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FDA Approves Ruxolitinib for Acute GVHD After Transplant
The FDA has approved ruxolitinib (Jakafi; Incyte) for the treatment of steroid-refractory acute graft-versus-host disease (GVHD) in patients aged ≥12 years who have undergone allogeneic hematopoietic stem-cell transplantation (HSCT).
The approval was based on results from the open-label, single-arm, multi-center Study INCB 18424-271, which enrolled 49 patients with grade 2 to 4 steroid-refractory acute GVHD after receipt of allogeneic HSCT. Patients received ruxolitinib 5 mg twice daily. If no toxicity occurred after 3 days, the dose could be increased to 10 mg twice daily.
The primary end points of the study were the day-28 overall response rate (ORR; defined as complete response, very good partial response, or partial response) and response duration calculated from day-28 response to progression, new salvage therapy for acute GVHD, or death from any cause.
The day-28 ORR was 100% for grade 2 acute GVHD, 40.7% for grade 3 acute GVHD, and 44.4% for grade 4 acute GVHD. The median response duration was 16 days (95% CI, 9-83), and the median time from day-28 response to death or new therapy for acute GVHD was 173 days (95% CI, 66-NE).
The most common (>50%) hematologic adverse reactions were anemia, thrombocytopenia, and neutropenia. The most common (>50%) nonhematologic adverse events were infections and edema.—Janelle Bradley
