Topical ruxolitinib for chronic cutaneous graft-versus-host disease: a single-centre, randomised, double-blind, intra-patient-controlled phase 2 trial.
Alina Markova, Jelena Ostojić, Veronica Rotemberg et al.
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In 30 seconds
This single-centre, randomised, double-blind, intra-patient-controlled phase 2 trial evaluated the efficacy and safety of topical ruxolitinib 1.5% cream in patients with chronic cutaneous graft-versus-host disease (cGVHD). Among 23 evaluable patients, the mean affected lesional body surface area decreased from 14.3% to 6.2% with ruxolitinib, compared to a decrease from 14.5% to 10.4% with placebo, resulting in a significant difference of -4.2 percentage points (p=0.0030).
Key findings
- Mean affected lesional body surface area decreased from 14.3% to 6.2% with ruxolitinib.
- Placebo group showed a decrease from 14.5% to 10.4%, resulting in a difference of -4.2 percentage points.
- Adjusted analysis confirmed a significant difference of -4.0 percentage points (p<0.0010).
- No treatment-related deaths occurred, with a favorable safety profile.
Why it matters
The findings suggest that topical ruxolitinib may provide a significant therapeutic option for patients with cutaneous cGVHD, potentially reducing reliance on corticosteroids and their associated adverse effects.