P068 Oral tofacitinib in comparison with dexamethasone oral mini-pulse therapy for the treatment of active nonsegmental vitiligo: a randomized controlled trial

Abstract Corticosteroid oral mini-pulse (OMP) therapy is the standard treatment modality for active vitiligo. Tofacitinib is being increasingly used in vitiligo; however, there is lack of literature on the stabilizing and repigmenting potential of oral tofacitinib in active vitiligo. Our objective was to compare the efficacy of oral tofacitinib with dexamethasone OMP therapy in active nonsegmental vitiligo. This was a prospective, randomized, investigator-blinded trial that was conducted at a tertiary care centre. Patients aged 18–60 years with active nonsegmental vitiligo were recruited. Participants were randomized to receive dexamethasone 2.5 mg twice a week (group A) or oral tofacitinib 5 mg twice daily (group B) for 24 weeks, followed by 12 weeks of observation. The primary outcome in both groups was the proportion of patients showing ≥ 50% improvement in Vitiligo Extent Score (VES) at 24 weeks. The secondary outcomes were proportions of patients showing disease stabilization (no new lesions for the last 12 weeks) at 24 weeks, patients maintaining disease stabilization at 36 weeks, patients experiencing treatment failure and relapse, extent of repigmentation (as assessed by mean reduction in VES scores at each visit) and incidence of treatment-related adverse effects in both groups. Sixty patients were recruited (30 in each group), among whom 49 patients completed 36 weeks of follow-up. There were four dropouts, two in each group. On intention-to-treat analysis, at 24 weeks the proportion of patients achieving ≥ 50% improvement in VES was similar in both groups (17% vs. 20%, P = 0.83). The proportions of patients achieving disease stabilization at 24 weeks (63% vs. 83%, P = 0.19) and maintaining stabilization at 36 weeks (84% vs. 100%, P = 0.07) were also statistically similar. However, at 36 weeks, the mean percentage repigmentation was significantly higher in group B (mean 31.5%, SD 24.9) than in group A (mean 16.7%, SD 34.8) (P = 0.03). Group A also had a significantly higher treatment failure rate at 12 weeks (20% vs. 3%, P = 0.05), but comparable relapse rates at 36 weeks (23% vs. 22%, P = 0.84). Treatment-related adverse effects were observed in 20% of patients in group A and 43% in group B (P = 0.08). Patients with acrofacial vitiligo (P = 0.05) and confetti lesions (P = 0.01) experienced significantly better repigmentation in group B. This study is limited by the small sample size, single-centre design and lack of double-blinding. In conclusion, tofacitinib is more effective than OMP in the treatment of active nonsegmental vitiligo, with greater extent of repigmentation and lower rates of treatment failure. However, both treatment modalities have statistically similar efficacy in terms of disease stabilization and relapse.