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Eczema causes substantial burden for many infants and preschoolers
INDIANAPOLIS – . Those are key findings from a large international web-based survey that was presented during a poster session at the annual meeting of the Society for Pediatric Dermatology.
“Improved knowledge of the AD-related burden may help reinforce the medical need in the pediatric population and contribute to better and earlier adequate management of the disease,” authors led by Stephan Weidinger , MD, PhD, vice head of the department of dermatology at University Hospital Schleswig-Holstein, Kiel, Germany, wrote in the abstract.
For the study, Dr. Weidinger and colleagues evaluated 1,486 infants and preschoolers with AD aged 6 months to under 6 years, who participated in the Epidemiology of Children with Atopic Dermatitis Reporting on their Experience (EPI-CARE), an international, cross-sectional, web-based survey of children and adolescents. The study population resided in 18 countries from five regions of the world, including North America, Latin America, Europe, Middle East/Eurasia, and East Asia. Parents or guardians answered all questions for infants/preschoolers younger than 4 years of age, while preschoolers aged 4 to younger than 6 years were asked to answer questions related to the impact of AD on their health-related quality of life.
AD severity was assessed using Patient Global Assessment (PtGA), where parents or guardians described their child’s eczema severity over the last week as mild, moderate, or severe. The researchers stratified outcomes by geographic region and AD severity, which included the following atopic comorbidities: worst itch, worst skin pain, and overall sleep disturbance in the past 24 hours as measured by the 0-10 numeric rating scale, where higher scores indicate worse severity; eczema-related hospitalization in the past 12 months; and frequency and average duration of flares over the past month.
The mean age of the study participants was 3 years and 61.6% had mild disease. The most common atopic comorbidities were hay fever, asthma, and seasonal allergies, and the incidence of atopic comorbidities increased with increasing AD severity. One or more atopic comorbidities was reported in 88.3% of patients with mild AD, compared with 92.1% of those with moderate disease and 95.8% of those with severe disease. In addition, infants and preschoolers with moderate or severe AD had worse itch, skin pain, and sleep disturbances over the past 24 hours, compared with those who had mild AD.
More than half of infants and preschoolers with severe AD (54.1%) were reported to have been hospitalized in the past 12 months (this ranged from 30.2% to 71.3% across regions), as did 35% of patients with moderate AD and 32.1% of those with mild AD. In addition, 50.6% of infants and preschoolers with severe AD had more than two flares in the past month, compared with 18.1% of those with moderate AD and 6.3% of those with mild disease.
In other findings, 50.7% of infants and preschoolers with severe AD had flares than lasted an average of 2 or more weeks, compared with 20.8% of those with moderate disease and 10% of those with mild disease. Also, 78.3% of preschoolers aged 4 to less than 6 years had missed one or more days of school in the previous 4 weeks: a mean of 5.1 days among those with mild AD, a mean of 7.3 days among those with moderate AD, and a mean of 12.1 days among those with severe disease.
Raj J. Chovatiya MD, PhD, of the department of dermatology at Northwestern University, Chicago, who was asked to comment on the study, said that infants and preschoolers remain an understudied group despite the high prevalence of AD in this age range. “The results of this study demonstrate a substantial burden of disease in this population, particularly among those with more severe disease,” said Dr. Chovatiya, who also directs the university’s Center for Eczema and Itch. “This includes longer and more frequent AD flares as well as high rates of inpatient hospitalization. These findings suggest that additional research is needed to better characterize disease burden and optimize outcomes for young children with AD.”
The study was funded by Regeneron Pharmaceuticals and Sanofi. Dr. Weidinger and other coauthors reported having received institutional research grants and consulting fees from many pharmaceutical companies that manufacture drugs used for the treatment of psoriasis and eczema.
Dr. Chovatiya disclosed that he has served as an advisory board member, consultant, speaker, and/or investigator for AbbVie, Arcutis, Arena, Beiersdorf, Bristol Myers Squibb, Dermavant, Eli Lilly, EPI Health, Incyte, L’Oréal, the National Eczema Association, Pfizer, Regeneron, Sanofi, and UCB.
INDIANAPOLIS – . Those are key findings from a large international web-based survey that was presented during a poster session at the annual meeting of the Society for Pediatric Dermatology.
“Improved knowledge of the AD-related burden may help reinforce the medical need in the pediatric population and contribute to better and earlier adequate management of the disease,” authors led by Stephan Weidinger , MD, PhD, vice head of the department of dermatology at University Hospital Schleswig-Holstein, Kiel, Germany, wrote in the abstract.
For the study, Dr. Weidinger and colleagues evaluated 1,486 infants and preschoolers with AD aged 6 months to under 6 years, who participated in the Epidemiology of Children with Atopic Dermatitis Reporting on their Experience (EPI-CARE), an international, cross-sectional, web-based survey of children and adolescents. The study population resided in 18 countries from five regions of the world, including North America, Latin America, Europe, Middle East/Eurasia, and East Asia. Parents or guardians answered all questions for infants/preschoolers younger than 4 years of age, while preschoolers aged 4 to younger than 6 years were asked to answer questions related to the impact of AD on their health-related quality of life.
AD severity was assessed using Patient Global Assessment (PtGA), where parents or guardians described their child’s eczema severity over the last week as mild, moderate, or severe. The researchers stratified outcomes by geographic region and AD severity, which included the following atopic comorbidities: worst itch, worst skin pain, and overall sleep disturbance in the past 24 hours as measured by the 0-10 numeric rating scale, where higher scores indicate worse severity; eczema-related hospitalization in the past 12 months; and frequency and average duration of flares over the past month.
The mean age of the study participants was 3 years and 61.6% had mild disease. The most common atopic comorbidities were hay fever, asthma, and seasonal allergies, and the incidence of atopic comorbidities increased with increasing AD severity. One or more atopic comorbidities was reported in 88.3% of patients with mild AD, compared with 92.1% of those with moderate disease and 95.8% of those with severe disease. In addition, infants and preschoolers with moderate or severe AD had worse itch, skin pain, and sleep disturbances over the past 24 hours, compared with those who had mild AD.
More than half of infants and preschoolers with severe AD (54.1%) were reported to have been hospitalized in the past 12 months (this ranged from 30.2% to 71.3% across regions), as did 35% of patients with moderate AD and 32.1% of those with mild AD. In addition, 50.6% of infants and preschoolers with severe AD had more than two flares in the past month, compared with 18.1% of those with moderate AD and 6.3% of those with mild disease.
In other findings, 50.7% of infants and preschoolers with severe AD had flares than lasted an average of 2 or more weeks, compared with 20.8% of those with moderate disease and 10% of those with mild disease. Also, 78.3% of preschoolers aged 4 to less than 6 years had missed one or more days of school in the previous 4 weeks: a mean of 5.1 days among those with mild AD, a mean of 7.3 days among those with moderate AD, and a mean of 12.1 days among those with severe disease.
Raj J. Chovatiya MD, PhD, of the department of dermatology at Northwestern University, Chicago, who was asked to comment on the study, said that infants and preschoolers remain an understudied group despite the high prevalence of AD in this age range. “The results of this study demonstrate a substantial burden of disease in this population, particularly among those with more severe disease,” said Dr. Chovatiya, who also directs the university’s Center for Eczema and Itch. “This includes longer and more frequent AD flares as well as high rates of inpatient hospitalization. These findings suggest that additional research is needed to better characterize disease burden and optimize outcomes for young children with AD.”
The study was funded by Regeneron Pharmaceuticals and Sanofi. Dr. Weidinger and other coauthors reported having received institutional research grants and consulting fees from many pharmaceutical companies that manufacture drugs used for the treatment of psoriasis and eczema.
Dr. Chovatiya disclosed that he has served as an advisory board member, consultant, speaker, and/or investigator for AbbVie, Arcutis, Arena, Beiersdorf, Bristol Myers Squibb, Dermavant, Eli Lilly, EPI Health, Incyte, L’Oréal, the National Eczema Association, Pfizer, Regeneron, Sanofi, and UCB.
INDIANAPOLIS – . Those are key findings from a large international web-based survey that was presented during a poster session at the annual meeting of the Society for Pediatric Dermatology.
“Improved knowledge of the AD-related burden may help reinforce the medical need in the pediatric population and contribute to better and earlier adequate management of the disease,” authors led by Stephan Weidinger , MD, PhD, vice head of the department of dermatology at University Hospital Schleswig-Holstein, Kiel, Germany, wrote in the abstract.
For the study, Dr. Weidinger and colleagues evaluated 1,486 infants and preschoolers with AD aged 6 months to under 6 years, who participated in the Epidemiology of Children with Atopic Dermatitis Reporting on their Experience (EPI-CARE), an international, cross-sectional, web-based survey of children and adolescents. The study population resided in 18 countries from five regions of the world, including North America, Latin America, Europe, Middle East/Eurasia, and East Asia. Parents or guardians answered all questions for infants/preschoolers younger than 4 years of age, while preschoolers aged 4 to younger than 6 years were asked to answer questions related to the impact of AD on their health-related quality of life.
AD severity was assessed using Patient Global Assessment (PtGA), where parents or guardians described their child’s eczema severity over the last week as mild, moderate, or severe. The researchers stratified outcomes by geographic region and AD severity, which included the following atopic comorbidities: worst itch, worst skin pain, and overall sleep disturbance in the past 24 hours as measured by the 0-10 numeric rating scale, where higher scores indicate worse severity; eczema-related hospitalization in the past 12 months; and frequency and average duration of flares over the past month.
The mean age of the study participants was 3 years and 61.6% had mild disease. The most common atopic comorbidities were hay fever, asthma, and seasonal allergies, and the incidence of atopic comorbidities increased with increasing AD severity. One or more atopic comorbidities was reported in 88.3% of patients with mild AD, compared with 92.1% of those with moderate disease and 95.8% of those with severe disease. In addition, infants and preschoolers with moderate or severe AD had worse itch, skin pain, and sleep disturbances over the past 24 hours, compared with those who had mild AD.
More than half of infants and preschoolers with severe AD (54.1%) were reported to have been hospitalized in the past 12 months (this ranged from 30.2% to 71.3% across regions), as did 35% of patients with moderate AD and 32.1% of those with mild AD. In addition, 50.6% of infants and preschoolers with severe AD had more than two flares in the past month, compared with 18.1% of those with moderate AD and 6.3% of those with mild disease.
In other findings, 50.7% of infants and preschoolers with severe AD had flares than lasted an average of 2 or more weeks, compared with 20.8% of those with moderate disease and 10% of those with mild disease. Also, 78.3% of preschoolers aged 4 to less than 6 years had missed one or more days of school in the previous 4 weeks: a mean of 5.1 days among those with mild AD, a mean of 7.3 days among those with moderate AD, and a mean of 12.1 days among those with severe disease.
Raj J. Chovatiya MD, PhD, of the department of dermatology at Northwestern University, Chicago, who was asked to comment on the study, said that infants and preschoolers remain an understudied group despite the high prevalence of AD in this age range. “The results of this study demonstrate a substantial burden of disease in this population, particularly among those with more severe disease,” said Dr. Chovatiya, who also directs the university’s Center for Eczema and Itch. “This includes longer and more frequent AD flares as well as high rates of inpatient hospitalization. These findings suggest that additional research is needed to better characterize disease burden and optimize outcomes for young children with AD.”
The study was funded by Regeneron Pharmaceuticals and Sanofi. Dr. Weidinger and other coauthors reported having received institutional research grants and consulting fees from many pharmaceutical companies that manufacture drugs used for the treatment of psoriasis and eczema.
Dr. Chovatiya disclosed that he has served as an advisory board member, consultant, speaker, and/or investigator for AbbVie, Arcutis, Arena, Beiersdorf, Bristol Myers Squibb, Dermavant, Eli Lilly, EPI Health, Incyte, L’Oréal, the National Eczema Association, Pfizer, Regeneron, Sanofi, and UCB.
AT SPD 2022
Study explores gender differences in pediatric melanoma
INDIANAPOLIS – .
In addition, male gender was independently associated with increased mortality, but age was not.
Those are key findings from a retrospective cohort analysis of nearly 5,000 records from the National Cancer Database.
“There are multiple studies from primarily adult populations showing females with melanoma have a different presentation and better outcomes than males,” co-first author Rebecca M. Thiede, MD, a dermatologist at the University of Arizona, Tucson, said in an interview with this news organization in advance of the annual meeting of the Society for Pediatric Dermatology, where the abstract was presented during a poster session. “However, because melanoma is so rare in younger patients, little is known about gender differences in presentation and survival in pediatric and adolescent patients. To our knowledge, this is one of the largest studies to date in this population, and the first to explore gender differences in detail in pediatric and adolescent patients with melanoma.”
Working with co-first author Sabrina Dahak, a fourth-year medical student at the University of Arizona, Phoenix, Dr. Thiede and colleagues retrospectively analyzed the National Cancer Database to identify biopsy-confirmed invasive primary cutaneous melanoma cases diagnosed in patients 0-21 years of age between 2004 and 2018. The search yielded 4,645 cases, and the researchers used American Academy of Pediatrics definitions to categorize the patients by age, from infancy (birth to 2 years), to childhood (3-10 years), early adolescence (11-14 years), middle adolescence (15-17 years), and late adolescence (18-21 years). They used the Kaplan Meier analysis to determine overall survival and multivariate Cox regression to determine independent survival predictors.
Of the 4,645 pediatric melanoma cases, 63.4% were in females and 36.6% were in males, a difference that was significant (P < .001). Dr. Thiede and colleagues also observed a significant relationship between primary site and gender (P < .001). Primary sites included the trunk (34.3% of females vs. 32.9% of males, respectively), head and neck (16.4% vs. 30.9%), upper extremities (19.5% vs. 16%), lower extremities (27.9% vs. 16.5%), and “unspecified” (1.9% vs. 3.7%).
Females had higher rates of superficial spreading melanoma while males were affected by nodular melanoma more often. For example, the median Breslow depth was higher for males (1.05 mm; interquartile range [IQR] 0.50-2.31) than for females (0.80 mm; IQR, 0.40-1.67; P < .001).
Although females accounted for a higher percentage of cases than males overall, from birth to 17 years, a higher percentage of males than females were found to have later stage of melanoma at time of diagnosis: Females were more likely to be diagnosed with stage I disease (67.8%) than were males (53.6%), and males were more likely than were females to be diagnosed with stages II (15.9% vs. 12.3%), III (27.1% vs. 18.3%), and IV disease (3.3% vs. 1.6%; P < .001 for all).
In other findings, the 5- and 10-year overall survival rates were higher for females (95.9% and 93.9%, respectively) than for males (92.0% vs. 86.7%, respectively; P < .001). However, by age group, overall survival rates were similar between females and males among infants, children, and those in early adolescence – but not for those in middle adolescence (96.7% vs. 91.9%; P < .001) or late adolescence (95.7% vs. 90.4%; P < .001).
When the researchers adjusted for confounding variables, male gender was independently associated with an increased risk of death (adjusted hazard ratio 1.37; P < .001), but age was not.
“It was particularly surprising to see that even at such a young age, there is a significant difference in overall survival between males and females, where females have better outcomes than males,” Dr. Thiede said. “When examining pediatric and adolescent patients, it is essential to maintain cutaneous melanoma on the differential,” she advised. “It is important for clinicians to perform a thorough exam at annual visits particularly for those at high risk for melanoma to catch this rare but potentially devastating diagnosis.”
She acknowledged certain limitations of the study, including its reliance on one database, “as comparing multiple databases would strengthen the conclusions,” she said. “There was some missing data present in our dataset, and a large percentage of the histologic subtypes were unspecified, both of which are common issues with cancer registries. An additional limitation is related to the low death rates in adolescent and pediatric patients, which may impact the analysis related to survival and independent predictors of survival.”
Asked to comment on the study results, Carrie C. Coughlin, MD, who directs the section of pediatric dermatology Washington University/St. Louis Children’s Hospital, said that the finding that males were more likely to present with stage II or higher disease compared with females “could be related to their finding that females had more superficial spreading melanomas, whereas males had more nodular melanoma.” Those differences “could influence how providers evaluate melanocytic lesions in children,” she added.
Dr. Coughlin, who directs the pediatric dermatology fellowship at Washington University/St. Louis Children’s Hospital, said it was “interesting” that the authors found no association between older age and an increased risk of death. “It would be helpful to have more data about melanoma subtype, including information about Spitz or Spitzoid melanomas,” she said. “Also, knowing the distribution of melanoma across the age categories could provide more insight into their data.”
Ms. Dahak received an award from the National Cancer Institute to fund travel for presentation of this study at the SPD meeting. No other financial conflicts were reported by the researchers. Dr. Coughlin is on the board of the Pediatric Dermatology Research Alliance (PeDRA) and the International Immunosuppression and Transplant Skin Cancer Collaborative.
INDIANAPOLIS – .
In addition, male gender was independently associated with increased mortality, but age was not.
Those are key findings from a retrospective cohort analysis of nearly 5,000 records from the National Cancer Database.
“There are multiple studies from primarily adult populations showing females with melanoma have a different presentation and better outcomes than males,” co-first author Rebecca M. Thiede, MD, a dermatologist at the University of Arizona, Tucson, said in an interview with this news organization in advance of the annual meeting of the Society for Pediatric Dermatology, where the abstract was presented during a poster session. “However, because melanoma is so rare in younger patients, little is known about gender differences in presentation and survival in pediatric and adolescent patients. To our knowledge, this is one of the largest studies to date in this population, and the first to explore gender differences in detail in pediatric and adolescent patients with melanoma.”
Working with co-first author Sabrina Dahak, a fourth-year medical student at the University of Arizona, Phoenix, Dr. Thiede and colleagues retrospectively analyzed the National Cancer Database to identify biopsy-confirmed invasive primary cutaneous melanoma cases diagnosed in patients 0-21 years of age between 2004 and 2018. The search yielded 4,645 cases, and the researchers used American Academy of Pediatrics definitions to categorize the patients by age, from infancy (birth to 2 years), to childhood (3-10 years), early adolescence (11-14 years), middle adolescence (15-17 years), and late adolescence (18-21 years). They used the Kaplan Meier analysis to determine overall survival and multivariate Cox regression to determine independent survival predictors.
Of the 4,645 pediatric melanoma cases, 63.4% were in females and 36.6% were in males, a difference that was significant (P < .001). Dr. Thiede and colleagues also observed a significant relationship between primary site and gender (P < .001). Primary sites included the trunk (34.3% of females vs. 32.9% of males, respectively), head and neck (16.4% vs. 30.9%), upper extremities (19.5% vs. 16%), lower extremities (27.9% vs. 16.5%), and “unspecified” (1.9% vs. 3.7%).
Females had higher rates of superficial spreading melanoma while males were affected by nodular melanoma more often. For example, the median Breslow depth was higher for males (1.05 mm; interquartile range [IQR] 0.50-2.31) than for females (0.80 mm; IQR, 0.40-1.67; P < .001).
Although females accounted for a higher percentage of cases than males overall, from birth to 17 years, a higher percentage of males than females were found to have later stage of melanoma at time of diagnosis: Females were more likely to be diagnosed with stage I disease (67.8%) than were males (53.6%), and males were more likely than were females to be diagnosed with stages II (15.9% vs. 12.3%), III (27.1% vs. 18.3%), and IV disease (3.3% vs. 1.6%; P < .001 for all).
In other findings, the 5- and 10-year overall survival rates were higher for females (95.9% and 93.9%, respectively) than for males (92.0% vs. 86.7%, respectively; P < .001). However, by age group, overall survival rates were similar between females and males among infants, children, and those in early adolescence – but not for those in middle adolescence (96.7% vs. 91.9%; P < .001) or late adolescence (95.7% vs. 90.4%; P < .001).
When the researchers adjusted for confounding variables, male gender was independently associated with an increased risk of death (adjusted hazard ratio 1.37; P < .001), but age was not.
“It was particularly surprising to see that even at such a young age, there is a significant difference in overall survival between males and females, where females have better outcomes than males,” Dr. Thiede said. “When examining pediatric and adolescent patients, it is essential to maintain cutaneous melanoma on the differential,” she advised. “It is important for clinicians to perform a thorough exam at annual visits particularly for those at high risk for melanoma to catch this rare but potentially devastating diagnosis.”
She acknowledged certain limitations of the study, including its reliance on one database, “as comparing multiple databases would strengthen the conclusions,” she said. “There was some missing data present in our dataset, and a large percentage of the histologic subtypes were unspecified, both of which are common issues with cancer registries. An additional limitation is related to the low death rates in adolescent and pediatric patients, which may impact the analysis related to survival and independent predictors of survival.”
Asked to comment on the study results, Carrie C. Coughlin, MD, who directs the section of pediatric dermatology Washington University/St. Louis Children’s Hospital, said that the finding that males were more likely to present with stage II or higher disease compared with females “could be related to their finding that females had more superficial spreading melanomas, whereas males had more nodular melanoma.” Those differences “could influence how providers evaluate melanocytic lesions in children,” she added.
Dr. Coughlin, who directs the pediatric dermatology fellowship at Washington University/St. Louis Children’s Hospital, said it was “interesting” that the authors found no association between older age and an increased risk of death. “It would be helpful to have more data about melanoma subtype, including information about Spitz or Spitzoid melanomas,” she said. “Also, knowing the distribution of melanoma across the age categories could provide more insight into their data.”
Ms. Dahak received an award from the National Cancer Institute to fund travel for presentation of this study at the SPD meeting. No other financial conflicts were reported by the researchers. Dr. Coughlin is on the board of the Pediatric Dermatology Research Alliance (PeDRA) and the International Immunosuppression and Transplant Skin Cancer Collaborative.
INDIANAPOLIS – .
In addition, male gender was independently associated with increased mortality, but age was not.
Those are key findings from a retrospective cohort analysis of nearly 5,000 records from the National Cancer Database.
“There are multiple studies from primarily adult populations showing females with melanoma have a different presentation and better outcomes than males,” co-first author Rebecca M. Thiede, MD, a dermatologist at the University of Arizona, Tucson, said in an interview with this news organization in advance of the annual meeting of the Society for Pediatric Dermatology, where the abstract was presented during a poster session. “However, because melanoma is so rare in younger patients, little is known about gender differences in presentation and survival in pediatric and adolescent patients. To our knowledge, this is one of the largest studies to date in this population, and the first to explore gender differences in detail in pediatric and adolescent patients with melanoma.”
Working with co-first author Sabrina Dahak, a fourth-year medical student at the University of Arizona, Phoenix, Dr. Thiede and colleagues retrospectively analyzed the National Cancer Database to identify biopsy-confirmed invasive primary cutaneous melanoma cases diagnosed in patients 0-21 years of age between 2004 and 2018. The search yielded 4,645 cases, and the researchers used American Academy of Pediatrics definitions to categorize the patients by age, from infancy (birth to 2 years), to childhood (3-10 years), early adolescence (11-14 years), middle adolescence (15-17 years), and late adolescence (18-21 years). They used the Kaplan Meier analysis to determine overall survival and multivariate Cox regression to determine independent survival predictors.
Of the 4,645 pediatric melanoma cases, 63.4% were in females and 36.6% were in males, a difference that was significant (P < .001). Dr. Thiede and colleagues also observed a significant relationship between primary site and gender (P < .001). Primary sites included the trunk (34.3% of females vs. 32.9% of males, respectively), head and neck (16.4% vs. 30.9%), upper extremities (19.5% vs. 16%), lower extremities (27.9% vs. 16.5%), and “unspecified” (1.9% vs. 3.7%).
Females had higher rates of superficial spreading melanoma while males were affected by nodular melanoma more often. For example, the median Breslow depth was higher for males (1.05 mm; interquartile range [IQR] 0.50-2.31) than for females (0.80 mm; IQR, 0.40-1.67; P < .001).
Although females accounted for a higher percentage of cases than males overall, from birth to 17 years, a higher percentage of males than females were found to have later stage of melanoma at time of diagnosis: Females were more likely to be diagnosed with stage I disease (67.8%) than were males (53.6%), and males were more likely than were females to be diagnosed with stages II (15.9% vs. 12.3%), III (27.1% vs. 18.3%), and IV disease (3.3% vs. 1.6%; P < .001 for all).
In other findings, the 5- and 10-year overall survival rates were higher for females (95.9% and 93.9%, respectively) than for males (92.0% vs. 86.7%, respectively; P < .001). However, by age group, overall survival rates were similar between females and males among infants, children, and those in early adolescence – but not for those in middle adolescence (96.7% vs. 91.9%; P < .001) or late adolescence (95.7% vs. 90.4%; P < .001).
When the researchers adjusted for confounding variables, male gender was independently associated with an increased risk of death (adjusted hazard ratio 1.37; P < .001), but age was not.
“It was particularly surprising to see that even at such a young age, there is a significant difference in overall survival between males and females, where females have better outcomes than males,” Dr. Thiede said. “When examining pediatric and adolescent patients, it is essential to maintain cutaneous melanoma on the differential,” she advised. “It is important for clinicians to perform a thorough exam at annual visits particularly for those at high risk for melanoma to catch this rare but potentially devastating diagnosis.”
She acknowledged certain limitations of the study, including its reliance on one database, “as comparing multiple databases would strengthen the conclusions,” she said. “There was some missing data present in our dataset, and a large percentage of the histologic subtypes were unspecified, both of which are common issues with cancer registries. An additional limitation is related to the low death rates in adolescent and pediatric patients, which may impact the analysis related to survival and independent predictors of survival.”
Asked to comment on the study results, Carrie C. Coughlin, MD, who directs the section of pediatric dermatology Washington University/St. Louis Children’s Hospital, said that the finding that males were more likely to present with stage II or higher disease compared with females “could be related to their finding that females had more superficial spreading melanomas, whereas males had more nodular melanoma.” Those differences “could influence how providers evaluate melanocytic lesions in children,” she added.
Dr. Coughlin, who directs the pediatric dermatology fellowship at Washington University/St. Louis Children’s Hospital, said it was “interesting” that the authors found no association between older age and an increased risk of death. “It would be helpful to have more data about melanoma subtype, including information about Spitz or Spitzoid melanomas,” she said. “Also, knowing the distribution of melanoma across the age categories could provide more insight into their data.”
Ms. Dahak received an award from the National Cancer Institute to fund travel for presentation of this study at the SPD meeting. No other financial conflicts were reported by the researchers. Dr. Coughlin is on the board of the Pediatric Dermatology Research Alliance (PeDRA) and the International Immunosuppression and Transplant Skin Cancer Collaborative.
AT SPD 2022
Ruxolitinib found to benefit adolescents with vitiligo up to one year
INDIANAPOLIS – and a higher proportion responded at week 52, results from a pooled analysis of phase 3 data showed.
Currently, there is no treatment approved by the Food and Drug Administration to repigment patients with vitiligo, but the cream formulation of the Janus kinase inhibitor ruxolitinib was shown to be effective and have a favorable safety profile in patients aged 12 years and up in the phase 3 clinical trials, TRuE-V1 and TruE-V2. “We know that about half of patients will develop vitiligo by the age of 20, so there is a significant need to have treatments available for the pediatric population,” lead study author David Rosmarin, MD, told this news organization in advance of the annual meeting of the Society for Pediatric Dermatology.
In September 2021, topical ruxolitinib (Opzelura) was approved by the FDA for treating atopic dermatitis in nonimmunocompromised patients aged 12 years and older. The manufacturer, Incyte, has submitted an application for approval to the agency for treating vitiligo in patients ages 12 years and older based on 24-week results; the FDA is expected to make a decision by July 18.
For the current study, presented during a poster session at the meeting, Dr. Rosmarin, of the department of dermatology at Tufts Medical Center, Boston, and colleagues pooled efficacy and safety data for adolescent patients aged 12-17 years from the TRuE-V studies, which enrolled patients 12 years of age and older diagnosed with nonsegmental vitiligo with depigmentation covering up to 10% of total body surface area (BSA), including facial and total Vitiligo Area Scoring Index (F-VASI/T-VASI) scores of ≥ 0.5/≥ 3. Investigators randomized patients 2:1 to twice-daily 1.5% ruxolitinib cream or vehicle for 24 weeks, after which all patients could apply 1.5% ruxolitinib cream through week 52. Efficacy endpoints included the proportions of patients who achieved at least 75%, 50%, and 90% improvement from baseline in F-VASI scores (F-VASI75, F-VASI50, F-VASI90); the proportion of patients who achieved at least a 50% improvement from baseline in T-VASI (T-VASI50); the proportion of patients who achieved a Vitiligo Noticeability Scale (VNS) rating of 4 or 5; and percentage change from baseline in facial BSA (F-BSA). Safety and tolerability were also assessed.
For the pooled analysis, Dr. Rosmarin and colleagues reported results on 72 adolescents: 55 who received ruxolitinib cream and 17 who received vehicle. At week 24, 32.1% of adolescents treated with ruxolitinib cream achieved F-VASI75, compared with none of those in the vehicle group. Further, response rates at week 52 for patients who applied ruxolitinib cream from day 1 were as follows: F-VASI75, 48.0%; F-VASI50, 70.0%; F-VASI90, 24.0%; T-VASI50, 60.0%; VNS score of 4/5, 56.0%; and F-BSA mean percentage change from baseline, –41.9%.
Efficacy at week 52 among crossover patients (after 28 weeks of ruxolitinib cream) was consistent with week 24 data in patients who applied ruxolitinib cream from day 1.
“As we know that repigmentation takes time, about half of the patients achieved the F-VASI75 at the 52-week endpoint,” said Dr. Rosmarin, who is also vice-chair for research and education at Tufts Medical Center, Boston. “Particularly remarkable is that 60% of adolescents achieved a T-VASI50 [50% or more repigmentation of the whole body at the year mark] and over half the patients described their vitiligo as a lot less noticeable or no longer noticeable at the year mark.”
In terms of safety, treatment-related adverse events occurred in 12.9% of patients treated with ruxolitinib (no information was available on the specific events). Serious adverse events occurred in 1.4% of patients; none were considered related to treatment.
“Overall, these results are quite impressive,” Dr. Rosmarin said. “While it can be very challenging to repigment patients with vitiligo, ruxolitinib cream provides an effective option which can help many of my patients.” He acknowledged certain limitations of the analysis, including the fact that the TRuE-V studies were conducted during the COVID-19 pandemic, “which may have contributed to patients being lost to follow-up. Also, the majority of the patients had skin phototypes 1-3.”
Carrie C. Coughlin, MD, who was asked to comment on the study, said that patients with vitiligo need treatment options that are well-studied and covered by insurance. “This study is a great step forward in developing medications for this underserved patient population,” said Dr. Coughlin, who directs the section of pediatric dermatology at Washington University/St. Louis Children’s Hospital.
However, she continued, “the authors mention approximately 13% of patients had a treatment-related adverse reaction, but the abstract does not delineate these reactions.” In addition, the study was limited to children who had less than or equal to 10% body surface area involvement of vitiligo, she noted, adding that “more work is needed to learn about safety of application to larger surface areas.”
Going forward, “it will be important to learn the durability of response,” said Dr. Coughlin, who is also assistant professor of dermatology at Washington University in St. Louis. “Does the vitiligo return if patients stop applying the ruxolitinib cream?”
Dr. Rosmarin disclosed that he has received honoraria as a consultant for Incyte, AbbVie, Abcuro, AltruBio, Arena, Boehringer Ingelheim, Bristol Meyers Squibb, Celgene, Concert, CSL Behring, Dermavant, Dermira, Janssen, Kyowa Kirin, Lilly, Novartis, Pfizer, Regeneron, Revolo Biotherapeutics, Sanofi, Sun Pharmaceuticals, UCB, and VielaBio. He has also received research support from Incyte, AbbVie, Amgen, Bristol-Myers Squibb, Celgene, Dermira, Galderma, Janssen, Lilly, Merck, Novartis, Pfizer, and Regeneron; and has served as a paid speaker for Incyte, AbbVie, Amgen, Bristol-Myers Squibb, Celgene, Incyte, Janssen, Lilly, Novartis, Pfizer, Regeneron, and Sanofi. Dr. Coughlin is on the board of the Pediatric Dermatology Research Alliance and the International Immunosuppression and Transplant Skin Cancer Collaborative.
INDIANAPOLIS – and a higher proportion responded at week 52, results from a pooled analysis of phase 3 data showed.
Currently, there is no treatment approved by the Food and Drug Administration to repigment patients with vitiligo, but the cream formulation of the Janus kinase inhibitor ruxolitinib was shown to be effective and have a favorable safety profile in patients aged 12 years and up in the phase 3 clinical trials, TRuE-V1 and TruE-V2. “We know that about half of patients will develop vitiligo by the age of 20, so there is a significant need to have treatments available for the pediatric population,” lead study author David Rosmarin, MD, told this news organization in advance of the annual meeting of the Society for Pediatric Dermatology.
In September 2021, topical ruxolitinib (Opzelura) was approved by the FDA for treating atopic dermatitis in nonimmunocompromised patients aged 12 years and older. The manufacturer, Incyte, has submitted an application for approval to the agency for treating vitiligo in patients ages 12 years and older based on 24-week results; the FDA is expected to make a decision by July 18.
For the current study, presented during a poster session at the meeting, Dr. Rosmarin, of the department of dermatology at Tufts Medical Center, Boston, and colleagues pooled efficacy and safety data for adolescent patients aged 12-17 years from the TRuE-V studies, which enrolled patients 12 years of age and older diagnosed with nonsegmental vitiligo with depigmentation covering up to 10% of total body surface area (BSA), including facial and total Vitiligo Area Scoring Index (F-VASI/T-VASI) scores of ≥ 0.5/≥ 3. Investigators randomized patients 2:1 to twice-daily 1.5% ruxolitinib cream or vehicle for 24 weeks, after which all patients could apply 1.5% ruxolitinib cream through week 52. Efficacy endpoints included the proportions of patients who achieved at least 75%, 50%, and 90% improvement from baseline in F-VASI scores (F-VASI75, F-VASI50, F-VASI90); the proportion of patients who achieved at least a 50% improvement from baseline in T-VASI (T-VASI50); the proportion of patients who achieved a Vitiligo Noticeability Scale (VNS) rating of 4 or 5; and percentage change from baseline in facial BSA (F-BSA). Safety and tolerability were also assessed.
For the pooled analysis, Dr. Rosmarin and colleagues reported results on 72 adolescents: 55 who received ruxolitinib cream and 17 who received vehicle. At week 24, 32.1% of adolescents treated with ruxolitinib cream achieved F-VASI75, compared with none of those in the vehicle group. Further, response rates at week 52 for patients who applied ruxolitinib cream from day 1 were as follows: F-VASI75, 48.0%; F-VASI50, 70.0%; F-VASI90, 24.0%; T-VASI50, 60.0%; VNS score of 4/5, 56.0%; and F-BSA mean percentage change from baseline, –41.9%.
Efficacy at week 52 among crossover patients (after 28 weeks of ruxolitinib cream) was consistent with week 24 data in patients who applied ruxolitinib cream from day 1.
“As we know that repigmentation takes time, about half of the patients achieved the F-VASI75 at the 52-week endpoint,” said Dr. Rosmarin, who is also vice-chair for research and education at Tufts Medical Center, Boston. “Particularly remarkable is that 60% of adolescents achieved a T-VASI50 [50% or more repigmentation of the whole body at the year mark] and over half the patients described their vitiligo as a lot less noticeable or no longer noticeable at the year mark.”
In terms of safety, treatment-related adverse events occurred in 12.9% of patients treated with ruxolitinib (no information was available on the specific events). Serious adverse events occurred in 1.4% of patients; none were considered related to treatment.
“Overall, these results are quite impressive,” Dr. Rosmarin said. “While it can be very challenging to repigment patients with vitiligo, ruxolitinib cream provides an effective option which can help many of my patients.” He acknowledged certain limitations of the analysis, including the fact that the TRuE-V studies were conducted during the COVID-19 pandemic, “which may have contributed to patients being lost to follow-up. Also, the majority of the patients had skin phototypes 1-3.”
Carrie C. Coughlin, MD, who was asked to comment on the study, said that patients with vitiligo need treatment options that are well-studied and covered by insurance. “This study is a great step forward in developing medications for this underserved patient population,” said Dr. Coughlin, who directs the section of pediatric dermatology at Washington University/St. Louis Children’s Hospital.
However, she continued, “the authors mention approximately 13% of patients had a treatment-related adverse reaction, but the abstract does not delineate these reactions.” In addition, the study was limited to children who had less than or equal to 10% body surface area involvement of vitiligo, she noted, adding that “more work is needed to learn about safety of application to larger surface areas.”
Going forward, “it will be important to learn the durability of response,” said Dr. Coughlin, who is also assistant professor of dermatology at Washington University in St. Louis. “Does the vitiligo return if patients stop applying the ruxolitinib cream?”
Dr. Rosmarin disclosed that he has received honoraria as a consultant for Incyte, AbbVie, Abcuro, AltruBio, Arena, Boehringer Ingelheim, Bristol Meyers Squibb, Celgene, Concert, CSL Behring, Dermavant, Dermira, Janssen, Kyowa Kirin, Lilly, Novartis, Pfizer, Regeneron, Revolo Biotherapeutics, Sanofi, Sun Pharmaceuticals, UCB, and VielaBio. He has also received research support from Incyte, AbbVie, Amgen, Bristol-Myers Squibb, Celgene, Dermira, Galderma, Janssen, Lilly, Merck, Novartis, Pfizer, and Regeneron; and has served as a paid speaker for Incyte, AbbVie, Amgen, Bristol-Myers Squibb, Celgene, Incyte, Janssen, Lilly, Novartis, Pfizer, Regeneron, and Sanofi. Dr. Coughlin is on the board of the Pediatric Dermatology Research Alliance and the International Immunosuppression and Transplant Skin Cancer Collaborative.
INDIANAPOLIS – and a higher proportion responded at week 52, results from a pooled analysis of phase 3 data showed.
Currently, there is no treatment approved by the Food and Drug Administration to repigment patients with vitiligo, but the cream formulation of the Janus kinase inhibitor ruxolitinib was shown to be effective and have a favorable safety profile in patients aged 12 years and up in the phase 3 clinical trials, TRuE-V1 and TruE-V2. “We know that about half of patients will develop vitiligo by the age of 20, so there is a significant need to have treatments available for the pediatric population,” lead study author David Rosmarin, MD, told this news organization in advance of the annual meeting of the Society for Pediatric Dermatology.
In September 2021, topical ruxolitinib (Opzelura) was approved by the FDA for treating atopic dermatitis in nonimmunocompromised patients aged 12 years and older. The manufacturer, Incyte, has submitted an application for approval to the agency for treating vitiligo in patients ages 12 years and older based on 24-week results; the FDA is expected to make a decision by July 18.
For the current study, presented during a poster session at the meeting, Dr. Rosmarin, of the department of dermatology at Tufts Medical Center, Boston, and colleagues pooled efficacy and safety data for adolescent patients aged 12-17 years from the TRuE-V studies, which enrolled patients 12 years of age and older diagnosed with nonsegmental vitiligo with depigmentation covering up to 10% of total body surface area (BSA), including facial and total Vitiligo Area Scoring Index (F-VASI/T-VASI) scores of ≥ 0.5/≥ 3. Investigators randomized patients 2:1 to twice-daily 1.5% ruxolitinib cream or vehicle for 24 weeks, after which all patients could apply 1.5% ruxolitinib cream through week 52. Efficacy endpoints included the proportions of patients who achieved at least 75%, 50%, and 90% improvement from baseline in F-VASI scores (F-VASI75, F-VASI50, F-VASI90); the proportion of patients who achieved at least a 50% improvement from baseline in T-VASI (T-VASI50); the proportion of patients who achieved a Vitiligo Noticeability Scale (VNS) rating of 4 or 5; and percentage change from baseline in facial BSA (F-BSA). Safety and tolerability were also assessed.
For the pooled analysis, Dr. Rosmarin and colleagues reported results on 72 adolescents: 55 who received ruxolitinib cream and 17 who received vehicle. At week 24, 32.1% of adolescents treated with ruxolitinib cream achieved F-VASI75, compared with none of those in the vehicle group. Further, response rates at week 52 for patients who applied ruxolitinib cream from day 1 were as follows: F-VASI75, 48.0%; F-VASI50, 70.0%; F-VASI90, 24.0%; T-VASI50, 60.0%; VNS score of 4/5, 56.0%; and F-BSA mean percentage change from baseline, –41.9%.
Efficacy at week 52 among crossover patients (after 28 weeks of ruxolitinib cream) was consistent with week 24 data in patients who applied ruxolitinib cream from day 1.
“As we know that repigmentation takes time, about half of the patients achieved the F-VASI75 at the 52-week endpoint,” said Dr. Rosmarin, who is also vice-chair for research and education at Tufts Medical Center, Boston. “Particularly remarkable is that 60% of adolescents achieved a T-VASI50 [50% or more repigmentation of the whole body at the year mark] and over half the patients described their vitiligo as a lot less noticeable or no longer noticeable at the year mark.”
In terms of safety, treatment-related adverse events occurred in 12.9% of patients treated with ruxolitinib (no information was available on the specific events). Serious adverse events occurred in 1.4% of patients; none were considered related to treatment.
“Overall, these results are quite impressive,” Dr. Rosmarin said. “While it can be very challenging to repigment patients with vitiligo, ruxolitinib cream provides an effective option which can help many of my patients.” He acknowledged certain limitations of the analysis, including the fact that the TRuE-V studies were conducted during the COVID-19 pandemic, “which may have contributed to patients being lost to follow-up. Also, the majority of the patients had skin phototypes 1-3.”
Carrie C. Coughlin, MD, who was asked to comment on the study, said that patients with vitiligo need treatment options that are well-studied and covered by insurance. “This study is a great step forward in developing medications for this underserved patient population,” said Dr. Coughlin, who directs the section of pediatric dermatology at Washington University/St. Louis Children’s Hospital.
However, she continued, “the authors mention approximately 13% of patients had a treatment-related adverse reaction, but the abstract does not delineate these reactions.” In addition, the study was limited to children who had less than or equal to 10% body surface area involvement of vitiligo, she noted, adding that “more work is needed to learn about safety of application to larger surface areas.”
Going forward, “it will be important to learn the durability of response,” said Dr. Coughlin, who is also assistant professor of dermatology at Washington University in St. Louis. “Does the vitiligo return if patients stop applying the ruxolitinib cream?”
Dr. Rosmarin disclosed that he has received honoraria as a consultant for Incyte, AbbVie, Abcuro, AltruBio, Arena, Boehringer Ingelheim, Bristol Meyers Squibb, Celgene, Concert, CSL Behring, Dermavant, Dermira, Janssen, Kyowa Kirin, Lilly, Novartis, Pfizer, Regeneron, Revolo Biotherapeutics, Sanofi, Sun Pharmaceuticals, UCB, and VielaBio. He has also received research support from Incyte, AbbVie, Amgen, Bristol-Myers Squibb, Celgene, Dermira, Galderma, Janssen, Lilly, Merck, Novartis, Pfizer, and Regeneron; and has served as a paid speaker for Incyte, AbbVie, Amgen, Bristol-Myers Squibb, Celgene, Incyte, Janssen, Lilly, Novartis, Pfizer, Regeneron, and Sanofi. Dr. Coughlin is on the board of the Pediatric Dermatology Research Alliance and the International Immunosuppression and Transplant Skin Cancer Collaborative.
AT SPD 2022