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 aged12. 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 aged12 years old.