Real-world outcomes of belumosudil in SR-cGvHD: Canadian compassionate use program

A multicenter, real-world study evaluated belumosudil therapy in 46 patients with steroid-refractory chronic graft-versus-host disease (cGvHD) under a Canadian compassionate program, including combination therapy with ruxolitinib. Results were published by Rodriguez-Rodriguez et al. in Annals of Hematology.

Key data: After a median follow-up of 11.4 months, the best overall response rate (ORR) was 52%. The failure-free survival (FFS) and overall survival (OS) rates at 12 months were 64.3% and 91.1%, respectively. No Grade 4 adverse events (AEs) occurred; 4% of patients had Grade 3 AEs, and 11% had Grade 2 AEs. Combination therapy with ruxolitinib exhibited equivalent treatment outcomes to belumosudil monotherapy, although patients treated with combination therapy presented with more advanced GvHD and had mostly failed ruxolitinib therapy.

Key learning: Belumosudil demonstrated durable efficacy and a favorable safety profile in heavily pretreated cGvHD. Future investigations should explore combination approaches and identify biomarkers to optimize patient selection.