Ruxolitinib Cream a Promising Option for Long-Term Vitiligo Treatment

Portrait of young man with vitiligo with eyes closed
The safety and efficacy of ruxolitinib cream following 104 weeks of treatment in a phase 2 study of adults is reported.

The following article is part of our coverage of the American Academy of Dermatology’s annual meeting (AAD 2021) that is being held virtually from April 23-25, 2021. Dermatology Advisor‘s staff will report on the top research in dermatologic advances and clinical care. Check back for the latest news from AAD 2021.


Ruxolitinib cream is both safe and effective during long-term treatment period and resulted in substantial repigmentation of vitiligo lesions, according to research results presented at the American Academy of Dermatology Virtual Meeting Experience (AAD VMX) 2021, held online from April 23 to April 25, 2021.

This phase 2, double-blind, open-label study (NCT03099304), investigated the safety and efficacy of treatment with ruxolitinib cream in adults with vitiligo during 104 weeks of treatment. Patients from 18 to 75 years of age with vitiligo that included depigmentation of 0.5% or more of the facial body surface area (BSA) and 3% or more BSA of nonfacial areas were included.

Patients were randomly assigned to receive 1 of 5 treatments: ruxolitinib cream 1.5% twice daily, ruxolitinib cream 1.5% once daily, ruxolitinib cream 0.5% once daily, ruxolitinib cream 0.15% once daily, or vehicle twice daily for a period of 24 weeks. At 24 weeks, patients in the vehicle or ruxolitinib 0.15% group who did not achieve 25% or greater improvement were either randomly reassigned to 1 of the 3 higher dosage treatment groups or to maintenance of their original dose through week 52. Patients who completed baseline, week 24, and week 52 assessments were eligible to receive open-label treatment with 1.5% ruxolitinib cream twice daily for an additional 104 weeks, for a total treatment duration of 156 weeks.

The total cohort included 157 patients. Of these participants, 94 were assigned to the 3 highest ruxolitinib doses. A total of 80 patients completed the initial 52-week period, and 77 entered the open-label period (mean age, 51.2±12 years; 57.1% men, 87% White), receiving ruxolitinib cream 1.5% twice daily for 52 weeks. Within this group, 71.4% (n=55) completed the week 104 study visit and were included in safety and efficacy analyses.

At week 104, facial Vitiligo Area Scoring Index (F-VASI) 50, F-VASI75, and F-VASI90 were achieved in 83.6%, 65.5%, and 52.7% of all patients, respectively, and total VASI (T-VASI) 50 and T-VASI75 were achieved by 58.2% and 27.3% of patients in the same time period. Responses on the Facial Physician’s Global Vitiligo Assessment (F-PhGVA) and the Patient Global Impression of Change for Vitiligo (PaGIC-V) were 43.6% and 55.6%, respectively, at week 104 with twice-daily ruxolitinib cream 1.5%.

Within the group of patients who were initially assigned to this group (n=33), the percentage of patients who achieved F-VASI50, 75, and 90 response demonstrated a steady increase between weeks 24 and 104.

In terms of safety, no significant safety signals were seen during the 52-week open-label treatment period. It was found that 51.9% of patients during the open-label period and 78.8% in the initially randomized group experienced treatment-emergent adverse events. No accumulation of adverse events was noted by investigators during the 104-week treatment period. In addition, no clinically relevant changes in either hemoglobin or platelet levels were seen during the 104-week treatment period regardless of ruxolitinib dose at randomization, the investigators noted.

“Treatment with ruxolitinib cream produced substantial facial and total body repigmentation of vitiligo lesions through 104 weeks of treatment,” the researchers concluded. “Ruxolitinib cream was well tolerated over a 2-year period, and no treatment-related serious AEs [adverse events] were reported.”

Disclosure: This clinical trial was supported by Incyte Corporation. Please see the original reference for a full list of authors’ disclosures.

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Harris JE, Pandya AG, Lebwohl M, et al. Safety and efficacy of ruxolitinib cream for the treatment of vitiligo: 104-week data from a phase 2 study. Presented at: AAD VMX 2021; April 23-25, 2021. Abstract/Poster 27535.