative medicine might be helpful.
For parents not fully comfortable with standard pharmacologic agents, Dr. Lio said there is evidence to support some of the complementary approaches, and these can be reassuring to parents with an interest in alternative medicines.
In Western medicine, it is common to hear terms like “attack,” “kill,” and “suppress,” disease, but alternative therapies are generally coupled with terms like “restore,” “strengthen,” and “tonify,” he said. “Who doesn’t want to be tonified?” he asked, noting that there are many sources of data suggesting that the number of patients seeking alternative medicine is “huge.” The alternative medicines are not generally taught in medical school and remain widely ignored in typical practice, but “our patients are interested even if we are not.”
Yet, there are data to support benefit from some of these alternative therapies, providing a win-win situation for patients who derive satisfaction from nontraditional therapies alone or combined with established pharmaceutical treatments.
Of these, Dr. Lio said there is support for the use of hempseed oil as a moisturizing agent and a strategy for improving barrier function in the skin of patients with AD. In a controlled crossover study, 2 teaspoons per day of dietary hempseed oil, a product that can be purchased in some grocery stores, was associated with significant reductions in skin dryness, itchiness, and use of topical medications relative to the same amount of olive oil, he noted.
Other examples include a compress made with black tea that was associated with an anti-inflammatory effect when followed by a moisturizer, a published study asserts. Although this was a trial in adults with facial dermatitis, Dr. Lio suggested that the same anti-inflammatory effect would be anticipated for other skin conditions, including AD in children.
As a third example, Dr. Lio said topical indigo, a traditional Chinese medicine used for a variety of dermatologic conditions, including psoriasis, has also demonstrated efficacy in a randomized trial, compared with vehicle for mild to severe AD.
Complementary medicines are not for everyone, but they may have a role when managing the expectations of parents who are not fully satisfied or express concern about regimens limited to mainstream therapies alone, according to Dr. Lio. In diseases that are not curable, such as AD, he thinks this is a strategy with potential for benefit and is reassuring to patients.
Another way to avoid moving to riskier or more expensive drugs quickly is to assure patients use the drugs that were prescribed first, according to Steven R. Feldman, MD, PhD, professor of dermatology, Wake Forest University, Winston-Salem, N.C.
Dr. Feldman believes that failure to adhere to therapy is basically the fault of the medical care system, not the patient. He made an analogy to a successful piano teacher, who provides a child with sheet music and then sees the child once a week to track progress. He juxtaposed this piano teacher to one who gives the child sheet music and tells the child to come back in 10 weeks for the recital. It is not hard to guess which approach would be more effective.
“Typically, doctors are worse than that second teacher,” he said. “Doctors are like a piano teacher that does not give you the sheet music but says, ‘Here is a prescription for some sheet music. Take this prescription to the sheet music store. I have no idea how much it will cost or whether your insurance will pay for it. But once you fill this prescription for sheet music, I want you to practice this every day,’ ” he said, adding, “Practicing this sheet music may cause rashes, diarrhea, or serious infection. When the patient next comes in 10-12 weeks later and is not better, the doctor says, ‘I will give you a harder piece of sheet music and maybe two or three other instruments to practice at the same time,’ ” said Dr. Feldman, expressing why the way clinicians practice might explain much of the poor adherence problem.
This largely explains why patients with AD do not immediately respond to the therapies doctors prescribe, Dr. Feldman implied, reiterating the theme that emerged from the AD panel: Better and more options are needed for AD of the most severe types, but better management, not better drugs, is typically what is needed for most patients.
Dr. Feldman, Dr. Lio, and Dr. Paller have financial relationships with more than 30 pharmaceutical and cosmetic companies, some of which manufacture therapies for atopic dermatitis.
This article was updated July 28, 2023, to clarify the comments and viewpoints of Dr. Amy Paller.
A version of this article first appeared on Medscape.com.
ASHEVILLE, N.C. – according to a three-member expert panel mulling over strategies at the annual meeting of the Society for Pediatric Dermatology.
In introductory remarks, the three panelists briefly addressed different aspects for controlling AD, including drugs in the pipeline, the potential value of alternative therapies, and whom to blame when compliance is poor.
But panel discussion following these presentations provided an opportunity for audience engagement on practical strategies for improving AD control.
In her formal remarks prior to the panel discussion, Amy S. Paller, MD, professor of dermatology and pediatrics and chair of dermatology, Northwestern University, Chicago, and a pediatric dermatologist at the Lurie Children’s Hospital of Chicago, described emerging AD treatments. This included an update on the status of the interleukin-13 (IL-13) inhibitors tralokinumab (Adbry), which was approved by the FDA for treating AD in adults in December 2021, and lebrikizumab, which is thought likely to be soon approved in the United States on the basis of two recently published phase 3 trials.
Along with dupilumab (Dupixent) for moderate-to-severe AD in children who do not respond to optimized use of topical therapies, these new biologics appear likely to further expand choices for AD control for adults (and for kids with AD too, if eventually licensed in children), according to the data from the phase 3 studies.
During a panel discussion that followed, Stephen Gellis, MD, pediatric dermatologist and former chief of pediatric dermatology at Boston Children’s Hospital and Harvard Medical School, raised the point of optimizing tried and true topical therapies before using systemic agents. He noted that parents sometimes pressure clinicians to use a biologic – and that moving too quickly to the latest and most expensive drugs may not be necessary.
Dr. Paller acknowledged that she, like many pediatric dermatologists, employed immunosuppressants as her drugs of choice for many years – commonly starting with a few months of cyclosporine before transitioning to methotrexate, which has a delayed onset of action. In fact, she still uses this regimen in some children.
However, she now prefers dupilumab, which is the first biologic available for children in the United States with an AD indication in children as young as 6 months. She said dupilumab has fewer potential risks than cyclosporine, and it offers clinically meaningful improvement in most children. She noted that current guidelines discourage the use of systemic corticosteroids for AD in children, given their potential toxicity.
She strongly agreed with Dr. Gellis that clinicians should resist pressure to use any systemic agent if children are responding well to topical medications. In her own practice, Dr. Paller moves to systemic medications only after ensuring that there has been adherence to appropriate therapy and that there is not another diagnosis that might explain the recalcitrance to topical agents.
When a systemic medication is considered the next step, Dr. Paller reminded the audience of the importance of presenting the benefits and risks of all the options for AD control, which could include dupilumab and immunosuppressants as initial systemic therapy.
“Many parents choose biologic treatment first, given its lack of requirement for blood monitoring and faster action than methotrexate,” Dr. Paller noted.
Nevertheless, “biologics are much more costly than immunosuppressants, require an injection – which is stressful for the child and the parents – and may not be accessible for our patients,” Dr. Paller said. Cyclosporine and methotrexate are effective and are often the best options for moderate to severe disease in areas of the world where dupilumab is not available, but Dr. Paller most commonly uses these therapies only when reimbursement for dupilumab cannot be secured, injection is not an option, or when dupilumab is not sufficiently effective and tolerated.
Providing different perspectives, the two other panelists discussing the treatment of pediatric AD also saw a role for ensuring that topical agents are not offering adequate AD control before turning to the latest and most sophisticated therapies for AD.
For meeting parent expectations when children are improving slowly on topical therapies, Peter A. Lio, MD, director of the Chicago Integrative Eczema Center and clinical assistant professor of dermatology and pediatrics at Northwestern University, suggested that integrative medicine might be helpful.
For parents not fully comfortable with standard pharmacologic agents, Dr. Lio said there is evidence to support some of the complementary approaches, and these can be reassuring to parents with an interest in alternative medicines.
In Western medicine, it is common to hear terms like “attack,” “kill,” and “suppress,” disease, but alternative therapies are generally coupled with terms like “restore,” “strengthen,” and “tonify,” he said. “Who doesn’t want to be tonified?” he asked, noting that there are many sources of data suggesting that the number of patients seeking alternative medicine is “huge.” The alternative medicines are not generally taught in medical school and remain widely ignored in typical practice, but “our patients are interested even if we are not.”
Yet, there are data to support benefit from some of these alternative therapies, providing a win-win situation for patients who derive satisfaction from nontraditional therapies alone or combined with established pharmaceutical treatments.
Of these, Dr. Lio said there is support for the use of hempseed oil as a moisturizing agent and a strategy for improving barrier function in the skin of patients with AD. In a controlled crossover study, 2 teaspoons per day of dietary hempseed oil, a product that can be purchased in some grocery stores, was associated with significant reductions in skin dryness, itchiness, and use of topical medications relative to the same amount of olive oil, he noted.
Other examples include a compress made with black tea that was associated with an anti-inflammatory effect when followed by a moisturizer, a published study asserts. Although this was a trial in adults with facial dermatitis, Dr. Lio suggested that the same anti-inflammatory effect would be anticipated for other skin conditions, including AD in children.
As a third example, Dr. Lio said topical indigo, a traditional Chinese medicine used for a variety of dermatologic conditions, including psoriasis, has also demonstrated efficacy in a randomized trial, compared with vehicle for mild to severe AD.
Complementary medicines are not for everyone, but they may have a role when managing the expectations of parents who are not fully satisfied or express concern about regimens limited to mainstream therapies alone, according to Dr. Lio. In diseases that are not curable, such as AD, he thinks this is a strategy with potential for benefit and is reassuring to patients.
Another way to avoid moving to riskier or more expensive drugs quickly is to assure patients use the drugs that were prescribed first, according to Steven R. Feldman, MD, PhD, professor of dermatology, Wake Forest University, Winston-Salem, N.C.
Dr. Feldman believes that failure to adhere to therapy is basically the fault of the medical care system, not the patient. He made an analogy to a successful piano teacher, who provides a child with sheet music and then sees the child once a week to track progress. He juxtaposed this piano teacher to one who gives the child sheet music and tells the child to come back in 10 weeks for the recital. It is not hard to guess which approach would be more effective.
“Typically, doctors are worse than that second teacher,” he said. “Doctors are like a piano teacher that does not give you the sheet music but says, ‘Here is a prescription for some sheet music. Take this prescription to the sheet music store. I have no idea how much it will cost or whether your insurance will pay for it. But once you fill this prescription for sheet music, I want you to practice this every day,’ ” he said, adding, “Practicing this sheet music may cause rashes, diarrhea, or serious infection. When the patient next comes in 10-12 weeks later and is not better, the doctor says, ‘I will give you a harder piece of sheet music and maybe two or three other instruments to practice at the same time,’ ” said Dr. Feldman, expressing why the way clinicians practice might explain much of the poor adherence problem.
This largely explains why patients with AD do not immediately respond to the therapies doctors prescribe, Dr. Feldman implied, reiterating the theme that emerged from the AD panel: Better and more options are needed for AD of the most severe types, but better management, not better drugs, is typically what is needed for most patients.
Dr. Feldman, Dr. Lio, and Dr. Paller have financial relationships with more than 30 pharmaceutical and cosmetic companies, some of which manufacture therapies for atopic dermatitis.
This article was updated July 28, 2023, to clarify the comments and viewpoints of Dr. Amy Paller.
A version of this article first appeared on Medscape.com.
AT SPD 2023
Ocular complications of dermatologic treatments: Advice from a pediatric ophthalmologist
ASHEVILLE, N.C. – The, according to one of several clinical messages from a pediatric ophthalmologist who spoke at the annual meeting of the Society for Pediatric Dermatology.
“There is a lot of steroid fear out there, which you can argue is actually harmful in itself, because not treating periorbital eczema is related to a lot of eye problems, including chronic discomfort and the eye rubbing that can cause corneal abrasions and keratoconus,” said Sara Grace, MD, a pediatric ophthalmologist who is on the clinical staff at Duke University, Durham, N.C. She maintains a practice at North Carolina Eye, Ear, Nose, and Throat in Durham.
Although the risks of periorbital steroid absorption are real, a limited course of low potency topical steroids is generally adequate for common periorbital indications, and these appear to be safe.
“There is insufficient evidence to link weak periocular topical corticosteroids such as desonide or hydrocortisone with ocular complications,” said Dr. Grace, suggesting that pediatric dermatologists can be reassured when using these medications at low concentrations.
“Potent periocular steroids have been associated with ocular complications, but this has typically involved exposures over months to years,” Dr. Grace specified.
When topical corticosteroids are applied at high concentrations on the face away from the periorbital area, glaucoma and other feared ophthalmic complications cannot be entirely ruled out, but, again, the risk is low in the absence of “very large quantities” of potent topical agents applied for lengthy periods of time, according to Dr. Grace, basing this observation on case studies.
In children, as in adults, the potential exception is a child with existing ocular disease. In such cases, or in children with risk factors for ocular disease, Dr. Grace recommends referral to an ophthalmologist for a baseline examination prior to a course of topical corticosteroids with the potential of periocular absorption. With a baseline assessment, adverse effects are more easily documented if exposure is prolonged.
The message, although not identical, is similar for use of dupilumab (Dupixent) or other biologics that target the interleukin-13 (IL-13) pathway. The potential for complications cannot be ignored but these are often time-limited and the benefit is likely to exceed the risk in children who have severe atopic dermatitis or other skin conditions for which these treatments are effective.
There are several potential mechanisms by which biologics targeting IL-13 might increase risk of ocular complications, one of which is the role that IL-13 plays in ocular mucus production, regulation of conjunctival goblet cells, and tear production, according to several published reports.
“Up to 30% of children will get some type of eye complication but, fortunately, most of them will not have to stop therapy,” Dr. Grace said. These side effects include conjunctivitis, blepharitis, keratitis, dry eye, and itching, but they are typically manageable. Topical steroids or calcineurin inhibitors can be offered if needed, but many of these conditions will self-resolve. Dr. Grace estimated that less than 1% of patients need to stop treatment because of ophthalmic side effects.
Lesions that obstruct vision
Dr. Grace urged pediatric dermatologists to be aware of the risk for amblyopia in young children with lesions that obstruct vision in one eye. In early development, prolonged obstruction of vision in one eye can alter neural communication with the brain, producing permanent vision impairment.
She explained that clearing the obstructed vision, whether from a capillary hemangioma or any periorbital growth, should be considered urgent to avoid irreversible damage.
Similarly, periorbital port-wine stains associated with Sturge-Weber syndrome, which is primarily a vascular disorder that predisposes children to glaucoma, represents a condition that requires prompt attention. Sturge-Weber syndrome is often but not always identified at birth, but it is a condition for which evaluation and treatment should involve the participation of an ophthalmologist.
Meibomian gland disease is another disorder that is often seen first by a pediatric dermatologist but also requires collaborative management. The challenge is sorting out the underlying cause or causes and initiating a therapy that unclogs the gland without having to resort to incision and drainage.
“Drainage is hard to do and is not necessarily effective,” explained Dr. Grace. While scrubs, warmth, and massage frequently are adequate to unclog the gland – which secretes meibum, a complex of lipids that perform several functions in protecting the eye – therapies specific to the cause, such as Demodex-related blepharitis, chalazions, and styes, might be needed.
Dr. Grace indicated that patience is often needed. The process of unclogging these glands often takes time, but she emphasized that a first-line conservative approach is always appropriate to avoid the difficulty and potential problems of incisions.
In general, these messages are not novel, but they provide a refresher for pediatric dermatologists who do not regularly confront complications that involve the eyes. According to session moderator, Elizabeth Neiman, MD, assistant professor of pediatric dermatology, University of North Carolina at Chapel Hill, the messages regarding topical steroids on the face and the eyes are “important” and worth emphasizing.
“It’s useful to reinforce the point that corticosteroids should be used when needed in the periorbital area [to control skin diseases] if they are used in low concentrations,” Dr. Neiman told this news organization.
Similarly, conjunctivitis and other ocular complications of dupilumab are a source of concern for parents as well as dermatologists. Dr. Neiman indicated that a review of the benefit-to-risk ratio is important when considering these treatments in patients with indications for severe skin disorders.
Dr. Grace and Dr. Nieman have no potential financial conflicts related to this topic.
A version of this article first appeared on Medscape.com.
ASHEVILLE, N.C. – The, according to one of several clinical messages from a pediatric ophthalmologist who spoke at the annual meeting of the Society for Pediatric Dermatology.
“There is a lot of steroid fear out there, which you can argue is actually harmful in itself, because not treating periorbital eczema is related to a lot of eye problems, including chronic discomfort and the eye rubbing that can cause corneal abrasions and keratoconus,” said Sara Grace, MD, a pediatric ophthalmologist who is on the clinical staff at Duke University, Durham, N.C. She maintains a practice at North Carolina Eye, Ear, Nose, and Throat in Durham.
Although the risks of periorbital steroid absorption are real, a limited course of low potency topical steroids is generally adequate for common periorbital indications, and these appear to be safe.
“There is insufficient evidence to link weak periocular topical corticosteroids such as desonide or hydrocortisone with ocular complications,” said Dr. Grace, suggesting that pediatric dermatologists can be reassured when using these medications at low concentrations.
“Potent periocular steroids have been associated with ocular complications, but this has typically involved exposures over months to years,” Dr. Grace specified.
When topical corticosteroids are applied at high concentrations on the face away from the periorbital area, glaucoma and other feared ophthalmic complications cannot be entirely ruled out, but, again, the risk is low in the absence of “very large quantities” of potent topical agents applied for lengthy periods of time, according to Dr. Grace, basing this observation on case studies.
In children, as in adults, the potential exception is a child with existing ocular disease. In such cases, or in children with risk factors for ocular disease, Dr. Grace recommends referral to an ophthalmologist for a baseline examination prior to a course of topical corticosteroids with the potential of periocular absorption. With a baseline assessment, adverse effects are more easily documented if exposure is prolonged.
The message, although not identical, is similar for use of dupilumab (Dupixent) or other biologics that target the interleukin-13 (IL-13) pathway. The potential for complications cannot be ignored but these are often time-limited and the benefit is likely to exceed the risk in children who have severe atopic dermatitis or other skin conditions for which these treatments are effective.
There are several potential mechanisms by which biologics targeting IL-13 might increase risk of ocular complications, one of which is the role that IL-13 plays in ocular mucus production, regulation of conjunctival goblet cells, and tear production, according to several published reports.
“Up to 30% of children will get some type of eye complication but, fortunately, most of them will not have to stop therapy,” Dr. Grace said. These side effects include conjunctivitis, blepharitis, keratitis, dry eye, and itching, but they are typically manageable. Topical steroids or calcineurin inhibitors can be offered if needed, but many of these conditions will self-resolve. Dr. Grace estimated that less than 1% of patients need to stop treatment because of ophthalmic side effects.
Lesions that obstruct vision
Dr. Grace urged pediatric dermatologists to be aware of the risk for amblyopia in young children with lesions that obstruct vision in one eye. In early development, prolonged obstruction of vision in one eye can alter neural communication with the brain, producing permanent vision impairment.
She explained that clearing the obstructed vision, whether from a capillary hemangioma or any periorbital growth, should be considered urgent to avoid irreversible damage.
Similarly, periorbital port-wine stains associated with Sturge-Weber syndrome, which is primarily a vascular disorder that predisposes children to glaucoma, represents a condition that requires prompt attention. Sturge-Weber syndrome is often but not always identified at birth, but it is a condition for which evaluation and treatment should involve the participation of an ophthalmologist.
Meibomian gland disease is another disorder that is often seen first by a pediatric dermatologist but also requires collaborative management. The challenge is sorting out the underlying cause or causes and initiating a therapy that unclogs the gland without having to resort to incision and drainage.
“Drainage is hard to do and is not necessarily effective,” explained Dr. Grace. While scrubs, warmth, and massage frequently are adequate to unclog the gland – which secretes meibum, a complex of lipids that perform several functions in protecting the eye – therapies specific to the cause, such as Demodex-related blepharitis, chalazions, and styes, might be needed.
Dr. Grace indicated that patience is often needed. The process of unclogging these glands often takes time, but she emphasized that a first-line conservative approach is always appropriate to avoid the difficulty and potential problems of incisions.
In general, these messages are not novel, but they provide a refresher for pediatric dermatologists who do not regularly confront complications that involve the eyes. According to session moderator, Elizabeth Neiman, MD, assistant professor of pediatric dermatology, University of North Carolina at Chapel Hill, the messages regarding topical steroids on the face and the eyes are “important” and worth emphasizing.
“It’s useful to reinforce the point that corticosteroids should be used when needed in the periorbital area [to control skin diseases] if they are used in low concentrations,” Dr. Neiman told this news organization.
Similarly, conjunctivitis and other ocular complications of dupilumab are a source of concern for parents as well as dermatologists. Dr. Neiman indicated that a review of the benefit-to-risk ratio is important when considering these treatments in patients with indications for severe skin disorders.
Dr. Grace and Dr. Nieman have no potential financial conflicts related to this topic.
A version of this article first appeared on Medscape.com.
ASHEVILLE, N.C. – The, according to one of several clinical messages from a pediatric ophthalmologist who spoke at the annual meeting of the Society for Pediatric Dermatology.
“There is a lot of steroid fear out there, which you can argue is actually harmful in itself, because not treating periorbital eczema is related to a lot of eye problems, including chronic discomfort and the eye rubbing that can cause corneal abrasions and keratoconus,” said Sara Grace, MD, a pediatric ophthalmologist who is on the clinical staff at Duke University, Durham, N.C. She maintains a practice at North Carolina Eye, Ear, Nose, and Throat in Durham.
Although the risks of periorbital steroid absorption are real, a limited course of low potency topical steroids is generally adequate for common periorbital indications, and these appear to be safe.
“There is insufficient evidence to link weak periocular topical corticosteroids such as desonide or hydrocortisone with ocular complications,” said Dr. Grace, suggesting that pediatric dermatologists can be reassured when using these medications at low concentrations.
“Potent periocular steroids have been associated with ocular complications, but this has typically involved exposures over months to years,” Dr. Grace specified.
When topical corticosteroids are applied at high concentrations on the face away from the periorbital area, glaucoma and other feared ophthalmic complications cannot be entirely ruled out, but, again, the risk is low in the absence of “very large quantities” of potent topical agents applied for lengthy periods of time, according to Dr. Grace, basing this observation on case studies.
In children, as in adults, the potential exception is a child with existing ocular disease. In such cases, or in children with risk factors for ocular disease, Dr. Grace recommends referral to an ophthalmologist for a baseline examination prior to a course of topical corticosteroids with the potential of periocular absorption. With a baseline assessment, adverse effects are more easily documented if exposure is prolonged.
The message, although not identical, is similar for use of dupilumab (Dupixent) or other biologics that target the interleukin-13 (IL-13) pathway. The potential for complications cannot be ignored but these are often time-limited and the benefit is likely to exceed the risk in children who have severe atopic dermatitis or other skin conditions for which these treatments are effective.
There are several potential mechanisms by which biologics targeting IL-13 might increase risk of ocular complications, one of which is the role that IL-13 plays in ocular mucus production, regulation of conjunctival goblet cells, and tear production, according to several published reports.
“Up to 30% of children will get some type of eye complication but, fortunately, most of them will not have to stop therapy,” Dr. Grace said. These side effects include conjunctivitis, blepharitis, keratitis, dry eye, and itching, but they are typically manageable. Topical steroids or calcineurin inhibitors can be offered if needed, but many of these conditions will self-resolve. Dr. Grace estimated that less than 1% of patients need to stop treatment because of ophthalmic side effects.
Lesions that obstruct vision
Dr. Grace urged pediatric dermatologists to be aware of the risk for amblyopia in young children with lesions that obstruct vision in one eye. In early development, prolonged obstruction of vision in one eye can alter neural communication with the brain, producing permanent vision impairment.
She explained that clearing the obstructed vision, whether from a capillary hemangioma or any periorbital growth, should be considered urgent to avoid irreversible damage.
Similarly, periorbital port-wine stains associated with Sturge-Weber syndrome, which is primarily a vascular disorder that predisposes children to glaucoma, represents a condition that requires prompt attention. Sturge-Weber syndrome is often but not always identified at birth, but it is a condition for which evaluation and treatment should involve the participation of an ophthalmologist.
Meibomian gland disease is another disorder that is often seen first by a pediatric dermatologist but also requires collaborative management. The challenge is sorting out the underlying cause or causes and initiating a therapy that unclogs the gland without having to resort to incision and drainage.
“Drainage is hard to do and is not necessarily effective,” explained Dr. Grace. While scrubs, warmth, and massage frequently are adequate to unclog the gland – which secretes meibum, a complex of lipids that perform several functions in protecting the eye – therapies specific to the cause, such as Demodex-related blepharitis, chalazions, and styes, might be needed.
Dr. Grace indicated that patience is often needed. The process of unclogging these glands often takes time, but she emphasized that a first-line conservative approach is always appropriate to avoid the difficulty and potential problems of incisions.
In general, these messages are not novel, but they provide a refresher for pediatric dermatologists who do not regularly confront complications that involve the eyes. According to session moderator, Elizabeth Neiman, MD, assistant professor of pediatric dermatology, University of North Carolina at Chapel Hill, the messages regarding topical steroids on the face and the eyes are “important” and worth emphasizing.
“It’s useful to reinforce the point that corticosteroids should be used when needed in the periorbital area [to control skin diseases] if they are used in low concentrations,” Dr. Neiman told this news organization.
Similarly, conjunctivitis and other ocular complications of dupilumab are a source of concern for parents as well as dermatologists. Dr. Neiman indicated that a review of the benefit-to-risk ratio is important when considering these treatments in patients with indications for severe skin disorders.
Dr. Grace and Dr. Nieman have no potential financial conflicts related to this topic.
A version of this article first appeared on Medscape.com.
AT SPD 2023
Case report describes pediatric RIME triggered by norovirus
, according to a newly published case report.
Lead author Anna Yasmine Kirkorian, MD, chief of dermatology at Children’s National Hospital in Washington, said she wanted to get the word out in part because it seems like RIME is occurring more frequently. “I do feel like we’re seeing more cases and from a more diverse number of pathogens,” Dr. Kirkorian told this news organization.
There was a decrease in RIME during the early stages of the COVID-19 pandemic when people were isolating more, Dr. Kirkorian said. SARS-CoV-2 has been a trigger for some cases, but she did not find that remarkable, given that respiratory viruses are known RIME precursors. The question is why RIME is being triggered more frequently now that people have essentially gone back to their normal lives, she said.
Dr. Kirkorian and colleagues at Children’s National Hospital and George Washington University, Washington, wrote about a 5-year-old boy with norovirus-triggered RIME in a case report published in Pediatric Dermatology.
RIME – previously known as Mycoplasma pneumoniae–induced rash and mucositis (MIRM) – tends to arise after a viral infection, with upper respiratory viruses such as mycoplasma and Chlamydophila pneumoniae, influenza, and enterovirus among the common triggers. “We think this is actually your own immune system overreacting to a pathogen,” Dr. Kirkorian said in an interview, adding that the mechanism of RIME is still not understood.
While the norovirus discovery was a surprise, it shows that much is still unknown about this rare condition. “I don’t think we know what is usual and what is unusual,” Dr. Kirkorian said.
In this case, the boy swiftly declined, with progressive conjunctivitis, high fever, and rapidly developing mucositis. By the time the 5-year-old got to Children’s National Hospital, he had a spreading, painful rash, including tense vesicles and bullae involving more than 30% of his total body surface area, and areas of denuded skin on both cheeks and the back of his neck.
He had hemorrhagic mucositis of the lips, a large erosion at the urethral meatus, and hemorrhagic conjunctivitis of both eyes with thick yellow crusting on the eyelids.
The clinicians intubated the boy and admitted him to the intensive care unit. He was given a one-time injection of etanercept (25 mg) followed by 8 days of intravenous cyclosporine at a dose of 5 mg per kilogram, divided twice daily, which helped calm the mucositis and stopped the rash from progressing. There is not an accepted protocol or list of evidence-based therapeutics for RIME, Dr. Kirkorian noted.
The severe eye damage required amniotic membrane grafts. The patient was extubated after 9 days but remained in the hospital for a total of 26 days because he needed to receive nutritional support (the mucositis kept him from eating), and for pain control and weaning of sedation.
As the clinicians searched for a potential triggering virus, they came up empty. Results were negative for adenovirus, Epstein Barr virus, cytomegalovirus, herpes simplex, and varicella zoster. But they noted that the child’s household contacts had all been sick a week before with presumed viral gastroenteritis. They decided to run a stool screen and the polymerase chain reaction for norovirus was positive. The boy never had GI symptoms.
Dr. Kirkorian said in the interview that she has seen other RIME cases where a child did not have symptoms associated with the original virus but did have a sudden onset of mucositis.
Although the definition of RIME is evolving, it is defined in part by mucositis in at least two of three areas: the mouth, eyes, and genitals. “Once you have the inflammation of the mucous membranes you should be on alert to think about more serious conditions,” like RIME, said Dr. Kirkorian. “Why does it manifest with the mucositis? I don’t think we know that,” she added.
RIME recurrence has also been vexing for patients, families and clinicians. In May, at the annual Atlantic Dermatology Conference, held in Baltimore, Dr. Kirkorian also discussed an 11-year-old patient who had RIME after SARS-CoV-2 infection early in the pandemic, resulting in a 22-day hospitalization and placement of a peripherally inserted central catheter and a feeding tube. He improved with cyclosporine and was discharged on systemic tacrolimus.
He was fine for several years, until another COVID infection. He again responded to medication. But not long after, an undetermined viral infection triggered another episode of RIME.
Dr. Kirkorian said there is no way to predict recurrence – making a devastating condition all the more worrisome. “Knowing that it might come back and it’s totally haphazard as to what might make it come back – that is very stressful for families,” she said in the interview.
“Some of the most perplexing patients with RIME are those with recurrent disease,” wrote Warren R. Heymann, MD, professor of dermatology and pediatrics at Rowan University, Camden, N.J., wrote in an online column on RIME in the American Academy of Dermatology’s “Dermatology World Insights and Inquiries”.
“Recurrent RIME is of particular interest, given that we could potentially intervene and prevent additional disease,” wrote Camille Introcaso, MD, associate professor of medicine at Rowan University, in response to Dr. Heymann’s remarks. “Although multiple possible mechanisms for the clinical findings of RIME have been proposed, including molecular mimicry between infectious agent proteins and keratinocyte antigens, immune complex deposition, and combinations of medication and infection, the pathophysiology is unknown,” she added.
In the interview, Dr. Kirkorian said that she and colleagues in the Pediatric Dermatology Research Alliance (PeDRA) are trying to assemble more multicenter trials to assess the underlying pathology of RIME, effectiveness of various treatments, and to “find some predictive factors.” Given that RIME is an acute-onset emergency, it is not easy to conduct randomized controlled trials, she added.
Dr. Kirkorian, Dr. Heymann, and Dr. Introcaso report no relevant financial relationships.
, according to a newly published case report.
Lead author Anna Yasmine Kirkorian, MD, chief of dermatology at Children’s National Hospital in Washington, said she wanted to get the word out in part because it seems like RIME is occurring more frequently. “I do feel like we’re seeing more cases and from a more diverse number of pathogens,” Dr. Kirkorian told this news organization.
There was a decrease in RIME during the early stages of the COVID-19 pandemic when people were isolating more, Dr. Kirkorian said. SARS-CoV-2 has been a trigger for some cases, but she did not find that remarkable, given that respiratory viruses are known RIME precursors. The question is why RIME is being triggered more frequently now that people have essentially gone back to their normal lives, she said.
Dr. Kirkorian and colleagues at Children’s National Hospital and George Washington University, Washington, wrote about a 5-year-old boy with norovirus-triggered RIME in a case report published in Pediatric Dermatology.
RIME – previously known as Mycoplasma pneumoniae–induced rash and mucositis (MIRM) – tends to arise after a viral infection, with upper respiratory viruses such as mycoplasma and Chlamydophila pneumoniae, influenza, and enterovirus among the common triggers. “We think this is actually your own immune system overreacting to a pathogen,” Dr. Kirkorian said in an interview, adding that the mechanism of RIME is still not understood.
While the norovirus discovery was a surprise, it shows that much is still unknown about this rare condition. “I don’t think we know what is usual and what is unusual,” Dr. Kirkorian said.
In this case, the boy swiftly declined, with progressive conjunctivitis, high fever, and rapidly developing mucositis. By the time the 5-year-old got to Children’s National Hospital, he had a spreading, painful rash, including tense vesicles and bullae involving more than 30% of his total body surface area, and areas of denuded skin on both cheeks and the back of his neck.
He had hemorrhagic mucositis of the lips, a large erosion at the urethral meatus, and hemorrhagic conjunctivitis of both eyes with thick yellow crusting on the eyelids.
The clinicians intubated the boy and admitted him to the intensive care unit. He was given a one-time injection of etanercept (25 mg) followed by 8 days of intravenous cyclosporine at a dose of 5 mg per kilogram, divided twice daily, which helped calm the mucositis and stopped the rash from progressing. There is not an accepted protocol or list of evidence-based therapeutics for RIME, Dr. Kirkorian noted.
The severe eye damage required amniotic membrane grafts. The patient was extubated after 9 days but remained in the hospital for a total of 26 days because he needed to receive nutritional support (the mucositis kept him from eating), and for pain control and weaning of sedation.
As the clinicians searched for a potential triggering virus, they came up empty. Results were negative for adenovirus, Epstein Barr virus, cytomegalovirus, herpes simplex, and varicella zoster. But they noted that the child’s household contacts had all been sick a week before with presumed viral gastroenteritis. They decided to run a stool screen and the polymerase chain reaction for norovirus was positive. The boy never had GI symptoms.
Dr. Kirkorian said in the interview that she has seen other RIME cases where a child did not have symptoms associated with the original virus but did have a sudden onset of mucositis.
Although the definition of RIME is evolving, it is defined in part by mucositis in at least two of three areas: the mouth, eyes, and genitals. “Once you have the inflammation of the mucous membranes you should be on alert to think about more serious conditions,” like RIME, said Dr. Kirkorian. “Why does it manifest with the mucositis? I don’t think we know that,” she added.
RIME recurrence has also been vexing for patients, families and clinicians. In May, at the annual Atlantic Dermatology Conference, held in Baltimore, Dr. Kirkorian also discussed an 11-year-old patient who had RIME after SARS-CoV-2 infection early in the pandemic, resulting in a 22-day hospitalization and placement of a peripherally inserted central catheter and a feeding tube. He improved with cyclosporine and was discharged on systemic tacrolimus.
He was fine for several years, until another COVID infection. He again responded to medication. But not long after, an undetermined viral infection triggered another episode of RIME.
Dr. Kirkorian said there is no way to predict recurrence – making a devastating condition all the more worrisome. “Knowing that it might come back and it’s totally haphazard as to what might make it come back – that is very stressful for families,” she said in the interview.
“Some of the most perplexing patients with RIME are those with recurrent disease,” wrote Warren R. Heymann, MD, professor of dermatology and pediatrics at Rowan University, Camden, N.J., wrote in an online column on RIME in the American Academy of Dermatology’s “Dermatology World Insights and Inquiries”.
“Recurrent RIME is of particular interest, given that we could potentially intervene and prevent additional disease,” wrote Camille Introcaso, MD, associate professor of medicine at Rowan University, in response to Dr. Heymann’s remarks. “Although multiple possible mechanisms for the clinical findings of RIME have been proposed, including molecular mimicry between infectious agent proteins and keratinocyte antigens, immune complex deposition, and combinations of medication and infection, the pathophysiology is unknown,” she added.
In the interview, Dr. Kirkorian said that she and colleagues in the Pediatric Dermatology Research Alliance (PeDRA) are trying to assemble more multicenter trials to assess the underlying pathology of RIME, effectiveness of various treatments, and to “find some predictive factors.” Given that RIME is an acute-onset emergency, it is not easy to conduct randomized controlled trials, she added.
Dr. Kirkorian, Dr. Heymann, and Dr. Introcaso report no relevant financial relationships.
, according to a newly published case report.
Lead author Anna Yasmine Kirkorian, MD, chief of dermatology at Children’s National Hospital in Washington, said she wanted to get the word out in part because it seems like RIME is occurring more frequently. “I do feel like we’re seeing more cases and from a more diverse number of pathogens,” Dr. Kirkorian told this news organization.
There was a decrease in RIME during the early stages of the COVID-19 pandemic when people were isolating more, Dr. Kirkorian said. SARS-CoV-2 has been a trigger for some cases, but she did not find that remarkable, given that respiratory viruses are known RIME precursors. The question is why RIME is being triggered more frequently now that people have essentially gone back to their normal lives, she said.
Dr. Kirkorian and colleagues at Children’s National Hospital and George Washington University, Washington, wrote about a 5-year-old boy with norovirus-triggered RIME in a case report published in Pediatric Dermatology.
RIME – previously known as Mycoplasma pneumoniae–induced rash and mucositis (MIRM) – tends to arise after a viral infection, with upper respiratory viruses such as mycoplasma and Chlamydophila pneumoniae, influenza, and enterovirus among the common triggers. “We think this is actually your own immune system overreacting to a pathogen,” Dr. Kirkorian said in an interview, adding that the mechanism of RIME is still not understood.
While the norovirus discovery was a surprise, it shows that much is still unknown about this rare condition. “I don’t think we know what is usual and what is unusual,” Dr. Kirkorian said.
In this case, the boy swiftly declined, with progressive conjunctivitis, high fever, and rapidly developing mucositis. By the time the 5-year-old got to Children’s National Hospital, he had a spreading, painful rash, including tense vesicles and bullae involving more than 30% of his total body surface area, and areas of denuded skin on both cheeks and the back of his neck.
He had hemorrhagic mucositis of the lips, a large erosion at the urethral meatus, and hemorrhagic conjunctivitis of both eyes with thick yellow crusting on the eyelids.
The clinicians intubated the boy and admitted him to the intensive care unit. He was given a one-time injection of etanercept (25 mg) followed by 8 days of intravenous cyclosporine at a dose of 5 mg per kilogram, divided twice daily, which helped calm the mucositis and stopped the rash from progressing. There is not an accepted protocol or list of evidence-based therapeutics for RIME, Dr. Kirkorian noted.
The severe eye damage required amniotic membrane grafts. The patient was extubated after 9 days but remained in the hospital for a total of 26 days because he needed to receive nutritional support (the mucositis kept him from eating), and for pain control and weaning of sedation.
As the clinicians searched for a potential triggering virus, they came up empty. Results were negative for adenovirus, Epstein Barr virus, cytomegalovirus, herpes simplex, and varicella zoster. But they noted that the child’s household contacts had all been sick a week before with presumed viral gastroenteritis. They decided to run a stool screen and the polymerase chain reaction for norovirus was positive. The boy never had GI symptoms.
Dr. Kirkorian said in the interview that she has seen other RIME cases where a child did not have symptoms associated with the original virus but did have a sudden onset of mucositis.
Although the definition of RIME is evolving, it is defined in part by mucositis in at least two of three areas: the mouth, eyes, and genitals. “Once you have the inflammation of the mucous membranes you should be on alert to think about more serious conditions,” like RIME, said Dr. Kirkorian. “Why does it manifest with the mucositis? I don’t think we know that,” she added.
RIME recurrence has also been vexing for patients, families and clinicians. In May, at the annual Atlantic Dermatology Conference, held in Baltimore, Dr. Kirkorian also discussed an 11-year-old patient who had RIME after SARS-CoV-2 infection early in the pandemic, resulting in a 22-day hospitalization and placement of a peripherally inserted central catheter and a feeding tube. He improved with cyclosporine and was discharged on systemic tacrolimus.
He was fine for several years, until another COVID infection. He again responded to medication. But not long after, an undetermined viral infection triggered another episode of RIME.
Dr. Kirkorian said there is no way to predict recurrence – making a devastating condition all the more worrisome. “Knowing that it might come back and it’s totally haphazard as to what might make it come back – that is very stressful for families,” she said in the interview.
“Some of the most perplexing patients with RIME are those with recurrent disease,” wrote Warren R. Heymann, MD, professor of dermatology and pediatrics at Rowan University, Camden, N.J., wrote in an online column on RIME in the American Academy of Dermatology’s “Dermatology World Insights and Inquiries”.
“Recurrent RIME is of particular interest, given that we could potentially intervene and prevent additional disease,” wrote Camille Introcaso, MD, associate professor of medicine at Rowan University, in response to Dr. Heymann’s remarks. “Although multiple possible mechanisms for the clinical findings of RIME have been proposed, including molecular mimicry between infectious agent proteins and keratinocyte antigens, immune complex deposition, and combinations of medication and infection, the pathophysiology is unknown,” she added.
In the interview, Dr. Kirkorian said that she and colleagues in the Pediatric Dermatology Research Alliance (PeDRA) are trying to assemble more multicenter trials to assess the underlying pathology of RIME, effectiveness of various treatments, and to “find some predictive factors.” Given that RIME is an acute-onset emergency, it is not easy to conduct randomized controlled trials, she added.
Dr. Kirkorian, Dr. Heymann, and Dr. Introcaso report no relevant financial relationships.
Humira biosimilars: Five things to know
The best-selling drug Humira (adalimumab) now faces competition in the United States after a 20-year monopoly. The first adalimumab biosimilar, Amjevita, launched in the United States on January 31, and in July, seven additional biosimilars became available. These drugs have the potential to lower prescription drug prices, but when and by how much remains to be seen.
Here’s what you need to know about adalimumab biosimilars.
What Humira biosimilars are now available?
Eight different biosimilars have launched in 2023 with discounts as large at 85% from Humira’s list price of $6,922. A few companies also offer two price points.
Three of these biosimilars – Hadlima, Hyrimoz, and Yuflyma – are available in high concentration formulations. This high concentration formulation makes up 85% of Humira prescriptions, according to a report from Goodroot, a collection of companies focused on lowering health care costs.
Cyltezo is currently the only adalimumab biosimilar with an interchangeability designation, meaning that a pharmacist can substitute the biosimilar for an equivalent Humira prescription without the intervention of a clinician. A total of 47 states allow for these substitutions without prior approval from a clinician, according to Goodroot, and the clinician must be notified of the switch within a certain time frame. A total of 40 states require that patients be notified of the switch before substitution.
However, it’s not clear if this interchangeability designation will prove an advantage for Cyltezo, as it is interchangeable with the lower concentration version of Humira that makes up just 15% of prescriptions.
Most of the companies behind these biosimilars are pursuing interchangeability designations for their drugs, except for Fresenius Kabi (Idacio) and Coherus (Yusimry).
A ninth biosimilar, Pfizer’s adalimumab-afzb (Abrilada), is not yet on the market and is currently awaiting an approval decision from the Food and Drug Administration to add an interchangeability designation to its prior approval for a low-concentration formulation.
Why are they priced differently?
The two price points offer different deals to payers. Pharmacy benefit managers make confidential agreements with drug manufacturers to get a discount – called a rebate – to get the drug on the PBM’s formulary. The PBM keeps a portion of that rebate, and the rest is passed on to the insurance company and patients. Biosimilars at a higher price point will likely offer larger rebates. Biosimilars offered at lower price points incorporate this discount up front in their list pricing and likely will not offer large rebates.
Will biosimilars be covered by payers?
Currently, biosimilars are being offered on formularies at parity with Humira, meaning they are on the same tier. The PBM companies OptumRx and Cigna Group’s Express Scripts will offer Amjevita (at both price points), Cyltezo, and Hyrimoz (at both price points).
“This decision allows our clients flexibility to provide access to the lower list price, so members in high-deductible plans and benefit designs with coinsurance can experience lower out-of-pocket costs,” said OptumRx spokesperson Isaac Sorensen in an email.
Mark Cuban Cost Plus Drug Company, which uses a direct-to-consumer model, will offer Yusimry for $567.27 on its website. SmithRx, a PBM based in San Francisco, announced it would partner with Cost Plus Drugs to offer Yusimry, adding that SmithRx members can use their insurance benefits to further reduce out-of-pocket costs. RxPreferred, another PBM, will also offer Yusimry through its partnership with Cuban’s company.
The news website Formulary Watch previously reported that CVS Caremark, another of the biggest PBMs, will be offering Amjevita, but as a nonpreferred brand, while Humira remains the preferred brand. CVS Caremark did not respond to a request for comment.
Will patients pay less?
Biosimilars have been touted as a potential solution to lower spending on biologic drugs, but it’s unknown if patients will ultimately benefit with lower out-of-pocket costs. It’s “impossible to predict” if the discount that third-party payers pay will be passed on to consumers, said Mark Fendrick, MD, who directs the University of Michigan Center for Value-based Insurance Design in Ann Arbor.
Generally, a consumer’s copay is a percentage of a drug’s list price, so it stands to reason that a low drug price would result in lower out-of-pocket payments. While this is mostly true, Humira has a successful copay assistance program to lower prescription costs for consumers. According to a 2022 IQVIA report, 82% of commercial prescriptions cost patients less than $10 for Humira because of this program.
To appeal to patients, biosimilar companies will need to offer similar savings, Dr. Fendrick added. “There will be some discontent if patients are actually asked to pay more out-of-pocket for a less expensive drug,” he said.
All eight companies behind these biosimilars are offering or will be launching copay saving programs, many which advertise copays as low as $0 per month for eligible patients.
How will Humira respond?
Marta Wosińska, PhD, a health care economist at the Brookings Institute, Washington, predicts payers will use these lower biosimilar prices to negotiate better deals with AbbVie, Humira’s manufacturer. “We have a lot of players coming into [the market] right now, so the competition is really fierce,” she said. In response, AbbVie will need to increase rebates on Humira and/or lower its price to compete with these biosimilars.
“The ball is in AbbVie’s court,” she said. “If [the company] is not willing to drop price sufficiently, then payers will start switching to biosimilars.”
Dr. Fendrick reported past financial relationships and consulting arrangements with AbbVie, Amgen, Arnold Ventures, Bayer, CareFirst, BlueCross BlueShield, and many other companies. Dr. Wosińska has received funding from Arnold Ventures and serves as an expert witness on antitrust cases involving generic medication.
A version of this article first appeared on Medscape.com.
The best-selling drug Humira (adalimumab) now faces competition in the United States after a 20-year monopoly. The first adalimumab biosimilar, Amjevita, launched in the United States on January 31, and in July, seven additional biosimilars became available. These drugs have the potential to lower prescription drug prices, but when and by how much remains to be seen.
Here’s what you need to know about adalimumab biosimilars.
What Humira biosimilars are now available?
Eight different biosimilars have launched in 2023 with discounts as large at 85% from Humira’s list price of $6,922. A few companies also offer two price points.
Three of these biosimilars – Hadlima, Hyrimoz, and Yuflyma – are available in high concentration formulations. This high concentration formulation makes up 85% of Humira prescriptions, according to a report from Goodroot, a collection of companies focused on lowering health care costs.
Cyltezo is currently the only adalimumab biosimilar with an interchangeability designation, meaning that a pharmacist can substitute the biosimilar for an equivalent Humira prescription without the intervention of a clinician. A total of 47 states allow for these substitutions without prior approval from a clinician, according to Goodroot, and the clinician must be notified of the switch within a certain time frame. A total of 40 states require that patients be notified of the switch before substitution.
However, it’s not clear if this interchangeability designation will prove an advantage for Cyltezo, as it is interchangeable with the lower concentration version of Humira that makes up just 15% of prescriptions.
Most of the companies behind these biosimilars are pursuing interchangeability designations for their drugs, except for Fresenius Kabi (Idacio) and Coherus (Yusimry).
A ninth biosimilar, Pfizer’s adalimumab-afzb (Abrilada), is not yet on the market and is currently awaiting an approval decision from the Food and Drug Administration to add an interchangeability designation to its prior approval for a low-concentration formulation.
Why are they priced differently?
The two price points offer different deals to payers. Pharmacy benefit managers make confidential agreements with drug manufacturers to get a discount – called a rebate – to get the drug on the PBM’s formulary. The PBM keeps a portion of that rebate, and the rest is passed on to the insurance company and patients. Biosimilars at a higher price point will likely offer larger rebates. Biosimilars offered at lower price points incorporate this discount up front in their list pricing and likely will not offer large rebates.
Will biosimilars be covered by payers?
Currently, biosimilars are being offered on formularies at parity with Humira, meaning they are on the same tier. The PBM companies OptumRx and Cigna Group’s Express Scripts will offer Amjevita (at both price points), Cyltezo, and Hyrimoz (at both price points).
“This decision allows our clients flexibility to provide access to the lower list price, so members in high-deductible plans and benefit designs with coinsurance can experience lower out-of-pocket costs,” said OptumRx spokesperson Isaac Sorensen in an email.
Mark Cuban Cost Plus Drug Company, which uses a direct-to-consumer model, will offer Yusimry for $567.27 on its website. SmithRx, a PBM based in San Francisco, announced it would partner with Cost Plus Drugs to offer Yusimry, adding that SmithRx members can use their insurance benefits to further reduce out-of-pocket costs. RxPreferred, another PBM, will also offer Yusimry through its partnership with Cuban’s company.
The news website Formulary Watch previously reported that CVS Caremark, another of the biggest PBMs, will be offering Amjevita, but as a nonpreferred brand, while Humira remains the preferred brand. CVS Caremark did not respond to a request for comment.
Will patients pay less?
Biosimilars have been touted as a potential solution to lower spending on biologic drugs, but it’s unknown if patients will ultimately benefit with lower out-of-pocket costs. It’s “impossible to predict” if the discount that third-party payers pay will be passed on to consumers, said Mark Fendrick, MD, who directs the University of Michigan Center for Value-based Insurance Design in Ann Arbor.
Generally, a consumer’s copay is a percentage of a drug’s list price, so it stands to reason that a low drug price would result in lower out-of-pocket payments. While this is mostly true, Humira has a successful copay assistance program to lower prescription costs for consumers. According to a 2022 IQVIA report, 82% of commercial prescriptions cost patients less than $10 for Humira because of this program.
To appeal to patients, biosimilar companies will need to offer similar savings, Dr. Fendrick added. “There will be some discontent if patients are actually asked to pay more out-of-pocket for a less expensive drug,” he said.
All eight companies behind these biosimilars are offering or will be launching copay saving programs, many which advertise copays as low as $0 per month for eligible patients.
How will Humira respond?
Marta Wosińska, PhD, a health care economist at the Brookings Institute, Washington, predicts payers will use these lower biosimilar prices to negotiate better deals with AbbVie, Humira’s manufacturer. “We have a lot of players coming into [the market] right now, so the competition is really fierce,” she said. In response, AbbVie will need to increase rebates on Humira and/or lower its price to compete with these biosimilars.
“The ball is in AbbVie’s court,” she said. “If [the company] is not willing to drop price sufficiently, then payers will start switching to biosimilars.”
Dr. Fendrick reported past financial relationships and consulting arrangements with AbbVie, Amgen, Arnold Ventures, Bayer, CareFirst, BlueCross BlueShield, and many other companies. Dr. Wosińska has received funding from Arnold Ventures and serves as an expert witness on antitrust cases involving generic medication.
A version of this article first appeared on Medscape.com.
The best-selling drug Humira (adalimumab) now faces competition in the United States after a 20-year monopoly. The first adalimumab biosimilar, Amjevita, launched in the United States on January 31, and in July, seven additional biosimilars became available. These drugs have the potential to lower prescription drug prices, but when and by how much remains to be seen.
Here’s what you need to know about adalimumab biosimilars.
What Humira biosimilars are now available?
Eight different biosimilars have launched in 2023 with discounts as large at 85% from Humira’s list price of $6,922. A few companies also offer two price points.
Three of these biosimilars – Hadlima, Hyrimoz, and Yuflyma – are available in high concentration formulations. This high concentration formulation makes up 85% of Humira prescriptions, according to a report from Goodroot, a collection of companies focused on lowering health care costs.
Cyltezo is currently the only adalimumab biosimilar with an interchangeability designation, meaning that a pharmacist can substitute the biosimilar for an equivalent Humira prescription without the intervention of a clinician. A total of 47 states allow for these substitutions without prior approval from a clinician, according to Goodroot, and the clinician must be notified of the switch within a certain time frame. A total of 40 states require that patients be notified of the switch before substitution.
However, it’s not clear if this interchangeability designation will prove an advantage for Cyltezo, as it is interchangeable with the lower concentration version of Humira that makes up just 15% of prescriptions.
Most of the companies behind these biosimilars are pursuing interchangeability designations for their drugs, except for Fresenius Kabi (Idacio) and Coherus (Yusimry).
A ninth biosimilar, Pfizer’s adalimumab-afzb (Abrilada), is not yet on the market and is currently awaiting an approval decision from the Food and Drug Administration to add an interchangeability designation to its prior approval for a low-concentration formulation.
Why are they priced differently?
The two price points offer different deals to payers. Pharmacy benefit managers make confidential agreements with drug manufacturers to get a discount – called a rebate – to get the drug on the PBM’s formulary. The PBM keeps a portion of that rebate, and the rest is passed on to the insurance company and patients. Biosimilars at a higher price point will likely offer larger rebates. Biosimilars offered at lower price points incorporate this discount up front in their list pricing and likely will not offer large rebates.
Will biosimilars be covered by payers?
Currently, biosimilars are being offered on formularies at parity with Humira, meaning they are on the same tier. The PBM companies OptumRx and Cigna Group’s Express Scripts will offer Amjevita (at both price points), Cyltezo, and Hyrimoz (at both price points).
“This decision allows our clients flexibility to provide access to the lower list price, so members in high-deductible plans and benefit designs with coinsurance can experience lower out-of-pocket costs,” said OptumRx spokesperson Isaac Sorensen in an email.
Mark Cuban Cost Plus Drug Company, which uses a direct-to-consumer model, will offer Yusimry for $567.27 on its website. SmithRx, a PBM based in San Francisco, announced it would partner with Cost Plus Drugs to offer Yusimry, adding that SmithRx members can use their insurance benefits to further reduce out-of-pocket costs. RxPreferred, another PBM, will also offer Yusimry through its partnership with Cuban’s company.
The news website Formulary Watch previously reported that CVS Caremark, another of the biggest PBMs, will be offering Amjevita, but as a nonpreferred brand, while Humira remains the preferred brand. CVS Caremark did not respond to a request for comment.
Will patients pay less?
Biosimilars have been touted as a potential solution to lower spending on biologic drugs, but it’s unknown if patients will ultimately benefit with lower out-of-pocket costs. It’s “impossible to predict” if the discount that third-party payers pay will be passed on to consumers, said Mark Fendrick, MD, who directs the University of Michigan Center for Value-based Insurance Design in Ann Arbor.
Generally, a consumer’s copay is a percentage of a drug’s list price, so it stands to reason that a low drug price would result in lower out-of-pocket payments. While this is mostly true, Humira has a successful copay assistance program to lower prescription costs for consumers. According to a 2022 IQVIA report, 82% of commercial prescriptions cost patients less than $10 for Humira because of this program.
To appeal to patients, biosimilar companies will need to offer similar savings, Dr. Fendrick added. “There will be some discontent if patients are actually asked to pay more out-of-pocket for a less expensive drug,” he said.
All eight companies behind these biosimilars are offering or will be launching copay saving programs, many which advertise copays as low as $0 per month for eligible patients.
How will Humira respond?
Marta Wosińska, PhD, a health care economist at the Brookings Institute, Washington, predicts payers will use these lower biosimilar prices to negotiate better deals with AbbVie, Humira’s manufacturer. “We have a lot of players coming into [the market] right now, so the competition is really fierce,” she said. In response, AbbVie will need to increase rebates on Humira and/or lower its price to compete with these biosimilars.
“The ball is in AbbVie’s court,” she said. “If [the company] is not willing to drop price sufficiently, then payers will start switching to biosimilars.”
Dr. Fendrick reported past financial relationships and consulting arrangements with AbbVie, Amgen, Arnold Ventures, Bayer, CareFirst, BlueCross BlueShield, and many other companies. Dr. Wosińska has received funding from Arnold Ventures and serves as an expert witness on antitrust cases involving generic medication.
A version of this article first appeared on Medscape.com.
Phenotypes drive antibiotic response in youth with bronchiectasis
Indigenous children or children with new abnormal auscultatory findings were significantly more likely than children in other categories to respond to oral antibiotics for exacerbations related to bronchiectasis, based on data from more than 200 individuals in New Zealand.
Children and adolescents with bronchiectasis are often treated with antibiotics for respiratory exacerbations, but the effects of antibiotics can vary among individuals, and phenotypic features associated with greater symptom resolution have not been identified, wrote Vikas Goyal, PhD, of the Centre for Children’s Health Research, Brisbane, Australia, and colleagues.
Previous studies have suggested that nearly half of exacerbations in children and adolescents resolve spontaneously after 14 days, and more data are needed to identify which patients are mostly likely to benefit from antibiotics, they noted.
In a study published in the journal Chest, the researchers reviewed secondary data from 217 children and adolescents aged 1-18 years with bronchiectasis enrolled in a pair of randomized, controlled trials comparing oral antibiotics with placebo (known as BEST-1 and BEST-2). The median age of the participants was 6.6 years, 52% were boys, and 41% were Indigenous (defined as Australian First Nations, New Zealand Maori, or Pacific Islander). All participants in the analysis received at least 14 days of oral antibiotics for treatment of nonhospitalized respiratory exacerbations.
Overall, 130 children had resolution of symptoms by day 14, and 87 were nonresponders.
In a multivariate analysis, children who were Indigenous or who had new abnormal auscultatory findings were significantly more likely to respond than children in other categories (odds ratios, 3.59 and 3.85, respectively).
Patients with multiple bronchiectatic lobes at the time of diagnosis and those with higher cough scores at the start of treatment were significantly less likely to respond to antibiotics than patients without these features (OR, 0.66 and 0.55, respectively).
The researchers conducted a further analysis to examine the association between Indigenous ethnicity and treatment response. They found no differences in the other response variables of number of affected lobes at diagnosis and cough scores at the start of treatment between Indigenous and non-Indigenous children.
Given the strong response to antibiotics among Indigenous children, the researchers also conducted a mediation analysis. “Respiratory bacterial pathogens were mediated by Indigenous ethnicity and associated with being an antibiotic ‘responder,’ ” they wrote. For new abnormal chest auscultatory findings, both direct and indirect effects on day 14 response to oral antibiotics were mediated by Indigenous ethnicity. However, neither cough scores at the start of treatment nor the number of affected lobes at diagnosis showed a mediation effect from Indigenous ethnicity.
Among the nonresponders, 59 of 87 resolved symptoms with continuing oral antibiotics over the next 2-4 weeks, and 21 improved without antibiotics.
Additionally, the detection of a respiratory virus at the start of an exacerbation was not associated with antibiotic failure at 14 days, the researchers noted.
The findings were limited by several factors including the use of data from randomized trials that were not designed to address the question in the current study, the researchers noted. Other limitations included incomplete clinical data and lack of data on inflammatory indices, potential antibiotic-resistant pathogens in nonresponders, and the follow-up period of only 14 days, they said.
However, the results suggest a role for patient and exacerbation phenotypes in management of bronchiectasis in clinical practice and promoting antimicrobial stewardship, the researchers wrote. “Although there is benefit in treating exacerbations early to avoid treatment failure and subsequent intravenous antibiotics, future research also needs to identify exacerbations that can be managed without antibiotics,” they concluded.
The BEST-1 and BEST-2 studies were supported by the Australian National Health and Medical Research Council and the NHMRC Centre for Research Excellence in Lung Health of Aboriginal and Torres Strait Islander Children. Dr. Goyal had no financial conflicts to disclose.
Indigenous children or children with new abnormal auscultatory findings were significantly more likely than children in other categories to respond to oral antibiotics for exacerbations related to bronchiectasis, based on data from more than 200 individuals in New Zealand.
Children and adolescents with bronchiectasis are often treated with antibiotics for respiratory exacerbations, but the effects of antibiotics can vary among individuals, and phenotypic features associated with greater symptom resolution have not been identified, wrote Vikas Goyal, PhD, of the Centre for Children’s Health Research, Brisbane, Australia, and colleagues.
Previous studies have suggested that nearly half of exacerbations in children and adolescents resolve spontaneously after 14 days, and more data are needed to identify which patients are mostly likely to benefit from antibiotics, they noted.
In a study published in the journal Chest, the researchers reviewed secondary data from 217 children and adolescents aged 1-18 years with bronchiectasis enrolled in a pair of randomized, controlled trials comparing oral antibiotics with placebo (known as BEST-1 and BEST-2). The median age of the participants was 6.6 years, 52% were boys, and 41% were Indigenous (defined as Australian First Nations, New Zealand Maori, or Pacific Islander). All participants in the analysis received at least 14 days of oral antibiotics for treatment of nonhospitalized respiratory exacerbations.
Overall, 130 children had resolution of symptoms by day 14, and 87 were nonresponders.
In a multivariate analysis, children who were Indigenous or who had new abnormal auscultatory findings were significantly more likely to respond than children in other categories (odds ratios, 3.59 and 3.85, respectively).
Patients with multiple bronchiectatic lobes at the time of diagnosis and those with higher cough scores at the start of treatment were significantly less likely to respond to antibiotics than patients without these features (OR, 0.66 and 0.55, respectively).
The researchers conducted a further analysis to examine the association between Indigenous ethnicity and treatment response. They found no differences in the other response variables of number of affected lobes at diagnosis and cough scores at the start of treatment between Indigenous and non-Indigenous children.
Given the strong response to antibiotics among Indigenous children, the researchers also conducted a mediation analysis. “Respiratory bacterial pathogens were mediated by Indigenous ethnicity and associated with being an antibiotic ‘responder,’ ” they wrote. For new abnormal chest auscultatory findings, both direct and indirect effects on day 14 response to oral antibiotics were mediated by Indigenous ethnicity. However, neither cough scores at the start of treatment nor the number of affected lobes at diagnosis showed a mediation effect from Indigenous ethnicity.
Among the nonresponders, 59 of 87 resolved symptoms with continuing oral antibiotics over the next 2-4 weeks, and 21 improved without antibiotics.
Additionally, the detection of a respiratory virus at the start of an exacerbation was not associated with antibiotic failure at 14 days, the researchers noted.
The findings were limited by several factors including the use of data from randomized trials that were not designed to address the question in the current study, the researchers noted. Other limitations included incomplete clinical data and lack of data on inflammatory indices, potential antibiotic-resistant pathogens in nonresponders, and the follow-up period of only 14 days, they said.
However, the results suggest a role for patient and exacerbation phenotypes in management of bronchiectasis in clinical practice and promoting antimicrobial stewardship, the researchers wrote. “Although there is benefit in treating exacerbations early to avoid treatment failure and subsequent intravenous antibiotics, future research also needs to identify exacerbations that can be managed without antibiotics,” they concluded.
The BEST-1 and BEST-2 studies were supported by the Australian National Health and Medical Research Council and the NHMRC Centre for Research Excellence in Lung Health of Aboriginal and Torres Strait Islander Children. Dr. Goyal had no financial conflicts to disclose.
Indigenous children or children with new abnormal auscultatory findings were significantly more likely than children in other categories to respond to oral antibiotics for exacerbations related to bronchiectasis, based on data from more than 200 individuals in New Zealand.
Children and adolescents with bronchiectasis are often treated with antibiotics for respiratory exacerbations, but the effects of antibiotics can vary among individuals, and phenotypic features associated with greater symptom resolution have not been identified, wrote Vikas Goyal, PhD, of the Centre for Children’s Health Research, Brisbane, Australia, and colleagues.
Previous studies have suggested that nearly half of exacerbations in children and adolescents resolve spontaneously after 14 days, and more data are needed to identify which patients are mostly likely to benefit from antibiotics, they noted.
In a study published in the journal Chest, the researchers reviewed secondary data from 217 children and adolescents aged 1-18 years with bronchiectasis enrolled in a pair of randomized, controlled trials comparing oral antibiotics with placebo (known as BEST-1 and BEST-2). The median age of the participants was 6.6 years, 52% were boys, and 41% were Indigenous (defined as Australian First Nations, New Zealand Maori, or Pacific Islander). All participants in the analysis received at least 14 days of oral antibiotics for treatment of nonhospitalized respiratory exacerbations.
Overall, 130 children had resolution of symptoms by day 14, and 87 were nonresponders.
In a multivariate analysis, children who were Indigenous or who had new abnormal auscultatory findings were significantly more likely to respond than children in other categories (odds ratios, 3.59 and 3.85, respectively).
Patients with multiple bronchiectatic lobes at the time of diagnosis and those with higher cough scores at the start of treatment were significantly less likely to respond to antibiotics than patients without these features (OR, 0.66 and 0.55, respectively).
The researchers conducted a further analysis to examine the association between Indigenous ethnicity and treatment response. They found no differences in the other response variables of number of affected lobes at diagnosis and cough scores at the start of treatment between Indigenous and non-Indigenous children.
Given the strong response to antibiotics among Indigenous children, the researchers also conducted a mediation analysis. “Respiratory bacterial pathogens were mediated by Indigenous ethnicity and associated with being an antibiotic ‘responder,’ ” they wrote. For new abnormal chest auscultatory findings, both direct and indirect effects on day 14 response to oral antibiotics were mediated by Indigenous ethnicity. However, neither cough scores at the start of treatment nor the number of affected lobes at diagnosis showed a mediation effect from Indigenous ethnicity.
Among the nonresponders, 59 of 87 resolved symptoms with continuing oral antibiotics over the next 2-4 weeks, and 21 improved without antibiotics.
Additionally, the detection of a respiratory virus at the start of an exacerbation was not associated with antibiotic failure at 14 days, the researchers noted.
The findings were limited by several factors including the use of data from randomized trials that were not designed to address the question in the current study, the researchers noted. Other limitations included incomplete clinical data and lack of data on inflammatory indices, potential antibiotic-resistant pathogens in nonresponders, and the follow-up period of only 14 days, they said.
However, the results suggest a role for patient and exacerbation phenotypes in management of bronchiectasis in clinical practice and promoting antimicrobial stewardship, the researchers wrote. “Although there is benefit in treating exacerbations early to avoid treatment failure and subsequent intravenous antibiotics, future research also needs to identify exacerbations that can be managed without antibiotics,” they concluded.
The BEST-1 and BEST-2 studies were supported by the Australian National Health and Medical Research Council and the NHMRC Centre for Research Excellence in Lung Health of Aboriginal and Torres Strait Islander Children. Dr. Goyal had no financial conflicts to disclose.
FROM THE JOURNAL CHEST
Goodbye, finger sticks; hello, CGMs
Nearly 90% of diabetes management in the United States is provided by primary care clinicians; diabetes is the fifth most common reason for a primary care visit. State-of-the-art technology such as continuous glucose monitors (CGMs) will inevitably transform the management of diabetes in primary care. Clinicians and staff must be ready to educate, counsel, and support primary care patients in the use of CGMs.
CGMs (also called glucose sensors) are small, minimally invasive devices that attach to the skin of the upper arm or trunk. A tiny electrode in the subcutaneous space prompts an enzyme reaction that measures the interstitial (rather than blood) glucose concentration, typically every 5 minutes. The results are displayed on an accompanying reader or transmitted to an app on the user’s mobile phone.
CGMs could eliminate the need for finger-stick blood glucose testing, which until now, has been the much-despised gold standard for self-monitoring of glucose levels in diabetes. Despite being relatively inexpensive and accurate, finger-stick glucose tests are inconvenient and often painful. But of greater significance is this downside: Finger-stick monitoring reveals the patient’s blood glucose concentration at a single point in time, which can be difficult to interpret. Is the blood glucose rising or falling? Multiple finger-stick tests are required to determine the trend of a patient’s glucose levels or the response to food or exercise.
In contrast, the graphic display from a CGM sensor is more like a movie, telling a story as it unfolds. Uninterrupted data provide valuable feedback to patients about the effects of diet, physical activity, stress, or pain on their glucose levels. And for the first time, it’s easy to determine the proportion of time the patient spends in or out of the target glucose range.
Incorporating new technology into your practice may seem like a burden, but the reward is better information that leads to better management of diabetes. If you’re new to glucose sensors, many excellent resources are available to learn how to use them.
I recommend starting with a website called diabeteswise.org, which has both a patient-facing and clinician-facing version. This unbranded site serves as a kind of Consumer Reports for diabetes technology, allowing both patients and professionals to compare and contrast currently available CGM devices.
DiabetesWisePro has information ranging from CGM device fundamentals and best practices to CGM prescribing and reimbursement.
Clinical Diabetes also provides multiple tools to help incorporate these devices into primary care clinical practice, including:
• Continuous Glucose Monitoring: Optimizing Diabetes Care (CME course).
• Diabetes Technology in Primary Care.
The next article in this series will cover two types of CGMs used in primary care: professional and personal devices.
Dr. Shubrook is a professor in the department of primary care, Touro University California College of Osteopathic Medicine, Vallejo, Calif., and director of diabetes services, Solano County Family Health Services, Fairfield, Calif. He disclosed ties with Abbott, Astra Zeneca, Bayer, Nevro, and Novo Nordisk.
A version of this article first appeared on Medscape.com.
Nearly 90% of diabetes management in the United States is provided by primary care clinicians; diabetes is the fifth most common reason for a primary care visit. State-of-the-art technology such as continuous glucose monitors (CGMs) will inevitably transform the management of diabetes in primary care. Clinicians and staff must be ready to educate, counsel, and support primary care patients in the use of CGMs.
CGMs (also called glucose sensors) are small, minimally invasive devices that attach to the skin of the upper arm or trunk. A tiny electrode in the subcutaneous space prompts an enzyme reaction that measures the interstitial (rather than blood) glucose concentration, typically every 5 minutes. The results are displayed on an accompanying reader or transmitted to an app on the user’s mobile phone.
CGMs could eliminate the need for finger-stick blood glucose testing, which until now, has been the much-despised gold standard for self-monitoring of glucose levels in diabetes. Despite being relatively inexpensive and accurate, finger-stick glucose tests are inconvenient and often painful. But of greater significance is this downside: Finger-stick monitoring reveals the patient’s blood glucose concentration at a single point in time, which can be difficult to interpret. Is the blood glucose rising or falling? Multiple finger-stick tests are required to determine the trend of a patient’s glucose levels or the response to food or exercise.
In contrast, the graphic display from a CGM sensor is more like a movie, telling a story as it unfolds. Uninterrupted data provide valuable feedback to patients about the effects of diet, physical activity, stress, or pain on their glucose levels. And for the first time, it’s easy to determine the proportion of time the patient spends in or out of the target glucose range.
Incorporating new technology into your practice may seem like a burden, but the reward is better information that leads to better management of diabetes. If you’re new to glucose sensors, many excellent resources are available to learn how to use them.
I recommend starting with a website called diabeteswise.org, which has both a patient-facing and clinician-facing version. This unbranded site serves as a kind of Consumer Reports for diabetes technology, allowing both patients and professionals to compare and contrast currently available CGM devices.
DiabetesWisePro has information ranging from CGM device fundamentals and best practices to CGM prescribing and reimbursement.
Clinical Diabetes also provides multiple tools to help incorporate these devices into primary care clinical practice, including:
• Continuous Glucose Monitoring: Optimizing Diabetes Care (CME course).
• Diabetes Technology in Primary Care.
The next article in this series will cover two types of CGMs used in primary care: professional and personal devices.
Dr. Shubrook is a professor in the department of primary care, Touro University California College of Osteopathic Medicine, Vallejo, Calif., and director of diabetes services, Solano County Family Health Services, Fairfield, Calif. He disclosed ties with Abbott, Astra Zeneca, Bayer, Nevro, and Novo Nordisk.
A version of this article first appeared on Medscape.com.
Nearly 90% of diabetes management in the United States is provided by primary care clinicians; diabetes is the fifth most common reason for a primary care visit. State-of-the-art technology such as continuous glucose monitors (CGMs) will inevitably transform the management of diabetes in primary care. Clinicians and staff must be ready to educate, counsel, and support primary care patients in the use of CGMs.
CGMs (also called glucose sensors) are small, minimally invasive devices that attach to the skin of the upper arm or trunk. A tiny electrode in the subcutaneous space prompts an enzyme reaction that measures the interstitial (rather than blood) glucose concentration, typically every 5 minutes. The results are displayed on an accompanying reader or transmitted to an app on the user’s mobile phone.
CGMs could eliminate the need for finger-stick blood glucose testing, which until now, has been the much-despised gold standard for self-monitoring of glucose levels in diabetes. Despite being relatively inexpensive and accurate, finger-stick glucose tests are inconvenient and often painful. But of greater significance is this downside: Finger-stick monitoring reveals the patient’s blood glucose concentration at a single point in time, which can be difficult to interpret. Is the blood glucose rising or falling? Multiple finger-stick tests are required to determine the trend of a patient’s glucose levels or the response to food or exercise.
In contrast, the graphic display from a CGM sensor is more like a movie, telling a story as it unfolds. Uninterrupted data provide valuable feedback to patients about the effects of diet, physical activity, stress, or pain on their glucose levels. And for the first time, it’s easy to determine the proportion of time the patient spends in or out of the target glucose range.
Incorporating new technology into your practice may seem like a burden, but the reward is better information that leads to better management of diabetes. If you’re new to glucose sensors, many excellent resources are available to learn how to use them.
I recommend starting with a website called diabeteswise.org, which has both a patient-facing and clinician-facing version. This unbranded site serves as a kind of Consumer Reports for diabetes technology, allowing both patients and professionals to compare and contrast currently available CGM devices.
DiabetesWisePro has information ranging from CGM device fundamentals and best practices to CGM prescribing and reimbursement.
Clinical Diabetes also provides multiple tools to help incorporate these devices into primary care clinical practice, including:
• Continuous Glucose Monitoring: Optimizing Diabetes Care (CME course).
• Diabetes Technology in Primary Care.
The next article in this series will cover two types of CGMs used in primary care: professional and personal devices.
Dr. Shubrook is a professor in the department of primary care, Touro University California College of Osteopathic Medicine, Vallejo, Calif., and director of diabetes services, Solano County Family Health Services, Fairfield, Calif. He disclosed ties with Abbott, Astra Zeneca, Bayer, Nevro, and Novo Nordisk.
A version of this article first appeared on Medscape.com.
In new era of gene therapy, PCPs are ‘boots on the ground’
In Colorado and Wyoming, nearly every baby born since 2020 is tested for signs of a mutation in the SMN1 gene, an indicator of spinal muscular atrophy (SMA). And in 4 years, genetic counselor Melissa Gibbons has seen 24 positive results. She has prepped 24 different pediatricians and family doctors to deliver the news: A seemingly perfect newborn likely has a lethal genetic disease.
Most of these clinicians had never cared for a child with SMA before, nor did they know that lifesaving gene therapy for the condition now exists. Still, the physicians were foundational to getting babies emergency treatment and monitoring the child’s safety after the fact.
“They are boots on the ground for this kind of [work],” Ms. Gibbons, who is the newborn screen coordinator for SMA in both states, told this news organization. “I’m not even sure they realize it.” As of today, the U.S. Food and Drug Administration has approved 16 gene therapies for the treatment of rare and debilitating diseases once considered lethal, such as SMA and cerebral adrenoleukodystrophy.
The newest addition to the list of approvals is Elevidys, Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD). These conditions can now be mitigated, abated for years at a time, and even cured using treatments that tweak a patient’s DNA or RNA.
Hundreds of treatments are under development using the same mechanism. Viruses, liposomes, and other vectors of all kinds are being used to usher new genes into cells, correcting faulty copies or equipping a cell to fight disease. Cells gain the ability to make lifesaving proteins – proteins that heal wounds, restore muscle function, and fight cancer.
Within the decade, a significant fraction of the pediatric population will have gone through gene therapy, experts told this news organization. And primary care stands to be a linchpin in the scale-up of this kind of precision genetic medicine. Pediatricians and general practitioners will be central to finding and monitoring the patients that need these treatments. But the time and support doctors will need to fill that role remain scarce.
“This is a world we are creating right now, quite literally,” said Stanley Nelson, MD, director of the center for Duchenne muscular dystrophy at the University of California, Los Angeles. These cases – some before gene therapy and some after – will show up in primary care offices before the textbook is written.
Unknown side effects, new diseases
Even now, gene therapy is sequestered away in large academic medical research centers. The diagnosis, decision-making, and aftercare are handled by subspecialists working on clinical trials. While the research is ongoing, trial sponsors are keeping a close eye on enrolled patients. But that’s only until these drugs get market approval, Phil Beales, MD, chief medical officer at Congenica, a digital health company specializing in genome analysis support, said. Afterward, “the trialists will no longer have a role in looking after those patients.”
At that point, the role of primary care clinicians will be critically important. Although they probably will not manage gene-therapy patients on their own – comanaging them instead with subspecialists – they will be involved in the ordering and monitoring of safety labs and other tests.
General practitioners “need to know side effects because they are going to deal with side effects when someone calls them in the middle of the night,” said Dr. Beales, who also is chief executive officer of Axovia Therapeutics, a biotech company developing gene therapies.
Some of the side effects that come with gene therapy are established. Adeno-associated virus (AAV) or AAV-mediated gene therapies carry an increased risk for damage to the heart and liver, Dr. Nelson said. Other side effects are less well known and could be specific to the treatment and the tissue it targets. Primary care will be critical in detecting these unexpected side effects and expediting visits with subspecialists, he said.
In rural Wyoming, pediatricians and family doctors are especially important, Ms. Gibbons said. In the 30-90 days after gene therapy, patients need a lot of follow-up for safety reasons.
But aftercare for gene therapy will be more than just monitoring and managing side effects. The diseases themselves will change. Patients will be living with conditions that once were lethal.
In some cases, gene therapy may largely eliminate the disease. The data suggest that thalassemia, for example, can be largely cured for decades with one infusion of a patient’s genetically modified hematopoietic stem cells made using bluebird bio’s Zynteglo, according to Christy Duncan, MD, medical director of clinical research at the gene therapy program at Boston Children’s Hospital.
But other gene therapies, like the one for DMD, will offer a “spectrum of benefits,” Dr. Nelson said. They will be lifesaving, but the signs of the disease will linger. Clinicians will be learning alongside specialists what the new disease state for DMD and other rare diseases looks like after gene therapy.
“As we get hundreds of such therapies, [post–gene therapy] will amount to a substantial part of the pediatric population,” Dr. Nelson said.
Finding patients
Many of these rare diseases that plague young patients are unmistakable. Children with moderate or severe dystrophic epidermolysis bullosa, for instance, carry a mutation that prevents them from making type VII collagen. The babies suffer wounds and excessive bleeding and tend to receive a quick diagnosis within the first 6 months of life, according to Andy Orth, chief commercial officer at Krystal Bio, manufacturer of a new wound-healing gene therapy, Vyjuvek, for the disorder.
Other rare neurologic or muscular diseases can go undiagnosed for years. Until recently, drug companies and researchers have had little motivation to speed up the timeline because early diagnosis of a disease like DMD would not change the outcome, Dr. Nelson said.
But with gene therapy, prognoses are changing. And finding diseases early could soon mean preserving muscular function or preventing neurologic damage, Dr. Duncan said.
Newborn sequencing “is not standard of care yet, but it’s certainly coming,” Josh Peterson, MD, MPH, director of the center for precision medicine at Vanderbilt University Medical Center, in Nashville, Tenn., told this news organization.
A recent survey of 238 specialists in rare diseases found that roughly 90% believe whole-genome sequencing should be available to all newborns. And 80% of those experts endorse 42 genes as disease predictors. Screening for rare diseases at birth could reveal a host of conditions in the first week of life and expedite treatment. But this strategy will often rely on primary care and pediatricians interpreting the results.
Most pediatricians think sequencing is a great idea, but they do not feel comfortable doing it themselves, Dr. Peterson said. The good news, he said, is that manufacturers have made screening tests straightforward. Some drug companies even offer free screenings for gene therapy candidates.
Dr. Peterson predicts pediatricians will need to be equipped to deliver negative results on their own, which will be the case for around 97%-99% of patients. They also will need to be clear on whether a negative result is definitive or if more testing is warranted.
Positive results are more nuanced. Genetic counseling is the ideal resource when delivering this kind of news to patients, but counselors are a scarce resource nationally – and particularly in rural areas, Dr. Nelson said. Physicians likely will have to rely on their own counseling training to some degree.
“I feel very strongly that genetic counselors are in short supply,” Ms. Gibbons in Colorado said. Patients need a friendly resource who can talk them through the disease and how it works. And that discussion is not a one-off, she said.
The number of board-certified genetic counselors in the United States has doubled to more than 6,000 in the past 10 years – a pace that is expected to continue, according to the National Society of Genetic Counselors. “However, the geographical distribution of genetic counselors is most concentrated in urban centers.”
Equally important to the counseling experience, according to Dr. Duncan at Boston Children’s, is a primary care physician’s network of connections. The best newborn screening rollouts across the country have succeeded because clinicians knew where to send people next and how to get families the help they needed, she said.
But she also cautioned that this learning curve will soon be overwhelming. As gene therapy expands, it may be difficult for primary care doctors to keep up with the science, treatment studies, and commercially available therapies. “It’s asking too much,” Dr. Duncan said.
The structure of primary care already stretches practitioners thin and will “affect how well precision medicine can be adopted and disseminated,” Dr. Peterson said. “I think that is a key issue.”
Artificial intelligence may offer a partial solution. Some genetic counseling models already exist, but their utility for clinicians so far is limited, Dr. Beales said. But he said he expects these tools to improve rapidly to help clinicians and patients. On the patient’s end, they may be able to answer questions and supplement basic genetic counseling. On the physician’s end, algorithms could help triage patients and help move them along to the next steps in the care pathway for these rare diseases.
The whole patient
Primary care physicians will not be expected to be experts in gene therapy or solely in charge of patient safety. They will have support from industry and subspecialists leading the development of these treatments, experts agreed.
But generalists should expect to be drawn into multidisciplinary care teams, be the sounding boards for patients making decisions about gene therapy, help arrange insurance coverage, and be the recipients of late-night phone calls about side effects.
All that, while never losing sight of the child’s holistic health. In children so sick, specialists, subspecialists, and even parents tend to focus only on the rare disease. The team can “get distracted from good normal routine care,” Dr. Nelson said. But these children aren’t exempt from check-ups, vaccine regimens, or the other diseases of childhood.
“In a world where we mitigate that core disease,” he said, “we need a partner in the general pediatrics community” investing in their long-term health.
A version of this article first appeared on Medscape.com.
In Colorado and Wyoming, nearly every baby born since 2020 is tested for signs of a mutation in the SMN1 gene, an indicator of spinal muscular atrophy (SMA). And in 4 years, genetic counselor Melissa Gibbons has seen 24 positive results. She has prepped 24 different pediatricians and family doctors to deliver the news: A seemingly perfect newborn likely has a lethal genetic disease.
Most of these clinicians had never cared for a child with SMA before, nor did they know that lifesaving gene therapy for the condition now exists. Still, the physicians were foundational to getting babies emergency treatment and monitoring the child’s safety after the fact.
“They are boots on the ground for this kind of [work],” Ms. Gibbons, who is the newborn screen coordinator for SMA in both states, told this news organization. “I’m not even sure they realize it.” As of today, the U.S. Food and Drug Administration has approved 16 gene therapies for the treatment of rare and debilitating diseases once considered lethal, such as SMA and cerebral adrenoleukodystrophy.
The newest addition to the list of approvals is Elevidys, Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD). These conditions can now be mitigated, abated for years at a time, and even cured using treatments that tweak a patient’s DNA or RNA.
Hundreds of treatments are under development using the same mechanism. Viruses, liposomes, and other vectors of all kinds are being used to usher new genes into cells, correcting faulty copies or equipping a cell to fight disease. Cells gain the ability to make lifesaving proteins – proteins that heal wounds, restore muscle function, and fight cancer.
Within the decade, a significant fraction of the pediatric population will have gone through gene therapy, experts told this news organization. And primary care stands to be a linchpin in the scale-up of this kind of precision genetic medicine. Pediatricians and general practitioners will be central to finding and monitoring the patients that need these treatments. But the time and support doctors will need to fill that role remain scarce.
“This is a world we are creating right now, quite literally,” said Stanley Nelson, MD, director of the center for Duchenne muscular dystrophy at the University of California, Los Angeles. These cases – some before gene therapy and some after – will show up in primary care offices before the textbook is written.
Unknown side effects, new diseases
Even now, gene therapy is sequestered away in large academic medical research centers. The diagnosis, decision-making, and aftercare are handled by subspecialists working on clinical trials. While the research is ongoing, trial sponsors are keeping a close eye on enrolled patients. But that’s only until these drugs get market approval, Phil Beales, MD, chief medical officer at Congenica, a digital health company specializing in genome analysis support, said. Afterward, “the trialists will no longer have a role in looking after those patients.”
At that point, the role of primary care clinicians will be critically important. Although they probably will not manage gene-therapy patients on their own – comanaging them instead with subspecialists – they will be involved in the ordering and monitoring of safety labs and other tests.
General practitioners “need to know side effects because they are going to deal with side effects when someone calls them in the middle of the night,” said Dr. Beales, who also is chief executive officer of Axovia Therapeutics, a biotech company developing gene therapies.
Some of the side effects that come with gene therapy are established. Adeno-associated virus (AAV) or AAV-mediated gene therapies carry an increased risk for damage to the heart and liver, Dr. Nelson said. Other side effects are less well known and could be specific to the treatment and the tissue it targets. Primary care will be critical in detecting these unexpected side effects and expediting visits with subspecialists, he said.
In rural Wyoming, pediatricians and family doctors are especially important, Ms. Gibbons said. In the 30-90 days after gene therapy, patients need a lot of follow-up for safety reasons.
But aftercare for gene therapy will be more than just monitoring and managing side effects. The diseases themselves will change. Patients will be living with conditions that once were lethal.
In some cases, gene therapy may largely eliminate the disease. The data suggest that thalassemia, for example, can be largely cured for decades with one infusion of a patient’s genetically modified hematopoietic stem cells made using bluebird bio’s Zynteglo, according to Christy Duncan, MD, medical director of clinical research at the gene therapy program at Boston Children’s Hospital.
But other gene therapies, like the one for DMD, will offer a “spectrum of benefits,” Dr. Nelson said. They will be lifesaving, but the signs of the disease will linger. Clinicians will be learning alongside specialists what the new disease state for DMD and other rare diseases looks like after gene therapy.
“As we get hundreds of such therapies, [post–gene therapy] will amount to a substantial part of the pediatric population,” Dr. Nelson said.
Finding patients
Many of these rare diseases that plague young patients are unmistakable. Children with moderate or severe dystrophic epidermolysis bullosa, for instance, carry a mutation that prevents them from making type VII collagen. The babies suffer wounds and excessive bleeding and tend to receive a quick diagnosis within the first 6 months of life, according to Andy Orth, chief commercial officer at Krystal Bio, manufacturer of a new wound-healing gene therapy, Vyjuvek, for the disorder.
Other rare neurologic or muscular diseases can go undiagnosed for years. Until recently, drug companies and researchers have had little motivation to speed up the timeline because early diagnosis of a disease like DMD would not change the outcome, Dr. Nelson said.
But with gene therapy, prognoses are changing. And finding diseases early could soon mean preserving muscular function or preventing neurologic damage, Dr. Duncan said.
Newborn sequencing “is not standard of care yet, but it’s certainly coming,” Josh Peterson, MD, MPH, director of the center for precision medicine at Vanderbilt University Medical Center, in Nashville, Tenn., told this news organization.
A recent survey of 238 specialists in rare diseases found that roughly 90% believe whole-genome sequencing should be available to all newborns. And 80% of those experts endorse 42 genes as disease predictors. Screening for rare diseases at birth could reveal a host of conditions in the first week of life and expedite treatment. But this strategy will often rely on primary care and pediatricians interpreting the results.
Most pediatricians think sequencing is a great idea, but they do not feel comfortable doing it themselves, Dr. Peterson said. The good news, he said, is that manufacturers have made screening tests straightforward. Some drug companies even offer free screenings for gene therapy candidates.
Dr. Peterson predicts pediatricians will need to be equipped to deliver negative results on their own, which will be the case for around 97%-99% of patients. They also will need to be clear on whether a negative result is definitive or if more testing is warranted.
Positive results are more nuanced. Genetic counseling is the ideal resource when delivering this kind of news to patients, but counselors are a scarce resource nationally – and particularly in rural areas, Dr. Nelson said. Physicians likely will have to rely on their own counseling training to some degree.
“I feel very strongly that genetic counselors are in short supply,” Ms. Gibbons in Colorado said. Patients need a friendly resource who can talk them through the disease and how it works. And that discussion is not a one-off, she said.
The number of board-certified genetic counselors in the United States has doubled to more than 6,000 in the past 10 years – a pace that is expected to continue, according to the National Society of Genetic Counselors. “However, the geographical distribution of genetic counselors is most concentrated in urban centers.”
Equally important to the counseling experience, according to Dr. Duncan at Boston Children’s, is a primary care physician’s network of connections. The best newborn screening rollouts across the country have succeeded because clinicians knew where to send people next and how to get families the help they needed, she said.
But she also cautioned that this learning curve will soon be overwhelming. As gene therapy expands, it may be difficult for primary care doctors to keep up with the science, treatment studies, and commercially available therapies. “It’s asking too much,” Dr. Duncan said.
The structure of primary care already stretches practitioners thin and will “affect how well precision medicine can be adopted and disseminated,” Dr. Peterson said. “I think that is a key issue.”
Artificial intelligence may offer a partial solution. Some genetic counseling models already exist, but their utility for clinicians so far is limited, Dr. Beales said. But he said he expects these tools to improve rapidly to help clinicians and patients. On the patient’s end, they may be able to answer questions and supplement basic genetic counseling. On the physician’s end, algorithms could help triage patients and help move them along to the next steps in the care pathway for these rare diseases.
The whole patient
Primary care physicians will not be expected to be experts in gene therapy or solely in charge of patient safety. They will have support from industry and subspecialists leading the development of these treatments, experts agreed.
But generalists should expect to be drawn into multidisciplinary care teams, be the sounding boards for patients making decisions about gene therapy, help arrange insurance coverage, and be the recipients of late-night phone calls about side effects.
All that, while never losing sight of the child’s holistic health. In children so sick, specialists, subspecialists, and even parents tend to focus only on the rare disease. The team can “get distracted from good normal routine care,” Dr. Nelson said. But these children aren’t exempt from check-ups, vaccine regimens, or the other diseases of childhood.
“In a world where we mitigate that core disease,” he said, “we need a partner in the general pediatrics community” investing in their long-term health.
A version of this article first appeared on Medscape.com.
In Colorado and Wyoming, nearly every baby born since 2020 is tested for signs of a mutation in the SMN1 gene, an indicator of spinal muscular atrophy (SMA). And in 4 years, genetic counselor Melissa Gibbons has seen 24 positive results. She has prepped 24 different pediatricians and family doctors to deliver the news: A seemingly perfect newborn likely has a lethal genetic disease.
Most of these clinicians had never cared for a child with SMA before, nor did they know that lifesaving gene therapy for the condition now exists. Still, the physicians were foundational to getting babies emergency treatment and monitoring the child’s safety after the fact.
“They are boots on the ground for this kind of [work],” Ms. Gibbons, who is the newborn screen coordinator for SMA in both states, told this news organization. “I’m not even sure they realize it.” As of today, the U.S. Food and Drug Administration has approved 16 gene therapies for the treatment of rare and debilitating diseases once considered lethal, such as SMA and cerebral adrenoleukodystrophy.
The newest addition to the list of approvals is Elevidys, Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD). These conditions can now be mitigated, abated for years at a time, and even cured using treatments that tweak a patient’s DNA or RNA.
Hundreds of treatments are under development using the same mechanism. Viruses, liposomes, and other vectors of all kinds are being used to usher new genes into cells, correcting faulty copies or equipping a cell to fight disease. Cells gain the ability to make lifesaving proteins – proteins that heal wounds, restore muscle function, and fight cancer.
Within the decade, a significant fraction of the pediatric population will have gone through gene therapy, experts told this news organization. And primary care stands to be a linchpin in the scale-up of this kind of precision genetic medicine. Pediatricians and general practitioners will be central to finding and monitoring the patients that need these treatments. But the time and support doctors will need to fill that role remain scarce.
“This is a world we are creating right now, quite literally,” said Stanley Nelson, MD, director of the center for Duchenne muscular dystrophy at the University of California, Los Angeles. These cases – some before gene therapy and some after – will show up in primary care offices before the textbook is written.
Unknown side effects, new diseases
Even now, gene therapy is sequestered away in large academic medical research centers. The diagnosis, decision-making, and aftercare are handled by subspecialists working on clinical trials. While the research is ongoing, trial sponsors are keeping a close eye on enrolled patients. But that’s only until these drugs get market approval, Phil Beales, MD, chief medical officer at Congenica, a digital health company specializing in genome analysis support, said. Afterward, “the trialists will no longer have a role in looking after those patients.”
At that point, the role of primary care clinicians will be critically important. Although they probably will not manage gene-therapy patients on their own – comanaging them instead with subspecialists – they will be involved in the ordering and monitoring of safety labs and other tests.
General practitioners “need to know side effects because they are going to deal with side effects when someone calls them in the middle of the night,” said Dr. Beales, who also is chief executive officer of Axovia Therapeutics, a biotech company developing gene therapies.
Some of the side effects that come with gene therapy are established. Adeno-associated virus (AAV) or AAV-mediated gene therapies carry an increased risk for damage to the heart and liver, Dr. Nelson said. Other side effects are less well known and could be specific to the treatment and the tissue it targets. Primary care will be critical in detecting these unexpected side effects and expediting visits with subspecialists, he said.
In rural Wyoming, pediatricians and family doctors are especially important, Ms. Gibbons said. In the 30-90 days after gene therapy, patients need a lot of follow-up for safety reasons.
But aftercare for gene therapy will be more than just monitoring and managing side effects. The diseases themselves will change. Patients will be living with conditions that once were lethal.
In some cases, gene therapy may largely eliminate the disease. The data suggest that thalassemia, for example, can be largely cured for decades with one infusion of a patient’s genetically modified hematopoietic stem cells made using bluebird bio’s Zynteglo, according to Christy Duncan, MD, medical director of clinical research at the gene therapy program at Boston Children’s Hospital.
But other gene therapies, like the one for DMD, will offer a “spectrum of benefits,” Dr. Nelson said. They will be lifesaving, but the signs of the disease will linger. Clinicians will be learning alongside specialists what the new disease state for DMD and other rare diseases looks like after gene therapy.
“As we get hundreds of such therapies, [post–gene therapy] will amount to a substantial part of the pediatric population,” Dr. Nelson said.
Finding patients
Many of these rare diseases that plague young patients are unmistakable. Children with moderate or severe dystrophic epidermolysis bullosa, for instance, carry a mutation that prevents them from making type VII collagen. The babies suffer wounds and excessive bleeding and tend to receive a quick diagnosis within the first 6 months of life, according to Andy Orth, chief commercial officer at Krystal Bio, manufacturer of a new wound-healing gene therapy, Vyjuvek, for the disorder.
Other rare neurologic or muscular diseases can go undiagnosed for years. Until recently, drug companies and researchers have had little motivation to speed up the timeline because early diagnosis of a disease like DMD would not change the outcome, Dr. Nelson said.
But with gene therapy, prognoses are changing. And finding diseases early could soon mean preserving muscular function or preventing neurologic damage, Dr. Duncan said.
Newborn sequencing “is not standard of care yet, but it’s certainly coming,” Josh Peterson, MD, MPH, director of the center for precision medicine at Vanderbilt University Medical Center, in Nashville, Tenn., told this news organization.
A recent survey of 238 specialists in rare diseases found that roughly 90% believe whole-genome sequencing should be available to all newborns. And 80% of those experts endorse 42 genes as disease predictors. Screening for rare diseases at birth could reveal a host of conditions in the first week of life and expedite treatment. But this strategy will often rely on primary care and pediatricians interpreting the results.
Most pediatricians think sequencing is a great idea, but they do not feel comfortable doing it themselves, Dr. Peterson said. The good news, he said, is that manufacturers have made screening tests straightforward. Some drug companies even offer free screenings for gene therapy candidates.
Dr. Peterson predicts pediatricians will need to be equipped to deliver negative results on their own, which will be the case for around 97%-99% of patients. They also will need to be clear on whether a negative result is definitive or if more testing is warranted.
Positive results are more nuanced. Genetic counseling is the ideal resource when delivering this kind of news to patients, but counselors are a scarce resource nationally – and particularly in rural areas, Dr. Nelson said. Physicians likely will have to rely on their own counseling training to some degree.
“I feel very strongly that genetic counselors are in short supply,” Ms. Gibbons in Colorado said. Patients need a friendly resource who can talk them through the disease and how it works. And that discussion is not a one-off, she said.
The number of board-certified genetic counselors in the United States has doubled to more than 6,000 in the past 10 years – a pace that is expected to continue, according to the National Society of Genetic Counselors. “However, the geographical distribution of genetic counselors is most concentrated in urban centers.”
Equally important to the counseling experience, according to Dr. Duncan at Boston Children’s, is a primary care physician’s network of connections. The best newborn screening rollouts across the country have succeeded because clinicians knew where to send people next and how to get families the help they needed, she said.
But she also cautioned that this learning curve will soon be overwhelming. As gene therapy expands, it may be difficult for primary care doctors to keep up with the science, treatment studies, and commercially available therapies. “It’s asking too much,” Dr. Duncan said.
The structure of primary care already stretches practitioners thin and will “affect how well precision medicine can be adopted and disseminated,” Dr. Peterson said. “I think that is a key issue.”
Artificial intelligence may offer a partial solution. Some genetic counseling models already exist, but their utility for clinicians so far is limited, Dr. Beales said. But he said he expects these tools to improve rapidly to help clinicians and patients. On the patient’s end, they may be able to answer questions and supplement basic genetic counseling. On the physician’s end, algorithms could help triage patients and help move them along to the next steps in the care pathway for these rare diseases.
The whole patient
Primary care physicians will not be expected to be experts in gene therapy or solely in charge of patient safety. They will have support from industry and subspecialists leading the development of these treatments, experts agreed.
But generalists should expect to be drawn into multidisciplinary care teams, be the sounding boards for patients making decisions about gene therapy, help arrange insurance coverage, and be the recipients of late-night phone calls about side effects.
All that, while never losing sight of the child’s holistic health. In children so sick, specialists, subspecialists, and even parents tend to focus only on the rare disease. The team can “get distracted from good normal routine care,” Dr. Nelson said. But these children aren’t exempt from check-ups, vaccine regimens, or the other diseases of childhood.
“In a world where we mitigate that core disease,” he said, “we need a partner in the general pediatrics community” investing in their long-term health.
A version of this article first appeared on Medscape.com.
‘Brain fitness program’ may aid memory loss, concussion, ADHD
new research shows.
The program, which consists of targeted cognitive training and EEG-based neurofeedback, coupled with meditation and diet/lifestyle coaching, led to improvements in memory, attention, mood, alertness, and sleep.
The program promotes “neuroplasticity and was equally effective for patients with all three conditions,” program creator Majid Fotuhi, MD, PhD, said in an interview.
Patients with mild to moderate cognitive symptoms often see “remarkable” results within 3 months of consistently following the program, said Dr. Fotuhi, adjunct professor of neuroscience at George Washington University, Washington, and medical director of NeuroGrow Brain Fitness Center, McLean, Va.
“It actually makes intuitive sense that a healthier and stronger brain would function better and that patients of all ages with various cognitive or emotional symptoms would all benefit from improving the biology of their brain,” Dr. Fotuhi added.
The study was published online in the Journal of Alzheimer’s Disease Reports.
Personalized program
The findings are based on 223 children and adults who completed the 12-week NeuroGrow Brain Fitness Program (NeuroGrow BFP), including 71 with ADHD, 88 with PCS, and 64 with memory loss, defined as diagnosed mild cognitive impairment or subjective cognitive decline.
As part of the program, participants undergo a complete neurocognitive evaluation, including tests for verbal memory, complex attention, processing speed, executive functioning, and the Neurocognitive Index.
They also complete questionnaires regarding sleep, mood, diet, exercise, and anxiety/depression, and they undergo quantitative EEG at the beginning and end of the program.
A comparison of before and after neurocognitive test scores showed that all three patient subgroups experienced statistically significant improvements on most measures, the study team reports.
After completing the program, 60%-90% of patients scored higher on cognitive tests and reported having fewer cognitive, sleep, and emotional symptoms.
In all subgroups, the most significant improvement was observed in executive functioning.
“These preliminary findings appear to show that multimodal interventions which are known to increase neuroplasticity in the brain, when personalized, can have benefits for patients with cognitive symptoms from a variety of neurological conditions,” the investigators wrote.
The study’s strengths include a large, community-based sample of patients of different ages who had disruptive symptoms and abnormalities as determined using objective cognitive tests whose progress was monitored by objective and subjective measures.
The chief limitation is the lack of a control or placebo group.
“Though it is difficult to find a comparable group of patients with the exact same profile of cognitive deficits and brain-related symptoms, studying a larger group of patients – and comparing them with a wait-list group – may make it possible to do a more definitive assessment of the NeuroGrow BFP,” the researchers noted.
Dr. Fotuhi said the “secret to the success” of the program is that it involves a full assessment of all cognitive and neurobehavioral symptoms for each patient. This allows for individualized and targeted interventions for specific concerns and symptoms.
He said there is a need to recognize that patients who present to a neurology practice with a single complaint, such as a problem with memory or attention, often have other problems, such as anxiety/depression, stress, insomnia, sedentary lifestyle, obesity, diabetes, sleep apnea, or alcohol overuse.
“Each of these factors can affect their cognitive abilities and need a multimodal set of interventions in order to see full resolution of their cognitive symptoms,” Dr. Fotuhi said.
He has created a series of educational videos to demonstrate the program’s benefits.
The self-pay cost for the NeuroGrow BFP assessment and treatment sessions is approximately $7,000.
Dr. Fotuhi said all of the interventions included in the program are readily available at low cost.
He suggested that health care professionals who lack time or staff for conducting a comprehensive neurocognitive assessment for their patients can provide them with a copy of the Brain Health Index.
“Patients can then be instructed to work on the individual components of their brain health on their own – and measure their brain health index on a weekly basis,” Dr. Fotuhi said. “Private practices or academic centers can use the detailed information I have provided in my paper to develop their own brain fitness program.”
Not ready for prime time
Commenting on the study, Percy Griffin, PhD, director of scientific engagement for the Alzheimer’s Association, noted that “nonpharmacologic interventions can help alleviate some of the symptoms associated with dementia.
“The current study investigates nonpharmacologic interventions in a small number of patients with ADHD, postconcussion syndrome, or memory loss. The researchers found improvements on most measures following the brain rehabilitation program.
“While this is interesting, more work is needed in larger, more diverse cohorts before these programs can be applied broadly. Nonpharmacologic interventions are a helpful tool that need to be studied further in future studies,” Dr. Griffin added.
Funding for the study was provided by the NeuroGrow Brain Fitness Center. Dr. Fotuhi, the owner of NeuroGrow, was involved in data analysis, writing, editing, approval, and decision to publish. Dr. Griffin reported no disclosures.
A version of this article appeared on Medscape.com.
new research shows.
The program, which consists of targeted cognitive training and EEG-based neurofeedback, coupled with meditation and diet/lifestyle coaching, led to improvements in memory, attention, mood, alertness, and sleep.
The program promotes “neuroplasticity and was equally effective for patients with all three conditions,” program creator Majid Fotuhi, MD, PhD, said in an interview.
Patients with mild to moderate cognitive symptoms often see “remarkable” results within 3 months of consistently following the program, said Dr. Fotuhi, adjunct professor of neuroscience at George Washington University, Washington, and medical director of NeuroGrow Brain Fitness Center, McLean, Va.
“It actually makes intuitive sense that a healthier and stronger brain would function better and that patients of all ages with various cognitive or emotional symptoms would all benefit from improving the biology of their brain,” Dr. Fotuhi added.
The study was published online in the Journal of Alzheimer’s Disease Reports.
Personalized program
The findings are based on 223 children and adults who completed the 12-week NeuroGrow Brain Fitness Program (NeuroGrow BFP), including 71 with ADHD, 88 with PCS, and 64 with memory loss, defined as diagnosed mild cognitive impairment or subjective cognitive decline.
As part of the program, participants undergo a complete neurocognitive evaluation, including tests for verbal memory, complex attention, processing speed, executive functioning, and the Neurocognitive Index.
They also complete questionnaires regarding sleep, mood, diet, exercise, and anxiety/depression, and they undergo quantitative EEG at the beginning and end of the program.
A comparison of before and after neurocognitive test scores showed that all three patient subgroups experienced statistically significant improvements on most measures, the study team reports.
After completing the program, 60%-90% of patients scored higher on cognitive tests and reported having fewer cognitive, sleep, and emotional symptoms.
In all subgroups, the most significant improvement was observed in executive functioning.
“These preliminary findings appear to show that multimodal interventions which are known to increase neuroplasticity in the brain, when personalized, can have benefits for patients with cognitive symptoms from a variety of neurological conditions,” the investigators wrote.
The study’s strengths include a large, community-based sample of patients of different ages who had disruptive symptoms and abnormalities as determined using objective cognitive tests whose progress was monitored by objective and subjective measures.
The chief limitation is the lack of a control or placebo group.
“Though it is difficult to find a comparable group of patients with the exact same profile of cognitive deficits and brain-related symptoms, studying a larger group of patients – and comparing them with a wait-list group – may make it possible to do a more definitive assessment of the NeuroGrow BFP,” the researchers noted.
Dr. Fotuhi said the “secret to the success” of the program is that it involves a full assessment of all cognitive and neurobehavioral symptoms for each patient. This allows for individualized and targeted interventions for specific concerns and symptoms.
He said there is a need to recognize that patients who present to a neurology practice with a single complaint, such as a problem with memory or attention, often have other problems, such as anxiety/depression, stress, insomnia, sedentary lifestyle, obesity, diabetes, sleep apnea, or alcohol overuse.
“Each of these factors can affect their cognitive abilities and need a multimodal set of interventions in order to see full resolution of their cognitive symptoms,” Dr. Fotuhi said.
He has created a series of educational videos to demonstrate the program’s benefits.
The self-pay cost for the NeuroGrow BFP assessment and treatment sessions is approximately $7,000.
Dr. Fotuhi said all of the interventions included in the program are readily available at low cost.
He suggested that health care professionals who lack time or staff for conducting a comprehensive neurocognitive assessment for their patients can provide them with a copy of the Brain Health Index.
“Patients can then be instructed to work on the individual components of their brain health on their own – and measure their brain health index on a weekly basis,” Dr. Fotuhi said. “Private practices or academic centers can use the detailed information I have provided in my paper to develop their own brain fitness program.”
Not ready for prime time
Commenting on the study, Percy Griffin, PhD, director of scientific engagement for the Alzheimer’s Association, noted that “nonpharmacologic interventions can help alleviate some of the symptoms associated with dementia.
“The current study investigates nonpharmacologic interventions in a small number of patients with ADHD, postconcussion syndrome, or memory loss. The researchers found improvements on most measures following the brain rehabilitation program.
“While this is interesting, more work is needed in larger, more diverse cohorts before these programs can be applied broadly. Nonpharmacologic interventions are a helpful tool that need to be studied further in future studies,” Dr. Griffin added.
Funding for the study was provided by the NeuroGrow Brain Fitness Center. Dr. Fotuhi, the owner of NeuroGrow, was involved in data analysis, writing, editing, approval, and decision to publish. Dr. Griffin reported no disclosures.
A version of this article appeared on Medscape.com.
new research shows.
The program, which consists of targeted cognitive training and EEG-based neurofeedback, coupled with meditation and diet/lifestyle coaching, led to improvements in memory, attention, mood, alertness, and sleep.
The program promotes “neuroplasticity and was equally effective for patients with all three conditions,” program creator Majid Fotuhi, MD, PhD, said in an interview.
Patients with mild to moderate cognitive symptoms often see “remarkable” results within 3 months of consistently following the program, said Dr. Fotuhi, adjunct professor of neuroscience at George Washington University, Washington, and medical director of NeuroGrow Brain Fitness Center, McLean, Va.
“It actually makes intuitive sense that a healthier and stronger brain would function better and that patients of all ages with various cognitive or emotional symptoms would all benefit from improving the biology of their brain,” Dr. Fotuhi added.
The study was published online in the Journal of Alzheimer’s Disease Reports.
Personalized program
The findings are based on 223 children and adults who completed the 12-week NeuroGrow Brain Fitness Program (NeuroGrow BFP), including 71 with ADHD, 88 with PCS, and 64 with memory loss, defined as diagnosed mild cognitive impairment or subjective cognitive decline.
As part of the program, participants undergo a complete neurocognitive evaluation, including tests for verbal memory, complex attention, processing speed, executive functioning, and the Neurocognitive Index.
They also complete questionnaires regarding sleep, mood, diet, exercise, and anxiety/depression, and they undergo quantitative EEG at the beginning and end of the program.
A comparison of before and after neurocognitive test scores showed that all three patient subgroups experienced statistically significant improvements on most measures, the study team reports.
After completing the program, 60%-90% of patients scored higher on cognitive tests and reported having fewer cognitive, sleep, and emotional symptoms.
In all subgroups, the most significant improvement was observed in executive functioning.
“These preliminary findings appear to show that multimodal interventions which are known to increase neuroplasticity in the brain, when personalized, can have benefits for patients with cognitive symptoms from a variety of neurological conditions,” the investigators wrote.
The study’s strengths include a large, community-based sample of patients of different ages who had disruptive symptoms and abnormalities as determined using objective cognitive tests whose progress was monitored by objective and subjective measures.
The chief limitation is the lack of a control or placebo group.
“Though it is difficult to find a comparable group of patients with the exact same profile of cognitive deficits and brain-related symptoms, studying a larger group of patients – and comparing them with a wait-list group – may make it possible to do a more definitive assessment of the NeuroGrow BFP,” the researchers noted.
Dr. Fotuhi said the “secret to the success” of the program is that it involves a full assessment of all cognitive and neurobehavioral symptoms for each patient. This allows for individualized and targeted interventions for specific concerns and symptoms.
He said there is a need to recognize that patients who present to a neurology practice with a single complaint, such as a problem with memory or attention, often have other problems, such as anxiety/depression, stress, insomnia, sedentary lifestyle, obesity, diabetes, sleep apnea, or alcohol overuse.
“Each of these factors can affect their cognitive abilities and need a multimodal set of interventions in order to see full resolution of their cognitive symptoms,” Dr. Fotuhi said.
He has created a series of educational videos to demonstrate the program’s benefits.
The self-pay cost for the NeuroGrow BFP assessment and treatment sessions is approximately $7,000.
Dr. Fotuhi said all of the interventions included in the program are readily available at low cost.
He suggested that health care professionals who lack time or staff for conducting a comprehensive neurocognitive assessment for their patients can provide them with a copy of the Brain Health Index.
“Patients can then be instructed to work on the individual components of their brain health on their own – and measure their brain health index on a weekly basis,” Dr. Fotuhi said. “Private practices or academic centers can use the detailed information I have provided in my paper to develop their own brain fitness program.”
Not ready for prime time
Commenting on the study, Percy Griffin, PhD, director of scientific engagement for the Alzheimer’s Association, noted that “nonpharmacologic interventions can help alleviate some of the symptoms associated with dementia.
“The current study investigates nonpharmacologic interventions in a small number of patients with ADHD, postconcussion syndrome, or memory loss. The researchers found improvements on most measures following the brain rehabilitation program.
“While this is interesting, more work is needed in larger, more diverse cohorts before these programs can be applied broadly. Nonpharmacologic interventions are a helpful tool that need to be studied further in future studies,” Dr. Griffin added.
Funding for the study was provided by the NeuroGrow Brain Fitness Center. Dr. Fotuhi, the owner of NeuroGrow, was involved in data analysis, writing, editing, approval, and decision to publish. Dr. Griffin reported no disclosures.
A version of this article appeared on Medscape.com.
FROM THE JOURNAL OF ALZHEIMER’S DISEASE REPORTS