Once- and twice-daily application of ruxolitinib cream at various doses led to repigmentation of vitiligo lesions at up to 52 weeks of treatment in patients with vitiligo, according to study finding published in the Lancet.
The study, which included 157 adult patients (mean age, 48.3 years) with depigmentation of ≥0.5% of their facial body surface area (BSA) and ≥3% of their non-facial BSA, was a phase 2 study conducted at 26 hospitals and medical centers across 18 states in the United States. Patients were randomly assigned to 4 different doses and dosing intervals of ruxolitinib cream, including 1.5% twice daily (n=33), 1.5% once daily (n=30), 0.5% once daily (n=31), or 0.15% once daily (n=31), or twice-daily vehicle control (n=32). Creams were applied to lesions that constituted ≤20% of patients’ total BSA for a total of 24 weeks.
In the control group and the once-daily 0.15% ruxolitinib group, those patients who did not experience a ≥25% improvement in the facial Vitiligo Area Scoring Index (F-VASI) by week 24 were randomly assigned again to ruxolitinib at either 0.5% once daily, 1.5% once daily, or 1.5% twice daily. Overall, patients received treatment for up to 52 weeks. All participants, investigators, and the sponsor were blinded to treatment assignment. The percentage of patients who achieved a ≥50% improvement in the F-VASI (F-VASI 50) at week 24 comprised the primary endpoint.
The F-VASI50 was achieved at 24 weeks in a significantly greater percentage of patients treated with ruxolitinib cream at 1.5% twice daily (odds ratio [OR], 24.7; 95% CI, 3.3–1121.4; P =.0001) and 1.5% once daily (OR, 28.5; 95% CI, 3.7–1305.2; P <.0001) compared with patients treated with vehicle control (3%). Serious treatment-emergent adverse events (TEAEs) were observed in 4 patients, including 1 patient randomly assigned to the 1.5% twice-daily group (subdural hematoma), 1 patient assigned to the 1.5% once-daily group (seizure), 1 patient in the 0.5% once-daily group (coronary artery occlusion), as well as 1 patient in the 0.5% once-daily arm (esophageal achalasia). All serious TEAEs were deemed unrelated to the ruxolitinib cream.
The most common treatment-related adverse event (TRAE) in ruxolitinib-treated patients was application site pruritus. Approximately 10% of the patients treated with the ruxolitinib cream experienced acne, whereas acne was reported in only 3% of patients who received the vehicle control. All TRAEs were considered mild or moderate in terms of their severity.
Limitations of the study were the inclusion of patients with mostly 1 to 3 Fitzpatrick skin phototypes, the small numbers of patients in each treatment group, and the lack of qualityof life assessments.
Findings from this study suggest that “the use of ruxolitinib cream monotherapy in patients with vitiligo could lead to effective and sustained repigmentation,” which is significant, it was noted, considering there is currently no approved therapy for repigmentation in these patients.
Disclosure: This clinical trial was supported by Incyte. Several study authors declared affiliations with the pharmaceutical industry. Please see the original reference for a full list of authors’ disclosures.
Rosmarin D, Pandya AG, Lebwohl M, et al. Ruxolitinib cream for treatment of vitiligo: a randomised, controlled, phase 2 trial. Lancet. 2020;396(10244):110-120.