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A topical cream could offer a gentler option for a tough-to-treat transplant complication. A phase 2 trial found that ruxolitinib 1.5% cream significantly reduced affected skin area in patients with cutaneous chronic graft-vs-host disease (cGVHD) compared to placebo — with no treatment-related deaths or dose modifications due to toxicity. Researchers say it could serve as a steroid-sparing, skin-directed alternative.
A randomized, double-blind, intra-patient–controlled phase 2 trial published in The Lancet Haematology found that ruxolitinib 1.5% cream (Opzelura) significantly reduced affected lesional body surface area (BSA) in patients with cutaneous chronic graft-vs-host disease (cGVHD) compared to placebo vehicle — and did so without serious treatment-related side effects.
The trial used a clever design: each patient had one body side treated with ruxolitinib cream and the other with placebo, allowing a direct head-to-head comparison within the same person. Improvements in clinician-assessed severity scores were already visible by day 14, and 74% of patients showed a clinical response on the ruxolitinib-treated side by day 28 — including 22% with complete responses.
By the Numbers:
Why it matters: Topical corticosteroids are the current standard for cutaneous cGVHD but carry risks like skin atrophy and systemic complications. Ruxolitinib cream — already approved for atopic dermatitis and vitiligo — could offer a steroid-sparing, targeted alternative, even for patients previously exposed to oral ruxolitinib.
