Ruxolitinib cream for cutaneous cGVHD clears phase II study

Topical ruxolitinib demonstrates significant clinical activity while having a favourable harms profile in the treatment of patients with cutaneous chronic graft-versus-host disease (cGVHD) in a phase II study.

The study included 24 patients aged ≥12 years (median age 47.5 years, 54 percent female) who had a history of allogenic haematopoietic stem cell transplantation and developed cutaneous cGVHD involving ≥2 percent body surface area (BSA). These patients received both ruxolitinib 1.5% cream and placebo vehicle, with the active treatment applied to one side of the body and the vehicle to the contralateral lesional skin as control. Treatment was applied twice daily for 28 days.

The absolute between-side difference in affected lesional BSA was measured as the primary endpoint. Only patients who completed the day 28 visit were included in the primary endpoint analysis, while all patients who received any study treatment were included in the safety analysis. The median follow-up was 128 days.

Among the 23 evaluable patients, mean affected lesional BSA decreased from 14.3 percent at baseline to 6.2 percent with ruxolitinib and from 14.5 percent to 10.4 percent with placebo vehicle (adjusted difference, –4 percentage points, 95 percent confidence interval, –5.5 to –2.4; p<0.0010).

In terms of safety, grade 3–5 treatment-emergent adverse events occurred in three separate patients, with individual cases of central nervous system leukaemia, sinusitis, and lung infection.

The findings point to the potential of ruxolitinib cream as a steroid-sparing, skin-directed alternative or second-line therapy for patients with cutaneous cGVHD.