Compared with a placebo vehicle in a pair of phase 3 trials, topical application of the JAK inhibitor ruxolitinib improved pigmentation of vitiligo lesions while causing adverse effects of pruritus and acne, researchers report. “Larger and longer trials are required to determine the effect and safety of ruxolitinib cream in patients with vitiligo,” the authors conclude.
The Topical Ruxolitinib Evaluation in Vitiligo Study 1 (TRuE-V1) and 2 (TRuE-V2) included, respectively, 330 and 344 patients aged 12 years or older with nonsegmental vitiligo with depigmentation covering 10% or less of total body-surface area. Participants were randomized to 1.5% ruxolitinib cream or vehicle control twice daily for 24 weeks to all vitiligo areas on the face and body; participants could continue the trial through week 52 with application of the active ingredient cream.
Based on a primary outcome of decrease (improvement) of at least 75% from baseline in the facial Vitiligo Area Scoring Index (F-VASI; range, 0–3), results showed the following: “The percentage of patients with an F-VASI75 response at week 24 was 29.8% in the ruxolitinib-cream group and 7.4% in the vehicle group (relative risk, 4.0; 95% confidence interval [CI], 1.9 to 8.4; P <0.001). In TRuE-V2, the percentages were 30.9% and 11.4%, respectively (relative risk, 2.7; 95% CI, 1.5 to 4.9; P <0.001). The results for key secondary end points showed superiority of ruxolitinib cream over vehicle control. Among patients who applied ruxolitinib cream throughout 52 weeks, adverse events occurred in 54.8% in TRuE-V1 and 62.3% in TRuE-V2; the most common adverse events were application-site acne (6.3% and 6.6%, respectively), nasopharyngitis (5.4% and 6.1%), and application-site pruritus (5.4% and 5.3%).”
Editorial: “With respect to the challenge of managing vitiligo in persons of color and the severe toll this disease has on the quality of their lives, future trials must do a better job of achieving truly representative patient cohorts by recruiting greater numbers of Black patients in the United States,” writes an editorialist. “The inclusion of trial sites in countries where vitiligo can be severely stigmatizing (e.g., India) will also be important to test the effect of immunomodulatory drugs in areas with a different burden of infectious disease. Despite these limitations, and in light of the revolutionary development of immunomodulatory drugs available for the treatment of psoriasis and atopic dermatitis, patients with vitiligo finally have the hope of efficient treatments, with several new immunomodulating drugs in different phases of clinical trials.”