Efficacy and Safety of Ruxolitinib Cream in Vitiligo by Patient Characteristic Subgroups: Descriptive Pooled Analysis From Two Phase 3 Studies

Two phase 3 trials (TRuE-V1 and TRuE-V2) demonstrated that a topical formulation of the Janus kinase (JAK) 1/JAK2 inhibitor ruxolitinib significantly improved repigmentation versus vehicle cream in adolescent and adult patients with vitiligo. This post hoc analysis of pooled TRuE-V1/TRuE-V2 data evaluated efficacy and safety by baseline demographics and clinical characteristics. Patients aged ≥ 12 years with nonsegmental vitiligo were randomized to vehicle cream or 1.5% ruxolitinib cream twice daily for 24 weeks, after which all patients could apply ruxolitinib cream through Week 52. Efficacy was evaluated using achievement of ≥ 75% improvement from baseline in facial Vitiligo Area Scoring Index [F-VASI75] at Week 52. Safety assessments included the frequency of treatment-emergent adverse events (AEs) and treatment-related AEs. The TRuE-V studies enrolled 674 patients. Week 52 F-VASI75 was achieved by 50.3% (176/350) of patients who applied ruxolitinib cream throughout and 28.2% (46/163) who crossed over from vehicle to ruxolitinib cream after Week 24. F-VASI75 responses were observed across subgroups regardless of patient age, sex, Fitzpatrick skin type, affected facial body surface area, disease duration, disease stability, and prior treatment status. Treatment-emergent AEs occurred in 52.1% (332/637) of patients, and treatment-related AEs occurred in 13.7% (87/637); rates were generally similar across demographic subgroups. Adolescent and adult patients with vitiligo who applied ruxolitinib cream could achieve clinically meaningful repigmentation per F-VASI75 response at 1 year, regardless of their baseline demographics or clinical characteristics. Ruxolitinib cream was well tolerated, with a similar incidence of treatment-emergent and treatment-related AEs across subgroups. NCT04052425/NCT04057573. Vitiligo is a disease that causes white patches on the skin. Two large clinical trials, each 1 year long, showed that applying a topical treatment called ruxolitinib cream resulted in a return of skin color to white patches. The analysis described here used data from these two clinical trials to determine whether ruxolitinib cream is safe and effective for treating vitiligo in different subgroups of people with vitiligo. These subgroups were based on age, sex (men/women), and skin tone, as well as features of their disease, such as how long they had vitiligo. The authors found that ruxolitinib cream recolored white patches on the faces of many people who treated their skin for 1 year. This was true regardless of how old they were, whether they were men or women, and how fair or dark their skin was. This was also true regardless of differences in the disease, including whether they had vitiligo for a short or a long time, how much of their face was originally covered in white patches, if they had white patches that stayed the same or changed a lot before the trial, and whether they tried other treatments before. Ruxolitinib cream was previously shown to have generally mild side effects. Importantly, the authors here found that side effects did not differ between patient subgroups. These results show that ruxolitinib cream is an effective and safe option to treat vitiligo across a diverse population of patients.