Clinical effectiveness and safety of belumosudil in chronic GVHD
Rathje K, Gagelmann N, Heidenreich S, et al. Belumosudil for Chronic Graft-Versus-Host Disease: A Systematic Review and Meta-Analysis. Transplantation and Cellular Therapy. 2025; (doi: 10.1016/j.jtct.2025.11.019).
Researchers agree on the robust potential of belumosudil as a treatment for chronic graft-versus-host disease (cGVHD), which can develop following allogeneic hematopoietic stem cell transplantation. Although the drug was cleared by the U.S. Food and Drug Administration in 2021 as a third-line intervention, a recent systematic review and meta-analysis showcases its safety and efficacy across diverse study designs and patient characteristics. The investigation included 16 studies, both prospective and retrospective in nature, and a total of 651 adult patients. One notable finding was the favorable overall response rate associated with belumosudil — as high as 73% among the prospective studies and 60% in the pooled analysis. Response was greatest in cGVHD cases with gastrointestinal or joint/fascia involvement, at 52% each, but low at 25% for patients with pulmonary cGVHD. Belumosudil was also noted for its safety profile. Pooled overall survival reached 87%, and adverse events were generally manageable despite their frequent occurrence. Additional research into patient-specific factors, including immune profiling and biomarkers of disease activity, could support personalized treatment strategies that improve patient outcomes, according to the analysis.