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Consider housing insecurity, other issues when managing challenging skin diseases in children, expert says
ASHEVILLE, N.C. – , according to a pediatric dermatologist who addressed the annual meeting of the Society for Pediatric Dermatology.
As a general principle for treating chronic skin conditions in children who are not doing well, it is reasonable to draw out information about a patient’s access to adequate housing, nutrition, and other basic needs, George Hightower, MD, PhD, of the division of pediatric and adolescent dermatology, University of California, San Diego, said at the meeting.
“We need conversations about where patients play, learn, and rest their heads at night,” said Dr. Hightower, who conducts research in this area. Fundamental components of well-being, such as stable housing and secure access to nutrition “are inseparable” from a child’s health, he noted.
“What are the stakes?” he asked. For many children, these factors might mean the difference between effective and poor control of the diseases for which the patient is seeking care.
To illustrate the point, Dr. Hightower used hidradenitis suppurativa (HS), a disease that appears to be on the rise among adolescents, as an example of why patient circumstances matter and should be considered. A complex disorder that is more prevalent in resource-poor communities, HS is difficult to control, often requiring extended periods of treatment with medications that can involve complex dosing or regular infusions.
“There is a need for medical providers to help the patient plan for this chronic illness,” said Dr. Hightower, referring to the importance of close follow-up. In adolescents, HS can be sufficiently disruptive from both the physical and psychological perspective that poor control can “derail future aspirations” by complicating educational endeavors and social interactions.
Dr. Hightower acknowledged that simply documenting housing insecurity or other issues does not solve these problems, but he does believe that developing a sensitivity to these obstacles to health care is a first step. It is a process that should permeate into medical training, health care research, and strategies to improve outcomes.
“The connections between fair housing and clinical practice may appear tenuous and inconsequential to the care provided by medical specialists,” Dr. Hightower said, but he emphasized that there are clear consequences when these factors contribute to inadequate control of such diseases as HS. As a source of missed appointments and disjointed care, an unstable home life can be an important barrier to disease control – and because of scarring nodules, fistulae, pain, school absences, and social isolation, complications can be dire.
Solutions to insecure housing are not typically available to an individual clinician, but the awareness that this can be a factor can help both physicians and patients begin to think about the role this plays in impairing recovery and what solutions might be found to modify the impact. Awareness not just among individual clinicians but a broader consortium of those working to improve health care outcomes is needed to “challenge the way we are doing medicine,” he said.
While conversations about the social determinants of health, including access to resources within patients’ neighborhoods, schools, and environment, can demonstrate concern about how to address obstacles, it can also be part of a reorientation to think beyond treatment for the underlying pathology alone. Eliciting trust and emphasizing the importance of environmental barriers to adequate care can be positive steps on the path to solutions.
Participatory action research
Relevant to this orientation, Dr. Hightower spoke about participatory action research (PAR), which provides a framework for patients to participate in the planning of clinical studies to effect change, not just serve as subjects in these studies.
The assumption of PAR is that “all people have valuable knowledge about their lives and experiences,” Dr. Hightower said. From this assumption, individuals who have been historically marginalized by race, income, or other factors can help define the problems from the patient’s perspective and, from there, create studies to seek solutions.
PAR is consistent with a patient-centered approach to medical care, which Dr. Hightower called “the future of medicine.” It involves a big-picture approach to look beyond disease pathology and symptoms to factors that might be creating susceptibility to disease and undermining health care.
Organized medicine alone cannot solve the cause of social inequities leading to disparate risks for disease and risks of inadequate health care, but Dr. Hightower argued that these inequities should not be ignored. He believes medical trainees should learn how to elicit information about the barriers to adequate health care and be aware of solutions, such as fair housing policies.
While he believes that PAR is an example of a pathway to problem solving, he suggested that a comprehensive approach requires an effective method of communication between providers and patients that would lead to a collaborative and mutually reinforcing approach.
“How do we ensure that individuals from communities most impacted by health disparities are treated fairly and empowered to address these disparities?” Dr. Hightower asked. He said that this is the direction of his own research and the issues that inhibit adequate treatment of many dermatologic diseases, as well as other types of disease, in childhood.
Craig Burkhart, MD, director of a private pediatric and adolescent dermatology practice in Cary, N.C., said that Dr. Hightower’s message is relevant. The value of considering and addressing the psychological well-being of patients of any age is not a new concept, but he acknowledged that he, for one, has not routinely inquired about obstacles to follow-up care if there is a signal that this might be an issue.
“As dermatologists, we focus on the acute complaints. We want to make the patient better,” said Dr. Burkhart, who moderated the session in which Dr. Hightower spoke. He agreed with Dr. Hightower that environmental factors make a difference on the road to recovery for a patient, and his presentation was a good reminder, he said, to consider the patient’s circumstances when response to treatment is inadequate, particularly in chronic diseases like HS, for which comprehensive care and close follow-up are needed.
Dr. Hightower and Dr. Burkhart report no potential conflicts of interest.
ASHEVILLE, N.C. – , according to a pediatric dermatologist who addressed the annual meeting of the Society for Pediatric Dermatology.
As a general principle for treating chronic skin conditions in children who are not doing well, it is reasonable to draw out information about a patient’s access to adequate housing, nutrition, and other basic needs, George Hightower, MD, PhD, of the division of pediatric and adolescent dermatology, University of California, San Diego, said at the meeting.
“We need conversations about where patients play, learn, and rest their heads at night,” said Dr. Hightower, who conducts research in this area. Fundamental components of well-being, such as stable housing and secure access to nutrition “are inseparable” from a child’s health, he noted.
“What are the stakes?” he asked. For many children, these factors might mean the difference between effective and poor control of the diseases for which the patient is seeking care.
To illustrate the point, Dr. Hightower used hidradenitis suppurativa (HS), a disease that appears to be on the rise among adolescents, as an example of why patient circumstances matter and should be considered. A complex disorder that is more prevalent in resource-poor communities, HS is difficult to control, often requiring extended periods of treatment with medications that can involve complex dosing or regular infusions.
“There is a need for medical providers to help the patient plan for this chronic illness,” said Dr. Hightower, referring to the importance of close follow-up. In adolescents, HS can be sufficiently disruptive from both the physical and psychological perspective that poor control can “derail future aspirations” by complicating educational endeavors and social interactions.
Dr. Hightower acknowledged that simply documenting housing insecurity or other issues does not solve these problems, but he does believe that developing a sensitivity to these obstacles to health care is a first step. It is a process that should permeate into medical training, health care research, and strategies to improve outcomes.
“The connections between fair housing and clinical practice may appear tenuous and inconsequential to the care provided by medical specialists,” Dr. Hightower said, but he emphasized that there are clear consequences when these factors contribute to inadequate control of such diseases as HS. As a source of missed appointments and disjointed care, an unstable home life can be an important barrier to disease control – and because of scarring nodules, fistulae, pain, school absences, and social isolation, complications can be dire.
Solutions to insecure housing are not typically available to an individual clinician, but the awareness that this can be a factor can help both physicians and patients begin to think about the role this plays in impairing recovery and what solutions might be found to modify the impact. Awareness not just among individual clinicians but a broader consortium of those working to improve health care outcomes is needed to “challenge the way we are doing medicine,” he said.
While conversations about the social determinants of health, including access to resources within patients’ neighborhoods, schools, and environment, can demonstrate concern about how to address obstacles, it can also be part of a reorientation to think beyond treatment for the underlying pathology alone. Eliciting trust and emphasizing the importance of environmental barriers to adequate care can be positive steps on the path to solutions.
Participatory action research
Relevant to this orientation, Dr. Hightower spoke about participatory action research (PAR), which provides a framework for patients to participate in the planning of clinical studies to effect change, not just serve as subjects in these studies.
The assumption of PAR is that “all people have valuable knowledge about their lives and experiences,” Dr. Hightower said. From this assumption, individuals who have been historically marginalized by race, income, or other factors can help define the problems from the patient’s perspective and, from there, create studies to seek solutions.
PAR is consistent with a patient-centered approach to medical care, which Dr. Hightower called “the future of medicine.” It involves a big-picture approach to look beyond disease pathology and symptoms to factors that might be creating susceptibility to disease and undermining health care.
Organized medicine alone cannot solve the cause of social inequities leading to disparate risks for disease and risks of inadequate health care, but Dr. Hightower argued that these inequities should not be ignored. He believes medical trainees should learn how to elicit information about the barriers to adequate health care and be aware of solutions, such as fair housing policies.
While he believes that PAR is an example of a pathway to problem solving, he suggested that a comprehensive approach requires an effective method of communication between providers and patients that would lead to a collaborative and mutually reinforcing approach.
“How do we ensure that individuals from communities most impacted by health disparities are treated fairly and empowered to address these disparities?” Dr. Hightower asked. He said that this is the direction of his own research and the issues that inhibit adequate treatment of many dermatologic diseases, as well as other types of disease, in childhood.
Craig Burkhart, MD, director of a private pediatric and adolescent dermatology practice in Cary, N.C., said that Dr. Hightower’s message is relevant. The value of considering and addressing the psychological well-being of patients of any age is not a new concept, but he acknowledged that he, for one, has not routinely inquired about obstacles to follow-up care if there is a signal that this might be an issue.
“As dermatologists, we focus on the acute complaints. We want to make the patient better,” said Dr. Burkhart, who moderated the session in which Dr. Hightower spoke. He agreed with Dr. Hightower that environmental factors make a difference on the road to recovery for a patient, and his presentation was a good reminder, he said, to consider the patient’s circumstances when response to treatment is inadequate, particularly in chronic diseases like HS, for which comprehensive care and close follow-up are needed.
Dr. Hightower and Dr. Burkhart report no potential conflicts of interest.
ASHEVILLE, N.C. – , according to a pediatric dermatologist who addressed the annual meeting of the Society for Pediatric Dermatology.
As a general principle for treating chronic skin conditions in children who are not doing well, it is reasonable to draw out information about a patient’s access to adequate housing, nutrition, and other basic needs, George Hightower, MD, PhD, of the division of pediatric and adolescent dermatology, University of California, San Diego, said at the meeting.
“We need conversations about where patients play, learn, and rest their heads at night,” said Dr. Hightower, who conducts research in this area. Fundamental components of well-being, such as stable housing and secure access to nutrition “are inseparable” from a child’s health, he noted.
“What are the stakes?” he asked. For many children, these factors might mean the difference between effective and poor control of the diseases for which the patient is seeking care.
To illustrate the point, Dr. Hightower used hidradenitis suppurativa (HS), a disease that appears to be on the rise among adolescents, as an example of why patient circumstances matter and should be considered. A complex disorder that is more prevalent in resource-poor communities, HS is difficult to control, often requiring extended periods of treatment with medications that can involve complex dosing or regular infusions.
“There is a need for medical providers to help the patient plan for this chronic illness,” said Dr. Hightower, referring to the importance of close follow-up. In adolescents, HS can be sufficiently disruptive from both the physical and psychological perspective that poor control can “derail future aspirations” by complicating educational endeavors and social interactions.
Dr. Hightower acknowledged that simply documenting housing insecurity or other issues does not solve these problems, but he does believe that developing a sensitivity to these obstacles to health care is a first step. It is a process that should permeate into medical training, health care research, and strategies to improve outcomes.
“The connections between fair housing and clinical practice may appear tenuous and inconsequential to the care provided by medical specialists,” Dr. Hightower said, but he emphasized that there are clear consequences when these factors contribute to inadequate control of such diseases as HS. As a source of missed appointments and disjointed care, an unstable home life can be an important barrier to disease control – and because of scarring nodules, fistulae, pain, school absences, and social isolation, complications can be dire.
Solutions to insecure housing are not typically available to an individual clinician, but the awareness that this can be a factor can help both physicians and patients begin to think about the role this plays in impairing recovery and what solutions might be found to modify the impact. Awareness not just among individual clinicians but a broader consortium of those working to improve health care outcomes is needed to “challenge the way we are doing medicine,” he said.
While conversations about the social determinants of health, including access to resources within patients’ neighborhoods, schools, and environment, can demonstrate concern about how to address obstacles, it can also be part of a reorientation to think beyond treatment for the underlying pathology alone. Eliciting trust and emphasizing the importance of environmental barriers to adequate care can be positive steps on the path to solutions.
Participatory action research
Relevant to this orientation, Dr. Hightower spoke about participatory action research (PAR), which provides a framework for patients to participate in the planning of clinical studies to effect change, not just serve as subjects in these studies.
The assumption of PAR is that “all people have valuable knowledge about their lives and experiences,” Dr. Hightower said. From this assumption, individuals who have been historically marginalized by race, income, or other factors can help define the problems from the patient’s perspective and, from there, create studies to seek solutions.
PAR is consistent with a patient-centered approach to medical care, which Dr. Hightower called “the future of medicine.” It involves a big-picture approach to look beyond disease pathology and symptoms to factors that might be creating susceptibility to disease and undermining health care.
Organized medicine alone cannot solve the cause of social inequities leading to disparate risks for disease and risks of inadequate health care, but Dr. Hightower argued that these inequities should not be ignored. He believes medical trainees should learn how to elicit information about the barriers to adequate health care and be aware of solutions, such as fair housing policies.
While he believes that PAR is an example of a pathway to problem solving, he suggested that a comprehensive approach requires an effective method of communication between providers and patients that would lead to a collaborative and mutually reinforcing approach.
“How do we ensure that individuals from communities most impacted by health disparities are treated fairly and empowered to address these disparities?” Dr. Hightower asked. He said that this is the direction of his own research and the issues that inhibit adequate treatment of many dermatologic diseases, as well as other types of disease, in childhood.
Craig Burkhart, MD, director of a private pediatric and adolescent dermatology practice in Cary, N.C., said that Dr. Hightower’s message is relevant. The value of considering and addressing the psychological well-being of patients of any age is not a new concept, but he acknowledged that he, for one, has not routinely inquired about obstacles to follow-up care if there is a signal that this might be an issue.
“As dermatologists, we focus on the acute complaints. We want to make the patient better,” said Dr. Burkhart, who moderated the session in which Dr. Hightower spoke. He agreed with Dr. Hightower that environmental factors make a difference on the road to recovery for a patient, and his presentation was a good reminder, he said, to consider the patient’s circumstances when response to treatment is inadequate, particularly in chronic diseases like HS, for which comprehensive care and close follow-up are needed.
Dr. Hightower and Dr. Burkhart report no potential conflicts of interest.
AT SPD 2023
Study highlights diagnostic challenges of differentiating lichen sclerosus from vitiligo
of cases.
Researchers who tallied symptoms and physical exam findings observed fewer statistically significant differences between LS and vitiligo patients than expected, and LS and vitiligo were sometimes misdiagnosed as each other.
“LS must be treated aggressively to prevent long-term sequelae such as permanent scarring and vulvar squamous cell carcinoma, making an accurate diagnosis crucial,” the authors write in a poster they presented at the annual meeting of the Society for Pediatric Dermatology.
LS is symptomatic and has multiple exam findings, but once treated or quiescent, the discoloration can persist and create diagnostic uncertainty, lead study author Kaiane Habeshian, MD, a pediatric dermatologist at Children’s National Hospital, Washington, told this news organization following the SPD meeting.
The diagnostic uncertainty is especially true in patients with darker skin tones, who may have vitiligoid LS, an LS variant that has overlapping features of both LS and vitiligo.
Vitiligoid LS “presents clinically as a depigmented symmetric white vulvar and perianal white patch, often with minimal signs of inflammation, but is symptomatic and appears consistent with LS on histopathology,” Dr. Habeshian said.
“In our experience, in patients with medium to dark skin tones, there is a variable amount of repigmentation after treating LS,” she added. “After use of high potency topical corticosteroids, some patients almost completely repigment, while others have minimal repigmentation, and this can fluctuate over time, sometimes independent of other signs or symptoms of a flare up. This can lead to diagnostic confusion. For example, if an LS patient is examined after treatment, and their symptoms have resolved, they may subsequently be given a diagnosis of vitiligo.”
In the study, Dr. Habeshian and her coauthors aimed to characterize differences in LS and vitiligo based on history, physical exam, and demographic findings at the time of the initial clinic visit. She and her colleagues extracted and reviewed the medical records of 98 patients with a diagnosis of LS or vitiligo who were seen at a joint pediatric dermatology-gynecology vulvar clinic over 6.8 years. The median and mean age of the study population at diagnosis was about 6 years, with ages ranging from 2 to 20. The team used descriptive statistics and Z tests for data analysis.
The researchers found that pruritus, constipation, and dysuria were the most common symptoms experienced by both LS and vitiligo patients. All were experienced more frequently by LS patients, but only pruritus reached statistical significance (P = .040). Other symptoms experienced only by LS patients included vulvar pain, bleeding, and pain with defecation.
Meanwhile, apart from hypopigmentation and erythema, all physical exam findings were more frequent in LS patients, compared with vitiligo patients, including fissures and purpura/petechiae, but only epidermal atrophy and figure-of-8 distribution of hypopigmentation reached statistical significance (P values of .047 and .036, respectively).
In other findings, LS and vitiligo were misdiagnosed as each other 15 times. Nearly half of the misdiagnoses (46.7%) were made in Black patients, who composed 38.8% of all patients in the study.
“I suspect that some vitiligo cases that were previously ‘misdiagnosed’ as LS were actually LS that just didn’t repigment and then were labeled as vitiligo in the chart,” Dr. Habeshian said.
“And some of those LS cases that previously were misdiagnosed as vitiligo likely had other more subtle LS findings that were missed (shininess and wrinkling of the skin, small fissures, constipation) or that were attributed to comorbid irritant contact dermatitis or another condition,” she said. “It was interesting to see that even in a vulvar dermatology clinic there can be confusion between these diagnoses because the literature on pediatric LS in darker skin tones is so sparse.”
She emphasized that a close exam and detailed history are needed to properly diagnose patients with anogenital skin conditions.
“Don’t forget to ask about constipation and urinary symptoms as well as psychosocial and, in the appropriate patient, sexual and reproductive function,” Dr. Habeshian said. “Based on my experience, pediatric LS is much more common in our community than the literature would suggest. Its psychosocial impact is tremendous but not well documented, particularly in pediatric patients. In my experience, the longer LS is misdiagnosed or mistreated, the more challenging it becomes to manage. You don’t want to miss LS.”
She acknowledged certain limitations of the study, including the fact that photographs were not available for review for many of the earlier years of the clinic. “Therefore, we had to depend on the diagnosis given at the time of the visit,” she said. “This likely accounts in part for the smaller number than expected of significant exam and history findings between LS and vitiligo. We need further studies utilizing a standardized approach to accurate diagnosis.”
Her coauthors were Nikita Menta, Aneka Khilnani, MS, and Tazim Dowlut-McElroy, MD. The researchers reported having no financial disclosures.
of cases.
Researchers who tallied symptoms and physical exam findings observed fewer statistically significant differences between LS and vitiligo patients than expected, and LS and vitiligo were sometimes misdiagnosed as each other.
“LS must be treated aggressively to prevent long-term sequelae such as permanent scarring and vulvar squamous cell carcinoma, making an accurate diagnosis crucial,” the authors write in a poster they presented at the annual meeting of the Society for Pediatric Dermatology.
LS is symptomatic and has multiple exam findings, but once treated or quiescent, the discoloration can persist and create diagnostic uncertainty, lead study author Kaiane Habeshian, MD, a pediatric dermatologist at Children’s National Hospital, Washington, told this news organization following the SPD meeting.
The diagnostic uncertainty is especially true in patients with darker skin tones, who may have vitiligoid LS, an LS variant that has overlapping features of both LS and vitiligo.
Vitiligoid LS “presents clinically as a depigmented symmetric white vulvar and perianal white patch, often with minimal signs of inflammation, but is symptomatic and appears consistent with LS on histopathology,” Dr. Habeshian said.
“In our experience, in patients with medium to dark skin tones, there is a variable amount of repigmentation after treating LS,” she added. “After use of high potency topical corticosteroids, some patients almost completely repigment, while others have minimal repigmentation, and this can fluctuate over time, sometimes independent of other signs or symptoms of a flare up. This can lead to diagnostic confusion. For example, if an LS patient is examined after treatment, and their symptoms have resolved, they may subsequently be given a diagnosis of vitiligo.”
In the study, Dr. Habeshian and her coauthors aimed to characterize differences in LS and vitiligo based on history, physical exam, and demographic findings at the time of the initial clinic visit. She and her colleagues extracted and reviewed the medical records of 98 patients with a diagnosis of LS or vitiligo who were seen at a joint pediatric dermatology-gynecology vulvar clinic over 6.8 years. The median and mean age of the study population at diagnosis was about 6 years, with ages ranging from 2 to 20. The team used descriptive statistics and Z tests for data analysis.
The researchers found that pruritus, constipation, and dysuria were the most common symptoms experienced by both LS and vitiligo patients. All were experienced more frequently by LS patients, but only pruritus reached statistical significance (P = .040). Other symptoms experienced only by LS patients included vulvar pain, bleeding, and pain with defecation.
Meanwhile, apart from hypopigmentation and erythema, all physical exam findings were more frequent in LS patients, compared with vitiligo patients, including fissures and purpura/petechiae, but only epidermal atrophy and figure-of-8 distribution of hypopigmentation reached statistical significance (P values of .047 and .036, respectively).
In other findings, LS and vitiligo were misdiagnosed as each other 15 times. Nearly half of the misdiagnoses (46.7%) were made in Black patients, who composed 38.8% of all patients in the study.
“I suspect that some vitiligo cases that were previously ‘misdiagnosed’ as LS were actually LS that just didn’t repigment and then were labeled as vitiligo in the chart,” Dr. Habeshian said.
“And some of those LS cases that previously were misdiagnosed as vitiligo likely had other more subtle LS findings that were missed (shininess and wrinkling of the skin, small fissures, constipation) or that were attributed to comorbid irritant contact dermatitis or another condition,” she said. “It was interesting to see that even in a vulvar dermatology clinic there can be confusion between these diagnoses because the literature on pediatric LS in darker skin tones is so sparse.”
She emphasized that a close exam and detailed history are needed to properly diagnose patients with anogenital skin conditions.
“Don’t forget to ask about constipation and urinary symptoms as well as psychosocial and, in the appropriate patient, sexual and reproductive function,” Dr. Habeshian said. “Based on my experience, pediatric LS is much more common in our community than the literature would suggest. Its psychosocial impact is tremendous but not well documented, particularly in pediatric patients. In my experience, the longer LS is misdiagnosed or mistreated, the more challenging it becomes to manage. You don’t want to miss LS.”
She acknowledged certain limitations of the study, including the fact that photographs were not available for review for many of the earlier years of the clinic. “Therefore, we had to depend on the diagnosis given at the time of the visit,” she said. “This likely accounts in part for the smaller number than expected of significant exam and history findings between LS and vitiligo. We need further studies utilizing a standardized approach to accurate diagnosis.”
Her coauthors were Nikita Menta, Aneka Khilnani, MS, and Tazim Dowlut-McElroy, MD. The researchers reported having no financial disclosures.
of cases.
Researchers who tallied symptoms and physical exam findings observed fewer statistically significant differences between LS and vitiligo patients than expected, and LS and vitiligo were sometimes misdiagnosed as each other.
“LS must be treated aggressively to prevent long-term sequelae such as permanent scarring and vulvar squamous cell carcinoma, making an accurate diagnosis crucial,” the authors write in a poster they presented at the annual meeting of the Society for Pediatric Dermatology.
LS is symptomatic and has multiple exam findings, but once treated or quiescent, the discoloration can persist and create diagnostic uncertainty, lead study author Kaiane Habeshian, MD, a pediatric dermatologist at Children’s National Hospital, Washington, told this news organization following the SPD meeting.
The diagnostic uncertainty is especially true in patients with darker skin tones, who may have vitiligoid LS, an LS variant that has overlapping features of both LS and vitiligo.
Vitiligoid LS “presents clinically as a depigmented symmetric white vulvar and perianal white patch, often with minimal signs of inflammation, but is symptomatic and appears consistent with LS on histopathology,” Dr. Habeshian said.
“In our experience, in patients with medium to dark skin tones, there is a variable amount of repigmentation after treating LS,” she added. “After use of high potency topical corticosteroids, some patients almost completely repigment, while others have minimal repigmentation, and this can fluctuate over time, sometimes independent of other signs or symptoms of a flare up. This can lead to diagnostic confusion. For example, if an LS patient is examined after treatment, and their symptoms have resolved, they may subsequently be given a diagnosis of vitiligo.”
In the study, Dr. Habeshian and her coauthors aimed to characterize differences in LS and vitiligo based on history, physical exam, and demographic findings at the time of the initial clinic visit. She and her colleagues extracted and reviewed the medical records of 98 patients with a diagnosis of LS or vitiligo who were seen at a joint pediatric dermatology-gynecology vulvar clinic over 6.8 years. The median and mean age of the study population at diagnosis was about 6 years, with ages ranging from 2 to 20. The team used descriptive statistics and Z tests for data analysis.
The researchers found that pruritus, constipation, and dysuria were the most common symptoms experienced by both LS and vitiligo patients. All were experienced more frequently by LS patients, but only pruritus reached statistical significance (P = .040). Other symptoms experienced only by LS patients included vulvar pain, bleeding, and pain with defecation.
Meanwhile, apart from hypopigmentation and erythema, all physical exam findings were more frequent in LS patients, compared with vitiligo patients, including fissures and purpura/petechiae, but only epidermal atrophy and figure-of-8 distribution of hypopigmentation reached statistical significance (P values of .047 and .036, respectively).
In other findings, LS and vitiligo were misdiagnosed as each other 15 times. Nearly half of the misdiagnoses (46.7%) were made in Black patients, who composed 38.8% of all patients in the study.
“I suspect that some vitiligo cases that were previously ‘misdiagnosed’ as LS were actually LS that just didn’t repigment and then were labeled as vitiligo in the chart,” Dr. Habeshian said.
“And some of those LS cases that previously were misdiagnosed as vitiligo likely had other more subtle LS findings that were missed (shininess and wrinkling of the skin, small fissures, constipation) or that were attributed to comorbid irritant contact dermatitis or another condition,” she said. “It was interesting to see that even in a vulvar dermatology clinic there can be confusion between these diagnoses because the literature on pediatric LS in darker skin tones is so sparse.”
She emphasized that a close exam and detailed history are needed to properly diagnose patients with anogenital skin conditions.
“Don’t forget to ask about constipation and urinary symptoms as well as psychosocial and, in the appropriate patient, sexual and reproductive function,” Dr. Habeshian said. “Based on my experience, pediatric LS is much more common in our community than the literature would suggest. Its psychosocial impact is tremendous but not well documented, particularly in pediatric patients. In my experience, the longer LS is misdiagnosed or mistreated, the more challenging it becomes to manage. You don’t want to miss LS.”
She acknowledged certain limitations of the study, including the fact that photographs were not available for review for many of the earlier years of the clinic. “Therefore, we had to depend on the diagnosis given at the time of the visit,” she said. “This likely accounts in part for the smaller number than expected of significant exam and history findings between LS and vitiligo. We need further studies utilizing a standardized approach to accurate diagnosis.”
Her coauthors were Nikita Menta, Aneka Khilnani, MS, and Tazim Dowlut-McElroy, MD. The researchers reported having no financial disclosures.
FROM SPD 2023
Roflumilast cream appears safe, effective for children with psoriasis, researchers report
In patients aged 2-11 years, roflumilast cream was well tolerated and improved signs and symptoms of psoriasis over 4 weeks, according to results from a pair of phase two studies.
“Limited topical treatments are approved for children younger than 12 years old with psoriasis,” researchers led by Adelaide A. Hebert, MD, wrote in their abstract. The results were presented during a poster session at the annual meeting of the Society for Pediatric Dermatology.
Roflumilast cream 0.3% (Zoryve) is a once-daily, topical nonsteroidal treatment from Arcutis Biotherapeutics. A phosphodiesterase-4 inhibitor, it was approved by the Food and Drug Administration in 2022 for mild, moderate and severe psoriasis in individuals aged 12 and older, including intertriginous psoriasis.
For the analysis, Dr. Hebert, chief of pediatric dermatology at the University of Texas, Houston, and colleagues conducted two 4-week, phase 2, open-label safety studies of roflumilast cream 0.3%.
One, study 216, enrolled 10 children aged 2-5, and all but one were Black. The other, study 215, enrolled 20 children aged 6-11, and half were Black and nearly half were White. At baseline, patients had 2% or greater body surface area (BSA) involvement and an Investigator Global Assessment (IGA) score of at least mild.
Caregivers applied roflumilast cream to all affected areas once daily for 28 days. The researchers collected pharmacokinetic samples at week 2 and week 4. The primary endpoints were pharmacokinetic, safety, and tolerability.
Efficacy was evaluated as exploratory endpoints: An IGA of clear or almost clear plus a 2-grade or more improvement from baseline, a 50% or greater improvement and a 75% or greater improvement on the Psoriasis Area and Severity Index (PASI-50 and PASI-75), a 4-point or greater reduction in the Worst Itch–Numeric Rating Scale (WI-NRS) in patients with a baseline score of 4 or greater, a mean change from baseline in BSA, and improvement in the Children’s Dermatology Life Quality Index (CDLQI).
At baseline, the mean BSA was similar for patients enrolled in studies 216 and 215 (9.6% and 8.8%, respectively), and 80% of all patients had baseline IGA of moderate. By week 2, the mean roflumilast and N-oxide predose plasma concentrations among patients in the younger group were 2.15 and 22.4 ng/mL, compared with 3.15 and 28.9 ng/mL among those in the older group. At week 4, the mean roflumilast and N-oxide predose concentrations were 2.04 and 15.8 ng/mL in the younger group (study 216), compared with 1.68 and 15.7 ng/mL in the older group (study 215).
As for efficacy, 90% and 40% of patients in studies 216 and 215 achieved IGA success at week 4, respectively, while 90% and 50% achieved PASI-75, 90% and 40% achieved WI-NRS success, and the mean BSA reductions at week 4 were 79.1% and 44.4%. Meanwhile, one younger patient in study 216 reported a treatment-emergent adverse event (TEAE) of headache, which was considered mild, while four older patients in study 215 reported 8 TEAEs, which were considered mild and ranged from back pain to nasal congestion.
“The rapid onset of action was surprising but exceedingly rewarding for the subjects enrolled in the study,” Dr. Hebert told this news organization after the meeting. “The PASI scores and itch scores were markedly improved at the end of the 4-week clinical trial. Patient and parents alike were pleased to use a steroid-free option with once-daily application and rapid onset of action to help control plaque psoriasis.”
In the poster abstract, she and her coauthors concluded that “under maximal use conditions in children aged 2-11 years, roflumilast cream 0.3% was well tolerated and improved signs and symptoms of psoriasis with measured improvements in IGA score, PASI score, BSA involvement, CDLQI, and WI-NRS. Overall, pharmacokinetics, safety, tolerability, and efficacy in patients aged 2-11 years were consistent with prior results in adults and adolescents.”
The study was funded by Arcutis Biotherapeutics. Dr. Hebert reported that she is an investigator for Arcutis. About half the coauthors are employees of Arcutis, and the other half disclosed grants, research funding and/or honoraria from the company. Research grants from the company for this study were paid to the McGovern Medical School at the University of Texas.
In patients aged 2-11 years, roflumilast cream was well tolerated and improved signs and symptoms of psoriasis over 4 weeks, according to results from a pair of phase two studies.
“Limited topical treatments are approved for children younger than 12 years old with psoriasis,” researchers led by Adelaide A. Hebert, MD, wrote in their abstract. The results were presented during a poster session at the annual meeting of the Society for Pediatric Dermatology.
Roflumilast cream 0.3% (Zoryve) is a once-daily, topical nonsteroidal treatment from Arcutis Biotherapeutics. A phosphodiesterase-4 inhibitor, it was approved by the Food and Drug Administration in 2022 for mild, moderate and severe psoriasis in individuals aged 12 and older, including intertriginous psoriasis.
For the analysis, Dr. Hebert, chief of pediatric dermatology at the University of Texas, Houston, and colleagues conducted two 4-week, phase 2, open-label safety studies of roflumilast cream 0.3%.
One, study 216, enrolled 10 children aged 2-5, and all but one were Black. The other, study 215, enrolled 20 children aged 6-11, and half were Black and nearly half were White. At baseline, patients had 2% or greater body surface area (BSA) involvement and an Investigator Global Assessment (IGA) score of at least mild.
Caregivers applied roflumilast cream to all affected areas once daily for 28 days. The researchers collected pharmacokinetic samples at week 2 and week 4. The primary endpoints were pharmacokinetic, safety, and tolerability.
Efficacy was evaluated as exploratory endpoints: An IGA of clear or almost clear plus a 2-grade or more improvement from baseline, a 50% or greater improvement and a 75% or greater improvement on the Psoriasis Area and Severity Index (PASI-50 and PASI-75), a 4-point or greater reduction in the Worst Itch–Numeric Rating Scale (WI-NRS) in patients with a baseline score of 4 or greater, a mean change from baseline in BSA, and improvement in the Children’s Dermatology Life Quality Index (CDLQI).
At baseline, the mean BSA was similar for patients enrolled in studies 216 and 215 (9.6% and 8.8%, respectively), and 80% of all patients had baseline IGA of moderate. By week 2, the mean roflumilast and N-oxide predose plasma concentrations among patients in the younger group were 2.15 and 22.4 ng/mL, compared with 3.15 and 28.9 ng/mL among those in the older group. At week 4, the mean roflumilast and N-oxide predose concentrations were 2.04 and 15.8 ng/mL in the younger group (study 216), compared with 1.68 and 15.7 ng/mL in the older group (study 215).
As for efficacy, 90% and 40% of patients in studies 216 and 215 achieved IGA success at week 4, respectively, while 90% and 50% achieved PASI-75, 90% and 40% achieved WI-NRS success, and the mean BSA reductions at week 4 were 79.1% and 44.4%. Meanwhile, one younger patient in study 216 reported a treatment-emergent adverse event (TEAE) of headache, which was considered mild, while four older patients in study 215 reported 8 TEAEs, which were considered mild and ranged from back pain to nasal congestion.
“The rapid onset of action was surprising but exceedingly rewarding for the subjects enrolled in the study,” Dr. Hebert told this news organization after the meeting. “The PASI scores and itch scores were markedly improved at the end of the 4-week clinical trial. Patient and parents alike were pleased to use a steroid-free option with once-daily application and rapid onset of action to help control plaque psoriasis.”
In the poster abstract, she and her coauthors concluded that “under maximal use conditions in children aged 2-11 years, roflumilast cream 0.3% was well tolerated and improved signs and symptoms of psoriasis with measured improvements in IGA score, PASI score, BSA involvement, CDLQI, and WI-NRS. Overall, pharmacokinetics, safety, tolerability, and efficacy in patients aged 2-11 years were consistent with prior results in adults and adolescents.”
The study was funded by Arcutis Biotherapeutics. Dr. Hebert reported that she is an investigator for Arcutis. About half the coauthors are employees of Arcutis, and the other half disclosed grants, research funding and/or honoraria from the company. Research grants from the company for this study were paid to the McGovern Medical School at the University of Texas.
In patients aged 2-11 years, roflumilast cream was well tolerated and improved signs and symptoms of psoriasis over 4 weeks, according to results from a pair of phase two studies.
“Limited topical treatments are approved for children younger than 12 years old with psoriasis,” researchers led by Adelaide A. Hebert, MD, wrote in their abstract. The results were presented during a poster session at the annual meeting of the Society for Pediatric Dermatology.
Roflumilast cream 0.3% (Zoryve) is a once-daily, topical nonsteroidal treatment from Arcutis Biotherapeutics. A phosphodiesterase-4 inhibitor, it was approved by the Food and Drug Administration in 2022 for mild, moderate and severe psoriasis in individuals aged 12 and older, including intertriginous psoriasis.
For the analysis, Dr. Hebert, chief of pediatric dermatology at the University of Texas, Houston, and colleagues conducted two 4-week, phase 2, open-label safety studies of roflumilast cream 0.3%.
One, study 216, enrolled 10 children aged 2-5, and all but one were Black. The other, study 215, enrolled 20 children aged 6-11, and half were Black and nearly half were White. At baseline, patients had 2% or greater body surface area (BSA) involvement and an Investigator Global Assessment (IGA) score of at least mild.
Caregivers applied roflumilast cream to all affected areas once daily for 28 days. The researchers collected pharmacokinetic samples at week 2 and week 4. The primary endpoints were pharmacokinetic, safety, and tolerability.
Efficacy was evaluated as exploratory endpoints: An IGA of clear or almost clear plus a 2-grade or more improvement from baseline, a 50% or greater improvement and a 75% or greater improvement on the Psoriasis Area and Severity Index (PASI-50 and PASI-75), a 4-point or greater reduction in the Worst Itch–Numeric Rating Scale (WI-NRS) in patients with a baseline score of 4 or greater, a mean change from baseline in BSA, and improvement in the Children’s Dermatology Life Quality Index (CDLQI).
At baseline, the mean BSA was similar for patients enrolled in studies 216 and 215 (9.6% and 8.8%, respectively), and 80% of all patients had baseline IGA of moderate. By week 2, the mean roflumilast and N-oxide predose plasma concentrations among patients in the younger group were 2.15 and 22.4 ng/mL, compared with 3.15 and 28.9 ng/mL among those in the older group. At week 4, the mean roflumilast and N-oxide predose concentrations were 2.04 and 15.8 ng/mL in the younger group (study 216), compared with 1.68 and 15.7 ng/mL in the older group (study 215).
As for efficacy, 90% and 40% of patients in studies 216 and 215 achieved IGA success at week 4, respectively, while 90% and 50% achieved PASI-75, 90% and 40% achieved WI-NRS success, and the mean BSA reductions at week 4 were 79.1% and 44.4%. Meanwhile, one younger patient in study 216 reported a treatment-emergent adverse event (TEAE) of headache, which was considered mild, while four older patients in study 215 reported 8 TEAEs, which were considered mild and ranged from back pain to nasal congestion.
“The rapid onset of action was surprising but exceedingly rewarding for the subjects enrolled in the study,” Dr. Hebert told this news organization after the meeting. “The PASI scores and itch scores were markedly improved at the end of the 4-week clinical trial. Patient and parents alike were pleased to use a steroid-free option with once-daily application and rapid onset of action to help control plaque psoriasis.”
In the poster abstract, she and her coauthors concluded that “under maximal use conditions in children aged 2-11 years, roflumilast cream 0.3% was well tolerated and improved signs and symptoms of psoriasis with measured improvements in IGA score, PASI score, BSA involvement, CDLQI, and WI-NRS. Overall, pharmacokinetics, safety, tolerability, and efficacy in patients aged 2-11 years were consistent with prior results in adults and adolescents.”
The study was funded by Arcutis Biotherapeutics. Dr. Hebert reported that she is an investigator for Arcutis. About half the coauthors are employees of Arcutis, and the other half disclosed grants, research funding and/or honoraria from the company. Research grants from the company for this study were paid to the McGovern Medical School at the University of Texas.
FROM SPD 2023
Progress seen on five fronts for substantially improving treatment of epidermolysis bullosa
ASHEVILLE, N.C. – , according to a prominent EB researcher.
Not only are recent developments in EB “exciting,” the progress on multiple fronts for control of disease or its symptoms suggests “we are on the cusp of a new era,” Jemima Mellerio, BSc, MD, a consultant dermatologist, St. John’s Institute of Dermatology, London, said at the annual meeting of the Society for Pediatric Dermatology.
Published clinical studies of cell therapies and gene therapies date back at least 15 years, according to a review by Dr. Mellerio on why developments are starting to move so quickly. The difference now is that many obstacles to routine use of these options are being resolved so that viable strategies have reached or are reaching phase 3 trials.
In addition to cell therapies and gene therapies, Dr. Mellerio discussed progress in three additional areas: gene editing, protein therapy, and drug repurposing.
Summarizing progress in each, she described improvement in levels of collagen VII, an important deficit in most types of EB, that were achieved with fibroblast injections that improved levels of collagen VII and anchoring fibrils in a study published in the Journal of Investigative Dermatology. Injection of mesenchymal stromal cells (MSC) have been associated with reduced pain and itch in a series of studies, one of the earliest of which was published in the New England Journal of Medicine.
Since that time, there have been several approaches using MSC.
Of these approaches, intravenous injection of ABCB5+ MSCs might be the first to gain regulatory approval. According to Dr. Mellerio, there is an ongoing phase 3 crossover trial evaluating this approach, which followed several earlier phase studies that demonstrated adequate safety and tolerability while reducing severity scores, relieving pain and itch, and improving wound closure in patients with EB.
In 2006, correction of junctional EB (JEB) was achieved by transplantation of genetically modified epidermal cells to replace the LAMB3 gene, thereby restoring production of laminin 332, which is an essential component of the dermal-epidermal junction, according to Dr. Mellerio, citing a study in Nature Medicine.
The next attempt with this approach did not take place until 2015, resurrected to save the life of a 7-year-old Syrian boy – to generate epidermal sheets that eventually covered 80% of his body. The success is supporting further work on this approach but has also been an inspiration to other gene therapies, including a topical gene therapy recently approved in the United States.
Topically applied beremagene geperpavec (Vyjuvek, formerly known as B-VEC) was approved by the FDA in May for treating wounds in patients 6 months of age and older, with recessive or dominant dystrophic EB, on the basis of a phase 3 trial published in the New England Journal of Medicine, but others are coming. Dr. Mellerio also described a recently completed phase 3 trial with introduction of ex vivo gene-corrected keratinocytes, which has been associated with long-term improvements among patients with recessive dystrophic EB (RDEB). The responses in early phase studies included wound healing and reduction in pain and itch.
Perhaps less advanced but still promising, protein therapy, gene editing, and repurposing of existing therapies are all approaches that are moving forward. Many are supported by at least some clinical data, according to Dr. Mellerio.
As an example of protein therapy, a completed phase I/II trial associated recombinant human collagen with wound healing and pain reduction in RDEB. This study provided proof of principle for a therapy that could be applied topically or intravenously. Further development is anticipated.
Multiple platforms for gene editing have been described with the goal of simply excising pathogenic mutations or antisense oligonucleotides for sustained or permanent control of EB expression. Clinical evidence is limited, but Dr. Mellerio suggested that the theoretical potential for eliminating the source of abnormal transcription is the restoration of functional proteins essential for reversing skin fragility.
In some cases, existing drugs have the same potential. Dr. Mellerio described efforts to use an aminoglycoside to circumvent nonsense mutations that produce messenger RNA decay and impaired production of the proteins that prevent EB. In a pilot study evaluating topical gentamicin in RDEB, there were substantial improvements at 1 month and 3 months in several measures of skin fragility and encouraged studies that are now ongoing in both RDEB and JEB.
More than promising, a multinational randomized phase 3 study with birch bark extract recently published in the British Journal of Dermatology, associated treatment with this topical gel, known as Oleogel-S10, with higher rates of complete wound closure at 45 days (41.3% vs. 28.9% in the control vehicle arm) and a low risk of adverse events.
“This therapy is now approved in Europe and the United Kingdom, although, unfortunately, it is not yet available in the United States,” Dr. Mellerio noted.
Importantly, none of these therapies are necessarily effective across subtypes of EB, which often have different underlying pathogenic mechanisms, she said. However, the growing sophistication with which the pathophysiology of these subtypes is understood makes the numerous treatments in the pipeline “exciting.”
“We are at a point where we can really start to think of personalized medicine in EB,” Dr. Mellerio said. With the clinical advances already available and those expected, she suggested the recently approved treatment options are just the beginning. She expects the treatment landscape to evolve quickly over the next few years.
This does not appear to be a personal opinion. Another prominent researcher in EB, M. Peter Marinkovich, MD, director of the Stanford Bullous Disease and Psoriasis Clinics at Stanford (Calif.) University, is seeing the same real-world promise of therapies that have been in gestation for a decade or more.
“Dr. Mellerio is right. This is an exciting time for EB patients,” Dr. Marinkovich said in an interview. While the approval of B-VEC, the first gene therapy for EB, is the proof, Dr. Marinkovich, the lead author of the NEJM paper on B-VEC, noted that “many other potential EB therapies are being studied right now.” Based on promise in earlier clinical studies with many of these agents, he, like Dr. Mellerio, expects progress in real-world treatments for EB to accelerate.
Dr. Mellerio reported financial relationships with Amryt Pharma and Krystal Biotech. Dr. Marinkovich receives research support from Abeona Therapeutics, Castle Creek Pharmaceuticals, Krystal Biotech, Phoenix Tissue Repair, and WINGS Therapeutics.
A version of this article first appeared on Medscape.com.
ASHEVILLE, N.C. – , according to a prominent EB researcher.
Not only are recent developments in EB “exciting,” the progress on multiple fronts for control of disease or its symptoms suggests “we are on the cusp of a new era,” Jemima Mellerio, BSc, MD, a consultant dermatologist, St. John’s Institute of Dermatology, London, said at the annual meeting of the Society for Pediatric Dermatology.
Published clinical studies of cell therapies and gene therapies date back at least 15 years, according to a review by Dr. Mellerio on why developments are starting to move so quickly. The difference now is that many obstacles to routine use of these options are being resolved so that viable strategies have reached or are reaching phase 3 trials.
In addition to cell therapies and gene therapies, Dr. Mellerio discussed progress in three additional areas: gene editing, protein therapy, and drug repurposing.
Summarizing progress in each, she described improvement in levels of collagen VII, an important deficit in most types of EB, that were achieved with fibroblast injections that improved levels of collagen VII and anchoring fibrils in a study published in the Journal of Investigative Dermatology. Injection of mesenchymal stromal cells (MSC) have been associated with reduced pain and itch in a series of studies, one of the earliest of which was published in the New England Journal of Medicine.
Since that time, there have been several approaches using MSC.
Of these approaches, intravenous injection of ABCB5+ MSCs might be the first to gain regulatory approval. According to Dr. Mellerio, there is an ongoing phase 3 crossover trial evaluating this approach, which followed several earlier phase studies that demonstrated adequate safety and tolerability while reducing severity scores, relieving pain and itch, and improving wound closure in patients with EB.
In 2006, correction of junctional EB (JEB) was achieved by transplantation of genetically modified epidermal cells to replace the LAMB3 gene, thereby restoring production of laminin 332, which is an essential component of the dermal-epidermal junction, according to Dr. Mellerio, citing a study in Nature Medicine.
The next attempt with this approach did not take place until 2015, resurrected to save the life of a 7-year-old Syrian boy – to generate epidermal sheets that eventually covered 80% of his body. The success is supporting further work on this approach but has also been an inspiration to other gene therapies, including a topical gene therapy recently approved in the United States.
Topically applied beremagene geperpavec (Vyjuvek, formerly known as B-VEC) was approved by the FDA in May for treating wounds in patients 6 months of age and older, with recessive or dominant dystrophic EB, on the basis of a phase 3 trial published in the New England Journal of Medicine, but others are coming. Dr. Mellerio also described a recently completed phase 3 trial with introduction of ex vivo gene-corrected keratinocytes, which has been associated with long-term improvements among patients with recessive dystrophic EB (RDEB). The responses in early phase studies included wound healing and reduction in pain and itch.
Perhaps less advanced but still promising, protein therapy, gene editing, and repurposing of existing therapies are all approaches that are moving forward. Many are supported by at least some clinical data, according to Dr. Mellerio.
As an example of protein therapy, a completed phase I/II trial associated recombinant human collagen with wound healing and pain reduction in RDEB. This study provided proof of principle for a therapy that could be applied topically or intravenously. Further development is anticipated.
Multiple platforms for gene editing have been described with the goal of simply excising pathogenic mutations or antisense oligonucleotides for sustained or permanent control of EB expression. Clinical evidence is limited, but Dr. Mellerio suggested that the theoretical potential for eliminating the source of abnormal transcription is the restoration of functional proteins essential for reversing skin fragility.
In some cases, existing drugs have the same potential. Dr. Mellerio described efforts to use an aminoglycoside to circumvent nonsense mutations that produce messenger RNA decay and impaired production of the proteins that prevent EB. In a pilot study evaluating topical gentamicin in RDEB, there were substantial improvements at 1 month and 3 months in several measures of skin fragility and encouraged studies that are now ongoing in both RDEB and JEB.
More than promising, a multinational randomized phase 3 study with birch bark extract recently published in the British Journal of Dermatology, associated treatment with this topical gel, known as Oleogel-S10, with higher rates of complete wound closure at 45 days (41.3% vs. 28.9% in the control vehicle arm) and a low risk of adverse events.
“This therapy is now approved in Europe and the United Kingdom, although, unfortunately, it is not yet available in the United States,” Dr. Mellerio noted.
Importantly, none of these therapies are necessarily effective across subtypes of EB, which often have different underlying pathogenic mechanisms, she said. However, the growing sophistication with which the pathophysiology of these subtypes is understood makes the numerous treatments in the pipeline “exciting.”
“We are at a point where we can really start to think of personalized medicine in EB,” Dr. Mellerio said. With the clinical advances already available and those expected, she suggested the recently approved treatment options are just the beginning. She expects the treatment landscape to evolve quickly over the next few years.
This does not appear to be a personal opinion. Another prominent researcher in EB, M. Peter Marinkovich, MD, director of the Stanford Bullous Disease and Psoriasis Clinics at Stanford (Calif.) University, is seeing the same real-world promise of therapies that have been in gestation for a decade or more.
“Dr. Mellerio is right. This is an exciting time for EB patients,” Dr. Marinkovich said in an interview. While the approval of B-VEC, the first gene therapy for EB, is the proof, Dr. Marinkovich, the lead author of the NEJM paper on B-VEC, noted that “many other potential EB therapies are being studied right now.” Based on promise in earlier clinical studies with many of these agents, he, like Dr. Mellerio, expects progress in real-world treatments for EB to accelerate.
Dr. Mellerio reported financial relationships with Amryt Pharma and Krystal Biotech. Dr. Marinkovich receives research support from Abeona Therapeutics, Castle Creek Pharmaceuticals, Krystal Biotech, Phoenix Tissue Repair, and WINGS Therapeutics.
A version of this article first appeared on Medscape.com.
ASHEVILLE, N.C. – , according to a prominent EB researcher.
Not only are recent developments in EB “exciting,” the progress on multiple fronts for control of disease or its symptoms suggests “we are on the cusp of a new era,” Jemima Mellerio, BSc, MD, a consultant dermatologist, St. John’s Institute of Dermatology, London, said at the annual meeting of the Society for Pediatric Dermatology.
Published clinical studies of cell therapies and gene therapies date back at least 15 years, according to a review by Dr. Mellerio on why developments are starting to move so quickly. The difference now is that many obstacles to routine use of these options are being resolved so that viable strategies have reached or are reaching phase 3 trials.
In addition to cell therapies and gene therapies, Dr. Mellerio discussed progress in three additional areas: gene editing, protein therapy, and drug repurposing.
Summarizing progress in each, she described improvement in levels of collagen VII, an important deficit in most types of EB, that were achieved with fibroblast injections that improved levels of collagen VII and anchoring fibrils in a study published in the Journal of Investigative Dermatology. Injection of mesenchymal stromal cells (MSC) have been associated with reduced pain and itch in a series of studies, one of the earliest of which was published in the New England Journal of Medicine.
Since that time, there have been several approaches using MSC.
Of these approaches, intravenous injection of ABCB5+ MSCs might be the first to gain regulatory approval. According to Dr. Mellerio, there is an ongoing phase 3 crossover trial evaluating this approach, which followed several earlier phase studies that demonstrated adequate safety and tolerability while reducing severity scores, relieving pain and itch, and improving wound closure in patients with EB.
In 2006, correction of junctional EB (JEB) was achieved by transplantation of genetically modified epidermal cells to replace the LAMB3 gene, thereby restoring production of laminin 332, which is an essential component of the dermal-epidermal junction, according to Dr. Mellerio, citing a study in Nature Medicine.
The next attempt with this approach did not take place until 2015, resurrected to save the life of a 7-year-old Syrian boy – to generate epidermal sheets that eventually covered 80% of his body. The success is supporting further work on this approach but has also been an inspiration to other gene therapies, including a topical gene therapy recently approved in the United States.
Topically applied beremagene geperpavec (Vyjuvek, formerly known as B-VEC) was approved by the FDA in May for treating wounds in patients 6 months of age and older, with recessive or dominant dystrophic EB, on the basis of a phase 3 trial published in the New England Journal of Medicine, but others are coming. Dr. Mellerio also described a recently completed phase 3 trial with introduction of ex vivo gene-corrected keratinocytes, which has been associated with long-term improvements among patients with recessive dystrophic EB (RDEB). The responses in early phase studies included wound healing and reduction in pain and itch.
Perhaps less advanced but still promising, protein therapy, gene editing, and repurposing of existing therapies are all approaches that are moving forward. Many are supported by at least some clinical data, according to Dr. Mellerio.
As an example of protein therapy, a completed phase I/II trial associated recombinant human collagen with wound healing and pain reduction in RDEB. This study provided proof of principle for a therapy that could be applied topically or intravenously. Further development is anticipated.
Multiple platforms for gene editing have been described with the goal of simply excising pathogenic mutations or antisense oligonucleotides for sustained or permanent control of EB expression. Clinical evidence is limited, but Dr. Mellerio suggested that the theoretical potential for eliminating the source of abnormal transcription is the restoration of functional proteins essential for reversing skin fragility.
In some cases, existing drugs have the same potential. Dr. Mellerio described efforts to use an aminoglycoside to circumvent nonsense mutations that produce messenger RNA decay and impaired production of the proteins that prevent EB. In a pilot study evaluating topical gentamicin in RDEB, there were substantial improvements at 1 month and 3 months in several measures of skin fragility and encouraged studies that are now ongoing in both RDEB and JEB.
More than promising, a multinational randomized phase 3 study with birch bark extract recently published in the British Journal of Dermatology, associated treatment with this topical gel, known as Oleogel-S10, with higher rates of complete wound closure at 45 days (41.3% vs. 28.9% in the control vehicle arm) and a low risk of adverse events.
“This therapy is now approved in Europe and the United Kingdom, although, unfortunately, it is not yet available in the United States,” Dr. Mellerio noted.
Importantly, none of these therapies are necessarily effective across subtypes of EB, which often have different underlying pathogenic mechanisms, she said. However, the growing sophistication with which the pathophysiology of these subtypes is understood makes the numerous treatments in the pipeline “exciting.”
“We are at a point where we can really start to think of personalized medicine in EB,” Dr. Mellerio said. With the clinical advances already available and those expected, she suggested the recently approved treatment options are just the beginning. She expects the treatment landscape to evolve quickly over the next few years.
This does not appear to be a personal opinion. Another prominent researcher in EB, M. Peter Marinkovich, MD, director of the Stanford Bullous Disease and Psoriasis Clinics at Stanford (Calif.) University, is seeing the same real-world promise of therapies that have been in gestation for a decade or more.
“Dr. Mellerio is right. This is an exciting time for EB patients,” Dr. Marinkovich said in an interview. While the approval of B-VEC, the first gene therapy for EB, is the proof, Dr. Marinkovich, the lead author of the NEJM paper on B-VEC, noted that “many other potential EB therapies are being studied right now.” Based on promise in earlier clinical studies with many of these agents, he, like Dr. Mellerio, expects progress in real-world treatments for EB to accelerate.
Dr. Mellerio reported financial relationships with Amryt Pharma and Krystal Biotech. Dr. Marinkovich receives research support from Abeona Therapeutics, Castle Creek Pharmaceuticals, Krystal Biotech, Phoenix Tissue Repair, and WINGS Therapeutics.
A version of this article first appeared on Medscape.com.
AT SPD 2023
Pediatric dermatologists encouraged to counter misinformation on TikTok, other social media sites
ASHEVILLE, N.C. – , warned an expert at the annual meeting of the Society for Pediatric Dermatology.
“If we don’t get involved, we are basically letting misinformation win. We need to be there,” said Angelo Landriscina, MD, director of dermatology at a Mount Sinai Doctors Clinic in New York.
Most of the content currently available on medical topics, including dermatology and pediatric dermatology, is not created by health care professionals, Dr. Landriscina noted. Not surprisingly, given that much of the content is based on personal opinion from individuals who have no expertise in medical care, he described the information as being of “low quality” when not fully erroneous.
Dr. Landriscina has been active on social media, including TikTok, for several years. Most of his posts involve responses to misinformation. When he sets the record straight on the basis of existing evidence, he often supports his counterargument with references.
He acknowledged that when he became involved in social media he faced criticism from colleagues about participating on an entertainment platform that many considered unworthy of providing objective information. If that was ever true, he argued, it is no longer the case.
“TikTok has adopted a new strategy. The goal is to unseat Google as a search tool, and it’s working,” he said. He explained that many people now use TikTok and other social media sites as their primary source of information on essentially every topic, from where to eat to whether to be screened for cancer.
The particular problem with TikTok – one of the most popular social media outlets – is that there is no mechanism for vetting the source of information. YouTube, by contrast, now requires some sort of validation for anyone who claims to have a medical degree or any other verifiable qualification, according to Dr. Landriscina. TikTok, like many other platforms, has no such requirement.
“Anyone can buy a pair of scrubs [implying expertise] and then post a video,” Dr. Landriscina said.
Even if information from one content provider is more valid than information from others, the TikTok algorithm is specifically designed to emphasize content that has the potential for going viral, which means it favors videos that are provocative over those that are not.
“The algorithm favors any content that is more controversial, more surprising, and keeps viewers engaged,” Dr. Landriscina pointed out.
This does not mean that objective and factual information is ignored, but the algorithm is indifferent to the validity of information, meaning that it allows videos to be posted without regard to whether the content is true, untrue, purposefully misleading, or utter nonsense. For that reason, it is often easier to attract attention by responding to a post that has already gone viral. Information that is clear and digestible can attract viewers and therefore is distributed more widely with the TikTok algorithm.
Parents are on Tiktok too
There is a misperception that the TikTok audience is younger, according to Dr. Landriscina. While peak use in the United States fell among people between the ages of 25 and 34 years in 2022, he said the number of users falls off relatively slowly with subsequent 10-year increments in age. In 2022, there were nearly 20 million users in the peak 10-year age range, but 7.5 million users were 55 years of age or older.
“Pediatric dermatologists should recognize that it is not just kids who are looking for information about their skin diseases, but also their parents,” Dr. Landriscina said.
The top three dermatology topics searched on TikTok in a recent period were acne, alopecia, and cysts. But top searches are very fluid and are extremely hard to quantify, because the basis of the algorithm, which is a proprietary secret, is not only unknown but produces different results for every user.
“The second you touch the app, it changes,” Dr. Landriscina said. He explained that an inquiry about any subject, including those that are medically related, yields content that is different, or at least ordered differently, “depending on how you behaved on the app in the past.”
The phenomenon that drives social media predates this technology. Dr. Landriscina cited a study in 1956 that described the “parasocial interaction theory.” The theory was based on the observation that those who consume media, such as television, which was relatively new in 1956, believed that they had a personal relationship with media figures.
“The users begin to trust influencers as a source, like a friend providing them advice,” Dr. Landriscina said. As an example, he suggested that a fan of the television show Friends who follows actor Jennifer Aniston on social media platforms may begin to think of her as a trusted source of information on any topic, including those for which she may not have expertise.
The reason that he urges medical professionals to become active on TikTok and other social media platforms is that they have a potentially critical role in responding to information that is not just wrong but harmful.
On TikTok and other social media platforms, “there is a lot of interest in content about dermatologic conditions in children. There is a real need for accurate information,” he said,
In the question-and-answer session following his presentation, Dr. Landriscina’s message was not uniformly embraced. One risk, according to an audience member, is that medical professionals will begin to express their own personal opinions rather than rely on evidence, with the result that they will “just add to the sea of misinformation.”
However, this opinion appeared to be the minority view. Most of those who commented took a “that-ship-has-sailed” stance, recognizing the irreversible ascendancy of social media.
“Whether you like it or not, social media is here to stay. We cannot fight it. Rather, we need to embrace it in a responsible way,” said Dakara R. Wright, MD, a dermatologist at the Mid-Atlantic Kaiser Permanente Group, Halethorpe, Md. She, like others, reported that she has come to recognize that social media is a major source of medical information for her patients.
“We need to be a presence on these platforms for the benefit of our patients and their parents,” she said. She acknowledged that she has not been active in posting on social media in the past but said that she has been speaking with administrators in her organization about how to become involved in a responsible way that can be useful to patients.
Candrice R. Heath, MD, assistant professor of dermatology at Temple University, Philadelphia, has been active on social media for several years, posting content on her own account, which is not related to her academic affiliation. She posts for many reasons, not least of which is drawing attention to her expertise.
Like Dr. Landriscina, she recognizes that users of these platforms are guided by the content to make decisions about health care. She also agreed that physicians should not ignore this phenomenon.
Tips on providing content
Given the fact that the algorithm is intended to produce posts that go viral, Dr. Landriscina urged clinicians to make their content easy to watch. He said it is not necessary to overthink content beyond providing accurate information, but he advised that videos be made with attention to adequate lighting and other simple factors to promote visual quality. He said that accurate information is not necessarily dull.
“Some facts can actually be surprising to patients,” he said. He noted that a calm, coherent video can be particularly effective in attracting an audience when it is in reaction to information that has gone viral but is misleading or patently incorrect.
Dr. Landriscina has been an influencer associated with multiple social media platforms, including TikTok. He has in the past been paid for consulting work for TikTok. Dr. Wright and Dr. Heath reported no potential conflicts of interest.
A version of this article first appeared on Medscape.com.
ASHEVILLE, N.C. – , warned an expert at the annual meeting of the Society for Pediatric Dermatology.
“If we don’t get involved, we are basically letting misinformation win. We need to be there,” said Angelo Landriscina, MD, director of dermatology at a Mount Sinai Doctors Clinic in New York.
Most of the content currently available on medical topics, including dermatology and pediatric dermatology, is not created by health care professionals, Dr. Landriscina noted. Not surprisingly, given that much of the content is based on personal opinion from individuals who have no expertise in medical care, he described the information as being of “low quality” when not fully erroneous.
Dr. Landriscina has been active on social media, including TikTok, for several years. Most of his posts involve responses to misinformation. When he sets the record straight on the basis of existing evidence, he often supports his counterargument with references.
He acknowledged that when he became involved in social media he faced criticism from colleagues about participating on an entertainment platform that many considered unworthy of providing objective information. If that was ever true, he argued, it is no longer the case.
“TikTok has adopted a new strategy. The goal is to unseat Google as a search tool, and it’s working,” he said. He explained that many people now use TikTok and other social media sites as their primary source of information on essentially every topic, from where to eat to whether to be screened for cancer.
The particular problem with TikTok – one of the most popular social media outlets – is that there is no mechanism for vetting the source of information. YouTube, by contrast, now requires some sort of validation for anyone who claims to have a medical degree or any other verifiable qualification, according to Dr. Landriscina. TikTok, like many other platforms, has no such requirement.
“Anyone can buy a pair of scrubs [implying expertise] and then post a video,” Dr. Landriscina said.
Even if information from one content provider is more valid than information from others, the TikTok algorithm is specifically designed to emphasize content that has the potential for going viral, which means it favors videos that are provocative over those that are not.
“The algorithm favors any content that is more controversial, more surprising, and keeps viewers engaged,” Dr. Landriscina pointed out.
This does not mean that objective and factual information is ignored, but the algorithm is indifferent to the validity of information, meaning that it allows videos to be posted without regard to whether the content is true, untrue, purposefully misleading, or utter nonsense. For that reason, it is often easier to attract attention by responding to a post that has already gone viral. Information that is clear and digestible can attract viewers and therefore is distributed more widely with the TikTok algorithm.
Parents are on Tiktok too
There is a misperception that the TikTok audience is younger, according to Dr. Landriscina. While peak use in the United States fell among people between the ages of 25 and 34 years in 2022, he said the number of users falls off relatively slowly with subsequent 10-year increments in age. In 2022, there were nearly 20 million users in the peak 10-year age range, but 7.5 million users were 55 years of age or older.
“Pediatric dermatologists should recognize that it is not just kids who are looking for information about their skin diseases, but also their parents,” Dr. Landriscina said.
The top three dermatology topics searched on TikTok in a recent period were acne, alopecia, and cysts. But top searches are very fluid and are extremely hard to quantify, because the basis of the algorithm, which is a proprietary secret, is not only unknown but produces different results for every user.
“The second you touch the app, it changes,” Dr. Landriscina said. He explained that an inquiry about any subject, including those that are medically related, yields content that is different, or at least ordered differently, “depending on how you behaved on the app in the past.”
The phenomenon that drives social media predates this technology. Dr. Landriscina cited a study in 1956 that described the “parasocial interaction theory.” The theory was based on the observation that those who consume media, such as television, which was relatively new in 1956, believed that they had a personal relationship with media figures.
“The users begin to trust influencers as a source, like a friend providing them advice,” Dr. Landriscina said. As an example, he suggested that a fan of the television show Friends who follows actor Jennifer Aniston on social media platforms may begin to think of her as a trusted source of information on any topic, including those for which she may not have expertise.
The reason that he urges medical professionals to become active on TikTok and other social media platforms is that they have a potentially critical role in responding to information that is not just wrong but harmful.
On TikTok and other social media platforms, “there is a lot of interest in content about dermatologic conditions in children. There is a real need for accurate information,” he said,
In the question-and-answer session following his presentation, Dr. Landriscina’s message was not uniformly embraced. One risk, according to an audience member, is that medical professionals will begin to express their own personal opinions rather than rely on evidence, with the result that they will “just add to the sea of misinformation.”
However, this opinion appeared to be the minority view. Most of those who commented took a “that-ship-has-sailed” stance, recognizing the irreversible ascendancy of social media.
“Whether you like it or not, social media is here to stay. We cannot fight it. Rather, we need to embrace it in a responsible way,” said Dakara R. Wright, MD, a dermatologist at the Mid-Atlantic Kaiser Permanente Group, Halethorpe, Md. She, like others, reported that she has come to recognize that social media is a major source of medical information for her patients.
“We need to be a presence on these platforms for the benefit of our patients and their parents,” she said. She acknowledged that she has not been active in posting on social media in the past but said that she has been speaking with administrators in her organization about how to become involved in a responsible way that can be useful to patients.
Candrice R. Heath, MD, assistant professor of dermatology at Temple University, Philadelphia, has been active on social media for several years, posting content on her own account, which is not related to her academic affiliation. She posts for many reasons, not least of which is drawing attention to her expertise.
Like Dr. Landriscina, she recognizes that users of these platforms are guided by the content to make decisions about health care. She also agreed that physicians should not ignore this phenomenon.
Tips on providing content
Given the fact that the algorithm is intended to produce posts that go viral, Dr. Landriscina urged clinicians to make their content easy to watch. He said it is not necessary to overthink content beyond providing accurate information, but he advised that videos be made with attention to adequate lighting and other simple factors to promote visual quality. He said that accurate information is not necessarily dull.
“Some facts can actually be surprising to patients,” he said. He noted that a calm, coherent video can be particularly effective in attracting an audience when it is in reaction to information that has gone viral but is misleading or patently incorrect.
Dr. Landriscina has been an influencer associated with multiple social media platforms, including TikTok. He has in the past been paid for consulting work for TikTok. Dr. Wright and Dr. Heath reported no potential conflicts of interest.
A version of this article first appeared on Medscape.com.
ASHEVILLE, N.C. – , warned an expert at the annual meeting of the Society for Pediatric Dermatology.
“If we don’t get involved, we are basically letting misinformation win. We need to be there,” said Angelo Landriscina, MD, director of dermatology at a Mount Sinai Doctors Clinic in New York.
Most of the content currently available on medical topics, including dermatology and pediatric dermatology, is not created by health care professionals, Dr. Landriscina noted. Not surprisingly, given that much of the content is based on personal opinion from individuals who have no expertise in medical care, he described the information as being of “low quality” when not fully erroneous.
Dr. Landriscina has been active on social media, including TikTok, for several years. Most of his posts involve responses to misinformation. When he sets the record straight on the basis of existing evidence, he often supports his counterargument with references.
He acknowledged that when he became involved in social media he faced criticism from colleagues about participating on an entertainment platform that many considered unworthy of providing objective information. If that was ever true, he argued, it is no longer the case.
“TikTok has adopted a new strategy. The goal is to unseat Google as a search tool, and it’s working,” he said. He explained that many people now use TikTok and other social media sites as their primary source of information on essentially every topic, from where to eat to whether to be screened for cancer.
The particular problem with TikTok – one of the most popular social media outlets – is that there is no mechanism for vetting the source of information. YouTube, by contrast, now requires some sort of validation for anyone who claims to have a medical degree or any other verifiable qualification, according to Dr. Landriscina. TikTok, like many other platforms, has no such requirement.
“Anyone can buy a pair of scrubs [implying expertise] and then post a video,” Dr. Landriscina said.
Even if information from one content provider is more valid than information from others, the TikTok algorithm is specifically designed to emphasize content that has the potential for going viral, which means it favors videos that are provocative over those that are not.
“The algorithm favors any content that is more controversial, more surprising, and keeps viewers engaged,” Dr. Landriscina pointed out.
This does not mean that objective and factual information is ignored, but the algorithm is indifferent to the validity of information, meaning that it allows videos to be posted without regard to whether the content is true, untrue, purposefully misleading, or utter nonsense. For that reason, it is often easier to attract attention by responding to a post that has already gone viral. Information that is clear and digestible can attract viewers and therefore is distributed more widely with the TikTok algorithm.
Parents are on Tiktok too
There is a misperception that the TikTok audience is younger, according to Dr. Landriscina. While peak use in the United States fell among people between the ages of 25 and 34 years in 2022, he said the number of users falls off relatively slowly with subsequent 10-year increments in age. In 2022, there were nearly 20 million users in the peak 10-year age range, but 7.5 million users were 55 years of age or older.
“Pediatric dermatologists should recognize that it is not just kids who are looking for information about their skin diseases, but also their parents,” Dr. Landriscina said.
The top three dermatology topics searched on TikTok in a recent period were acne, alopecia, and cysts. But top searches are very fluid and are extremely hard to quantify, because the basis of the algorithm, which is a proprietary secret, is not only unknown but produces different results for every user.
“The second you touch the app, it changes,” Dr. Landriscina said. He explained that an inquiry about any subject, including those that are medically related, yields content that is different, or at least ordered differently, “depending on how you behaved on the app in the past.”
The phenomenon that drives social media predates this technology. Dr. Landriscina cited a study in 1956 that described the “parasocial interaction theory.” The theory was based on the observation that those who consume media, such as television, which was relatively new in 1956, believed that they had a personal relationship with media figures.
“The users begin to trust influencers as a source, like a friend providing them advice,” Dr. Landriscina said. As an example, he suggested that a fan of the television show Friends who follows actor Jennifer Aniston on social media platforms may begin to think of her as a trusted source of information on any topic, including those for which she may not have expertise.
The reason that he urges medical professionals to become active on TikTok and other social media platforms is that they have a potentially critical role in responding to information that is not just wrong but harmful.
On TikTok and other social media platforms, “there is a lot of interest in content about dermatologic conditions in children. There is a real need for accurate information,” he said,
In the question-and-answer session following his presentation, Dr. Landriscina’s message was not uniformly embraced. One risk, according to an audience member, is that medical professionals will begin to express their own personal opinions rather than rely on evidence, with the result that they will “just add to the sea of misinformation.”
However, this opinion appeared to be the minority view. Most of those who commented took a “that-ship-has-sailed” stance, recognizing the irreversible ascendancy of social media.
“Whether you like it or not, social media is here to stay. We cannot fight it. Rather, we need to embrace it in a responsible way,” said Dakara R. Wright, MD, a dermatologist at the Mid-Atlantic Kaiser Permanente Group, Halethorpe, Md. She, like others, reported that she has come to recognize that social media is a major source of medical information for her patients.
“We need to be a presence on these platforms for the benefit of our patients and their parents,” she said. She acknowledged that she has not been active in posting on social media in the past but said that she has been speaking with administrators in her organization about how to become involved in a responsible way that can be useful to patients.
Candrice R. Heath, MD, assistant professor of dermatology at Temple University, Philadelphia, has been active on social media for several years, posting content on her own account, which is not related to her academic affiliation. She posts for many reasons, not least of which is drawing attention to her expertise.
Like Dr. Landriscina, she recognizes that users of these platforms are guided by the content to make decisions about health care. She also agreed that physicians should not ignore this phenomenon.
Tips on providing content
Given the fact that the algorithm is intended to produce posts that go viral, Dr. Landriscina urged clinicians to make their content easy to watch. He said it is not necessary to overthink content beyond providing accurate information, but he advised that videos be made with attention to adequate lighting and other simple factors to promote visual quality. He said that accurate information is not necessarily dull.
“Some facts can actually be surprising to patients,” he said. He noted that a calm, coherent video can be particularly effective in attracting an audience when it is in reaction to information that has gone viral but is misleading or patently incorrect.
Dr. Landriscina has been an influencer associated with multiple social media platforms, including TikTok. He has in the past been paid for consulting work for TikTok. Dr. Wright and Dr. Heath reported no potential conflicts of interest.
A version of this article first appeared on Medscape.com.
AT SPD 2023
Study examines pediatric skin biopsy trends at a tertiary care center
.
In addition, fewer biopsies were performed in the first 3 years of the global COVID-19 pandemic than in the previous 3 years.
These findings from a retrospective analysis were presented during a poster session at the annual meeting of the Society for Pediatric Dermatology. The analysis set out to evaluate which patients required biopsy, which skin conditions were sampled, and if practice patterns changed following the start of the COVID-19 pandemic.
“The work is important because very few pediatric patients, relative to adult patients seen in dermatology clinics, have a biopsy done,” Kelly M. Cordoro, MD, one of the study authors, told this news organization.
“Approximately 1%-4% of pediatric patients visiting a dermatology clinic will have a biopsy done as compared to 30%-50% of adult patients. Understanding what is being biopsied in children sheds light on the medical decision-making required to decide when a biopsy is necessary,” said Dr. Cordoro, chief of pediatric dermatology at UCSF.
For the study, the researchers retrospectively reviewed 1,196 biopsy specimens from 1,080 unique patients that were performed by pediatric dermatologists at UCSF from 2017 to 2022. Half of the patients were female, their mean age was 11.5 years, and they ranged in age from 1 day to 61 years. Nearly half of biopsies (47%) were performed in patients aged 12-18 years and one-quarter (25.6%) were performed in those aged 6-11 years. In the remaining biopsies, 6.6% came from patients younger than 1 year, 5.8% of those aged 1-2 years, 7.3% from those aged 3-5 years, and 3.9% each in those aged 19-21 years and in those older than 21 years.
The five most common biopsy results were compound nevus (99 biopsies), pyogenic granuloma (96), spongiotic dermatitis (57), intradermal nevus (53), and pilomatricoma (40).
The researchers identified 30 malignant diagnoses in 28 unique patients, most commonly mycosis fungoides (in 16 patients with a median age of 12.5 years), basal cell carcinoma (in 5 patients with a median age of 9 years), and dermatofibrosarcoma protuberans (in 4 patients with a median age of 2 years).
There was no significant sex-based difference in the number of biopsies performed at a given age (P = .47), but Dr. Cordoro and colleagues noted a statistically significant decrease in the number of biopsies during the pandemic compared with the 3 years prior to the pandemic (P = .04).
“There was a slight uptick in 2022, although it remains to be seen whether this trend will continue,” they wrote in their abstract. “While the most common diagnoses in the years leading up to – versus following the start of the pandemic – were similar, there was one clear outlier. The histopathologic diagnosis of pernio spiked in 2020, reflecting the ‘COVID toes’ phenomenon”.
In an interview, Dr. Cordoro said that growths and rashes in children of all ages can, and should, be biopsied, but special considerations are necessary depending on the patient’s age and context.
“Our data showed that neoplastic conditions were biopsied more often than inflammatory conditions, with an emphasis on lesions that required removal (such as pyogenic granuloma), raised concerns for atypia (nevi), or had implications for systemic management (such as Langerhans cell histiocytosis and graft-versus-host disease). Importantly, cutaneous malignancies in children are rare but do occur, and a high index of suspicion is required when approaching any child with a complex neoplasm or rash.”
Dr. Cordoro characterized the medical decision making and rationale for biopsying skin lesions and rashes in children as “a complex process that involves weighing the risks of the biopsy itself against the benefit of the information it will provide; shared decision-making with the caregivers, the patient (if age-appropriate), and other members of the health care team; age of the child and clinical context; and whether the biopsy can be done at the bedside or requires sedation.”
Based on the study results, Dr. Cordoro said, the rationale to proceed with a biopsy boils down to three main goals: To make or confirm a diagnosis, to make decisions about management, and/or the biopsy itself is therapeutic.
UCSF dermatopathology fellow Suzanne W. Birmingham, MD, performed the study in collaboration with Dr. Cordoro and UCSF dermatopathologist Thaddeus W. Mully, MD. Additional analyses of this data set are in progress. The researchers reported having no relevant financial disclosures.
.
In addition, fewer biopsies were performed in the first 3 years of the global COVID-19 pandemic than in the previous 3 years.
These findings from a retrospective analysis were presented during a poster session at the annual meeting of the Society for Pediatric Dermatology. The analysis set out to evaluate which patients required biopsy, which skin conditions were sampled, and if practice patterns changed following the start of the COVID-19 pandemic.
“The work is important because very few pediatric patients, relative to adult patients seen in dermatology clinics, have a biopsy done,” Kelly M. Cordoro, MD, one of the study authors, told this news organization.
“Approximately 1%-4% of pediatric patients visiting a dermatology clinic will have a biopsy done as compared to 30%-50% of adult patients. Understanding what is being biopsied in children sheds light on the medical decision-making required to decide when a biopsy is necessary,” said Dr. Cordoro, chief of pediatric dermatology at UCSF.
For the study, the researchers retrospectively reviewed 1,196 biopsy specimens from 1,080 unique patients that were performed by pediatric dermatologists at UCSF from 2017 to 2022. Half of the patients were female, their mean age was 11.5 years, and they ranged in age from 1 day to 61 years. Nearly half of biopsies (47%) were performed in patients aged 12-18 years and one-quarter (25.6%) were performed in those aged 6-11 years. In the remaining biopsies, 6.6% came from patients younger than 1 year, 5.8% of those aged 1-2 years, 7.3% from those aged 3-5 years, and 3.9% each in those aged 19-21 years and in those older than 21 years.
The five most common biopsy results were compound nevus (99 biopsies), pyogenic granuloma (96), spongiotic dermatitis (57), intradermal nevus (53), and pilomatricoma (40).
The researchers identified 30 malignant diagnoses in 28 unique patients, most commonly mycosis fungoides (in 16 patients with a median age of 12.5 years), basal cell carcinoma (in 5 patients with a median age of 9 years), and dermatofibrosarcoma protuberans (in 4 patients with a median age of 2 years).
There was no significant sex-based difference in the number of biopsies performed at a given age (P = .47), but Dr. Cordoro and colleagues noted a statistically significant decrease in the number of biopsies during the pandemic compared with the 3 years prior to the pandemic (P = .04).
“There was a slight uptick in 2022, although it remains to be seen whether this trend will continue,” they wrote in their abstract. “While the most common diagnoses in the years leading up to – versus following the start of the pandemic – were similar, there was one clear outlier. The histopathologic diagnosis of pernio spiked in 2020, reflecting the ‘COVID toes’ phenomenon”.
In an interview, Dr. Cordoro said that growths and rashes in children of all ages can, and should, be biopsied, but special considerations are necessary depending on the patient’s age and context.
“Our data showed that neoplastic conditions were biopsied more often than inflammatory conditions, with an emphasis on lesions that required removal (such as pyogenic granuloma), raised concerns for atypia (nevi), or had implications for systemic management (such as Langerhans cell histiocytosis and graft-versus-host disease). Importantly, cutaneous malignancies in children are rare but do occur, and a high index of suspicion is required when approaching any child with a complex neoplasm or rash.”
Dr. Cordoro characterized the medical decision making and rationale for biopsying skin lesions and rashes in children as “a complex process that involves weighing the risks of the biopsy itself against the benefit of the information it will provide; shared decision-making with the caregivers, the patient (if age-appropriate), and other members of the health care team; age of the child and clinical context; and whether the biopsy can be done at the bedside or requires sedation.”
Based on the study results, Dr. Cordoro said, the rationale to proceed with a biopsy boils down to three main goals: To make or confirm a diagnosis, to make decisions about management, and/or the biopsy itself is therapeutic.
UCSF dermatopathology fellow Suzanne W. Birmingham, MD, performed the study in collaboration with Dr. Cordoro and UCSF dermatopathologist Thaddeus W. Mully, MD. Additional analyses of this data set are in progress. The researchers reported having no relevant financial disclosures.
.
In addition, fewer biopsies were performed in the first 3 years of the global COVID-19 pandemic than in the previous 3 years.
These findings from a retrospective analysis were presented during a poster session at the annual meeting of the Society for Pediatric Dermatology. The analysis set out to evaluate which patients required biopsy, which skin conditions were sampled, and if practice patterns changed following the start of the COVID-19 pandemic.
“The work is important because very few pediatric patients, relative to adult patients seen in dermatology clinics, have a biopsy done,” Kelly M. Cordoro, MD, one of the study authors, told this news organization.
“Approximately 1%-4% of pediatric patients visiting a dermatology clinic will have a biopsy done as compared to 30%-50% of adult patients. Understanding what is being biopsied in children sheds light on the medical decision-making required to decide when a biopsy is necessary,” said Dr. Cordoro, chief of pediatric dermatology at UCSF.
For the study, the researchers retrospectively reviewed 1,196 biopsy specimens from 1,080 unique patients that were performed by pediatric dermatologists at UCSF from 2017 to 2022. Half of the patients were female, their mean age was 11.5 years, and they ranged in age from 1 day to 61 years. Nearly half of biopsies (47%) were performed in patients aged 12-18 years and one-quarter (25.6%) were performed in those aged 6-11 years. In the remaining biopsies, 6.6% came from patients younger than 1 year, 5.8% of those aged 1-2 years, 7.3% from those aged 3-5 years, and 3.9% each in those aged 19-21 years and in those older than 21 years.
The five most common biopsy results were compound nevus (99 biopsies), pyogenic granuloma (96), spongiotic dermatitis (57), intradermal nevus (53), and pilomatricoma (40).
The researchers identified 30 malignant diagnoses in 28 unique patients, most commonly mycosis fungoides (in 16 patients with a median age of 12.5 years), basal cell carcinoma (in 5 patients with a median age of 9 years), and dermatofibrosarcoma protuberans (in 4 patients with a median age of 2 years).
There was no significant sex-based difference in the number of biopsies performed at a given age (P = .47), but Dr. Cordoro and colleagues noted a statistically significant decrease in the number of biopsies during the pandemic compared with the 3 years prior to the pandemic (P = .04).
“There was a slight uptick in 2022, although it remains to be seen whether this trend will continue,” they wrote in their abstract. “While the most common diagnoses in the years leading up to – versus following the start of the pandemic – were similar, there was one clear outlier. The histopathologic diagnosis of pernio spiked in 2020, reflecting the ‘COVID toes’ phenomenon”.
In an interview, Dr. Cordoro said that growths and rashes in children of all ages can, and should, be biopsied, but special considerations are necessary depending on the patient’s age and context.
“Our data showed that neoplastic conditions were biopsied more often than inflammatory conditions, with an emphasis on lesions that required removal (such as pyogenic granuloma), raised concerns for atypia (nevi), or had implications for systemic management (such as Langerhans cell histiocytosis and graft-versus-host disease). Importantly, cutaneous malignancies in children are rare but do occur, and a high index of suspicion is required when approaching any child with a complex neoplasm or rash.”
Dr. Cordoro characterized the medical decision making and rationale for biopsying skin lesions and rashes in children as “a complex process that involves weighing the risks of the biopsy itself against the benefit of the information it will provide; shared decision-making with the caregivers, the patient (if age-appropriate), and other members of the health care team; age of the child and clinical context; and whether the biopsy can be done at the bedside or requires sedation.”
Based on the study results, Dr. Cordoro said, the rationale to proceed with a biopsy boils down to three main goals: To make or confirm a diagnosis, to make decisions about management, and/or the biopsy itself is therapeutic.
UCSF dermatopathology fellow Suzanne W. Birmingham, MD, performed the study in collaboration with Dr. Cordoro and UCSF dermatopathologist Thaddeus W. Mully, MD. Additional analyses of this data set are in progress. The researchers reported having no relevant financial disclosures.
FROM SPD 2023
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