Real-Life Effectiveness, Safety, and Growth Outcomes of Dupilumab in Children Aged 6 Months to 5 Years With Moderate-to-Severe Atopic Dermatitis: A Multicenter Retrospective Study from an Italian NPP Program.

Abstract

Introduction: Real-world evidence on the long-term use of dupilumab in very young children with moderate-to-severe atopic dermatitis (AD) remains limited. Observational data are needed to complement clinical trial findings by describing treatment outcomes in routine clinical practice.

Methods: This multicenter retrospective study included children aged 6 months to 5 years with moderate-to-severe AD treated with dupilumab through the Italian Named Patient Program. Clinical assessments were performed at baseline and at weeks (W) 16, 24, 36, and 52. Disease severity, quality of life, and symptom burden were evaluated using the Eczema Area and Severity Index (EASI), Children's Dermatology Life Quality Index (c-DLQI), pruritus-numeric rating scale (P-NRS), and sleep-numeric rating scale (S-NRS). EASI-50/75/90 responder rates were calculated at each time point. Safety data were collected throughout treatment. Growth parameters were monitored between baseline and W52.

Results: Forty-seven children were included. Dupilumab led to rapid and progressive improvement of AD severity, with mean EASI decreasing from 26.1 at baseline to 2.7 at W52 (-89.7%). Marked improvements were also observed in quality of life (-91.3% in c-DLQI), itch intensity (-82.5% in P-NRS), and sleep disturbance (-82.7% in S-NRS). At W52, EASI-75 and EASI-90 responses were achieved by 79.5% and 59.0% of evaluable patients, respectively. Dupilumab was well tolerated, with treatment-emergent adverse events occurring in 10.6% of patients, all mild or moderate and none leading to discontinuation. Weight- and height-for-age z-scores significantly increased over 52 weeks; no child newly developed values below -2 standard deviations, although 1 child remained below this threshold at W52. Percentile-based analyses yielded consistent results, confirming the absence of negative effects on growth.

Conclusion: Dupilumab was effective and well tolerated over 52 weeks in children aged 6 months to 5 years with moderate-to-severe AD, providing sustained skin clearance, symptom relief, and quality-of-life improvement. These findings support dupilumab as a valuable long-term therapeutic option in very young children with uncontrolled AD in clinical practice.