TOPLINE:
Ruxolitinib demonstrated superior long-term outcomes in steroid-refractory/dependent chronic graft-vs-host disease (SR/D-cGVHD) with median failure-free survival of 38.4 months compared with 5.7 months for the best available therapy (BAT). The treatment maintained efficacy through 3 years, with 59.6% of ruxolitinib-treated patients sustaining response at 36 months compared with 26.7% with BAT.
METHODOLOGY:
Researchers conducted a randomized, phase 3, open-label, multicenter study involving 329 patients aged ≥ 12 years with moderate/severe steroid-refractory/dependent chronic GVHD.
Participants received either ruxolitinib (10 mg twice daily) or investigator-selected BAT in a 1:1 randomization for 24 weeks during the primary efficacy period.
Analysis included a follow-up period