Treatment of vitiligo with the topical Janus kinase inhibitor ruxolitinib

Publication date: Available online 5 April 2017
Source:Journal of the American Academy of Dermatology
Author(s): Brooke Rothstein, Deep Joshipura, Ami Saraiya, Rana Abdat, Huda Ashkar, Yana Turkowski, Vaneeta Sheth, Victor Huang, Shiu Chung Au, Courtney Kachuk, Nicole Dumont, Alice B. Gottlieb, David Rosmarin
BackgroundExisting therapies for vitiligo are limited in efficacy and can be associated with undesirable side effects. Topical Janus kinase inhibitors may offer a new therapeutic option for vitiligo.ObjectiveWe sought to assess the role of topical ruxolitinib 1.5% cream, a Janus kinase inhibitor, in vitiligo treatment.MethodsThis 20-week, open-label, proof-of-concept trial of twice-daily topical ruxolitinib 1.5% cream was conducted in 12 patients with a minimum of 1% affected body surface area of vitiligo. The primary outcome was percent improvement in Vitiligo Area Scoring Index from baseline to week 20.ResultsOf 12 patients screened, 11 were enrolled and 9 completed the study (54.5% men; mean age, 52 years). Four patients with significant facial involvement at baseline had a 76% improvement in facial Vitiligo Area Scoring Index scores at week 20 (95% confidence interval, 53-99%; P = .001). A 23% improvement in overall Vitiligo Area Scoring Index scores was observed in all enrolled patients at week 20 (95% confidence interval, 4-43%; P = .02). Three of 8 patients responded on body surfaces and 1 of 8 patients responded on acral surfaces. Adverse events were minor, including erythema, hyperpigmentation, and transient acne.LimitationsLimitations of the study include the small sample size and open-label study design.ConclusionsTopical ruxolitinib 1.5% cream provided significant repigmentation in facial vitiligo and may offer a valuable new treatment for vitiligo.



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