Efficacy and safety of dupilumab treatment with concomitant topical corticosteroids in children aged 6 months to 5 years with severe atopic dermatitis

Approved systemic treatments for children with atopic dermatitis (AD) are limited. To report dupilumab (DPL) efficacy and safety in a subgroup of children with severe AD. LIBERTY AD PRESCHOOL (NCT03346434 part B), a double-blind, randomized, placebo (PBO)-controlled, 16-week (wk) phase 3 study, enrolled patients (pts; 6 months–5 years) with inadequately controlled moderate-to-severe AD. Pts received DPL 200/300 mg (baseline [BL] weight 5– < 15 kg/15– < 30 kg: 200/300 mg) or PBO every 4 weeks with standardized low-potency topical corticosteroids. Seventy-five percent reduction from BL in Eczema Area and Severity Index (EASI-75), Worst Scratch/Itch Numerical Rating Scale (WSI-NRS), Children's Dermatology Life Quality Index (CDLQI), and Infant's Dermatitis Quality of Life (IDQOL) in children with severe AD (Investigator's Global Assessment = 4) are reported. In total, 125 pts were included (DPL/PBO, n = 63/62). BL disease characteristics were similar between DPL/PBO. Dupilumab significantly increased the proportion of pts achieving EASI-75 vs. PBO by Wk4 (27% vs. 4.8%; P = 0.0009), and Wk16 (46% vs. 7%; P < 0.0001). At Wk16, DPL resulted in significantly greater percent reduction from BL in WSI-NRS vs. PBO (LS mean (SE) – 41.8 [5.4] vs. 0.5 [5.4]; P < 0.0001); and significantly improved change from BL in CDLQI (−9.1 [1.1] vs. −2.6 [1.2]; P < 0.0001); and IDQOL (−9.1 [1.3] vs. −0.6 [1.1]; P < 0.0001). Treatment-emergent adverse events (TEAEs) were reported in 42 (66.7%)/45 (73.8%) pts (DPL/PBO); most were mild–moderate and deemed unrelated to study drug by the investigator. The most common TEAE was AD (10 [15.9%]/16 [26.2%]). TEAEs in the conjunctivitis cluster were reported by 4 (6.4%)/0 pts; 3 (4.9%) serious adverse events (AE) were reported with PBO. No DPL-related AE were serious or led to treatment discontinuation. DPL significantly improved AD signs, symptoms, and quality of life in children aged 6 months–5 years with severe AD and had a safety profile consistent with that previously seen in older age groups.