Belumosudil in pediatric patients with steroid-refractory chronic graft-versus-host disease

Abstract Belumosudil has been introduced to treat steroid-refractory chronic graft-versus-host disease (SR-cGVHD) in adult patients. Similarly, cGVHD occurred in nearly 25% of pediatric allogeneic hematopoietic cell transplant (HCT) recipients. But the lack of data in pediatric SR-cGVHD patients contributes to poor prognosis, especially in young children aged<12. We aim to investigate the efficacy and safety of belumosudil in pediatric patients. This study included 13 HCT pediatric patients with SR-cGVHD failing multiple lines of treatment (LOTs), and all patients received belumosudil.The primary endpoint was the overall response rate (ORR), failure-free survival (FFS) and overall survival (OS), and adverse effects (AEs) during belumosudil administration were documented. The median time to response was 7 weeks (range, 3-18), and the ORR was 76.9%. The overall FFS and OS was 68.6% and 100% in patients received belumosudil, while 32.8% and 71.3% in the control group (P=0.048 and P=0.086). Belumosudil was well tolerated and the AE rates were comparable between the belumosudil and the control groups. Belumosudil was effective and well tolerated in pediatric patients with SR-cGVHD, and similar efficacy and safety was also confirmed in the patients aged<12 years old.