Efficiency and Toxicity of Ruxolitinib as the Salvage Treatment in Steroid-Refractory Acute Graft-Versus-Host Disease after Haplo-Identical Stem Cell Transplantation.

Haplo-identical stem cell transplantation (haplo-SCT) for hematological malignancies has ushered in a new era in which everyone has a potential donor. However, the occurrence of steroid-refractory acute graft-versus-host disease (SR-aGVHD), with no priority among second-line therapies, leads to late mortality after haplo-SCT. Ruxolitinib is the first drug recommended for SR-aGVHD. Here, we report the outcome data from 40 patients after haplo-SCT following the Beijing Protocol who had received ruxolitinib as a salvage therapy for grades II to IV SR-aGVHD in our center between November 2017 and May 2019. The overall response rate was 85% (34/40; 95% confidence interval [CI], 73.4% to 96.6%), including 25 patients with complete response. The median time to first response was 10 days. The levels of inflammatory cytokines and T cell activation declined, and the percentage of regulatory T cells increased. The rate of GVHD relapse was 26.5% (9/34; 95% CI, 10.8% to 42.1%) in responders. Cytomegalovirus reactivation and cytopenia were the major adverse events after ruxolitinib was begun (57.5% and 60%, respectively). The 6-month overall survival estimate was 56.8% (95% CI, 41.5% to 72.1%), and the event-free survival was 45% (95% CI, 29.7% to 60.3%). Liver GVHD was associated with a worse response rate and poor survival. Collectively, ruxolitinib could be an effective treatment for SR-aGVHD patients after haplo-SCT.