Belumosudil for pediatric cGvHD: A retrospective analysis

Results from a single-institution, retrospective analysis evaluating the efficacy and safety of belumosudil in pediatric patients with steroid-refractory or progressive chronic graft-versus-host disease (cGvHD) were published in Transplantation and Cellular Therapy by Anerao et al. A total of 11 patients were included in the analysis and had received treatment with belumosudil between September 2021 and July 2025.

Key data: All 11 patients responded to belumosudil, with 10 partial responses (PRs) and 1 complete response (CR). Dose reduction or discontinuation of corticosteroids or other cGvHD treatments was reported in 8 patients. Among patients with lung GvHD, forced expiratory volume in 1 second (FEV1) and diffusing capacity of the lungs for carbon monoxide (DLCO) remained stable throughout the belumosudil treatment course. There were no reports of systemic viremia, bacteremia, or fungemia. No immediate increases in serum creatinine above the upper limit of normal were observed, indicating that there was no nephrotoxicity or glomerular filtration rate (GFR) reduction.

Key learning: In this retrospective analysis, belumosudil demonstrated high response rates, a corticosteroid-sparing effect, and an acceptable safety profile in pediatric patients with steroid-refractory or progressive cGvHD. Prospective, multicenter studies are needed to further evaluate the efficacy and safety of belumosudil in this population.