Ruxolitinib cream effective for vitiligo treatment
USA: Ruxolitinib cream might be an effective treatment option for patients with vitiligo, suggests a recent study published in the journal LANCET. The study found ruxolitinib cream to be associated with substantial repigmentation of vitiligo lesions up to 52 weeks of treatment, and all doses were well tolerated.
Vitiligo is a chronic autoimmune disease that causes skin depigmentation and reduces the quality of life. No treatment has been approved until now for vitiligo repigmentation. Current off-label therapies have limited efficacies which emphasizes the need for improved treatment options. David Rosmarin, Tufts Medical Center, Boston, MA, USA, and colleagues investigated the therapeutic potential of ruxolitinib cream in vitiligo patients and reported the efficacy and safety results of up to 52 weeks of double-blind treatment.
The researchers performed a multicentre, randomized, double-blind, phase 2 study for vitiligo patients in 2 US hospitals and medical centers in 18 states. Patients were having a depigmentation of 0·5% or more of their facial body surface area (BSA) and 3% or more of their non-facial BSA. They were randomly assigned in the ratio 1:1:1:1:1 by use of an interactive response technology system to receive ruxolitinib cream (1·5% twice daily, 1·5% once-daily, 0·5% once daily, or 0·15% once daily) or vehicle (control group) twice daily on lesions constituting 20% or less of their total BSA for 24 weeks.
Control groups patients in addition to the patients in the 0·15% once-daily group who did not show a 25% or higher improvement from baseline in facial Vitiligo Area Scoring Index (F-VASI) at week 24 were re-randomized to one of three higher ruxolitinib cream doses (0·5% once-daily, 1·5% once-daily, 1·5% twice daily). Patients in the 0·5% once-daily, 1·5% once daily, or 1·5% twice-daily groups remained at their original dose up to week 52. Patients, investigators, and the study sponsor remained masked to treatment assignment throughout the study.
The primary endpoint was the proportion of patients achieving a 50% or higher improvement from baseline in F-VASI (F-VASI50) at week 24, assessed in the intention-to-treat population.
205 patients were screened for eligibility between June 7, 2017, and March 21, 2018. In all 48 were excluded and 157 patients (mean age, 48•3 years [SD 12•9]; 73 [46%] male and 84 [54%] female) were randomly assigned to either an intervention group or the control group.
Key findings of the study include:
- 32 (20%) of 157 were assigned to the control group, 31 (20%) to the 0·15% once-daily group, 31 (20%) to the 0·5% once-daily group, 30 (19%) to the 1·5% once-daily group, and 33 (21%) to 1·5% twice-daily group.
- F-VASI50 at week 24 was reached by significantly more patients given ruxolitinib cream at 1·5% twice daily (15 [45%] of 33) and 1·5% once daily (15 [50%] of 30) than were treated with vehicle (one [3%] of 32).
- Four patients had serious treatment-emergent adverse events (one patient in the 1·5% twice the daily group developed subdural hematoma; one patient in the 1·5% once daily group had a seizure; one patient in the 0·5% once-daily group had coronary artery occlusion; and one patient in the 0·5% once-daily group had oesophageal achalasia), all of which were unrelated to study treatment.
- Application site pruritus was the most common treatment-related adverse event among patients given ruxolitinib cream (one [3%] of 33 in the 1·5% twice-daily group; three [10%] of 30 in the 1·5% once-daily group; three [10%] of 31 in the 0·5% once-daily group; and six [19%] of 31 in the 0·15% once-daily group)with three [9%] of 32 patients showing application site pruritis in the control group.
- Acne was noted as a treatment-related adverse event in 13 (10%) of 125 patients who received ruxolitinib cream and one (3%) of 32 patients who received vehicle cream.
- All treatment-related adverse events were mild or moderate in severity and similar across treatment groups.
"Treatment with ruxolitinib cream was associated with substantial repigmentation of vitiligo lesions up to 52 weeks of treatment, and all doses were well tolerated. These data suggest that ruxolitinib cream might be an effective treatment option for patients with vitiligo," concluded the authors.
The study, "Ruxolitinib cream for treatment of vitiligo: a randomised, controlled, phase 2 trial," is published in the journal Lancet.