Abstract

Background: Hematopoietic stem cell transplantation (HSCT) offers curative potential for hematologic malignancies, but chronic graft-versus-host disease (cGVHD) remains a common and debilitating complication. Belumosudil, recently approved for cGVHD after two or more prior therapies, has shown promise in clinical trials, but real-world data are limited. Case series: This single-center case series evaluates the real-world tolerability, safety, and accessibility of belumosudil, particularly in combination with other therapies for cGVHD. We reviewed outcomes in 33 patients treated with belumosudil between July 2021 and August 2022. Data on patient demographics, treatment history, adverse effects, drug interactions, and cost/accessibility were collected and analyzed. Belumosudil was generally well tolerated; 45% reported adverse effects, most commonly diarrhea, dizziness, and appetite loss. Less than 10% discontinued due to side effects. Notably, blood glucose fluctuations and elevated sirolimus/tacrolimus levels were observed. Belumosudil was frequently used in combination with other therapies, including ruxolitinib and ibrutinib, with 21% of patients eventually discontinuing all other systemic therapies. The majority of patients had low or no out-of-pocket cost due to secondary payor assistance. Conclusion: Belumosudil appears to be a well-tolerated, accessible treatment option in real-world settings for patients with cGVHD, including those on combination therapies. Clinicians should monitor for potential drug interactions and metabolic disturbances. Further real-world research is needed to confirm these findings

Keywords

belumosudil, real-world data, combination therapy, immunosuppressant interactions, drug safety, ruxolitinib, ibrutinib, tacrolimus, sirolimus