Global Sr Medical Director Sanofi Cambridge, Massachusetts, United States
Background: Dupilumab has proven to be effective in improving patient-reported outcomes for atopic dermatitis (AD) in pediatric patients in clinical trials. However, few real-world daily practice studies are available to show the treatment effect on patient-reported disease symptoms and quality of life in children with moderate-to-severe AD. Objective: To report the effect of dupilumab treatment on patient-reported outcomes for AD in pediatric patients in real-world practice. Design/Methods: PEDISTAD (NCT03687359) is an ongoing, international, longitudinal, observational 5-year registry study in patients aged 6 months to 11 years at enrollment with moderate-to-severe AD, whose disease is not adequately controlled by topical prescription therapies or for whom those therapies are medically inadvisable. This interim analysis assesses the effect of dupilumab on patient-reported disease severity using Patient-Oriented Eczema Measure (POEM) and on quality of life (QoL) using Children’s Dermatology Life Quality Index (CDLQI), for up to 3 years. Overall safety was also evaluated. Results: A total of 214 patients received dupilumab (median treatment observation period: 16.1 months; accumulated 3-year discontinuation rate: 13.3%). The proportion of patients with severe/very severe disease (POEM score > 17 [range 0–28]) decreased from 63.2% at therapy start to 16.3% at last observation. The mean (± SE) POEM score also decreased with dupilumab use, from 17.9 ± 0.5 at therapy start to 9.1 ± 0.7 at last observation. Similarly, the mean (± SE) CDLQI score decreased with dupilumab use, from 12.9 ± 0.5 at therapy start to 7.3 ± 0.7 at last observation. 24% of patients had adverse events, including 2.4% serious adverse events.
Conclusion(s): Dupilumab significantly improved patient-reported symptoms of AD and QoL in children aged 6 months to 11 years in real-world daily practice.
