Efficacy and safety of ruxolitinib cream for the treatment of vitiligo by patient demographics and baseline clinical characteristics: Week 52 pooled subgroup analysis from two randomized phase 3 studies

Abstract Vitiligo is a chronic autoimmune disease that targets melanocytes, causing skin depigmentation. Disease pathogenesis largely regulated by interferon-? activation of the Janus kinase (JAK) signalling pathway. A cream formulation JAK1/JAK2 inhibitor ruxolitinib demonstrated substantial re-pigmentation in two randomized, double-blind, vehicle-controlled phase 3 studies adults and adolescents with vitiligo (TruE-V1 TruE-V2). Pooled 52-week efficacy safety data based on baseline demographics clinical characteristics from TruE-V are analysed reported here. TruE-V1 (NCT04052425) TruE-V2 (NCT04057573) were conducted North America Europe. Patients ? 12 years old diagnosed nonsegmental (NSV) depigmentation covering ? 10% total body surface area (BSA), including facial Area Scoring Index (F-VASI/T-VASI) scores 0·5/? 3, eligible for enrolment. randomized 2 : 1 to twice-daily 1·5% or vehicle 24 weeks, after which all patients could apply through Week 52 (open-label extension). At 52, who achieved 75% improvement F-VASI (F-VASI75) evaluated sex, age group, Fitzpatrick type, BSA (F-BSA), investigator-assessed stability previous therapy. Safety tolerability also assessed. as observed. In total, 674 TRuE-V (ruxolitinib cream, n = 450; vehicle, 224); 673 661 included analyses, respectively, 569 continued open-label extension Day 1, 385; crossover 184). Mean (SD) at was 39·5 (15·1) years, 27·9% had types IV–VI. Baseline mean T-VASI values 0·92 6·69, respectively. 50·3% applied F-VASI75. Efficacy F-VASI75 response identified demographic characteristic subgroups. Substantive responses seen men [(n 149), 43·6%] women 201), 55·2%], groups [12–17 (n 50), 48·0%; 18–64 275), 52·0%; 65 25), 36·0%]. generally consistent type [I–III 249), 47·4%; IV–VI 101), 57·4%], F-BSA [<1·5% 283), 49·1%; 67), [stable 257), 49·4%; progressive 93), 52·7%] therapy [topical corticosteroids 110), 50·9%; topical calcineurin inhibitors 126), 49·2%; phototherapy 113), 54·0%]. Treatment-related adverse events (AEs) occurred 13·7% any time during study, similar rates among subgroups cream. Adolescent adult NSV applying per regardless characteristics. Ruxolitinib well tolerated, incidence treatment-related AEs across