Efficacy and Safety of Dupilumab Monotherapy in Patients With Dystrophic Epidermolysis Bullosa: A Retrospective Study of 8 Cases

Dystrophic epidermolysis bullosa (DEB) is a genetic skin disease with limited therapeutic options for managing pruritus. This study retrospectively evaluated the efficacy and safety of Dupilumab monotherapy in patients with DEB suffering from severe pruritus. DEB patients treated with Dupilumab between June 2021 and June 2023 were reviewed. DEB severity and pruritus intensity were assessed using the Birmingham Epidermolysis Bullosa Severity (BEBS, range 0-100) scores and the Numerical Rating Scale (NRS, range 0-10), respectively. A linear mixed model was used to evaluate the changes in NRS scores of Dupilumab monotherapy over time. The Wilcoxon signed-rank test was used to compare the differences in BEBS scores between pretreatment and posttreatment. Statistical significance was defined as p < 0.05. Eight patients were included (four males and four females; aged 5-38 years). Subtypes included severe (n = 4), intermediate (n = 2), pruriginosa RDEB (n = 1), or pruriginosa DDEB (n = 1). The baseline NRS scores averaged 8.75 ± 1.04, with significant reductions of 58.51%, 60.00%, and 49.94% at 4, 8, and 12 weeks, respectively (p < 0.001). The mean BEBS score at baseline was 35.36 ± 21.47, decreased by 22.29% at 12 weeks (p = 0.049), while the mean area of damaged skin score was 19.63 ± 1.71 at baseline, decreased by 31.23% after 12 weeks (p = 0.048). The mean duration of Dupilumab treatment was 44.50 (12-130) weeks. One DDEB-Pr patient discontinued Dupilumab for planned conception, with no adverse effects reported for the pregnant woman or newborn. Dupilumab appears to be a safe and effective treatment for pruritus in patients with DEB. Preliminary findings also support its potential use in women of reproductive age. Further larger prospective studies are needed to confirm these results.