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FDA approves topical ruxolitinib for nonsegmental vitiligo
The on July 18. The treatment, which was approved for treating mild to moderate atopic dermatitis in September 2021, is a cream formulation of ruxolitinib, a Janus kinase 1 (JAK1)/JAK2 inhibitor.
Previously, no treatment was approved to repigment patients with vitiligo, says David Rosmarin, MD, vice chair for research and education in the department of dermatology at Tufts Medical Center, Boston. “It’s important to have options that we can give to patients that are both safe and effective to get them the desired results,” Dr. Rosmarin, the lead investigator of the phase 3 clinical trials of topical ruxolitinib, said in an interview. Vitiligo is “a disease that can really affect quality of life. Some people [with vitiligo] feel as if they’re being stared at or they’re being bullied; they don’t feel confident. It can affect relationships and intimacy.”
Approval was based on the results of two phase 3 trials (TruE-V1 and TruE-V2) in 674 patients with nonsegmental vitiligo aged 12 years or older. At 24 weeks, about 30% of the patients on treatment, applied twice a day, achieved at least a 75% improvement in the facial Vitiligo Area Scoring Index (F-VASI75), compared with about 8% and 13% among those in the vehicle groups in the two trials.
At 52 weeks, about 50% of the patients treated with topical ruxolitinib achieved F-VASI75.
Also, using self-reporting as measured by the Vitiligo Noticeability Scale, about 30%-40% of patients described their vitiligo as being “a lot less noticeable” or “no longer noticeable” at week 52. Dr. Rosmarin reported the 52-week results at the 2022 annual meeting of the American Academy of Dermatology.
The trial group used 1.5% ruxolitinib cream twice daily for the full year. The vehicle group began using ruxolitinib halfway through the trial. In this group, 26.8% and 29.6% achieved F-VASI 75 at 52 weeks in the two trials.
For treating vitiligo, patients are advised to apply a thin layer of topical ruxolitinib to affected areas twice a day, “up to 10% body surface area,” according to the prescribing information, which adds: “Satisfactory patient response may require treatment … for more than 24 weeks. If the patient does not find the repigmentation meaningful by 24 weeks, the patient should be reevaluated by the health care provider.”
The most common side effects during the vehicle-controlled part of the trials were development of acne and pruritus at the application site, headache, urinary tract infections, erythema at the application site, and pyrexia, according to the company.
The approved label for topical ruxolitinib includes a boxed warning about serious infections, mortality, cancer, major adverse cardiovascular events, and thrombosis – which, the warning notes, is based on reports in patients treated with oral JAK inhibitors for inflammatory conditions.
Dr. Rosmarin believes that using this drug with other therapies, like light treatment, might yield even better responses. The available data are in patients treated with ruxolitinib as monotherapy, without complementary therapies.
William Damsky, MD, PhD, professor of dermatology and dermatopathology at Yale University, New Haven, who was not involved in the trials, said what is most exciting about this drug is its novelty. Although some topical steroids are used off-label to treat vitiligo, their efficacy is far from what’s been observed in these trials of topical ruxolitinib, he told this news organization. “It’s huge for a number of reasons. … One very big reason is it just provides some hope” for the many patients with vitiligo who, over the years, have been told “that there’s nothing that could be done for their disease, and this really changes that.”
Dr. Rosmarin reports financial relationships with over 20 pharmaceutical companies. Dr. Damsky disclosed no relevant financial relationships.
A version of this article first appeared on Medscape.com.
The on July 18. The treatment, which was approved for treating mild to moderate atopic dermatitis in September 2021, is a cream formulation of ruxolitinib, a Janus kinase 1 (JAK1)/JAK2 inhibitor.
Previously, no treatment was approved to repigment patients with vitiligo, says David Rosmarin, MD, vice chair for research and education in the department of dermatology at Tufts Medical Center, Boston. “It’s important to have options that we can give to patients that are both safe and effective to get them the desired results,” Dr. Rosmarin, the lead investigator of the phase 3 clinical trials of topical ruxolitinib, said in an interview. Vitiligo is “a disease that can really affect quality of life. Some people [with vitiligo] feel as if they’re being stared at or they’re being bullied; they don’t feel confident. It can affect relationships and intimacy.”
Approval was based on the results of two phase 3 trials (TruE-V1 and TruE-V2) in 674 patients with nonsegmental vitiligo aged 12 years or older. At 24 weeks, about 30% of the patients on treatment, applied twice a day, achieved at least a 75% improvement in the facial Vitiligo Area Scoring Index (F-VASI75), compared with about 8% and 13% among those in the vehicle groups in the two trials.
At 52 weeks, about 50% of the patients treated with topical ruxolitinib achieved F-VASI75.
Also, using self-reporting as measured by the Vitiligo Noticeability Scale, about 30%-40% of patients described their vitiligo as being “a lot less noticeable” or “no longer noticeable” at week 52. Dr. Rosmarin reported the 52-week results at the 2022 annual meeting of the American Academy of Dermatology.
The trial group used 1.5% ruxolitinib cream twice daily for the full year. The vehicle group began using ruxolitinib halfway through the trial. In this group, 26.8% and 29.6% achieved F-VASI 75 at 52 weeks in the two trials.
For treating vitiligo, patients are advised to apply a thin layer of topical ruxolitinib to affected areas twice a day, “up to 10% body surface area,” according to the prescribing information, which adds: “Satisfactory patient response may require treatment … for more than 24 weeks. If the patient does not find the repigmentation meaningful by 24 weeks, the patient should be reevaluated by the health care provider.”
The most common side effects during the vehicle-controlled part of the trials were development of acne and pruritus at the application site, headache, urinary tract infections, erythema at the application site, and pyrexia, according to the company.
The approved label for topical ruxolitinib includes a boxed warning about serious infections, mortality, cancer, major adverse cardiovascular events, and thrombosis – which, the warning notes, is based on reports in patients treated with oral JAK inhibitors for inflammatory conditions.
Dr. Rosmarin believes that using this drug with other therapies, like light treatment, might yield even better responses. The available data are in patients treated with ruxolitinib as monotherapy, without complementary therapies.
William Damsky, MD, PhD, professor of dermatology and dermatopathology at Yale University, New Haven, who was not involved in the trials, said what is most exciting about this drug is its novelty. Although some topical steroids are used off-label to treat vitiligo, their efficacy is far from what’s been observed in these trials of topical ruxolitinib, he told this news organization. “It’s huge for a number of reasons. … One very big reason is it just provides some hope” for the many patients with vitiligo who, over the years, have been told “that there’s nothing that could be done for their disease, and this really changes that.”
Dr. Rosmarin reports financial relationships with over 20 pharmaceutical companies. Dr. Damsky disclosed no relevant financial relationships.
A version of this article first appeared on Medscape.com.
The on July 18. The treatment, which was approved for treating mild to moderate atopic dermatitis in September 2021, is a cream formulation of ruxolitinib, a Janus kinase 1 (JAK1)/JAK2 inhibitor.
Previously, no treatment was approved to repigment patients with vitiligo, says David Rosmarin, MD, vice chair for research and education in the department of dermatology at Tufts Medical Center, Boston. “It’s important to have options that we can give to patients that are both safe and effective to get them the desired results,” Dr. Rosmarin, the lead investigator of the phase 3 clinical trials of topical ruxolitinib, said in an interview. Vitiligo is “a disease that can really affect quality of life. Some people [with vitiligo] feel as if they’re being stared at or they’re being bullied; they don’t feel confident. It can affect relationships and intimacy.”
Approval was based on the results of two phase 3 trials (TruE-V1 and TruE-V2) in 674 patients with nonsegmental vitiligo aged 12 years or older. At 24 weeks, about 30% of the patients on treatment, applied twice a day, achieved at least a 75% improvement in the facial Vitiligo Area Scoring Index (F-VASI75), compared with about 8% and 13% among those in the vehicle groups in the two trials.
At 52 weeks, about 50% of the patients treated with topical ruxolitinib achieved F-VASI75.
Also, using self-reporting as measured by the Vitiligo Noticeability Scale, about 30%-40% of patients described their vitiligo as being “a lot less noticeable” or “no longer noticeable” at week 52. Dr. Rosmarin reported the 52-week results at the 2022 annual meeting of the American Academy of Dermatology.
The trial group used 1.5% ruxolitinib cream twice daily for the full year. The vehicle group began using ruxolitinib halfway through the trial. In this group, 26.8% and 29.6% achieved F-VASI 75 at 52 weeks in the two trials.
For treating vitiligo, patients are advised to apply a thin layer of topical ruxolitinib to affected areas twice a day, “up to 10% body surface area,” according to the prescribing information, which adds: “Satisfactory patient response may require treatment … for more than 24 weeks. If the patient does not find the repigmentation meaningful by 24 weeks, the patient should be reevaluated by the health care provider.”
The most common side effects during the vehicle-controlled part of the trials were development of acne and pruritus at the application site, headache, urinary tract infections, erythema at the application site, and pyrexia, according to the company.
The approved label for topical ruxolitinib includes a boxed warning about serious infections, mortality, cancer, major adverse cardiovascular events, and thrombosis – which, the warning notes, is based on reports in patients treated with oral JAK inhibitors for inflammatory conditions.
Dr. Rosmarin believes that using this drug with other therapies, like light treatment, might yield even better responses. The available data are in patients treated with ruxolitinib as monotherapy, without complementary therapies.
William Damsky, MD, PhD, professor of dermatology and dermatopathology at Yale University, New Haven, who was not involved in the trials, said what is most exciting about this drug is its novelty. Although some topical steroids are used off-label to treat vitiligo, their efficacy is far from what’s been observed in these trials of topical ruxolitinib, he told this news organization. “It’s huge for a number of reasons. … One very big reason is it just provides some hope” for the many patients with vitiligo who, over the years, have been told “that there’s nothing that could be done for their disease, and this really changes that.”
Dr. Rosmarin reports financial relationships with over 20 pharmaceutical companies. Dr. Damsky disclosed no relevant financial relationships.
A version of this article first appeared on Medscape.com.
Commentary: Reversal of Roe v. Wade affects adolescents
The Supreme Court decision to strike down the 50-year ruling on Roe v. Wade, which allowed legal abortion, will affect all patients and families seeking care in pediatric and adolescent medicine clinics. Regardless of how you view abortion, the reality is your adolescent female patients and their parents will seek your counsel.
The overturning of Roe has resulted in much confusion for both patients and providers. The overall effect of this decision in Wisconsin is yet to be known but currently we have had to create road maps to direct adolescent patients who experience an unplanned pregnancy and wish to abort. Unfortunately, these road maps include only resources out of state or online. Providing adolescents confidential care may be challenged as the teens may need to disclose the unplanned pregnancy to an adult to access resources.
Providers remain unsettled regarding their risk of assisting an adolescent who discloses an unplanned pregnancy. Recently, many questions arose regarding dispensing Plan B and the risk to prescribers. Communication was needed to assure providers that Plan B is contraception and at this time contraception remains legal in our state.
Daily I educate adolescent females on the risks of unplanned pregnancy and what the Supreme Court decision will mean to them if they become pregnant. Unfortunately, many teens do not understand the ruling and how this decision affects them personally. Education is needed today more than ever regarding pregnancy prevention.
The recent AAP policy statement reaffirms its position that the rights of adolescents to seek confidential care when considering abortion must be protected.1 It further reaffirms access to safe and legal abortion is a core tenant of sexual and reproductive health care.
A recent article published in AAP News by Elise D. Berlan, MD, “AAP’s teen reproductive health policies reaffirm right to comprehensive care,” further advises on the role of the pediatric provider.2 Pediatric providers should continue offering option counseling for pregnant adolescents, be prepared to provide accurate information regarding these options with awareness that some options such as the IUD may no longer be available, remain supportive of the decision they choose, and encourage discussion with a family member to support their decisions. It is imperative that we familiarize ourselves with the abortion policies in our states, advocate to prevent government interference with the patient-doctor relationship, and recognize the impact restrictive abortion has regarding marginalized individuals, she stated. Finally we must recognize our own bias regarding option counseling and refer appropriately to another professional if we are unable to confidently offer guidance.
Ms. Thew is the medical director of the department of adolescent medicine at Children’s Wisconsin in Milwaukee.
References
1. AAP Committee on Adolescence. Pediatrics. 2022. doi: 10.1542/peds.2022-058780.
2. Berlan ED. AAP’s teen reproductive health policies reaffirm right to comprehensive care. 2022. AAP News.
The Supreme Court decision to strike down the 50-year ruling on Roe v. Wade, which allowed legal abortion, will affect all patients and families seeking care in pediatric and adolescent medicine clinics. Regardless of how you view abortion, the reality is your adolescent female patients and their parents will seek your counsel.
The overturning of Roe has resulted in much confusion for both patients and providers. The overall effect of this decision in Wisconsin is yet to be known but currently we have had to create road maps to direct adolescent patients who experience an unplanned pregnancy and wish to abort. Unfortunately, these road maps include only resources out of state or online. Providing adolescents confidential care may be challenged as the teens may need to disclose the unplanned pregnancy to an adult to access resources.
Providers remain unsettled regarding their risk of assisting an adolescent who discloses an unplanned pregnancy. Recently, many questions arose regarding dispensing Plan B and the risk to prescribers. Communication was needed to assure providers that Plan B is contraception and at this time contraception remains legal in our state.
Daily I educate adolescent females on the risks of unplanned pregnancy and what the Supreme Court decision will mean to them if they become pregnant. Unfortunately, many teens do not understand the ruling and how this decision affects them personally. Education is needed today more than ever regarding pregnancy prevention.
The recent AAP policy statement reaffirms its position that the rights of adolescents to seek confidential care when considering abortion must be protected.1 It further reaffirms access to safe and legal abortion is a core tenant of sexual and reproductive health care.
A recent article published in AAP News by Elise D. Berlan, MD, “AAP’s teen reproductive health policies reaffirm right to comprehensive care,” further advises on the role of the pediatric provider.2 Pediatric providers should continue offering option counseling for pregnant adolescents, be prepared to provide accurate information regarding these options with awareness that some options such as the IUD may no longer be available, remain supportive of the decision they choose, and encourage discussion with a family member to support their decisions. It is imperative that we familiarize ourselves with the abortion policies in our states, advocate to prevent government interference with the patient-doctor relationship, and recognize the impact restrictive abortion has regarding marginalized individuals, she stated. Finally we must recognize our own bias regarding option counseling and refer appropriately to another professional if we are unable to confidently offer guidance.
Ms. Thew is the medical director of the department of adolescent medicine at Children’s Wisconsin in Milwaukee.
References
1. AAP Committee on Adolescence. Pediatrics. 2022. doi: 10.1542/peds.2022-058780.
2. Berlan ED. AAP’s teen reproductive health policies reaffirm right to comprehensive care. 2022. AAP News.
The Supreme Court decision to strike down the 50-year ruling on Roe v. Wade, which allowed legal abortion, will affect all patients and families seeking care in pediatric and adolescent medicine clinics. Regardless of how you view abortion, the reality is your adolescent female patients and their parents will seek your counsel.
The overturning of Roe has resulted in much confusion for both patients and providers. The overall effect of this decision in Wisconsin is yet to be known but currently we have had to create road maps to direct adolescent patients who experience an unplanned pregnancy and wish to abort. Unfortunately, these road maps include only resources out of state or online. Providing adolescents confidential care may be challenged as the teens may need to disclose the unplanned pregnancy to an adult to access resources.
Providers remain unsettled regarding their risk of assisting an adolescent who discloses an unplanned pregnancy. Recently, many questions arose regarding dispensing Plan B and the risk to prescribers. Communication was needed to assure providers that Plan B is contraception and at this time contraception remains legal in our state.
Daily I educate adolescent females on the risks of unplanned pregnancy and what the Supreme Court decision will mean to them if they become pregnant. Unfortunately, many teens do not understand the ruling and how this decision affects them personally. Education is needed today more than ever regarding pregnancy prevention.
The recent AAP policy statement reaffirms its position that the rights of adolescents to seek confidential care when considering abortion must be protected.1 It further reaffirms access to safe and legal abortion is a core tenant of sexual and reproductive health care.
A recent article published in AAP News by Elise D. Berlan, MD, “AAP’s teen reproductive health policies reaffirm right to comprehensive care,” further advises on the role of the pediatric provider.2 Pediatric providers should continue offering option counseling for pregnant adolescents, be prepared to provide accurate information regarding these options with awareness that some options such as the IUD may no longer be available, remain supportive of the decision they choose, and encourage discussion with a family member to support their decisions. It is imperative that we familiarize ourselves with the abortion policies in our states, advocate to prevent government interference with the patient-doctor relationship, and recognize the impact restrictive abortion has regarding marginalized individuals, she stated. Finally we must recognize our own bias regarding option counseling and refer appropriately to another professional if we are unable to confidently offer guidance.
Ms. Thew is the medical director of the department of adolescent medicine at Children’s Wisconsin in Milwaukee.
References
1. AAP Committee on Adolescence. Pediatrics. 2022. doi: 10.1542/peds.2022-058780.
2. Berlan ED. AAP’s teen reproductive health policies reaffirm right to comprehensive care. 2022. AAP News.
Think of pediatric morphea as a systemic, chronic disease, expert advises
INDIANAPOLIS – In the opinion of Elena Pope, MD, MSc,
“There is no correlation between the extent and activity of skin lesions and the presence, severity, and activity of extracutaneous manifestations,” Dr. Pope, professor of pediatrics at the University of Toronto and division head of pediatric dermatology at the Hospital for Sick Children in Toronto, said during the annual meeting of the Society for Pediatric Dermatology. “Treatment needs to be tailored to the extent of cutaneous manifestations, and I think we need to be aware of and address the impact on patients’ quality of life,” she added. There is also a need for more research “on targeted and better-tolerated therapies to put a stop to the progression of disease.”
Congenital morphea is a form of localized scleroderma that presents at birth but can be confused with port wine stain. Results from a multicenter retrospective review of 25 cases conducted by Dr. Pope and colleagues found that the median age at diagnosis was 2.9 years and 76% had linear-type lesions. In addition, 48% had extracutaneous involvement (all of these patients had linear morphea), most commonly of the central nervous system.
“It’s important to realize these lesions may become active over time,” Dr. Pope said. “In my experience, there are two different courses. Either you have innocuous lesions when the patients are born and they may become active around 3-4 years of age, or you have early intrauterine involvement, with lesions inactive at birth but with potential for significant damage in utero.”
She cautioned against treating a suspected port wine stain lesion with laser until congenital morphea is ruled out. “I’m aware of at least one lawsuit of a child where someone used a laser in a child who had progression with significant sclerosis,” she said. “The parents assumed it was the use of the laser that led to the progression, not the actual disease.”
Extracutaneous manifestations are common in morphea patients. A multicenter study of 750 patients with juvenile scleroderma found that 22% had extracutaneous manifestations. Almost half of patients (47%) had arthritis, but 17% had neurologic findings such as seizures and headaches, 9% had vascular manifestations, and 8% had uveitis. Subsequent studies found that neurological disease affects between 11% and 19% of cases, especially in those involving the head and neck.
“There is a wide range of manifestations from headache and neuropsychiatric changes to brain atrophy, seizures, and CNS cavernoma,” Dr. Pope said. “There also can be orthodental involvement such as malocclusion. It’s important to do a brain MRI, eye exam for uveitis, and don’t forget the orthodental assessment.”
She recalled a 10-year-old boy who presented to the Hospital for Sick Children with tissue loss on the forehead and eyebrow and eyelashes. He had no other congenital morphea symptoms and the MRI was normal, but the eye exam revealed uveitis. “It’s important to remember that uveitis is asymptomatic, so unless you look for it, you’re not going to find it,” she said.
According to unpublished data in 42 congenital morphea patients with lesions limited to the head and neck, who underwent MRI imaging at the Hospital for Sick Children, 57% had CNS changes that were ipsilateral in 68% of cases. “White matter changes were the most common, and to our surprise, there were patients who had progressive CNS disease, including CNS vasculitis, new lesions, and enhancement of prior stable lesions,” Dr. Pope said.
She recalled the case of an 8-year-old boy who presented to the hospital with intractable seizures. Upon completion of the MRI, one of the radiologists noted that the imaging showed subtle thinning of the forehead, and he was referred to Dr. Pope and colleagues for assessment. In the span of 4 years, despite aggressive treatment, the boy’s CNS disease progressed. “There was more enhancement, more tissue loss, his seizures are very hard to control, and he has many neurodevelopmental changes,” she recalled. “What I learned from this case is that skin activity does not correlate with imaging. Don’t assume that just because the skin is burnt out that the CNS will be the same. Also, the extent of skin disease does not predict involvement or progression of the CNS.”
Linear lesions on the lower extremities are a harbinger of orthopedic complications, which can occur in about half of patients. Joint contractures in this subset of patients are seen in about 81% of cases, while other sequelae can include arthritis, limb atrophy, leg-leg discrepancy, and angular deformity. “About 14% of patients require intervention,” Dr. Pope said. “In terms of working those patients up, you need to do an MRI and assess the extent of muscle and fascial involvement. Early physiotherapy and an orthopedic evaluation are also recommended.”
As for possible markers of morphea, antinuclear antibody is positive in 22%-68% of cases and correlates with disease severity, extracutaneous manifestations, and disease flare-up. Antihistone antibodies (AHA) are positive in about 47% of cases, “and that tends to correlate with the extent of skin and muscle involvement,” Dr. Pope said. “Anti–double-stranded DNA correlates with extent of disease, but the only known biomarker to date that correlates with disease activity is CXCL9/10. This has been documented in the skin as well as in the blood. So, this marker may help us determine if the patient needs to be treated or not.”
Treatments
For treatment of active localized disease, topical medications are helpful in some cases. Options include topical steroids, calcipotriol with or without betamethasone, imiquimod, and tacrolimus. “In my experience the combination of calcipotriol with betamethasone is best,” she said. “It really shuts down the activity fairly soon, and you can scale down to calcipotriol alone. I don’t find imiquimod very helpful for active lesions, although it has a role for inactive lesions.”
For patients with linear or generalized/mixed disease, “the combination of methotrexate and corticosteroids or methotrexate alone is probably the way to go,” Dr. Pope said. “The addition of steroids really depends on where the lesion is and how worried you are about other problems.”
According to the best available literature, 88% of patients should respond to treatment with methotrexate (MTX) and/or steroids within 3-6 months, and 74% within 3 months. “If they don’t, you have to wonder if the patient’s taking the medication, or you need to think about other alternative treatments,” she said. “Complete remission is possible in most of the patients, and the longer you treat the more you will see that. On average, most of us treat patients for about 3 years, but there are treatment failures as well. This can occur in up to 16% of patients.”
As for second-line treatment agents for congenital morphea, clinicians often turn to mycophenolate mofetil (MMF). Results from a retrospective longitudinal study of juvenile localized scleroderma patients found that after a mean of 9 years 91% of patients on MMF and 100% of patients on MTX had inactive disease. “There were no differences in relapse rates, although MMF seems to have a more sustained long-term effect and overall is better tolerated,” said Dr. Pope, who was not involved with the study. “However, it’s more immunosuppressive than MTX, which is important, especially in the era of COVID-19. You also need to think about the potential for more hematological suppression with MMF use.” If standard therapy fails, there is anecdotal data supporting the use of abatacept (which suppresses the T-cell activity in affected patients), tofacitinib (which inhibits transforming growth factor–beta), or dupilumab (which inhibits interleukin-4).
Dr. Pope emphasized the effect congenital morphea has on quality of life. Remarks from patients with facial morphea and their parents who participated in a focus group on the topic organized by the Hospital for Sick Children included, “You just want to stay inside because you are afraid of what people will say,” “They laugh at her. They make fun of her, and it’s terrible,” and “MTX makes me feel weird. I would throw up, feel dizzy.”
“You have to take that into consideration, because we cannot make the treatment worse than the disease,” Dr. Pope said. “There are many domains where patients could be affected, including skin symptoms, physical functioning, body image and social support, side effects of medication, and presence of extracutaneous manifestations. Predictors of poor quality of life include female sex and involvement of hands and feet.”
Dr. Pope disclosed that she has received grants/research support from AbbVie, Centocor, and Amgen. She has also received consulting fees from AbbVie, Sanofi, Novartis, Boehringer-Ingelheim, Phoenix, Amryt Pharma, and Timber Pharmaceuticals.
INDIANAPOLIS – In the opinion of Elena Pope, MD, MSc,
“There is no correlation between the extent and activity of skin lesions and the presence, severity, and activity of extracutaneous manifestations,” Dr. Pope, professor of pediatrics at the University of Toronto and division head of pediatric dermatology at the Hospital for Sick Children in Toronto, said during the annual meeting of the Society for Pediatric Dermatology. “Treatment needs to be tailored to the extent of cutaneous manifestations, and I think we need to be aware of and address the impact on patients’ quality of life,” she added. There is also a need for more research “on targeted and better-tolerated therapies to put a stop to the progression of disease.”
Congenital morphea is a form of localized scleroderma that presents at birth but can be confused with port wine stain. Results from a multicenter retrospective review of 25 cases conducted by Dr. Pope and colleagues found that the median age at diagnosis was 2.9 years and 76% had linear-type lesions. In addition, 48% had extracutaneous involvement (all of these patients had linear morphea), most commonly of the central nervous system.
“It’s important to realize these lesions may become active over time,” Dr. Pope said. “In my experience, there are two different courses. Either you have innocuous lesions when the patients are born and they may become active around 3-4 years of age, or you have early intrauterine involvement, with lesions inactive at birth but with potential for significant damage in utero.”
She cautioned against treating a suspected port wine stain lesion with laser until congenital morphea is ruled out. “I’m aware of at least one lawsuit of a child where someone used a laser in a child who had progression with significant sclerosis,” she said. “The parents assumed it was the use of the laser that led to the progression, not the actual disease.”
Extracutaneous manifestations are common in morphea patients. A multicenter study of 750 patients with juvenile scleroderma found that 22% had extracutaneous manifestations. Almost half of patients (47%) had arthritis, but 17% had neurologic findings such as seizures and headaches, 9% had vascular manifestations, and 8% had uveitis. Subsequent studies found that neurological disease affects between 11% and 19% of cases, especially in those involving the head and neck.
“There is a wide range of manifestations from headache and neuropsychiatric changes to brain atrophy, seizures, and CNS cavernoma,” Dr. Pope said. “There also can be orthodental involvement such as malocclusion. It’s important to do a brain MRI, eye exam for uveitis, and don’t forget the orthodental assessment.”
She recalled a 10-year-old boy who presented to the Hospital for Sick Children with tissue loss on the forehead and eyebrow and eyelashes. He had no other congenital morphea symptoms and the MRI was normal, but the eye exam revealed uveitis. “It’s important to remember that uveitis is asymptomatic, so unless you look for it, you’re not going to find it,” she said.
According to unpublished data in 42 congenital morphea patients with lesions limited to the head and neck, who underwent MRI imaging at the Hospital for Sick Children, 57% had CNS changes that were ipsilateral in 68% of cases. “White matter changes were the most common, and to our surprise, there were patients who had progressive CNS disease, including CNS vasculitis, new lesions, and enhancement of prior stable lesions,” Dr. Pope said.
She recalled the case of an 8-year-old boy who presented to the hospital with intractable seizures. Upon completion of the MRI, one of the radiologists noted that the imaging showed subtle thinning of the forehead, and he was referred to Dr. Pope and colleagues for assessment. In the span of 4 years, despite aggressive treatment, the boy’s CNS disease progressed. “There was more enhancement, more tissue loss, his seizures are very hard to control, and he has many neurodevelopmental changes,” she recalled. “What I learned from this case is that skin activity does not correlate with imaging. Don’t assume that just because the skin is burnt out that the CNS will be the same. Also, the extent of skin disease does not predict involvement or progression of the CNS.”
Linear lesions on the lower extremities are a harbinger of orthopedic complications, which can occur in about half of patients. Joint contractures in this subset of patients are seen in about 81% of cases, while other sequelae can include arthritis, limb atrophy, leg-leg discrepancy, and angular deformity. “About 14% of patients require intervention,” Dr. Pope said. “In terms of working those patients up, you need to do an MRI and assess the extent of muscle and fascial involvement. Early physiotherapy and an orthopedic evaluation are also recommended.”
As for possible markers of morphea, antinuclear antibody is positive in 22%-68% of cases and correlates with disease severity, extracutaneous manifestations, and disease flare-up. Antihistone antibodies (AHA) are positive in about 47% of cases, “and that tends to correlate with the extent of skin and muscle involvement,” Dr. Pope said. “Anti–double-stranded DNA correlates with extent of disease, but the only known biomarker to date that correlates with disease activity is CXCL9/10. This has been documented in the skin as well as in the blood. So, this marker may help us determine if the patient needs to be treated or not.”
Treatments
For treatment of active localized disease, topical medications are helpful in some cases. Options include topical steroids, calcipotriol with or without betamethasone, imiquimod, and tacrolimus. “In my experience the combination of calcipotriol with betamethasone is best,” she said. “It really shuts down the activity fairly soon, and you can scale down to calcipotriol alone. I don’t find imiquimod very helpful for active lesions, although it has a role for inactive lesions.”
For patients with linear or generalized/mixed disease, “the combination of methotrexate and corticosteroids or methotrexate alone is probably the way to go,” Dr. Pope said. “The addition of steroids really depends on where the lesion is and how worried you are about other problems.”
According to the best available literature, 88% of patients should respond to treatment with methotrexate (MTX) and/or steroids within 3-6 months, and 74% within 3 months. “If they don’t, you have to wonder if the patient’s taking the medication, or you need to think about other alternative treatments,” she said. “Complete remission is possible in most of the patients, and the longer you treat the more you will see that. On average, most of us treat patients for about 3 years, but there are treatment failures as well. This can occur in up to 16% of patients.”
As for second-line treatment agents for congenital morphea, clinicians often turn to mycophenolate mofetil (MMF). Results from a retrospective longitudinal study of juvenile localized scleroderma patients found that after a mean of 9 years 91% of patients on MMF and 100% of patients on MTX had inactive disease. “There were no differences in relapse rates, although MMF seems to have a more sustained long-term effect and overall is better tolerated,” said Dr. Pope, who was not involved with the study. “However, it’s more immunosuppressive than MTX, which is important, especially in the era of COVID-19. You also need to think about the potential for more hematological suppression with MMF use.” If standard therapy fails, there is anecdotal data supporting the use of abatacept (which suppresses the T-cell activity in affected patients), tofacitinib (which inhibits transforming growth factor–beta), or dupilumab (which inhibits interleukin-4).
Dr. Pope emphasized the effect congenital morphea has on quality of life. Remarks from patients with facial morphea and their parents who participated in a focus group on the topic organized by the Hospital for Sick Children included, “You just want to stay inside because you are afraid of what people will say,” “They laugh at her. They make fun of her, and it’s terrible,” and “MTX makes me feel weird. I would throw up, feel dizzy.”
“You have to take that into consideration, because we cannot make the treatment worse than the disease,” Dr. Pope said. “There are many domains where patients could be affected, including skin symptoms, physical functioning, body image and social support, side effects of medication, and presence of extracutaneous manifestations. Predictors of poor quality of life include female sex and involvement of hands and feet.”
Dr. Pope disclosed that she has received grants/research support from AbbVie, Centocor, and Amgen. She has also received consulting fees from AbbVie, Sanofi, Novartis, Boehringer-Ingelheim, Phoenix, Amryt Pharma, and Timber Pharmaceuticals.
INDIANAPOLIS – In the opinion of Elena Pope, MD, MSc,
“There is no correlation between the extent and activity of skin lesions and the presence, severity, and activity of extracutaneous manifestations,” Dr. Pope, professor of pediatrics at the University of Toronto and division head of pediatric dermatology at the Hospital for Sick Children in Toronto, said during the annual meeting of the Society for Pediatric Dermatology. “Treatment needs to be tailored to the extent of cutaneous manifestations, and I think we need to be aware of and address the impact on patients’ quality of life,” she added. There is also a need for more research “on targeted and better-tolerated therapies to put a stop to the progression of disease.”
Congenital morphea is a form of localized scleroderma that presents at birth but can be confused with port wine stain. Results from a multicenter retrospective review of 25 cases conducted by Dr. Pope and colleagues found that the median age at diagnosis was 2.9 years and 76% had linear-type lesions. In addition, 48% had extracutaneous involvement (all of these patients had linear morphea), most commonly of the central nervous system.
“It’s important to realize these lesions may become active over time,” Dr. Pope said. “In my experience, there are two different courses. Either you have innocuous lesions when the patients are born and they may become active around 3-4 years of age, or you have early intrauterine involvement, with lesions inactive at birth but with potential for significant damage in utero.”
She cautioned against treating a suspected port wine stain lesion with laser until congenital morphea is ruled out. “I’m aware of at least one lawsuit of a child where someone used a laser in a child who had progression with significant sclerosis,” she said. “The parents assumed it was the use of the laser that led to the progression, not the actual disease.”
Extracutaneous manifestations are common in morphea patients. A multicenter study of 750 patients with juvenile scleroderma found that 22% had extracutaneous manifestations. Almost half of patients (47%) had arthritis, but 17% had neurologic findings such as seizures and headaches, 9% had vascular manifestations, and 8% had uveitis. Subsequent studies found that neurological disease affects between 11% and 19% of cases, especially in those involving the head and neck.
“There is a wide range of manifestations from headache and neuropsychiatric changes to brain atrophy, seizures, and CNS cavernoma,” Dr. Pope said. “There also can be orthodental involvement such as malocclusion. It’s important to do a brain MRI, eye exam for uveitis, and don’t forget the orthodental assessment.”
She recalled a 10-year-old boy who presented to the Hospital for Sick Children with tissue loss on the forehead and eyebrow and eyelashes. He had no other congenital morphea symptoms and the MRI was normal, but the eye exam revealed uveitis. “It’s important to remember that uveitis is asymptomatic, so unless you look for it, you’re not going to find it,” she said.
According to unpublished data in 42 congenital morphea patients with lesions limited to the head and neck, who underwent MRI imaging at the Hospital for Sick Children, 57% had CNS changes that were ipsilateral in 68% of cases. “White matter changes were the most common, and to our surprise, there were patients who had progressive CNS disease, including CNS vasculitis, new lesions, and enhancement of prior stable lesions,” Dr. Pope said.
She recalled the case of an 8-year-old boy who presented to the hospital with intractable seizures. Upon completion of the MRI, one of the radiologists noted that the imaging showed subtle thinning of the forehead, and he was referred to Dr. Pope and colleagues for assessment. In the span of 4 years, despite aggressive treatment, the boy’s CNS disease progressed. “There was more enhancement, more tissue loss, his seizures are very hard to control, and he has many neurodevelopmental changes,” she recalled. “What I learned from this case is that skin activity does not correlate with imaging. Don’t assume that just because the skin is burnt out that the CNS will be the same. Also, the extent of skin disease does not predict involvement or progression of the CNS.”
Linear lesions on the lower extremities are a harbinger of orthopedic complications, which can occur in about half of patients. Joint contractures in this subset of patients are seen in about 81% of cases, while other sequelae can include arthritis, limb atrophy, leg-leg discrepancy, and angular deformity. “About 14% of patients require intervention,” Dr. Pope said. “In terms of working those patients up, you need to do an MRI and assess the extent of muscle and fascial involvement. Early physiotherapy and an orthopedic evaluation are also recommended.”
As for possible markers of morphea, antinuclear antibody is positive in 22%-68% of cases and correlates with disease severity, extracutaneous manifestations, and disease flare-up. Antihistone antibodies (AHA) are positive in about 47% of cases, “and that tends to correlate with the extent of skin and muscle involvement,” Dr. Pope said. “Anti–double-stranded DNA correlates with extent of disease, but the only known biomarker to date that correlates with disease activity is CXCL9/10. This has been documented in the skin as well as in the blood. So, this marker may help us determine if the patient needs to be treated or not.”
Treatments
For treatment of active localized disease, topical medications are helpful in some cases. Options include topical steroids, calcipotriol with or without betamethasone, imiquimod, and tacrolimus. “In my experience the combination of calcipotriol with betamethasone is best,” she said. “It really shuts down the activity fairly soon, and you can scale down to calcipotriol alone. I don’t find imiquimod very helpful for active lesions, although it has a role for inactive lesions.”
For patients with linear or generalized/mixed disease, “the combination of methotrexate and corticosteroids or methotrexate alone is probably the way to go,” Dr. Pope said. “The addition of steroids really depends on where the lesion is and how worried you are about other problems.”
According to the best available literature, 88% of patients should respond to treatment with methotrexate (MTX) and/or steroids within 3-6 months, and 74% within 3 months. “If they don’t, you have to wonder if the patient’s taking the medication, or you need to think about other alternative treatments,” she said. “Complete remission is possible in most of the patients, and the longer you treat the more you will see that. On average, most of us treat patients for about 3 years, but there are treatment failures as well. This can occur in up to 16% of patients.”
As for second-line treatment agents for congenital morphea, clinicians often turn to mycophenolate mofetil (MMF). Results from a retrospective longitudinal study of juvenile localized scleroderma patients found that after a mean of 9 years 91% of patients on MMF and 100% of patients on MTX had inactive disease. “There were no differences in relapse rates, although MMF seems to have a more sustained long-term effect and overall is better tolerated,” said Dr. Pope, who was not involved with the study. “However, it’s more immunosuppressive than MTX, which is important, especially in the era of COVID-19. You also need to think about the potential for more hematological suppression with MMF use.” If standard therapy fails, there is anecdotal data supporting the use of abatacept (which suppresses the T-cell activity in affected patients), tofacitinib (which inhibits transforming growth factor–beta), or dupilumab (which inhibits interleukin-4).
Dr. Pope emphasized the effect congenital morphea has on quality of life. Remarks from patients with facial morphea and their parents who participated in a focus group on the topic organized by the Hospital for Sick Children included, “You just want to stay inside because you are afraid of what people will say,” “They laugh at her. They make fun of her, and it’s terrible,” and “MTX makes me feel weird. I would throw up, feel dizzy.”
“You have to take that into consideration, because we cannot make the treatment worse than the disease,” Dr. Pope said. “There are many domains where patients could be affected, including skin symptoms, physical functioning, body image and social support, side effects of medication, and presence of extracutaneous manifestations. Predictors of poor quality of life include female sex and involvement of hands and feet.”
Dr. Pope disclosed that she has received grants/research support from AbbVie, Centocor, and Amgen. She has also received consulting fees from AbbVie, Sanofi, Novartis, Boehringer-Ingelheim, Phoenix, Amryt Pharma, and Timber Pharmaceuticals.
AT SPD 2022
Berdazimer gel beats vehicle for molluscum contagiosum in phase 3 study
Treatment with .
Molluscum contagiosum (MC) remains a common infection that, despite being self-limiting, may persist for months or years, and is associated with quality of life concerns and the need for ongoing therapy, wrote John C. Browning, MD, of Texas Dermatology and Laser Specialists, San Antonio, and colleagues, who conducted the phase 3 randomized study.
The infection is most common in children aged 1-14 years, and treatment may be needed in part to avoid infecting peers and family members, they said. No treatments for molluscum are currently approved by the Food and Drug Administration.
In the study, which was published in JAMA Dermatology, the researchers randomized 444 patients to berdazimer gel 10.3% and 447 to a placebo gel, applied once daily in a thin layer on all MC lesions for 12 weeks. The study was conducted at 55 clinics across the United States between Sept. 1, 2020, and July 21, 2021. The mean age of the patients was about 6.5 years (range was 0.9-49 years), and about 85% were White. Participants had 3-70 raised MC lesions; those with sexually transmitted MC or MC in the periocular area were excluded. The primary endpoint was complete clearance of MC lesions after 12 weeks of treatment. At 12 weeks, significantly more patients treated with berdazimer gel achieved complete clearance than those on vehicle (32.4% vs. 19.7%; P < .001). A total of 64 (14.4%) patients in the berdazimer group discontinued treatment because of MC clearance, compared with 40 patients (8.9%) in the vehicle group.
Most adverse events were mild or moderate, and rates of adverse events resulting in treatment discontinuation were low overall for both groups; the most common adverse events were application-site pain and erythema, which were mostly mild. Overall, 4.1% of berdazimer-treated patients and 0.7% of placebo patients discontinued the study because of adverse events.
The study findings were limited by several factors, including the small number of patients in subgroups for race, ethnicity, and age; and the lack of data on patients with sexually transmitted MC and on concomitant use with other topical MC therapies, the researchers noted.
However, the results represent the largest randomized clinical trial of berdazimer 10.3% to date, and support its potential as a first-line therapy for MC patients aged 6 months and older, according to the authors. “Berdazimer is under consideration as a first in-class therapeutic agent for MC and may provide a topical prescription alternative to other therapies used for this highly contagious and psychosocially challenging skin condition,” they said.
Having a reliable, steroid-free, safe, and efficacious medication to treat molluscum in the pediatric population, as early as age 6 months, that can be used at home would “change the whole therapeutic paradigm,” one of the study authors, Adelaide Hebert, MD, said in an interview at the Society for Pediatric Dermatology annual meeting in July, where she presented phase 2 data on berdazimer gel. “This is a common problem and the rate of infections among siblings if it goes untreated is 41%. Affected kids have a sense of isolation; they don’t get invited to swimming parties.”
The lack of a safe and effective topical therapy “has been challenging,” added Dr. Hebert, professor of dermatology and pediatrics, and chief of pediatric dermatology at the University of Texas, Houston. She noted that treatments that have been used but have not been successful include imiquimod. “I’m not impressed with tretinoin,” although it is prescribed for MC, and the most common treatment prescribed by pediatricians for molluscum – mupirocin – is “usually not effective,” she said.
Another MC treatment in trials
Another investigative treatment for molluscum contagiosum, VP-102, a drug-device combination of cantharidin 0.7% administered through a single-use precision applicator, has been evaluated in phase 3 studies of patients with MC aged 2 years and older. The results of two phase 3 studies were published in 2020.
In May 2022, Verrica Pharmaceuticals, which is developing VP-102, announced that Food and Drug Administration approval had been delayed because of deficiencies identified at a contract manufacturing organization, and that the company was working with the agency to bring VP-102 to the market as soon as possible.
A step in the right direction
Although MC is self-resolving, cases last an average of 13.5 months, and “many families look to fast-forward their child’s experience with the infection,” Vikash S. Oza, MD, a pediatric dermatologist at New York University, New York, wrote in an editorial that accompanied the berdazimer study.
“To truly create a paradigm shift in the decision to treat MC, a therapeutic treatment would need to be developed that would lead to resolution of the infection over a short time frame (ideally, weeks) with minimal discomfort,” Dr. Oza noted. “Both VP-102 and berdazimer gel, 10.3%, have the potential to be the first-ever MC therapies approved by the U.S. Food and Drug Administration,” and families seeking to reduce MC in visible areas would welcome this option for a home therapy, he said.
However, Dr. Oza emphasized that potential barriers to widespread use of these therapies include whether the efficacy can be maintained in patients who fail to comply with daily application, and the ongoing need for office-based therapy to manage sexually transmitted MC in adults and periocular and perianal MC in children. The study was funded by Novan. Lead author Dr. Browning disclosed grants from Novan during the conduct of the study; Dr. Hebert reported grants from the University of Texas Health Science Center McGovern Medical School-Houston during the conduct of the study. Disclosures of other authors included having reported equity in Novan during the conduct of the study and receiving a grant from Novan. Dr. Oza had no financial conflicts to disclose.
Treatment with .
Molluscum contagiosum (MC) remains a common infection that, despite being self-limiting, may persist for months or years, and is associated with quality of life concerns and the need for ongoing therapy, wrote John C. Browning, MD, of Texas Dermatology and Laser Specialists, San Antonio, and colleagues, who conducted the phase 3 randomized study.
The infection is most common in children aged 1-14 years, and treatment may be needed in part to avoid infecting peers and family members, they said. No treatments for molluscum are currently approved by the Food and Drug Administration.
In the study, which was published in JAMA Dermatology, the researchers randomized 444 patients to berdazimer gel 10.3% and 447 to a placebo gel, applied once daily in a thin layer on all MC lesions for 12 weeks. The study was conducted at 55 clinics across the United States between Sept. 1, 2020, and July 21, 2021. The mean age of the patients was about 6.5 years (range was 0.9-49 years), and about 85% were White. Participants had 3-70 raised MC lesions; those with sexually transmitted MC or MC in the periocular area were excluded. The primary endpoint was complete clearance of MC lesions after 12 weeks of treatment. At 12 weeks, significantly more patients treated with berdazimer gel achieved complete clearance than those on vehicle (32.4% vs. 19.7%; P < .001). A total of 64 (14.4%) patients in the berdazimer group discontinued treatment because of MC clearance, compared with 40 patients (8.9%) in the vehicle group.
Most adverse events were mild or moderate, and rates of adverse events resulting in treatment discontinuation were low overall for both groups; the most common adverse events were application-site pain and erythema, which were mostly mild. Overall, 4.1% of berdazimer-treated patients and 0.7% of placebo patients discontinued the study because of adverse events.
The study findings were limited by several factors, including the small number of patients in subgroups for race, ethnicity, and age; and the lack of data on patients with sexually transmitted MC and on concomitant use with other topical MC therapies, the researchers noted.
However, the results represent the largest randomized clinical trial of berdazimer 10.3% to date, and support its potential as a first-line therapy for MC patients aged 6 months and older, according to the authors. “Berdazimer is under consideration as a first in-class therapeutic agent for MC and may provide a topical prescription alternative to other therapies used for this highly contagious and psychosocially challenging skin condition,” they said.
Having a reliable, steroid-free, safe, and efficacious medication to treat molluscum in the pediatric population, as early as age 6 months, that can be used at home would “change the whole therapeutic paradigm,” one of the study authors, Adelaide Hebert, MD, said in an interview at the Society for Pediatric Dermatology annual meeting in July, where she presented phase 2 data on berdazimer gel. “This is a common problem and the rate of infections among siblings if it goes untreated is 41%. Affected kids have a sense of isolation; they don’t get invited to swimming parties.”
The lack of a safe and effective topical therapy “has been challenging,” added Dr. Hebert, professor of dermatology and pediatrics, and chief of pediatric dermatology at the University of Texas, Houston. She noted that treatments that have been used but have not been successful include imiquimod. “I’m not impressed with tretinoin,” although it is prescribed for MC, and the most common treatment prescribed by pediatricians for molluscum – mupirocin – is “usually not effective,” she said.
Another MC treatment in trials
Another investigative treatment for molluscum contagiosum, VP-102, a drug-device combination of cantharidin 0.7% administered through a single-use precision applicator, has been evaluated in phase 3 studies of patients with MC aged 2 years and older. The results of two phase 3 studies were published in 2020.
In May 2022, Verrica Pharmaceuticals, which is developing VP-102, announced that Food and Drug Administration approval had been delayed because of deficiencies identified at a contract manufacturing organization, and that the company was working with the agency to bring VP-102 to the market as soon as possible.
A step in the right direction
Although MC is self-resolving, cases last an average of 13.5 months, and “many families look to fast-forward their child’s experience with the infection,” Vikash S. Oza, MD, a pediatric dermatologist at New York University, New York, wrote in an editorial that accompanied the berdazimer study.
“To truly create a paradigm shift in the decision to treat MC, a therapeutic treatment would need to be developed that would lead to resolution of the infection over a short time frame (ideally, weeks) with minimal discomfort,” Dr. Oza noted. “Both VP-102 and berdazimer gel, 10.3%, have the potential to be the first-ever MC therapies approved by the U.S. Food and Drug Administration,” and families seeking to reduce MC in visible areas would welcome this option for a home therapy, he said.
However, Dr. Oza emphasized that potential barriers to widespread use of these therapies include whether the efficacy can be maintained in patients who fail to comply with daily application, and the ongoing need for office-based therapy to manage sexually transmitted MC in adults and periocular and perianal MC in children. The study was funded by Novan. Lead author Dr. Browning disclosed grants from Novan during the conduct of the study; Dr. Hebert reported grants from the University of Texas Health Science Center McGovern Medical School-Houston during the conduct of the study. Disclosures of other authors included having reported equity in Novan during the conduct of the study and receiving a grant from Novan. Dr. Oza had no financial conflicts to disclose.
Treatment with .
Molluscum contagiosum (MC) remains a common infection that, despite being self-limiting, may persist for months or years, and is associated with quality of life concerns and the need for ongoing therapy, wrote John C. Browning, MD, of Texas Dermatology and Laser Specialists, San Antonio, and colleagues, who conducted the phase 3 randomized study.
The infection is most common in children aged 1-14 years, and treatment may be needed in part to avoid infecting peers and family members, they said. No treatments for molluscum are currently approved by the Food and Drug Administration.
In the study, which was published in JAMA Dermatology, the researchers randomized 444 patients to berdazimer gel 10.3% and 447 to a placebo gel, applied once daily in a thin layer on all MC lesions for 12 weeks. The study was conducted at 55 clinics across the United States between Sept. 1, 2020, and July 21, 2021. The mean age of the patients was about 6.5 years (range was 0.9-49 years), and about 85% were White. Participants had 3-70 raised MC lesions; those with sexually transmitted MC or MC in the periocular area were excluded. The primary endpoint was complete clearance of MC lesions after 12 weeks of treatment. At 12 weeks, significantly more patients treated with berdazimer gel achieved complete clearance than those on vehicle (32.4% vs. 19.7%; P < .001). A total of 64 (14.4%) patients in the berdazimer group discontinued treatment because of MC clearance, compared with 40 patients (8.9%) in the vehicle group.
Most adverse events were mild or moderate, and rates of adverse events resulting in treatment discontinuation were low overall for both groups; the most common adverse events were application-site pain and erythema, which were mostly mild. Overall, 4.1% of berdazimer-treated patients and 0.7% of placebo patients discontinued the study because of adverse events.
The study findings were limited by several factors, including the small number of patients in subgroups for race, ethnicity, and age; and the lack of data on patients with sexually transmitted MC and on concomitant use with other topical MC therapies, the researchers noted.
However, the results represent the largest randomized clinical trial of berdazimer 10.3% to date, and support its potential as a first-line therapy for MC patients aged 6 months and older, according to the authors. “Berdazimer is under consideration as a first in-class therapeutic agent for MC and may provide a topical prescription alternative to other therapies used for this highly contagious and psychosocially challenging skin condition,” they said.
Having a reliable, steroid-free, safe, and efficacious medication to treat molluscum in the pediatric population, as early as age 6 months, that can be used at home would “change the whole therapeutic paradigm,” one of the study authors, Adelaide Hebert, MD, said in an interview at the Society for Pediatric Dermatology annual meeting in July, where she presented phase 2 data on berdazimer gel. “This is a common problem and the rate of infections among siblings if it goes untreated is 41%. Affected kids have a sense of isolation; they don’t get invited to swimming parties.”
The lack of a safe and effective topical therapy “has been challenging,” added Dr. Hebert, professor of dermatology and pediatrics, and chief of pediatric dermatology at the University of Texas, Houston. She noted that treatments that have been used but have not been successful include imiquimod. “I’m not impressed with tretinoin,” although it is prescribed for MC, and the most common treatment prescribed by pediatricians for molluscum – mupirocin – is “usually not effective,” she said.
Another MC treatment in trials
Another investigative treatment for molluscum contagiosum, VP-102, a drug-device combination of cantharidin 0.7% administered through a single-use precision applicator, has been evaluated in phase 3 studies of patients with MC aged 2 years and older. The results of two phase 3 studies were published in 2020.
In May 2022, Verrica Pharmaceuticals, which is developing VP-102, announced that Food and Drug Administration approval had been delayed because of deficiencies identified at a contract manufacturing organization, and that the company was working with the agency to bring VP-102 to the market as soon as possible.
A step in the right direction
Although MC is self-resolving, cases last an average of 13.5 months, and “many families look to fast-forward their child’s experience with the infection,” Vikash S. Oza, MD, a pediatric dermatologist at New York University, New York, wrote in an editorial that accompanied the berdazimer study.
“To truly create a paradigm shift in the decision to treat MC, a therapeutic treatment would need to be developed that would lead to resolution of the infection over a short time frame (ideally, weeks) with minimal discomfort,” Dr. Oza noted. “Both VP-102 and berdazimer gel, 10.3%, have the potential to be the first-ever MC therapies approved by the U.S. Food and Drug Administration,” and families seeking to reduce MC in visible areas would welcome this option for a home therapy, he said.
However, Dr. Oza emphasized that potential barriers to widespread use of these therapies include whether the efficacy can be maintained in patients who fail to comply with daily application, and the ongoing need for office-based therapy to manage sexually transmitted MC in adults and periocular and perianal MC in children. The study was funded by Novan. Lead author Dr. Browning disclosed grants from Novan during the conduct of the study; Dr. Hebert reported grants from the University of Texas Health Science Center McGovern Medical School-Houston during the conduct of the study. Disclosures of other authors included having reported equity in Novan during the conduct of the study and receiving a grant from Novan. Dr. Oza had no financial conflicts to disclose.
FROM JAMA DERMATOLOGY
LGBTQ+ Youth Consult: Let’s talk about PrEP!
As pediatricians, almost all of our clinic visits include some anticipatory guidance and recommendations on ways to promote well-being and prevent illness and injury for our patients. Because of minority stress, discrimination, and increased exposure to adverse childhood experiences, LGBTQ+ patients are disproportionately affected by certain health conditions including depression, anxiety, substance use, homelessness, as well as HIV and other sexually transmitted infections (STIs).1 While LGBTQ+ youth could benefit from additional guidance, counseling, and interventions related to these health disparities and have expressed interest in talking about these topics with their providers, sexual and gender minority youth also stress that they want to be treated as any other youth.2 Extending counseling for preventive care measures such as preexposure prophylaxis (PrEP) for HIV to all sexually active youth could help to destigmatize LGBTQ+ youth as being “different” from other youth and also help to increase overall access to HIV prevention services.3
Described by some as the “birth control” for HIV infection, PrEP is taken on an ongoing basis by those who are HIV negative before potential exposures to HIV in order to prevent new HIV infections. PrEP was first approved as a daily pill for adults in 2015 by the Food and Drug Administration with extension in 2018 to all individuals at risk for HIV weighing at least 35 kg after safety and efficacy data showed it could be used routinely for adolescents.4 When taken daily, oral PrEP can decrease the risk of HIV from sexual contact by more than 90% and from injection drug use by around 70%. As PrEP is highly effective with low risk for side effects, the U.S. Preventive Services Task Force (USPSTF) gave PrEP a “Grade A” recommendation for use in those at high risk for HIV infection in 2019.5 Since efficacy is closely tied to adherence, the first injectable PrEP (given at 0, 1, and 2 months with dosing then every 2 months) was also recently FDA approved in late 2021.6
Since HIV infection disproportionately affects LBGTQ+ individuals, and particularly LBGTQ+ youth of color, counseling related to PrEP has been largely targeted to these groups.7 Insurance and financial barriers to PrEP have been greatly reduced over the past several years through changes in insurance coverage (strengthened by the USPSTF recommendation), supplemental insurance programs, and pharmaceutical copay programs. Many states (but not all) also include HIV in the definition of STIs and allow minors to consent to PrEP services without a parent or guardian. Unfortunately, despite the high efficacy of PrEP and efforts to decrease barriers, rates of PrEP use continue to be extremely low, especially in youth, with only 15.6% of those aged 16-24 who are at risk for HIV in the United States actually taking PrEP in 2019.8 Many barriers to PrEP continue to exist including lack of awareness of PrEP, stigma surrounding HIV and PrEP, and lack of PrEP providers.
In order to address these low rates of PrEP uptake, the Centers for Disease Control and Prevention now recommends that medical providers discuss PrEP with all sexually active patients.6 PrEP should not be seen or discussed as something only relevant to LBGTQ+ populations, but rather as another tool in everyone’s “sexual health toolbox” that can allow us to experience human connection and pleasure through sexual activity while also having more control over what happens to our bodies. Not only will this allow more patients to access PrEP directly, it will also decrease the stigma of talking about HIV and PrEP and strengthen youths’ sense of autonomy and control over their own sexual health.
Since PrEP is a relatively new medical service, many providers will need to learn more about PrEP to at least have initial discussions with patients and to feel comfortable prescribing this themselves (See Resources). Below are also some suggestions to incorporate into your practice in order to advocate for the health and well-being of all your patients, including LGBTQ+ youth.
- Once your patients are 13 years and older, spend time with them alone to confidentially discuss more sensitive topics such as sexual health, mental health, and substance use.
- For all patients who are sexually active or considering sexual activity in the near future, discuss topics to help them control what happens to their bodies including consent, condoms, birth control, PrEP, and routine STI screening.
- Recommend PrEP to anyone who is sexually active and may be at increased risk for HIV infection or who is interested in taking PrEP for HIV prevention.
- Learn more about PrEP and start prescribing it to your own patients or become familiar with providers in your area to whom you could refer patients who are interested. While no certification is needed to prescribe PrEP, programs exist to help providers become more familiar with how to prescribe PrEP.
Dr. Warus is an adolescent medicine physician who specializes in care for transgender and gender-nonconforming youth, HIV prevention for adolescents and young adults, and LGBTQ health for youth at Children’s Hospital of Los Angeles. He is an assistant professor of clinical pediatrics and a University of Southern California faculty member.
Resources
CDC PrEP resources for clinicians: www.cdc.gov/hiv/clinicians/prevention/prep.html.Health HIV’s HIV Prevention Certified Provider Certification Program: https://healthhiv.org/programs/hpcp/.PrEP providers in the United States: https://preplocator.org/.Adolescent Health Working Group’s Sexual and Reproductive Health Toolkit for Adolescent Providers: https://ahwg.org/download/sexual-and-reproductive-health-toolkit-for-adolescent-providers/.
References
1. Lund EM and Burgess CM. Prim Care Clin Office Pract. 2021:48:179-89.
2. Hoffman ND et al. J Adolesc Health. 2009;45:222-9.
3. Mayer KH et al. Adv Ther. 2020;37:1778-811.
4. Hosek SG et al. JAMA Pediatr. 2017;171(11):1063-71.
5. U.S. Preventive Services Task Force; Owens DK et al. JAMA. 2019;321(22):2203-13.
6. Centers for Disease Control and Prevention: U.S. Public Health Service: Preexposure Prophylaxis for the Prevention of HIV Infection in the United States – 2021 Update: A Clinical Practice Guideline. Published 2021. Accessed July 10, 2022.
7. Centers for Disease Control and Prevention. Estimated HIV Incidence and Prevalence in the United States, 2015-2019. HIV Surveillance Supplemental Report. 2021;26(1). Published May 2021. Accessed July 10, 2022.
8. Centers for Disease Control and Prevention. Monitoring Selected National HIV Prevention and Care Objectives by Using HIV Surveillance Data–United States and 6 Dependent Areas, 2020. HIV Surveillance Supplemental Report. 2022;27(3).
As pediatricians, almost all of our clinic visits include some anticipatory guidance and recommendations on ways to promote well-being and prevent illness and injury for our patients. Because of minority stress, discrimination, and increased exposure to adverse childhood experiences, LGBTQ+ patients are disproportionately affected by certain health conditions including depression, anxiety, substance use, homelessness, as well as HIV and other sexually transmitted infections (STIs).1 While LGBTQ+ youth could benefit from additional guidance, counseling, and interventions related to these health disparities and have expressed interest in talking about these topics with their providers, sexual and gender minority youth also stress that they want to be treated as any other youth.2 Extending counseling for preventive care measures such as preexposure prophylaxis (PrEP) for HIV to all sexually active youth could help to destigmatize LGBTQ+ youth as being “different” from other youth and also help to increase overall access to HIV prevention services.3
Described by some as the “birth control” for HIV infection, PrEP is taken on an ongoing basis by those who are HIV negative before potential exposures to HIV in order to prevent new HIV infections. PrEP was first approved as a daily pill for adults in 2015 by the Food and Drug Administration with extension in 2018 to all individuals at risk for HIV weighing at least 35 kg after safety and efficacy data showed it could be used routinely for adolescents.4 When taken daily, oral PrEP can decrease the risk of HIV from sexual contact by more than 90% and from injection drug use by around 70%. As PrEP is highly effective with low risk for side effects, the U.S. Preventive Services Task Force (USPSTF) gave PrEP a “Grade A” recommendation for use in those at high risk for HIV infection in 2019.5 Since efficacy is closely tied to adherence, the first injectable PrEP (given at 0, 1, and 2 months with dosing then every 2 months) was also recently FDA approved in late 2021.6
Since HIV infection disproportionately affects LBGTQ+ individuals, and particularly LBGTQ+ youth of color, counseling related to PrEP has been largely targeted to these groups.7 Insurance and financial barriers to PrEP have been greatly reduced over the past several years through changes in insurance coverage (strengthened by the USPSTF recommendation), supplemental insurance programs, and pharmaceutical copay programs. Many states (but not all) also include HIV in the definition of STIs and allow minors to consent to PrEP services without a parent or guardian. Unfortunately, despite the high efficacy of PrEP and efforts to decrease barriers, rates of PrEP use continue to be extremely low, especially in youth, with only 15.6% of those aged 16-24 who are at risk for HIV in the United States actually taking PrEP in 2019.8 Many barriers to PrEP continue to exist including lack of awareness of PrEP, stigma surrounding HIV and PrEP, and lack of PrEP providers.
In order to address these low rates of PrEP uptake, the Centers for Disease Control and Prevention now recommends that medical providers discuss PrEP with all sexually active patients.6 PrEP should not be seen or discussed as something only relevant to LBGTQ+ populations, but rather as another tool in everyone’s “sexual health toolbox” that can allow us to experience human connection and pleasure through sexual activity while also having more control over what happens to our bodies. Not only will this allow more patients to access PrEP directly, it will also decrease the stigma of talking about HIV and PrEP and strengthen youths’ sense of autonomy and control over their own sexual health.
Since PrEP is a relatively new medical service, many providers will need to learn more about PrEP to at least have initial discussions with patients and to feel comfortable prescribing this themselves (See Resources). Below are also some suggestions to incorporate into your practice in order to advocate for the health and well-being of all your patients, including LGBTQ+ youth.
- Once your patients are 13 years and older, spend time with them alone to confidentially discuss more sensitive topics such as sexual health, mental health, and substance use.
- For all patients who are sexually active or considering sexual activity in the near future, discuss topics to help them control what happens to their bodies including consent, condoms, birth control, PrEP, and routine STI screening.
- Recommend PrEP to anyone who is sexually active and may be at increased risk for HIV infection or who is interested in taking PrEP for HIV prevention.
- Learn more about PrEP and start prescribing it to your own patients or become familiar with providers in your area to whom you could refer patients who are interested. While no certification is needed to prescribe PrEP, programs exist to help providers become more familiar with how to prescribe PrEP.
Dr. Warus is an adolescent medicine physician who specializes in care for transgender and gender-nonconforming youth, HIV prevention for adolescents and young adults, and LGBTQ health for youth at Children’s Hospital of Los Angeles. He is an assistant professor of clinical pediatrics and a University of Southern California faculty member.
Resources
CDC PrEP resources for clinicians: www.cdc.gov/hiv/clinicians/prevention/prep.html.Health HIV’s HIV Prevention Certified Provider Certification Program: https://healthhiv.org/programs/hpcp/.PrEP providers in the United States: https://preplocator.org/.Adolescent Health Working Group’s Sexual and Reproductive Health Toolkit for Adolescent Providers: https://ahwg.org/download/sexual-and-reproductive-health-toolkit-for-adolescent-providers/.
References
1. Lund EM and Burgess CM. Prim Care Clin Office Pract. 2021:48:179-89.
2. Hoffman ND et al. J Adolesc Health. 2009;45:222-9.
3. Mayer KH et al. Adv Ther. 2020;37:1778-811.
4. Hosek SG et al. JAMA Pediatr. 2017;171(11):1063-71.
5. U.S. Preventive Services Task Force; Owens DK et al. JAMA. 2019;321(22):2203-13.
6. Centers for Disease Control and Prevention: U.S. Public Health Service: Preexposure Prophylaxis for the Prevention of HIV Infection in the United States – 2021 Update: A Clinical Practice Guideline. Published 2021. Accessed July 10, 2022.
7. Centers for Disease Control and Prevention. Estimated HIV Incidence and Prevalence in the United States, 2015-2019. HIV Surveillance Supplemental Report. 2021;26(1). Published May 2021. Accessed July 10, 2022.
8. Centers for Disease Control and Prevention. Monitoring Selected National HIV Prevention and Care Objectives by Using HIV Surveillance Data–United States and 6 Dependent Areas, 2020. HIV Surveillance Supplemental Report. 2022;27(3).
As pediatricians, almost all of our clinic visits include some anticipatory guidance and recommendations on ways to promote well-being and prevent illness and injury for our patients. Because of minority stress, discrimination, and increased exposure to adverse childhood experiences, LGBTQ+ patients are disproportionately affected by certain health conditions including depression, anxiety, substance use, homelessness, as well as HIV and other sexually transmitted infections (STIs).1 While LGBTQ+ youth could benefit from additional guidance, counseling, and interventions related to these health disparities and have expressed interest in talking about these topics with their providers, sexual and gender minority youth also stress that they want to be treated as any other youth.2 Extending counseling for preventive care measures such as preexposure prophylaxis (PrEP) for HIV to all sexually active youth could help to destigmatize LGBTQ+ youth as being “different” from other youth and also help to increase overall access to HIV prevention services.3
Described by some as the “birth control” for HIV infection, PrEP is taken on an ongoing basis by those who are HIV negative before potential exposures to HIV in order to prevent new HIV infections. PrEP was first approved as a daily pill for adults in 2015 by the Food and Drug Administration with extension in 2018 to all individuals at risk for HIV weighing at least 35 kg after safety and efficacy data showed it could be used routinely for adolescents.4 When taken daily, oral PrEP can decrease the risk of HIV from sexual contact by more than 90% and from injection drug use by around 70%. As PrEP is highly effective with low risk for side effects, the U.S. Preventive Services Task Force (USPSTF) gave PrEP a “Grade A” recommendation for use in those at high risk for HIV infection in 2019.5 Since efficacy is closely tied to adherence, the first injectable PrEP (given at 0, 1, and 2 months with dosing then every 2 months) was also recently FDA approved in late 2021.6
Since HIV infection disproportionately affects LBGTQ+ individuals, and particularly LBGTQ+ youth of color, counseling related to PrEP has been largely targeted to these groups.7 Insurance and financial barriers to PrEP have been greatly reduced over the past several years through changes in insurance coverage (strengthened by the USPSTF recommendation), supplemental insurance programs, and pharmaceutical copay programs. Many states (but not all) also include HIV in the definition of STIs and allow minors to consent to PrEP services without a parent or guardian. Unfortunately, despite the high efficacy of PrEP and efforts to decrease barriers, rates of PrEP use continue to be extremely low, especially in youth, with only 15.6% of those aged 16-24 who are at risk for HIV in the United States actually taking PrEP in 2019.8 Many barriers to PrEP continue to exist including lack of awareness of PrEP, stigma surrounding HIV and PrEP, and lack of PrEP providers.
In order to address these low rates of PrEP uptake, the Centers for Disease Control and Prevention now recommends that medical providers discuss PrEP with all sexually active patients.6 PrEP should not be seen or discussed as something only relevant to LBGTQ+ populations, but rather as another tool in everyone’s “sexual health toolbox” that can allow us to experience human connection and pleasure through sexual activity while also having more control over what happens to our bodies. Not only will this allow more patients to access PrEP directly, it will also decrease the stigma of talking about HIV and PrEP and strengthen youths’ sense of autonomy and control over their own sexual health.
Since PrEP is a relatively new medical service, many providers will need to learn more about PrEP to at least have initial discussions with patients and to feel comfortable prescribing this themselves (See Resources). Below are also some suggestions to incorporate into your practice in order to advocate for the health and well-being of all your patients, including LGBTQ+ youth.
- Once your patients are 13 years and older, spend time with them alone to confidentially discuss more sensitive topics such as sexual health, mental health, and substance use.
- For all patients who are sexually active or considering sexual activity in the near future, discuss topics to help them control what happens to their bodies including consent, condoms, birth control, PrEP, and routine STI screening.
- Recommend PrEP to anyone who is sexually active and may be at increased risk for HIV infection or who is interested in taking PrEP for HIV prevention.
- Learn more about PrEP and start prescribing it to your own patients or become familiar with providers in your area to whom you could refer patients who are interested. While no certification is needed to prescribe PrEP, programs exist to help providers become more familiar with how to prescribe PrEP.
Dr. Warus is an adolescent medicine physician who specializes in care for transgender and gender-nonconforming youth, HIV prevention for adolescents and young adults, and LGBTQ health for youth at Children’s Hospital of Los Angeles. He is an assistant professor of clinical pediatrics and a University of Southern California faculty member.
Resources
CDC PrEP resources for clinicians: www.cdc.gov/hiv/clinicians/prevention/prep.html.Health HIV’s HIV Prevention Certified Provider Certification Program: https://healthhiv.org/programs/hpcp/.PrEP providers in the United States: https://preplocator.org/.Adolescent Health Working Group’s Sexual and Reproductive Health Toolkit for Adolescent Providers: https://ahwg.org/download/sexual-and-reproductive-health-toolkit-for-adolescent-providers/.
References
1. Lund EM and Burgess CM. Prim Care Clin Office Pract. 2021:48:179-89.
2. Hoffman ND et al. J Adolesc Health. 2009;45:222-9.
3. Mayer KH et al. Adv Ther. 2020;37:1778-811.
4. Hosek SG et al. JAMA Pediatr. 2017;171(11):1063-71.
5. U.S. Preventive Services Task Force; Owens DK et al. JAMA. 2019;321(22):2203-13.
6. Centers for Disease Control and Prevention: U.S. Public Health Service: Preexposure Prophylaxis for the Prevention of HIV Infection in the United States – 2021 Update: A Clinical Practice Guideline. Published 2021. Accessed July 10, 2022.
7. Centers for Disease Control and Prevention. Estimated HIV Incidence and Prevalence in the United States, 2015-2019. HIV Surveillance Supplemental Report. 2021;26(1). Published May 2021. Accessed July 10, 2022.
8. Centers for Disease Control and Prevention. Monitoring Selected National HIV Prevention and Care Objectives by Using HIV Surveillance Data–United States and 6 Dependent Areas, 2020. HIV Surveillance Supplemental Report. 2022;27(3).
Two congressmen targeting ‘gender transition’ physicians
Two GOP congressmen have introduced legislation aimed at holding doctors who perform gender transition procedures on minors liable for their actions, says a story reported on KATV.com, among other news sites.
The two GOP lawmakers – Rep. Jim Banks (IN) and Sen. Tom Cotton (AR) – introduced the Protecting Minors from Medical Malpractice Act in their respective chambers.
If passed, the House and Senate bills would make doctors liable for any gender transition surgery on a minor that results in injury, whether physical, psychological, emotional, or physiological. Minors who believe they’ve been harmed would have up to 30 years from when they turn 18 to file a claim.
The House proposal would also strip federal funding from states that require health care professionals to provide gender transition procedures, including puberty blockers, cross-sex hormones, and gender reassignment surgeries.
A companion House bill, also sponsored by Banks, targets another issue related to gender transitioning for minors: parental consent.
If passed, the Empower Parents to Protect Their Kids Act of 2022 would deny federal funding to any elementary and secondary schools that initiate a minor’s gender transition without first securing parental consent. (Last October, Sen. Cotton released a similar bill in the Senate.)
The content contained in this article is for informational purposes only and does not constitute legal advice. Reliance on any information provided in this article is solely at your own risk.
A version of this article first appeared on Medscape.com.
Two GOP congressmen have introduced legislation aimed at holding doctors who perform gender transition procedures on minors liable for their actions, says a story reported on KATV.com, among other news sites.
The two GOP lawmakers – Rep. Jim Banks (IN) and Sen. Tom Cotton (AR) – introduced the Protecting Minors from Medical Malpractice Act in their respective chambers.
If passed, the House and Senate bills would make doctors liable for any gender transition surgery on a minor that results in injury, whether physical, psychological, emotional, or physiological. Minors who believe they’ve been harmed would have up to 30 years from when they turn 18 to file a claim.
The House proposal would also strip federal funding from states that require health care professionals to provide gender transition procedures, including puberty blockers, cross-sex hormones, and gender reassignment surgeries.
A companion House bill, also sponsored by Banks, targets another issue related to gender transitioning for minors: parental consent.
If passed, the Empower Parents to Protect Their Kids Act of 2022 would deny federal funding to any elementary and secondary schools that initiate a minor’s gender transition without first securing parental consent. (Last October, Sen. Cotton released a similar bill in the Senate.)
The content contained in this article is for informational purposes only and does not constitute legal advice. Reliance on any information provided in this article is solely at your own risk.
A version of this article first appeared on Medscape.com.
Two GOP congressmen have introduced legislation aimed at holding doctors who perform gender transition procedures on minors liable for their actions, says a story reported on KATV.com, among other news sites.
The two GOP lawmakers – Rep. Jim Banks (IN) and Sen. Tom Cotton (AR) – introduced the Protecting Minors from Medical Malpractice Act in their respective chambers.
If passed, the House and Senate bills would make doctors liable for any gender transition surgery on a minor that results in injury, whether physical, psychological, emotional, or physiological. Minors who believe they’ve been harmed would have up to 30 years from when they turn 18 to file a claim.
The House proposal would also strip federal funding from states that require health care professionals to provide gender transition procedures, including puberty blockers, cross-sex hormones, and gender reassignment surgeries.
A companion House bill, also sponsored by Banks, targets another issue related to gender transitioning for minors: parental consent.
If passed, the Empower Parents to Protect Their Kids Act of 2022 would deny federal funding to any elementary and secondary schools that initiate a minor’s gender transition without first securing parental consent. (Last October, Sen. Cotton released a similar bill in the Senate.)
The content contained in this article is for informational purposes only and does not constitute legal advice. Reliance on any information provided in this article is solely at your own risk.
A version of this article first appeared on Medscape.com.
Link between pediatric hepatitis and adenovirus 41 still unclear
While two new studies reiterate a possible relationship between adenovirus 41 and acute hepatitis of unknown cause in children, whether these infections are significant or merely bystanders remains unclear.
In both studies – one conducted in Alabama and the other conducted in the United Kingdom – researchers found that 90% of children with acute hepatitis of unknown cause tested positive for adenovirus 41. The virus subtype is not an uncommon infection, but it usually causes gastroenteritis in children.
“Across the world, adenovirus continues to be a common signal” in these pediatric hepatitis cases, said Helena Gutierrez, MD, the medical director of the Pediatric Liver Transplant Program at the University of Alabama at Birmingham, in an interview. She led one of the studies. More data are necessary to understand what role this virus may play in these cases, she said.
In November, the Alabama Department of Public Health and the U.S. Centers for Disease Control and Prevention began investigating a cluster of severe pediatric hepatitis cases at the Children’s of Alabama hospital in Birmingham. These children also tested positive for adenovirus. In April, the United Kingdom announced they were investigating similar cases, and the CDC expanded their search nationally. As of July 8, 1,010 cases in 35 countries have been reported to the World Health Organization. There are 263 confirmed cases in the United Kingdom and 332 cases under investigation by the CDC in the United States, according to the most recent counts.
The two studies, both published in the New England Journal of Medicine, provide additional clinical data on a number of these mysterious hepatitis cases. Dr. Gutierrez’s study looked at nine children admitted for hepatitis of unknown origin between October 1 and February 28. Patients had a median age of 2 years 11 months and two required liver transplants, and there were no deaths.
Eight out of nine patients (89%) tested positive for adenovirus, and all five of the samples that were of sufficient quality for gene sequencing tested positive for adenovirus 41. None of the six liver biopsies performed found signs of adenovirus infection, but the liver tissue samples of three patients tested positive for adenovirus via PCR.
The second study involved 44 children referred to a liver transplantation center in the United Kingdom between January 1 and April 11, 2022. The median age for patients was 4 years. Six children required liver transplants, and there were no deaths. Of the 30 patients who underwent molecular adenovirus testing, 27 (90%) were positive for adenovirus 41. Liver samples of nine children (3 from biopsies and 6 from explanted livers) all tested negative for adenovirus antibodies.
In both studies, however, the median adenovirus viral load of patients needing a transplant was much higher than the viral loads in children who did not require liver transplants.
Although most of the clinical features and test results of these cases suggest that adenovirus may be involved, the negative results in histology are “intriguing,” Chayarani Kelgeri, MD, a consultant pediatric hepatologist at the Birmingham Women’s and Children’s Hospital, U.K., said in an email. She is the lead author of the U.K. study. “Whether this is because the liver injury we see is an aftermath of the viral infection, the mechanism of injury is immune mediated, and if other cofactors are involved is being explored,” she added. “Further investigations being undertaken by UK Health Security Agency will add to our understanding of this illness.”
Although there is a high adenovirus positivity rate amongst these cases, there is not enough evidence yet to say adenovirus 41 is a new cause of pediatric hepatitis in previously healthy children, said Saul Karpen, MD, PhD, the division chief of pediatric gastroenterology, hepatology, and nutrition at Emory University School of Medicine, Atlanta. He wrote an editorial accompanying the two NEJM studies.
The CDC has not yet found an increase in pediatric hepatitis cases, according to a recent analysis, though the United Kingdom has found an uptick in cases this year, he told this news organization. Also, the cases highlighted in both articles showed no histological evidence of adenovirus in liver biopsies. “That’s completely opposite of what we generally see in adenoviral hepatitis that can be quite severe,” he said, adding that in general, there are detectable viral particles and antigens in affected livers.
“These two important reports indicate to those inside and outside the field of pediatric hepatology that registries and clinical studies of acute hepatitis in children are sorely needed,” Dr. Karpen writes in the editorial; “It is likely that with greater attention to collecting data on cases and biospecimens from children with acute hepatitis, we will be able to determine whether this one virus, human adenovirus 41, is of relevance to this important and serious condition in children.”
Dr. Gutierrez, Dr. Kelgeri, and Dr. Karpen report no relevant financial relationships.
A version of this article first appeared on Medscape.com.
While two new studies reiterate a possible relationship between adenovirus 41 and acute hepatitis of unknown cause in children, whether these infections are significant or merely bystanders remains unclear.
In both studies – one conducted in Alabama and the other conducted in the United Kingdom – researchers found that 90% of children with acute hepatitis of unknown cause tested positive for adenovirus 41. The virus subtype is not an uncommon infection, but it usually causes gastroenteritis in children.
“Across the world, adenovirus continues to be a common signal” in these pediatric hepatitis cases, said Helena Gutierrez, MD, the medical director of the Pediatric Liver Transplant Program at the University of Alabama at Birmingham, in an interview. She led one of the studies. More data are necessary to understand what role this virus may play in these cases, she said.
In November, the Alabama Department of Public Health and the U.S. Centers for Disease Control and Prevention began investigating a cluster of severe pediatric hepatitis cases at the Children’s of Alabama hospital in Birmingham. These children also tested positive for adenovirus. In April, the United Kingdom announced they were investigating similar cases, and the CDC expanded their search nationally. As of July 8, 1,010 cases in 35 countries have been reported to the World Health Organization. There are 263 confirmed cases in the United Kingdom and 332 cases under investigation by the CDC in the United States, according to the most recent counts.
The two studies, both published in the New England Journal of Medicine, provide additional clinical data on a number of these mysterious hepatitis cases. Dr. Gutierrez’s study looked at nine children admitted for hepatitis of unknown origin between October 1 and February 28. Patients had a median age of 2 years 11 months and two required liver transplants, and there were no deaths.
Eight out of nine patients (89%) tested positive for adenovirus, and all five of the samples that were of sufficient quality for gene sequencing tested positive for adenovirus 41. None of the six liver biopsies performed found signs of adenovirus infection, but the liver tissue samples of three patients tested positive for adenovirus via PCR.
The second study involved 44 children referred to a liver transplantation center in the United Kingdom between January 1 and April 11, 2022. The median age for patients was 4 years. Six children required liver transplants, and there were no deaths. Of the 30 patients who underwent molecular adenovirus testing, 27 (90%) were positive for adenovirus 41. Liver samples of nine children (3 from biopsies and 6 from explanted livers) all tested negative for adenovirus antibodies.
In both studies, however, the median adenovirus viral load of patients needing a transplant was much higher than the viral loads in children who did not require liver transplants.
Although most of the clinical features and test results of these cases suggest that adenovirus may be involved, the negative results in histology are “intriguing,” Chayarani Kelgeri, MD, a consultant pediatric hepatologist at the Birmingham Women’s and Children’s Hospital, U.K., said in an email. She is the lead author of the U.K. study. “Whether this is because the liver injury we see is an aftermath of the viral infection, the mechanism of injury is immune mediated, and if other cofactors are involved is being explored,” she added. “Further investigations being undertaken by UK Health Security Agency will add to our understanding of this illness.”
Although there is a high adenovirus positivity rate amongst these cases, there is not enough evidence yet to say adenovirus 41 is a new cause of pediatric hepatitis in previously healthy children, said Saul Karpen, MD, PhD, the division chief of pediatric gastroenterology, hepatology, and nutrition at Emory University School of Medicine, Atlanta. He wrote an editorial accompanying the two NEJM studies.
The CDC has not yet found an increase in pediatric hepatitis cases, according to a recent analysis, though the United Kingdom has found an uptick in cases this year, he told this news organization. Also, the cases highlighted in both articles showed no histological evidence of adenovirus in liver biopsies. “That’s completely opposite of what we generally see in adenoviral hepatitis that can be quite severe,” he said, adding that in general, there are detectable viral particles and antigens in affected livers.
“These two important reports indicate to those inside and outside the field of pediatric hepatology that registries and clinical studies of acute hepatitis in children are sorely needed,” Dr. Karpen writes in the editorial; “It is likely that with greater attention to collecting data on cases and biospecimens from children with acute hepatitis, we will be able to determine whether this one virus, human adenovirus 41, is of relevance to this important and serious condition in children.”
Dr. Gutierrez, Dr. Kelgeri, and Dr. Karpen report no relevant financial relationships.
A version of this article first appeared on Medscape.com.
While two new studies reiterate a possible relationship between adenovirus 41 and acute hepatitis of unknown cause in children, whether these infections are significant or merely bystanders remains unclear.
In both studies – one conducted in Alabama and the other conducted in the United Kingdom – researchers found that 90% of children with acute hepatitis of unknown cause tested positive for adenovirus 41. The virus subtype is not an uncommon infection, but it usually causes gastroenteritis in children.
“Across the world, adenovirus continues to be a common signal” in these pediatric hepatitis cases, said Helena Gutierrez, MD, the medical director of the Pediatric Liver Transplant Program at the University of Alabama at Birmingham, in an interview. She led one of the studies. More data are necessary to understand what role this virus may play in these cases, she said.
In November, the Alabama Department of Public Health and the U.S. Centers for Disease Control and Prevention began investigating a cluster of severe pediatric hepatitis cases at the Children’s of Alabama hospital in Birmingham. These children also tested positive for adenovirus. In April, the United Kingdom announced they were investigating similar cases, and the CDC expanded their search nationally. As of July 8, 1,010 cases in 35 countries have been reported to the World Health Organization. There are 263 confirmed cases in the United Kingdom and 332 cases under investigation by the CDC in the United States, according to the most recent counts.
The two studies, both published in the New England Journal of Medicine, provide additional clinical data on a number of these mysterious hepatitis cases. Dr. Gutierrez’s study looked at nine children admitted for hepatitis of unknown origin between October 1 and February 28. Patients had a median age of 2 years 11 months and two required liver transplants, and there were no deaths.
Eight out of nine patients (89%) tested positive for adenovirus, and all five of the samples that were of sufficient quality for gene sequencing tested positive for adenovirus 41. None of the six liver biopsies performed found signs of adenovirus infection, but the liver tissue samples of three patients tested positive for adenovirus via PCR.
The second study involved 44 children referred to a liver transplantation center in the United Kingdom between January 1 and April 11, 2022. The median age for patients was 4 years. Six children required liver transplants, and there were no deaths. Of the 30 patients who underwent molecular adenovirus testing, 27 (90%) were positive for adenovirus 41. Liver samples of nine children (3 from biopsies and 6 from explanted livers) all tested negative for adenovirus antibodies.
In both studies, however, the median adenovirus viral load of patients needing a transplant was much higher than the viral loads in children who did not require liver transplants.
Although most of the clinical features and test results of these cases suggest that adenovirus may be involved, the negative results in histology are “intriguing,” Chayarani Kelgeri, MD, a consultant pediatric hepatologist at the Birmingham Women’s and Children’s Hospital, U.K., said in an email. She is the lead author of the U.K. study. “Whether this is because the liver injury we see is an aftermath of the viral infection, the mechanism of injury is immune mediated, and if other cofactors are involved is being explored,” she added. “Further investigations being undertaken by UK Health Security Agency will add to our understanding of this illness.”
Although there is a high adenovirus positivity rate amongst these cases, there is not enough evidence yet to say adenovirus 41 is a new cause of pediatric hepatitis in previously healthy children, said Saul Karpen, MD, PhD, the division chief of pediatric gastroenterology, hepatology, and nutrition at Emory University School of Medicine, Atlanta. He wrote an editorial accompanying the two NEJM studies.
The CDC has not yet found an increase in pediatric hepatitis cases, according to a recent analysis, though the United Kingdom has found an uptick in cases this year, he told this news organization. Also, the cases highlighted in both articles showed no histological evidence of adenovirus in liver biopsies. “That’s completely opposite of what we generally see in adenoviral hepatitis that can be quite severe,” he said, adding that in general, there are detectable viral particles and antigens in affected livers.
“These two important reports indicate to those inside and outside the field of pediatric hepatology that registries and clinical studies of acute hepatitis in children are sorely needed,” Dr. Karpen writes in the editorial; “It is likely that with greater attention to collecting data on cases and biospecimens from children with acute hepatitis, we will be able to determine whether this one virus, human adenovirus 41, is of relevance to this important and serious condition in children.”
Dr. Gutierrez, Dr. Kelgeri, and Dr. Karpen report no relevant financial relationships.
A version of this article first appeared on Medscape.com.
Recommendations on breastfeeding: A case of too much information
The American Academy of Pediatrics is built on good intentions. It wants the best for children in the world, and it hopes to support its members in their efforts to achieve this goal. But from time to time, the academy loses sight of reality and makes recommendations that are counterproductive to its stated goals.
The recent release of its new policy “Breastfeeding and the Use of Human Milk” is another unfortunate example of poorly aimed recommendations. A careful reading of the document reveals it to be a well-researched treatise on breastfeeding and the value of human milk, including a discussion of the numerous impediments to the universal adoption of breastfeeding in our society. However, when a document of this breadth and complexity is released to the public it is never surprising that the messages deserving the most attention are lost in the press coverage. Most of the headlines I saw mentioned pediatricians supporting breastfeeding for a year or 2.
Who was the target audience? If it was pediatricians, most of us don’t need a longer list of the health benefits of breastfeeding. We already believe it is the best nutritional source for human babies and realize that the institutional framework in this country continues to be unfriendly to women who intend to breastfeed.
If the audience is politicians and public health decision-makers, the new policy contains a wealth of supportive evidence. However, most pediatricians I know are too busy or lack the skills and enthusiasm to become political activists. For the rest of population, including parents, the recommendations represent a collection of TMI (too much information).
If the audience is women who are considering breastfeeding I suspect nearly 100% already know pediatricians think it is the preferred way to feed their babies. And, likewise, a longer list won’t convince them to try nursing. Additional evidence may simply make them feel more guilty when they aren’t successful.
Many pregnant women have already been told that breastfeeding can be a challenge and given their situation breast milk alone for the first 6 months may sound like an unreasonable goal. The new recommendation that breastfeeding for a year or 2 is good is not a message they want to hear.
On the other hand, if the target audience is women who will be comforted to hear an official statement that normalizes breastfeeding longer than a year, the new policy statement has hit the nail on the head.
Of course the new policy document is sprinkled with caveats that vaguely hint at the possibility that pediatricians are sensitive human beings who under certain circumstances may be able to compromise when it comes to the duration of breastfeeding and the introduction of formula. But this whiff of reality is certainly not the dominant odor in these new recommendations.
Don’t get me wrong: I think the academy was overdue for a policy revision on breastfeeding. However, it should have been one that was reality based. It should acknowledge that there are institutional and societal biases against breastfeeding, and it should remind pediatricians that they can effect change by discussing these realities honestly with parents, while making it clear that we are there for them and their children regardless of how they feed their baby. Pediatricians believe that breastfeeding is the best but not the only way to feed a baby. We have (or will provide) the skills to assist parents succeed in whatever method they choose and strive to minimize the impediments that are within our power to change.
If the academy had chosen to release a separate statement simply supporting mothers who chose to nurse longer than a year, then that would have been a good idea. However, when presented as part of the larger document, that message dominated in the media and only served to fuel the guilt that many new mothers must endure.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].
The American Academy of Pediatrics is built on good intentions. It wants the best for children in the world, and it hopes to support its members in their efforts to achieve this goal. But from time to time, the academy loses sight of reality and makes recommendations that are counterproductive to its stated goals.
The recent release of its new policy “Breastfeeding and the Use of Human Milk” is another unfortunate example of poorly aimed recommendations. A careful reading of the document reveals it to be a well-researched treatise on breastfeeding and the value of human milk, including a discussion of the numerous impediments to the universal adoption of breastfeeding in our society. However, when a document of this breadth and complexity is released to the public it is never surprising that the messages deserving the most attention are lost in the press coverage. Most of the headlines I saw mentioned pediatricians supporting breastfeeding for a year or 2.
Who was the target audience? If it was pediatricians, most of us don’t need a longer list of the health benefits of breastfeeding. We already believe it is the best nutritional source for human babies and realize that the institutional framework in this country continues to be unfriendly to women who intend to breastfeed.
If the audience is politicians and public health decision-makers, the new policy contains a wealth of supportive evidence. However, most pediatricians I know are too busy or lack the skills and enthusiasm to become political activists. For the rest of population, including parents, the recommendations represent a collection of TMI (too much information).
If the audience is women who are considering breastfeeding I suspect nearly 100% already know pediatricians think it is the preferred way to feed their babies. And, likewise, a longer list won’t convince them to try nursing. Additional evidence may simply make them feel more guilty when they aren’t successful.
Many pregnant women have already been told that breastfeeding can be a challenge and given their situation breast milk alone for the first 6 months may sound like an unreasonable goal. The new recommendation that breastfeeding for a year or 2 is good is not a message they want to hear.
On the other hand, if the target audience is women who will be comforted to hear an official statement that normalizes breastfeeding longer than a year, the new policy statement has hit the nail on the head.
Of course the new policy document is sprinkled with caveats that vaguely hint at the possibility that pediatricians are sensitive human beings who under certain circumstances may be able to compromise when it comes to the duration of breastfeeding and the introduction of formula. But this whiff of reality is certainly not the dominant odor in these new recommendations.
Don’t get me wrong: I think the academy was overdue for a policy revision on breastfeeding. However, it should have been one that was reality based. It should acknowledge that there are institutional and societal biases against breastfeeding, and it should remind pediatricians that they can effect change by discussing these realities honestly with parents, while making it clear that we are there for them and their children regardless of how they feed their baby. Pediatricians believe that breastfeeding is the best but not the only way to feed a baby. We have (or will provide) the skills to assist parents succeed in whatever method they choose and strive to minimize the impediments that are within our power to change.
If the academy had chosen to release a separate statement simply supporting mothers who chose to nurse longer than a year, then that would have been a good idea. However, when presented as part of the larger document, that message dominated in the media and only served to fuel the guilt that many new mothers must endure.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].
The American Academy of Pediatrics is built on good intentions. It wants the best for children in the world, and it hopes to support its members in their efforts to achieve this goal. But from time to time, the academy loses sight of reality and makes recommendations that are counterproductive to its stated goals.
The recent release of its new policy “Breastfeeding and the Use of Human Milk” is another unfortunate example of poorly aimed recommendations. A careful reading of the document reveals it to be a well-researched treatise on breastfeeding and the value of human milk, including a discussion of the numerous impediments to the universal adoption of breastfeeding in our society. However, when a document of this breadth and complexity is released to the public it is never surprising that the messages deserving the most attention are lost in the press coverage. Most of the headlines I saw mentioned pediatricians supporting breastfeeding for a year or 2.
Who was the target audience? If it was pediatricians, most of us don’t need a longer list of the health benefits of breastfeeding. We already believe it is the best nutritional source for human babies and realize that the institutional framework in this country continues to be unfriendly to women who intend to breastfeed.
If the audience is politicians and public health decision-makers, the new policy contains a wealth of supportive evidence. However, most pediatricians I know are too busy or lack the skills and enthusiasm to become political activists. For the rest of population, including parents, the recommendations represent a collection of TMI (too much information).
If the audience is women who are considering breastfeeding I suspect nearly 100% already know pediatricians think it is the preferred way to feed their babies. And, likewise, a longer list won’t convince them to try nursing. Additional evidence may simply make them feel more guilty when they aren’t successful.
Many pregnant women have already been told that breastfeeding can be a challenge and given their situation breast milk alone for the first 6 months may sound like an unreasonable goal. The new recommendation that breastfeeding for a year or 2 is good is not a message they want to hear.
On the other hand, if the target audience is women who will be comforted to hear an official statement that normalizes breastfeeding longer than a year, the new policy statement has hit the nail on the head.
Of course the new policy document is sprinkled with caveats that vaguely hint at the possibility that pediatricians are sensitive human beings who under certain circumstances may be able to compromise when it comes to the duration of breastfeeding and the introduction of formula. But this whiff of reality is certainly not the dominant odor in these new recommendations.
Don’t get me wrong: I think the academy was overdue for a policy revision on breastfeeding. However, it should have been one that was reality based. It should acknowledge that there are institutional and societal biases against breastfeeding, and it should remind pediatricians that they can effect change by discussing these realities honestly with parents, while making it clear that we are there for them and their children regardless of how they feed their baby. Pediatricians believe that breastfeeding is the best but not the only way to feed a baby. We have (or will provide) the skills to assist parents succeed in whatever method they choose and strive to minimize the impediments that are within our power to change.
If the academy had chosen to release a separate statement simply supporting mothers who chose to nurse longer than a year, then that would have been a good idea. However, when presented as part of the larger document, that message dominated in the media and only served to fuel the guilt that many new mothers must endure.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].
Study eyes characteristics of pediatric patients with hidradenitis suppurativa
INDIANAPOLIS – in a study presented at the annual meeting of the Society for Pediatric Dermatology.
In addition, 44% presented with scarring, which suggests that HS may be underdiagnosed in this patient population. Those are the key findings from the study, a single-center retrospective chart review presented by Stephanie Sanchez during a poster session at the meeting.
“There is limited research on HS within the pediatric population,” said Ms. Sanchez, a fourth-year medical student at Boston University. “It’s not very well defined or characterized.” The “unusually high number of pediatric patients with HS” at Boston Medical Center provided “a unique opportunity to study this topic.”
Working with her mentor, Lisa Shen, MD, associate medical director of pediatric dermatology at Boston University, Ms. Sanchez and colleagues retrospectively reviewed the medical records of 303 patients aged 4-18 years who were diagnosed with HS at Boston Medical Center from 2012 to 2021. Boston Medical Center is the largest safety net hospital in New England. All data points and outcome measures were collected within 6 months of the patient’s HS diagnosis date.
Of the 303 patients with HS, 84% were female and 16% were male. Complete information about race was available in 286 patients. Of these, 65% were Black/African American, 11% were White, and the rest were from other racial groups. The mean age at symptom onset was 13 years, while the mean age at diagnosis was 15 years, and the mean delay to diagnosis was 2 years. A family history of HS was reported in 36% of patients.
The most common clinical features in these HS patients were pain/tenderness (90%), pustules/papules (65%), discharge/drainage (62%), and deep-seated nodules (51%). Scarring was present in 44% of patients at the time of diagnosis. The three most common sites of involvement were the axillary area (79%), the pubic area (36%), and the inguinal folds/inner thighs (34%).
Obesity was the most common comorbidity at the time of diagnosis, with 64% of patients affected. The next most common comorbidities were acne vulgaris (36%), acanthosis nigricans (25%), depression (18%), being overweight (17%), polycystic ovary syndrome (16%) and anxiety (13%). None had type 1 diabetes or metabolic syndrome.
Referring to the large population of underserved minority patients at Boston Medical Center, Dr. Shen noted, “we have to make sure not to underestimate the prevalence of obesity in this population as they get older. We need to start from a younger age to incorporate multidisciplinary care such as weight management, nutrition, and working with our pediatric surgery colleagues in trying to tackle [HS] because there is data to suggest that the earlier we intervene, the better outcomes they have. That makes sense.”
Adam Friedman, MD, professor and chair of dermatology at George Washington University, Washington, who was asked to comment on the findings, said that the study “highlights the impressive and concerning gap and delays in diagnosis, not too dissimilar to what the literature shows in adult HS patients, which unfortunately has tremendous ramifications, both physically and emotionally/psychosocially.”
While this single-center study identified potential risk factors, such as obesity and self-identifying as Black, he said, “it is important to note that this condition does not discriminate and therefore it is important not to miss the cases that don’t follow the textbook nor stigmatize this condition as one that only impacts certain demographics.”
The researchers reported having no financial disclosures. Dr. Friedman, who was not involved with the study, reported that he serves as a consultant and/or advisor to numerous pharmaceutical companies. He is a speaker for companies including, Regeneron, Sanofi, AbbVie, Janssen, Incyte, and Brickell Biotech, and has received grants from Pfizer, the Dermatology Foundation, Almirall, Incyte, Galderma, and Janssen.
INDIANAPOLIS – in a study presented at the annual meeting of the Society for Pediatric Dermatology.
In addition, 44% presented with scarring, which suggests that HS may be underdiagnosed in this patient population. Those are the key findings from the study, a single-center retrospective chart review presented by Stephanie Sanchez during a poster session at the meeting.
“There is limited research on HS within the pediatric population,” said Ms. Sanchez, a fourth-year medical student at Boston University. “It’s not very well defined or characterized.” The “unusually high number of pediatric patients with HS” at Boston Medical Center provided “a unique opportunity to study this topic.”
Working with her mentor, Lisa Shen, MD, associate medical director of pediatric dermatology at Boston University, Ms. Sanchez and colleagues retrospectively reviewed the medical records of 303 patients aged 4-18 years who were diagnosed with HS at Boston Medical Center from 2012 to 2021. Boston Medical Center is the largest safety net hospital in New England. All data points and outcome measures were collected within 6 months of the patient’s HS diagnosis date.
Of the 303 patients with HS, 84% were female and 16% were male. Complete information about race was available in 286 patients. Of these, 65% were Black/African American, 11% were White, and the rest were from other racial groups. The mean age at symptom onset was 13 years, while the mean age at diagnosis was 15 years, and the mean delay to diagnosis was 2 years. A family history of HS was reported in 36% of patients.
The most common clinical features in these HS patients were pain/tenderness (90%), pustules/papules (65%), discharge/drainage (62%), and deep-seated nodules (51%). Scarring was present in 44% of patients at the time of diagnosis. The three most common sites of involvement were the axillary area (79%), the pubic area (36%), and the inguinal folds/inner thighs (34%).
Obesity was the most common comorbidity at the time of diagnosis, with 64% of patients affected. The next most common comorbidities were acne vulgaris (36%), acanthosis nigricans (25%), depression (18%), being overweight (17%), polycystic ovary syndrome (16%) and anxiety (13%). None had type 1 diabetes or metabolic syndrome.
Referring to the large population of underserved minority patients at Boston Medical Center, Dr. Shen noted, “we have to make sure not to underestimate the prevalence of obesity in this population as they get older. We need to start from a younger age to incorporate multidisciplinary care such as weight management, nutrition, and working with our pediatric surgery colleagues in trying to tackle [HS] because there is data to suggest that the earlier we intervene, the better outcomes they have. That makes sense.”
Adam Friedman, MD, professor and chair of dermatology at George Washington University, Washington, who was asked to comment on the findings, said that the study “highlights the impressive and concerning gap and delays in diagnosis, not too dissimilar to what the literature shows in adult HS patients, which unfortunately has tremendous ramifications, both physically and emotionally/psychosocially.”
While this single-center study identified potential risk factors, such as obesity and self-identifying as Black, he said, “it is important to note that this condition does not discriminate and therefore it is important not to miss the cases that don’t follow the textbook nor stigmatize this condition as one that only impacts certain demographics.”
The researchers reported having no financial disclosures. Dr. Friedman, who was not involved with the study, reported that he serves as a consultant and/or advisor to numerous pharmaceutical companies. He is a speaker for companies including, Regeneron, Sanofi, AbbVie, Janssen, Incyte, and Brickell Biotech, and has received grants from Pfizer, the Dermatology Foundation, Almirall, Incyte, Galderma, and Janssen.
INDIANAPOLIS – in a study presented at the annual meeting of the Society for Pediatric Dermatology.
In addition, 44% presented with scarring, which suggests that HS may be underdiagnosed in this patient population. Those are the key findings from the study, a single-center retrospective chart review presented by Stephanie Sanchez during a poster session at the meeting.
“There is limited research on HS within the pediatric population,” said Ms. Sanchez, a fourth-year medical student at Boston University. “It’s not very well defined or characterized.” The “unusually high number of pediatric patients with HS” at Boston Medical Center provided “a unique opportunity to study this topic.”
Working with her mentor, Lisa Shen, MD, associate medical director of pediatric dermatology at Boston University, Ms. Sanchez and colleagues retrospectively reviewed the medical records of 303 patients aged 4-18 years who were diagnosed with HS at Boston Medical Center from 2012 to 2021. Boston Medical Center is the largest safety net hospital in New England. All data points and outcome measures were collected within 6 months of the patient’s HS diagnosis date.
Of the 303 patients with HS, 84% were female and 16% were male. Complete information about race was available in 286 patients. Of these, 65% were Black/African American, 11% were White, and the rest were from other racial groups. The mean age at symptom onset was 13 years, while the mean age at diagnosis was 15 years, and the mean delay to diagnosis was 2 years. A family history of HS was reported in 36% of patients.
The most common clinical features in these HS patients were pain/tenderness (90%), pustules/papules (65%), discharge/drainage (62%), and deep-seated nodules (51%). Scarring was present in 44% of patients at the time of diagnosis. The three most common sites of involvement were the axillary area (79%), the pubic area (36%), and the inguinal folds/inner thighs (34%).
Obesity was the most common comorbidity at the time of diagnosis, with 64% of patients affected. The next most common comorbidities were acne vulgaris (36%), acanthosis nigricans (25%), depression (18%), being overweight (17%), polycystic ovary syndrome (16%) and anxiety (13%). None had type 1 diabetes or metabolic syndrome.
Referring to the large population of underserved minority patients at Boston Medical Center, Dr. Shen noted, “we have to make sure not to underestimate the prevalence of obesity in this population as they get older. We need to start from a younger age to incorporate multidisciplinary care such as weight management, nutrition, and working with our pediatric surgery colleagues in trying to tackle [HS] because there is data to suggest that the earlier we intervene, the better outcomes they have. That makes sense.”
Adam Friedman, MD, professor and chair of dermatology at George Washington University, Washington, who was asked to comment on the findings, said that the study “highlights the impressive and concerning gap and delays in diagnosis, not too dissimilar to what the literature shows in adult HS patients, which unfortunately has tremendous ramifications, both physically and emotionally/psychosocially.”
While this single-center study identified potential risk factors, such as obesity and self-identifying as Black, he said, “it is important to note that this condition does not discriminate and therefore it is important not to miss the cases that don’t follow the textbook nor stigmatize this condition as one that only impacts certain demographics.”
The researchers reported having no financial disclosures. Dr. Friedman, who was not involved with the study, reported that he serves as a consultant and/or advisor to numerous pharmaceutical companies. He is a speaker for companies including, Regeneron, Sanofi, AbbVie, Janssen, Incyte, and Brickell Biotech, and has received grants from Pfizer, the Dermatology Foundation, Almirall, Incyte, Galderma, and Janssen.
AT SPD 2022
In the quest for a cure for type 1 diabetes, two companies merge
The $320 million cash purchase “will accelerate our goal of transforming, if not curing, type 1 diabetes by expanding our capabilities and bringing additional tools, technologies, and assets to our current stem cell-based programs,” said Vertex Chief Executive Officer and President Reshma Kewalramani, MD, in a company statement.
Last month, Vertex reported on a phase 1/2 multicenter clinical trial for two patients with type 1 diabetes who experienced improved blood glucose control with half doses of the company’s investigational allogeneic stem cell-derived islets (VX-880).
The first person to receive the product remained completely insulin-independent at 9 months post-transplant. A third patient has received the full targeted dose, but the data for this participant have yet to be reported.
For Viacyte’s part, last week the company announced that a clinical hold placed by the U.S. Food and Drug Administration on the trial has been lifted, allowing it to move forward with a planned total enrollment of 17 patients.
“The FDA requested additional information on the program, which we provided expeditiously. We are pleased that the hold has been lifted and look forward to continuing the Phase 1/2 trial in the U.S.,” a Vertex spokesperson told this news organization.
And a company official for ViaCyte presented results for three patients who received pancreatic precursor (PEC-01) cells derived from the company’s proprietary pluripotent stem cell line at the annual meeting of the Endocrine Society held in June. The cells are housed in an open delivery device implanted into a patient’s forearm. All three participants experienced improved blood glucose levels.
That presentation followed ViaCyte’s announcement in February that the first patient with type 1 diabetes had been dosed in a Phase 1 clinical trial of its investigational allogeneic, gene-edited, stem cell-derived product, VCTX210, developed in collaboration with CRISPR Therapeutics’ gene-editing technology. The aim is to generate islet cells that will produce insulin while avoiding recognition by the immune system, thus rendering immunosuppressive drugs unnecessary.
According to Vertex’s announcement, “The acquisition of ViaCyte provides Vertex with complementary assets, capabilities, and technologies, including additional human stem cell lines, intellectual property around stem cell differentiation, and Good Manufacturing Practice ... facilities for cell-based therapies that could accelerate Vertex’s ongoing type 1 diabetes programs. The acquisition also provides access to novel hypoimmune stem cell assets via the ViaCyte collaboration with CRISPR Therapeutics.”
In response to the announcement, the type 1 diabetes advocacy organization JDRF, which has funded the work of both companies, said in a statement that the acquisition “represents a significant stride in cures research for the type 1 diabetes community.”
“The coming together of two leaders in the cell-derived therapies field will undoubtedly accelerate the development of VX-880 by combining their resources, technologies, intellectual property, and more,” it added.
A third company developing stem cell–derived islet cell therapies, Sernova, said in a statement provided to this news organization: “We are very confident that bringing important game-changing technologies together, as we are seeing across the industry, will result in several viable technologies for the millions of people with type 1 diabetes ... We are thrilled that there are several technologies under development using different approaches that have the potential to provide a ‘functional cure’ for this disease.”
Vertex anticipates the acquisition will close later in 2022.
A version of this article first appeared on Medscape.com.
The $320 million cash purchase “will accelerate our goal of transforming, if not curing, type 1 diabetes by expanding our capabilities and bringing additional tools, technologies, and assets to our current stem cell-based programs,” said Vertex Chief Executive Officer and President Reshma Kewalramani, MD, in a company statement.
Last month, Vertex reported on a phase 1/2 multicenter clinical trial for two patients with type 1 diabetes who experienced improved blood glucose control with half doses of the company’s investigational allogeneic stem cell-derived islets (VX-880).
The first person to receive the product remained completely insulin-independent at 9 months post-transplant. A third patient has received the full targeted dose, but the data for this participant have yet to be reported.
For Viacyte’s part, last week the company announced that a clinical hold placed by the U.S. Food and Drug Administration on the trial has been lifted, allowing it to move forward with a planned total enrollment of 17 patients.
“The FDA requested additional information on the program, which we provided expeditiously. We are pleased that the hold has been lifted and look forward to continuing the Phase 1/2 trial in the U.S.,” a Vertex spokesperson told this news organization.
And a company official for ViaCyte presented results for three patients who received pancreatic precursor (PEC-01) cells derived from the company’s proprietary pluripotent stem cell line at the annual meeting of the Endocrine Society held in June. The cells are housed in an open delivery device implanted into a patient’s forearm. All three participants experienced improved blood glucose levels.
That presentation followed ViaCyte’s announcement in February that the first patient with type 1 diabetes had been dosed in a Phase 1 clinical trial of its investigational allogeneic, gene-edited, stem cell-derived product, VCTX210, developed in collaboration with CRISPR Therapeutics’ gene-editing technology. The aim is to generate islet cells that will produce insulin while avoiding recognition by the immune system, thus rendering immunosuppressive drugs unnecessary.
According to Vertex’s announcement, “The acquisition of ViaCyte provides Vertex with complementary assets, capabilities, and technologies, including additional human stem cell lines, intellectual property around stem cell differentiation, and Good Manufacturing Practice ... facilities for cell-based therapies that could accelerate Vertex’s ongoing type 1 diabetes programs. The acquisition also provides access to novel hypoimmune stem cell assets via the ViaCyte collaboration with CRISPR Therapeutics.”
In response to the announcement, the type 1 diabetes advocacy organization JDRF, which has funded the work of both companies, said in a statement that the acquisition “represents a significant stride in cures research for the type 1 diabetes community.”
“The coming together of two leaders in the cell-derived therapies field will undoubtedly accelerate the development of VX-880 by combining their resources, technologies, intellectual property, and more,” it added.
A third company developing stem cell–derived islet cell therapies, Sernova, said in a statement provided to this news organization: “We are very confident that bringing important game-changing technologies together, as we are seeing across the industry, will result in several viable technologies for the millions of people with type 1 diabetes ... We are thrilled that there are several technologies under development using different approaches that have the potential to provide a ‘functional cure’ for this disease.”
Vertex anticipates the acquisition will close later in 2022.
A version of this article first appeared on Medscape.com.
The $320 million cash purchase “will accelerate our goal of transforming, if not curing, type 1 diabetes by expanding our capabilities and bringing additional tools, technologies, and assets to our current stem cell-based programs,” said Vertex Chief Executive Officer and President Reshma Kewalramani, MD, in a company statement.
Last month, Vertex reported on a phase 1/2 multicenter clinical trial for two patients with type 1 diabetes who experienced improved blood glucose control with half doses of the company’s investigational allogeneic stem cell-derived islets (VX-880).
The first person to receive the product remained completely insulin-independent at 9 months post-transplant. A third patient has received the full targeted dose, but the data for this participant have yet to be reported.
For Viacyte’s part, last week the company announced that a clinical hold placed by the U.S. Food and Drug Administration on the trial has been lifted, allowing it to move forward with a planned total enrollment of 17 patients.
“The FDA requested additional information on the program, which we provided expeditiously. We are pleased that the hold has been lifted and look forward to continuing the Phase 1/2 trial in the U.S.,” a Vertex spokesperson told this news organization.
And a company official for ViaCyte presented results for three patients who received pancreatic precursor (PEC-01) cells derived from the company’s proprietary pluripotent stem cell line at the annual meeting of the Endocrine Society held in June. The cells are housed in an open delivery device implanted into a patient’s forearm. All three participants experienced improved blood glucose levels.
That presentation followed ViaCyte’s announcement in February that the first patient with type 1 diabetes had been dosed in a Phase 1 clinical trial of its investigational allogeneic, gene-edited, stem cell-derived product, VCTX210, developed in collaboration with CRISPR Therapeutics’ gene-editing technology. The aim is to generate islet cells that will produce insulin while avoiding recognition by the immune system, thus rendering immunosuppressive drugs unnecessary.
According to Vertex’s announcement, “The acquisition of ViaCyte provides Vertex with complementary assets, capabilities, and technologies, including additional human stem cell lines, intellectual property around stem cell differentiation, and Good Manufacturing Practice ... facilities for cell-based therapies that could accelerate Vertex’s ongoing type 1 diabetes programs. The acquisition also provides access to novel hypoimmune stem cell assets via the ViaCyte collaboration with CRISPR Therapeutics.”
In response to the announcement, the type 1 diabetes advocacy organization JDRF, which has funded the work of both companies, said in a statement that the acquisition “represents a significant stride in cures research for the type 1 diabetes community.”
“The coming together of two leaders in the cell-derived therapies field will undoubtedly accelerate the development of VX-880 by combining their resources, technologies, intellectual property, and more,” it added.
A third company developing stem cell–derived islet cell therapies, Sernova, said in a statement provided to this news organization: “We are very confident that bringing important game-changing technologies together, as we are seeing across the industry, will result in several viable technologies for the millions of people with type 1 diabetes ... We are thrilled that there are several technologies under development using different approaches that have the potential to provide a ‘functional cure’ for this disease.”
Vertex anticipates the acquisition will close later in 2022.
A version of this article first appeared on Medscape.com.