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31st October 2022
Topical ruxolitinib produces significantly greater re-pigmentation over 52 weeks compared to placebo in patients with non-segmental vitiligo according to the combined results of two randomised, double-blind, placebo-controlled trials by researchers from the TRuE-V study Group.
Vitiligo describes an autoimmune disease in which there is progressive destruction of melanocytes in the skin leading to patchy depigmentation and the condition has an estimated prevalence of 0.5% in the general population. Due to the visible nature of vitiligo, not surprisingly, patients experience a reduction in many aspects of quality of life including feelings of stigmatisation, adjustment disorders, sleep disturbance, relationship difficulties and avoidance or restriction behaviour. Studies suggest that active vitiligo peri-lesional skin is characterised by prominent type 1 and 2 associated immune responses and induces melanocyte dysfunction and an epidermal inflammatory response through Janus Kinase signalling. In fact, in a phase 2 trial, use of topical ruxolitinib was associated with substantial re-pigmentation of vitiligo lesions up to 52 weeks of treatment. Based on these early positive data, in the current study, researchers undertook two identical, randomised, double-blind, placebo-controlled trials, Topical Ruxolitinib Evaluation in Vitiligo Study 1 (TRuE-V1 and 2 (TRuE-V2), using a formulation containing 1.5% ruxolitinib. Individuals 12 years of age and older with non-segmental vitiligo (i.e., where symptoms are bilateral) and depigmentation covering 10% or less of their body surface area and at least 0.5% of the body surface area of the face, were enrolled and randomised, 2:1 to topical ruxolitinib 1.5% or a matching placebo. Treatments were applied twice daily for 24 weeks to all affected areas and after this point, all of the patients were eligible to apply the active treatment up until week 52. The study primary endpoint was a 75% reduction (i.e., improvement) from baseline in the facial vitiligo area scoring index (F-VASI), which ranges from 0 to 3 and where higher scores are indicative of greater facial depigmentation. The primary outcome was assessed at week 24 and secondary endpoints included a F-VASI50 and F-VASI90.
Topical ruxolitinib and improvement of vitiligo
A total of 330 patients with a mean age of 40.2 years (56.4% female) were enrolled in TRuE-V1, of whom, 221 received ruxolitinib. There were 343 participants randomised in TRuE-V2 with a slightly lower mean age (38.9 years, 50.1% female). The baseline mean F-VASI score was 0.95 in TRuE-V1 and 0.88 in TRuE-V2.
In TRuE-V1 the primary outcome was achieved by 29.8% of those receiving ruxolitinib and 7.4% of those using placebo (relative risk, RR = 4, 95% CI 1.9 – 8.4, p < 0.001). Similarly, in TRuE-V2, there was a significant difference between the active and placebo arms (RR = 2.7, 95% CI 1.5 – 4.9, p < 0.001).
For the secondary outcomes, in TRuE-V1, 15.3% of those assigned to ruxolitinib achieved a F-VASI90 (i.e., 90% improvement in score from baseline) compared to only 2.2% of placebo patients.
In terms of safety, mild or moderate adverse events occurred in a similar proportion of ruxolitinib patients in both trials when assessed up to 52 weeks (54.8% and 62.3%). The most common adverse events in ruxolitinib patients were application site acne (6.3%) and application site pruritus (5.4%).
The authors concluded that topical ruxolitinib was superior to placebo for re-pigmentation in vitiligo, adding that longer trials were needed to determine the effect and risks of ruxolitinib in vitiligo.
Rosemarin D et al. Two Phase 3, Randomized, Controlled Trials of Ruxolitinib Cream for Vitiligo N Eng J Med 2022