On May 24, the U.S. Food and Drug Administration (FDA) approved ruxolitinib (Jakafi) for steroid-refractory acute graft-vs-host disease (GVHD) in adult and pediatric patients 12 years and older.
Approval was based on the REACH1 trial/Study INCB 18424-271, an open-label, single-arm, multicenter study of ruxolitinib that enrolled 49 patients with steroid-refractory acute GVHD grades 2 to 4 (Mount Sinai Acute GVHD International Consortium criteria) occurring after allogeneic hematopoietic stem cell transplantation. Ruxolitinib was administered at 5 mg twice daily, and the dose could be increased to 10 mg twice daily after 3 days in the absence of toxicity.
The day-28 overall response rate was 100% for grade 2 GVHD, 40.7% for grade 3 GVHD, and 44.4% for grade 4 GVHD. The median response duration—calculated from day-28 response to disease progression, new salvage therapy for acute GVHD, or death from any cause (with disease progression being defined as worsening by one stage in any organ without improvement in other organs in comparison to prior response assessment)—was 16 days, and the median time from day-28 response to either death or the need for new therapy for acute GVHD (additional salvage therapy or increase in steroids) was 173 days.
In acute GVHD, the most common hematologic adverse reactions (incidence > 50%) are anemia, thrombocytopenia, and neutropenia. The most common nonhematologic adverse reactions (incidence > 50%) are infections and edema. ■