Real‐World Effectiveness and Safety of Topical Ruxolitinib in Adolescents With Vitiligo: A 52‐Week Retrospective Study

Abstract

Topical ruxolitinib, a selective JAK1/2 inhibitor, is the first approved topical therapy for repigmentation in non‐segmental vitiligo in patients aged 12 years and older. Real‐world data in adolescents remains limited. To evaluate the real‐world effectiveness and safety of topical ruxolitinib 1.5% cream in adolescents with vitiligo over 24 and 52 weeks. Single‐centre retrospective study of 39 adolescents (12–17 years) treated with ruxolitinib 1.5% cream twice daily. Disease severity was assessed by body surface area (BSA), total Vitiligo Area Scoring Index (T‐VASI) and facial VASI (F‐VASI) at baseline, week 24 and week 52. Continuous variables are reported as median (interquartile range [IQR]). Within‐patient changes were analysed with the Wilcoxon signed‐rank test, and a sensitivity analysis restricted to patients with complete data at all three time points was performed. Median T‐VASI decreased from 1.80 (IQR 1.03–3.38) to 1.50 (0.75–1.94) at week 24 ( n  = 34; median difference −0.56, 95% CI − 0.90 to −0.31; p  = 0.0002) and from 1.50 (0.76–2.00) to 1.25 (0.50–1.44) at week 52 ( n  = 18; −0.62, 95% CI − 1.02 to −0.25; p  = 0.006), with reductions in BSA and F‐VASI. Among the 18 patients assessed at week 52, 8 (44.4%) achieved T‐VASI50 and 6 (33.3%) achieved F‐VASI75, with repigmentation most pronounced on the face. In a sensitivity analysis restricted to these 18 patients, F‐VASI decreased further between weeks 24 and 52 ( p  = 0.008), whereas T‐VASI did not ( p  = 0.19). Few adverse events were recorded: mild application‐site reactions in 4 of 39 patients (10.3%), with no serious adverse events. Retrospective, single‐centre design with a small sample size. Attrition by week 52 was high (18 of 39 patients, 46.2%) and was not independent of baseline severity, as patients assessed at week 52 had milder facial involvement at baseline; week 52 estimates are therefore susceptible to attrition bias and responder rates at the two time points are not directly comparable. Adherence was not measured, residual confounding cannot be excluded, no patient‐reported outcomes were collected, and the small number of adverse events precludes conclusions on the safety profile. Topical ruxolitinib was associated with a reduction in vitiligo severity scores and was well tolerated. The magnitude and time course of the response cannot be established with precision within the present design; these findings are hypothesis‐generating and require confirmation in larger prospective studies.