A phase 3, randomized, open-label trial of 329 patients (≥12 years) with steroid-refractory/dependent chronic graft-vs-host disease (SR/D-cGVHD) found that ruxolitinib significantly extended median failure-free survival (FFS) to 38.4 months versus 5.7 months with best available therapy (BAT) (HR 0.36).
At 36 months, 59.6% of ruxolitinib-treated patients maintained response vs 26.7% with BAT. Among BAT patients who crossed over, the week-24 overall response rate was 50%, with best overall response reaching 81.4%. Nonrelapse mortality was slightly lower with ruxolitinib (17.8% vs 22.0%).
The treatment showed sustained efficacy and manageable safety over 3 years, though some long-term endpoints (like quality of life) couldn’t be fully assessed due to dropouts.
Study led by Robert Zeiser, MD; funded by Novartis and Incyte; published June 25 in Journal of Clinical Oncology.