Ruxolitinib Versus Best Available Therapy in Patients With Steroid-Refractory Acute Graft-Versus-Host Disease: Final Analysis From the Randomized Phase III REACH2 Trial

Abstract

Approximately 30%-50% of patients develop acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic cell transplantation (Allo-HCT), representing a major limitation of this treatment. Although corticosteroids remain the standard first-line therapy for aGVHD, up to 50% of patients become steroid-refractory (SR). REACH2 is a phase III study of ruxolitinib versus best available therapy (BAT) in patients age 12 years and older with SR-aGVHD after Allo-HCT. We present the final efficacy and safety outcomes from REACH2 after 24 months of treatment. Cumulative median (range) duration of response was 167 (22-677) days with ruxolitinib and 106 (10-526) days with BAT. Median overall survival and event-free survival were 10.7 and 8.3 months for ruxolitinib, compared with 5.8 and 4.2 months, respectively, with BAT. Median failure-free survival was significantly longer with ruxolitinib than with BAT (4.86 v 1.02 months, P