Long-Term Data Confirm Belumosudil's Durable Efficacy and Safety in Chronic GVHD
A pooled analysis of 3 clinical studies has confirmed the long-term efficacy and safety of belumosudil, an oral ROCK2 inhibitor approved for treatment of chronic graft-versus-host disease (cGVHD) after failure of at least 2 prior systemic therapies.
The integrated dataset included 208 patients from the KD025-208, ROCKstar, and KD025-217 studies, with a median follow-up of 31.4 months. Patients were treated with belumosudil at 3 dosing regimens. The best overall response rate (ORR) in the modified intent-to-treat population was 72%, with median duration of response (DOR) of 62.3 weeks and median failure-free survival (FFS) of 15.1 months.
“The long-term results from this pooled analysis... demonstrated belumosudil was associated with durable responses, and it remained well tolerated with no new safety concerns,” the authors wrote.
Belumosudil reduced corticosteroid use in 68% of patients and enabled 25% to discontinue steroids entirely. A similar reduction was observed in calcineurin inhibitor use. The median time to the next treatment (TTNT) was 22.1 months, with 47% of patients avoiding new systemic therapy for at least 3 years.
Responses were consistent across subgroups, including patients previously treated with ibrutinib or ruxolitinib, those with severe cGVHD, and those with multiple organ involvement. Patients receiving the FDA-approved 200 mg once-daily dose achieved a 73% ORR and showed durable clinical benefit.
Safety outcomes were consistent with earlier reports. Grade ≥3 adverse events included pneumonia, hypertension, and hyperglycemia. Serious adverse events (SAEs) occurred in 44% of patients, and drug-related SAEs in 7%.
The data support belumosudil as a long-term treatment option for patients with cGVHD, demonstrating durable clinical responses and a manageable safety profile. Further research is needed to define optimal sequencing with other cGVHD therapies.
Reference
Lee SJ, Pavletic S, Blazar BR, et al. Belumosudil for chronic graft-versus-host disease: analysis of long-term results from the KD025-208 and ROCKstar studies. Transplant Cell Ther. Published online May 1, 2025. doi:10.1016/j.jtct.2025.04.020.
