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

Abstract

Background/objective: 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.

Method: 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.

Results: 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.

Conclusions: 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.