Belumosudil in pediatric patients with SR-cGvHD

At the European Hematology Association (EHA) 2026 Congress, June 11–14, 2026, Stockholm, SE, Lipeng Liu presented results from a case series evaluating the efficacy and safety of belumosudil in pediatric patients with steroid-refractory (SR) chronic graft-versus-host disease (cGvHD) after multiple lines of therapy (LoT) (N = 49). The primary endpoint was overall response rate (ORR), with failure-free survival (FFS), overall survival (OS), and adverse events (AEs) also evaluated.

Key data: The best ORR was 81.6%, including a complete response (CR) in 38.7% of patients. The median time to response (TTR) was 6 weeks (range, 2–16). At 24 weeks, the duration of response (DoR) rate was 74.1% (95% confidence interval [CI], 66.5–81.7). Organ-specific ORRs were 94.9% in the gastrointestinal (GI) tract, 93.1% in the skin, 90.9% in the liver, 90.0% in the mouth, 87.5% in the eyes, and 64.3% in the lung. Overall FFS and OS rates were 77.0% (95% CI, 70.1–83.9) and 100%, respectively. No treatment-related discontinuations or deaths occurred. Most patients discontinued or reduced corticosteroids and other immunosuppressive regimens.

Key learning: Belumosudil demonstrated clinical activity, durable responses, and corticosteroid- and immunosuppressive-regimen-sparing potential in heavily pretreated pediatric patients with SR-cGvHD, supporting further prospective evaluation in this population.