Vitiligo is a chronic autoimmune disease that targets melanocytes, resulting in patches of skin depigmentation. [1] The pathogenesis of vitiligo is driven by signalling through JAK1/JAK23, and a cream formulation of ruxolitinib, a JAK1/JAK2 inhibitor, [2] is under investigation for the treatment of the condition. [3] Ruxolitinib cream provided significant repigmentation of facial vitiligo lesions after 24 weeks of double-blind, vehicle-controlled treatment (NCT03099304). [4]

• This trial further investigates the therapeutic potential of ruxolitinib cream in patients with vitiligo after 52 weeks of double-blind treatment.

Type of study, patients, and inclusion criteria

• Trial NCT03099304 was a randomised, double-blind, dose-ranging study of ruxolitinib phosphate cream in subjects with vitiligo.
• Patients had to have a clinical diagnosis of vitiligo with depigmented areas including ≥0.5% of total Body Surface Area (BSA) on the face and ≥3% of total BSA on non-facial areas.
• Patients in the previous groups (0.5% QD, 1.5% QD, 1.5% BID) of trial NCT03099304 continued treatment.
• Subjects on vehicle and those in the 0.15% group were rerandomised if <25% improvement in facial Vitiligo Area Scoring Index (F-VASI) was seen at week 24.
• Re-randomisation was to 0.5% QD, 1.5% QD, or 1.5% BID.
• A total of 157 patients were studied.

Primary outcome measure

• The primary endpoint was proportion of patients treated with ruxolitinib cream who achieved a ≥50% improvement from baseline in F-VASI (F-VASI50) at week 24 compared with patients treated with vehicle.

• At week 24, F-VASI50 was achieved by a significantly greater proportion of patients receiving ruxolitinib cream (25.8%–50.0% across doses) vs vehicle (3.1%).
• At week 52, the proportion of patients achieving an F-VASI50 response was highest in the 1.5% BID group (Figure).
• At week 52, the proportions of patients achieving F-VASI75 and F-VASI90 responses were highest in the 1.5% BID group.
• T-VASI50 at week 52 was achieved by patients in a dose-dependent manner.
• Among patients who treated all depigmented skin (baseline total BSA ≤20%), T-VASI50 response was 45.0% with the 1.5% BID regimen at week 52.
• The proportion of patients who attained Physician's Global Vitiligo Assessment (F-PhGVA) scores of clear or almost clear at week 24 increased by week 52.
• Ruxolitinib cream was not associated with clinically significant application site reactions or serious treatment-related adverse events.
• Most relevant TEAEs include acne and possible application site reactions.

• Ruxolitinib cream monotherapy produced substantial facial and total body repigmentation of vitiligo lesions after week 24.
• Continued improvement was seen through 52 weeks of treatment (highest responses with 1.5% BID), suggesting that ruxolitinib cream is an effective treatment option for patients with vitiligo.
• A longer duration of therapy was associated with greater repigmentation, objectively assessed using the VASI.
  • Near-complete facial repigmentation as assessed by F-VASI75.
  • Substantial total body repigmentation as assessed by T-VASI50.
• All doses of ruxolitinib cream were well tolerated, and no treatment-related serious AEs were reported.