Topical ruxolitinib effective in vitiligo

Vitiligo pathogenesis is driven by signalling through Janus kinase (JAK) 1 and 2. In a clinical trial, topical ruxolitinib cream produced substantial facial and total body repigmentation up to 1 year [1].

Vitiligo is a chronic autoimmune disease of the skin that targets melanocytes, resulting in patches of skin depigmentation [2]. Expression of interferon (IFN)-γ is increased in the lesional skin of patients. Neutralisation of IFN-γ prevents CD8(+) T-cell accumulation and depigmentation, suggesting a therapeutic potential for this approach [3]. Thus, JAK1/JAK2 inhibitors appear effective in vitiligo, presumably via inhibition of IFN-γ signalling in the skin [4].

Ruxolitinib cream across a dose range of 0.15 to 1.5% provided significant repigmentation of facial vitiligo lesion after 24 weeks of double-blind, vehicle-controlled treatment [5]. After the blinded study phase, all patients were re-randomised and then treated with 1.5% ruxolitinib cream for 52 weeks. Primary endpoint was the proportion of patients who achieved a ≥ 50% improvement in the Vitiligo Area Scoring Index (VASI) in the face at week 24 compared with patients treated with vehicle. The proportion of patients achieving ≥ 50% improvement in facial VASI at week 52 was a secondary endpoint. Participants (n=157) had depigmented areas of ≥ 0.5% of total body surface area (BSA) on the face and ≥ 3% on non-facial areas. Most of the participants were middle-aged (mean age 48 years) and 84% of them were Caucasian.

At week 24, a ≥ 50% improvement in the facial VASI (primary endpoint) was achieved by a significantly greater proportion of patients receiving ruxolitinib cream versus vehicle. Continued improvement was seen through 52 weeks of treatment. At week 52, the proportion of patients achieving this response was highest in the 1.5% ruxolitinib group: 57.6% of patients reached this endpoint. At week 52, more than half of the patients (51.5%) even achieved an improvement in VASI of the face by 75%, and a third of patients by 90%. In addition, the total VASI improvement by 50% was noticed in a dose-dependent manner. Ruxolitinib cream was not associated with clinically significant application site reactions or serious treatment-related adverse events.

“I have waited 30 years for a study in vitiligo with these results,” said Prof. Amit Pandya (University of Texas Southwestern Medical Center, USA) during the presentation of the results.

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   1. Pandya A. Late-breaking abstract D3T01.1L, EADV 2019, 9-13 Oct, Madrid, Spain.
   2. Taieb A, Picardo M. N Engl J Med 2009;360:160-9.
   3. Harris JE. J Invest Dermatol 2012;132:1869-76.
   4. Liu LY, et al. J Am Acad Dermatol 2017;77:675-82.
   5. Rosmarin D, et al. Poster presented at WCD 2019, 10-15 June, Milan, Italy.

Posted on 9 December, 201916 February, 2024