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

Abstract

Effective nonsteroidal treatment options for patients with lichen sclerosus (LS) are limited. To evaluate the efficacy and safety of 1.5% ruxolitinib cream in adult women with anogenital LS. 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. 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. Treatment duration was relatively short. Ruxolitinib cream significantly improved clinical signs of LS, but not itch, versus vehicle and was generally well tolerated in women with LS.