Long-Term Effectiveness, Safety, and Survival of Dupilumab in Pediatric Atopic Dermatitis.

Abstract

Limited data are available on the long-term effectiveness and safety of dupilumab in pediatric patients with atopic dermatitis (AD) in a clinical setting. To assess the clinical effectiveness, safety, drug survival, and reasons and associated factors for discontinuation of dupilumab across pediatric age groups with up to 3 years of follow-up. This multicenter, prospective cohort study (BioDay Registry) was conducted across 4 academic and 3 nonacademic hospitals in the Netherlands from November 2019 to October 2025 and included pediatric patients (≤16 years) with moderate to severe AD who initiated treatment with dupilumab. Data were analyzed from September 2025 to January 2026. Dupilumab was administered at the label-recommended dose. Dose reduction was initiated if clinically indicated. Clinical effectiveness was evaluated by the Eczema Area and Severity Index (EASI) and Investigator Global Assessment (IGA). Additional outcomes included the Numeric Rating Scale (NRS) for pruritus, NRS for pain, and quality of life (QoL) scores. Safety was assessed by adverse events (AEs). Drug survival, reasons, and factors associated with discontinuation were evaluated. Overall, 309 pediatric patients with AD (mean [SD] age, 10.2 [4.2] years; 150 female individuals [48.5%] and 159 male individuals [51.5%]) were treated with dupilumab; 51 (16.5%) were aged 6 months to 5 years (median follow-up, 45.0 [IQR, 26.0-101.0] weeks), 120 (38.8%) were aged 6 to 11 years (median follow-up, 80.0 [IQR, 32.0-135.0] weeks), and 138 (44.7%) were aged 12 to 16 years (median follow-up, 89.5 [IQR, 48.0-143.0] weeks) at baseline. Most patients maintained controlled AD, with mean EASI and NRS scores for pruritus remaining low after 3 years (3.4 [95% CI, 2.4-4.5] and 3.6 [95% CI, 3.1-4.1], respectively). Age groups differed significantly in NRS for pruritus and QoL scores over time, with children aged 6 months to 5 years consistently reporting higher scores than older children. The most frequently reported AE was dupilumab-associated ocular surface disease (113 [36.6%]). Fifty patients (50 [16.2%]) discontinued dupilumab, mainly due to ineffectiveness (19 [38.0%]) and/or AEs (15 [30.0%]), or administration problems (11 [22.0%]); most discontinuations occurred within 1.5 years. Three-year drug survival was 80.1%; allergic asthma, allergic rhinitis, and being age 6 to 11 years were associated with a lower risk of discontinuation. The results of this cohort study suggest that dupilumab demonstrates long-term effectiveness and safety in pediatric AD in a clinical setting. Younger children show higher NRS scores for pruritus and greater QoL impairment and discontinue treatment more often due to administration problems, highlighting age-specific challenges.