Ruxolitinib Cream in Lichen Sclerosus: Phase 2 Trial Results

Key Takeaways

• Greater improvement in total Clinical Lichen Sclerosus Score was observed with ruxolitinib than with vehicle at week 12.
• The proportion reaching Itch NRS4 at week 12 was similar in the ruxolitinib and vehicle groups.
• Application-site reactions were infrequent, no serious adverse events were reported with ruxolitinib cream, and treatment duration was relatively short.

In a phase 2 trial of ruxolitinib cream in anogenital lichen sclerosus, 1.5% ruxolitinib reduced total Clinical Lichen Sclerosus Score more than vehicle at week 12, -5.79 versus -3.03. The randomized, double-blind, vehicle-controlled study enrolled adult women with anogenital lichen sclerosus and focused on week 12 outcomes. Between-group separation was clearer for measured clinical signs than for patient-reported itch across the controlled comparison. The primary itch endpoint did not differ from vehicle at week 12.

Investigators randomized 61 adult women 1:1 to twice-daily 1.5% ruxolitinib cream or vehicle for 12 weeks during the controlled phase. Entry criteria required anogenital lichen sclerosus affecting 10% or less body surface area and an Investigator’s Global Assessment score of at least 2. Patients also needed a baseline Itch Numerical Rating Scale score of at least 4 before randomization. After week 12, all patients moved to ruxolitinib cream once or twice daily through week 24 after the randomized phase ended. The primary endpoint was a 4-point or greater improvement from baseline in Itch NRS at week 12.

At week 12, Itch NRS4 was achieved by 35.7% of the ruxolitinib group and 40.0% of the vehicle group, with no between-group difference (P=0.737). Over the same interval, total Clinical Lichen Sclerosus Score changed by -5.79 with ruxolitinib and -3.03 with vehicle (P=0.019). The prespecified symptom endpoint and the sign-based assessment showed different comparative patterns in the primary analysis, and the numeric itch response proportions did not favor active treatment. Clinical signs improved more with ruxolitinib than with vehicle, while itch responses remained similar between groups. That contrast defined the week 12 results for the randomized phase.

Application-site reactions were infrequent, occurring in 4 patients and limited to grade 1 or 2 events. No serious adverse events were reported with ruxolitinib cream during the summarized period. The treatment was described as generally well tolerated in adult women with anogenital disease. The authors also noted that treatment duration was relatively short for this study. Over 12 weeks, the reported pattern remained clearer for vehicle-controlled improvement in clinical signs than for itch, alongside infrequent local reactions and no serious adverse events.