Efficacy and Safety of Ruxolitinib Cream in Patients With Lichen Sclerosus: Results From a Phase 2, Randomized, Double-Blind, Vehicle-Controlled Study.

Abstract

Background: Effective nonsteroidal treatment options for patients with lichen sclerosus (LS) are limited.

Objective: To evaluate the efficacy and safety of 1.5% ruxolitinib cream in adult women with anogenital LS.

Methods: This phase 2 study (NCT05593445) enrolled women with LS affecting ≤10% of their body surface area, Investigator's Global Assessment score ≥2, and baseline Itch Numerical Rating Scale (NRS) ≥4. Patients were randomized 1:1 to twice-daily application of 1.5% ruxolitinib cream or vehicle for 12 weeks, after which all patients applied ruxolitinib cream (once or twice daily) through Week 24. The primary endpoint was ≥4-point improvement from baseline in Itch NRS (Itch NRS4) at Week 12.

Results: Sixty-one patients were randomized. At Week 12, similar proportions of patients in the ruxolitinib cream and vehicle groups achieved Itch NRS4 (35.7% vs 40.0%, P=0.737). The ruxolitinib cream group had greater reductions in total Clinical Lichen Sclerosus Score versus vehicle (-5.79 vs -3.03; P=0.019). Application site reactions were infrequent (n=4; grade 1/2), and no serious adverse events were reported with ruxolitinib cream.

Limitations: Treatment duration was relatively short.

Conclusion: Ruxolitinib cream significantly improved clinical signs of LS, but not itch, versus vehicle and was generally well tolerated in women with LS.