Comparative efficacy of topical tofacitinib versus topical tacrolimus in the treatment of localized vitiligo: a randomized investigator-blinded intraindividual trial.

Abstract

Topical ruxolitinib was recently approved by the US Food and Drug Administration for the treatment of vitiligo. Studies comparing other topical Janus kinase inhibitors with established topical therapies like tacrolimus are lacking. To compare the efficacy and tolerability of topical tofacitinib and topical tacrolimus in patients with localized vitiligo using patient- and investigator-reported outcome measures. This was a prospective randomized investigator-blinded intraindividual single-centre comparative trial conducted over 16 weeks between January and December 2024. Thirty patients with 60 symmetrical vitiligo patches were enrolled. Eligible participants had slowly spreading, nonsegmental vitiligo affecting ≤ 5% of their body surface area. Patches were randomized to receive either topical tofacitinib 2% ointment or topical tacrolimus 0.1% ointment twice daily for 16 weeks. The primary outcome was percentage of patches achieving treatment success, defined as a Vitiligo Noticeability Scale (VNS) score of 4 ('a lot less noticeable') or 5 ('no longer noticeable'). Secondary outcomes included time to treatment success, trends of VNS among groups, extent of repigmentation and adverse effects. The study was registered with the Clinical Trial Registry of India (CTRI/2023/12/060431). Of the patches treated with tofacitinib, 47% (n = 14/30) achieved treatment success vs. 37% (n = 11/30) of those treated with tacrolimus (P = 0.60). The median time to treatment success was shorter for patches treated with tofacitinib [8 weeks; 95% confidence interval (CI) 4.333-11.667] than for those treated with tacrolimus [12 weeks; 95% CI 8.301-15.699 (P = 0.18)]. Significant repigmentation was seen with both treatments, with 33% (n = 10) of tofacitinib-treated patches and 20% (n = 6) of those treated with tacrolimus achieving > 80% repigmentation. There were fewer adverse events with tofacitinib (n = 2) than with tacrolimus (n = 7). Facial lesions responded better than acral or trunk lesions with both treatments. Topical tofacitinib demonstrated comparable efficacy to tacrolimus for localized vitiligo but showed trends toward earlier patient-reported response and a more favourable safety profile, a finding that could be validated in future studies with larger sample sizes.