Real-World Systemic Treatment Patterns and Outcomes of Children With Severe Alopecia Areata: A French Nationwide Study (ESTAPED Cohort)

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Severe forms of alopecia areata (AA) often require off-label systemic treatments (ST) for which data on effectiveness and safety are limited. This study aimed to provide a comprehensive real-world overview of ST patterns in children with severe AA. We conducted a retrospective, longitudinal, multicenter study of children with AA (aged <18 years) receiving at least one ST (2010–2023), identified using a keyword search in Ouest Data Hub Warehouse (a hospital data network in France with a critical mass of 5.1 million patients) or by members of the Société Française de Dermatologie Pédiatrique. The primary outcome was drug survival (DS), defined as time on ST, and the secondary outcomes included reasons for treatment discontinuation and long-term observations. Among the 262 included children (median age at AA onset: 9 years; 58.4% girls, atopic dermatitis in 32.4%), the first-line ST most frequently initiated (median age: 12 years, alopecia totalis in 28.6%) was intravenous corticosteroids (35.9%, median duration: 3 months), followed by methotrexate + intravenous corticosteroids (24.0%, 12.5 months), oral corticosteroids (17.9%, 1 month), methotrexate (10.7%, 9.5 months), methotrexate + oral corticosteroids (8.8%, 9 months), and lastly baricitinib (2.7%, 2.5 months). Specific maintenance treatments were not significantly associated with DS. Psychological impact, atopic dermatitis, later childhood onset and female sex were associated with longer first-line systemic treatment drug survival. Treatment failure was the main reason for discontinuation (51.9%) and no serious adverse events were reported. Five years after starting the first ST (n = 62), 14.5% of patients had achieved remission. In this national, retrospective real-world study, drug survival analyses highlighted two treatment profiles: pulse therapy (corticosteroids) for flare-ups and maintenance treatments (methotrexate, baricitinib) for long-term management. Overall, the effectiveness of therapies appears limited with frequent relapse and a likely need for prolonged treatment. However, data on long-term treatment outcomes of JAKi in children are lacking. This study provides real-life data which could help improve management strategies in children with severe AA. Alopecia areata is a common chronic disease causing hair loss. Severe forms often require systemic treatments, for which data concerning efficacy and safety are limited. This study aimed to provide an overview of systemic treatment patterns in children with severe alopecia areata in France. We conducted a retrospective study using medical records from west regional French hospitals or obtained by members of the Société Française de Dermatologie Pédiatrique. We analyzed how long treatments were continued and the reasons why they were discontinued. A total of 262 children (58.4% girls) treated with at least one systemic treatment between 2010 and 2023 were included. Intravenous corticosteroids were the most frequently prescribed first-line systemic treatment (35.9%, median duration: 3 months), followed by methotrexate combined with intravenous corticosteroids (24.0%, 12.5 months), oral corticosteroids (17.9%, 1 month), methotrexate alone (10.7%, 9.5 months), methotrexate combined with oral corticosteroids (8.8%, 9 months), and baricitinib (2.7%, 2.5 months). The type of maintenance treatment was not significantly associated with treatment duration. Psychological impact, atopic dermatitis, later onset and female sex were associated with longer treatment duration. Treatment failure was the main reason for discontinuing treatment (51.9%); no serious adverse events were reported. Five years after starting the first systemic treatment, 14.5% of children with available data had achieved remission. Systemic treatment use could be categorized into pulse therapy (corticosteroids) for flare-ups and maintenance treatments (methotrexate and baricitinib) to maintain hair regrowth. This study provides real-life data which may help improve treatment strategies in children with severe alopecia areata.

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