Systemic Treatments in Moderate-to-Severe Atopic Dermatitis in Pediatric Patients up to 12 Years of Age: Real-World Treatment Outcomes from the PEDISTAD Registry

Atopic dermatitis (AD), a chronic systemic disease, can cause intense skin itching and negatively impact sleep, mood, and quality of life (QoL) for patients and families. PEDISTAD is an ongoing, 10-year, observational registry describing disease characteristics, atopic comorbidities, and treatment patterns in pediatric patients (aged <12 years at enrollment) with moderate-to-severe AD. This 3-year interim analysis evaluates clinician-reported and caregiver-reported/patient-reported outcomes (Eczema Area and Severity Index [EASI], percent body surface area affected, worst itching/scratching, Children's Dermatology Life Quality Index, and Dermatitis Family Impact) in children treated with dupilumab, methotrexate, and/or cyclosporine. Outcomes were assessed as change from therapy start to last observation (either data cutoff date or treatment discontinuation). Mean (±SE) EASI scores at the time of the last 3-year interim observation were consistent with mild disease in the dupilumab cohort and moderate disease in the methotrexate and cyclosporine cohorts. Improvements in pruritus were numerically greater in the dupilumab cohort relative to the methotrexate and cyclosporine cohorts, while improvements in QoL were similar in the dupilumab and methotrexate cohorts, with no significant change in the cyclosporine cohort. Rates of AD exacerbation were numerically lower with dupilumab treatment relative to methotrexate treatment which were numerically lower than cyclosporine treatment. Dupilumab discontinuation rates were numerically lower relative to methotrexate which were numerically lower than cyclosporine. This PEDISTAD 3-year interim analysis of dupilumab, methotrexate, and cyclosporine treatment in children with AD demonstrates numerically greater improvements in AD signs, symptoms and QoL with dupilumab treatment relative to methotrexate and cyclosporine [Video abstract and graphical abstract available]. NCT03687359. Atopic dermatitis (AD) is a chronic inflammatory skin disease, often diagnosed before 5 years of age. AD causes intense itching and negatively impacts the sleep, mood, and quality of life of affected children and their families. Children may be treated with skin creams and ointments, which are often not enough to treat all AD signs (extent and severity of disease assessed by a clinician) and symptoms (burden of disease reported by a patient and/or caregiver). The drug dupilumab is approved for use in children as young as 6 months with moderate-to-severe AD and has shown improvements in signs and symptoms in clinical trials (sponsored by Regeneron Pharmaceuticals Inc., and Sanofi). We compared the use of dupilumab, methotrexate, and cyclosporine in routine clinical practice in children under the age of 12 years with moderate-to-severe AD using 3-year data from the PEDISTAD study. Compared with children treated with methotrexate and cyclosporine, children treated with dupilumab had greater improvements in the extent and severity of their AD, as assessed by their doctor. Children treated with dupilumab also showed greater improvements in how they described the severity of their symptoms, such as daytime/nighttime itching, and their quality of life. Fewer children using dupilumab stopped treatment and fewer reported side effects compared with those using methotrexate and cyclosporine. In summary, dupilumab-treated children had greater improvements in AD signs, AD symptoms, and quality of life, were more likely to continue treatment, and had fewer reported side effects than those given cyclosporine and methotrexate.