Efficacy and safety of ruxolitinib cream in patients with lichen sclerosus: Results from a phase 2, randomized, double-blind, vehicle-controlled study

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 at week 12. RESULTS: Sixty-one patients were randomized. At week 12, similar proportions of patients in the ruxolitinib cream and vehicle groups achieved ≥4-point improvement from baseline in Itch NRS (35.7% vs 40.0%, P = .737). The ruxolitinib cream group had greater reductions in total Clinical Lichen Sclerosus Score versus vehicle (-5.79 vs -3.03; P = .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.