A phase 2, randomized, multicenter study found belumosudil was well-tolerated and resulted in clinically meaningful responses among patients with chronic graft-versus-host disease (cGVHD) who had 2 or more treatment failures. These findings were published in the journal Blood.1
cGVHD affects up to 70% of patients undergoing allogeneic hematopoietic cell transplantation and is the leading cause of late nonrelapse mortality, morbidity, and impaired quality of life. As it can be difficult to treat, new therapies are needed to improve clinical trajectories. Belumosudil is an oral kinase inhibitor that affects the adaptive immune system and fibrosis, 2 mechanisms that occur during the cGVHD course.
Researchers recruited 132 patients with cGVHD who had received 2 to 5 lines of therapy from 28 centers in the United States between 2018 and 2020. Participants were randomly assigned to receive 200 mg belumosudil once a day (QD cohort) or twice a day (BID cohort).
The participants were median age 56 years (range, 21 to 77), 57% were men, 67% had severe cGVHD, and median number of involved organs was 4 (range, 0 to 7).
The overall response rate was 74% in the QD cohort and 77% in the BID cohort, and overall clinically significant improvement was observed in 59% and 62%, respectively. At 6 and 12 months, failure-free survival rates were 75% and 56%, respectively, across all study participants.
Grade 3 or greater adverse events were reported by 56% in the QD cohort and 52% in the BID cohort. Death occurred in 11% of the study population. Drug-related serious adverse events were reported for 5 participants in the QD cohort and 2 in the BID cohort.
This study was limited by not having a comparator group that did not receive belumosudil.
Belumosudil (Rezurock; Kadmon Pharmaceuticals LLC) was granted US Food and Drug Administration (FDA) approval on July 16, 2021 for the treatment of patients 12 years and older with cGVHD after failure of at least 2 prior lines of systemic therapy.2
Disclosure: Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.
- Cutler CS, Lee SJ, Arai S, et al. Belumosudil for chronic graft-versus-host disease (cGVHD) after 2 or more prior lines of therapy: the ROCKstar Study. Blood. 2021;blood.2021012021.doi:10.1182/blood.2021012021
- US Food and Drug Administration. FDA approves belumosudil for chronic grafter-versus-host disease [news release]. US Food & Drug Administration; July 16, 2021. Accessed July 23, 2021. https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-belumosudil-chronic-graft-versus-host-disease