October 2021: After failure of one or two lines of systemic therapy, the Food and Drug Administration approved ruxolitinib (Jakafi, Incyte Corp.) for chronic graft-versus-host disease (cGVHD) in adults and children aged 12 and up.
REACH-3 (NCT03112603) compared ruxolitinib to best available therapy (BAT) in a randomised, open-label, multicenter clinical trial for corticosteroid-refractory cGVHD after allogeneic stem cell transplantation. 329 individuals (1:1) were randomly assigned to receive either ruxolitinib 10 mg twice daily or BAT in this study.
Overall response rate (ORR) (2014 NIH Response Criteria) through cycle 7 day 1 was the key efficacy result utilised to support approval. The ORR for the ruxolitinib arm was 70% (95 percent CI 63 percent, 77 percent) and the ORR for the BAT arm was 57 percent (95 percent CI 49 percent, 65 percent), a difference of 13%. (95 percent CI 3 percent , 23 percent ). The median durations of response for the ruxolitinib and BAT arms, computed from first response to progression, death, or new systemic treatments for chronic GVHD, were 4.2 months (95% CI 3.2, 6.7) and 2.1 months (95% CI 1.6, 3.2), respectively. The median durations from first response to death or new systemic therapy for cGVHD in the ruxolitinib and BAT arms were 25 months (95 percent CI 16.8, NE) and 5.6 months (95 percent CI 4.1, 7.8), respectively.
Anemia and thrombocytopenia were the most common hematologic adverse responses of ruxolitinib in patients with cGVHD (incidence > 35 percent). Infections and viral infection were the most prevalent nonhematologic side effects (incidence 20%).
For cGVHD, a beginning dose of ruxolitinib of 10 mg twice daily is indicated.