Ruxolitinib Beneficial Across Demographics of Patients with Vitiligo

Julien Seneschal, MD, PhD

Patients with nonsegmental vitiligo across all tested demographic groups saw positive results when using ruxolitinib cream, according to new findings from a yearlong trial.

Nonsegmental vitiligo is the most common form of vitiligo, an autoimmune condition that affects melanocytes and results in patches of skin depigmentation. The condition’s pathogenesis is regulated primarily by activation of the Janus kinase (JAK) signaling pathway by interferon-γ.

The efficacy of this cream formulation of ruxolitinib – a JAK1/JAK2 inhibitor designed to treat nonsegmental vitiligo– was examined in a study presented at the European Academy of Dermatology and Venerology (EADV) 2022 Congress this week by a team led by Julien Seneschal, MD, PhD, and Albert Wolkerstorfer, MD, PhD.

The goal of Seneschal and colleagues’ research sought to determine the efficacy and overall safety of this treatment in both adolescent and adult vitiligo patients using a pool of data following 52 weeks of ruxolitinib use.

Research and Methods

The key exclusion criteria used by the researchers were the existence of confounding dermatologic diseases, use of JAK inhibitors prior to the study, complete leukotrichia within facial lesions, biologic or experimental therapy use, immunomodulation treatments, topical skin treatments, and phototherapy.

The investigators arranged the patients’ data by both Fitzpatrick skin type and geographic area. The participants aged ≥12 years and diagnosed with nonsegmental vitiligo were eligible for the trial. Eligible patients also had depigmented areas covering ≤10% of their total body surface area, and Facial Vitiligo Area Scoring Index (F-VASI) scores ≥0.5.

The investigators assessed the benefit of ruxolitinib over 52 weeks and examined both baseline demographics and clinical characteristics for the adolescent and adult patients. The data collected was pooled from 2 previous TRuE-V1/TRuE-V2 phase 3 studies, during which ruxolitinib cream was found to be statistically superior by week 24.

For 28 more weeks, the investigators allowed the patients to then apply 1.5% ruxolitinib cream twice daily in the open-label treatment extension.

Study Results

Following week 52, the investigators summarized the analyses of subgroups, including patients who reported applying ≥1 dose. The TRuE-V studies randomized 674 participants, and the mean age at baseline for the patients was 39.5 years.

By 52 weeks , patients with ≥75% improvement from baseline in F-VASI (F-VASI75) were evaluated by sex, age group, geographic area (North America or Europe), race, Fitzpatrick skin type (I–III or IV–VI), facial body surface area (F-BSA; <1.5%, ≥1.5%), disease stability, other autoimmune disorder presence, previous therapy, and disease duration.

Investigators found that 50.3% of those who used ruxolitinib cream from the first day achieved F-VASI75, and that F-VASI75 response efficacy was identified in every subgroup, including all sexes and age groups.

The researchers found that ruxolitinib “was well tolerated, and the incidence of treatment-related AEs was similar across demographic subgroups.”

The study, “Efficacy and Safety of Ruxolitinib Cream for the Treatment of Vitiligo by Patient Demographics and Baseline Clinical Characteristics: Week 52 Pooled Subgroup Analysis From Two Randomized Phase 3 Studies,” was presented at EADV 2022.