Researchers evaluated the rate of failure-free survival (FFS) after belumosudil treatment for chronic graft-versus-host disease (cGHVD) and, according to primary investigator Aleksandr Lazaryan, found that “treatment with belumosudil resulted in high FFS rates compared with historic benchmarks in cGVHD refractory to prior LOTs.” The analysis was presented at the Transplantation & Cellular Therapy 2022 Tandem Meetings from the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).
The authors’ conclusions were based on an analysis of two belumosudil clinical trials, the dose-finding KD025-208 trail and the pivotal ROCKstar trials. A total of 186 patients treated with delumosudil 200mg QD, 200 mg BID, or 400 mg QD were included, of which 70% had severe cGVHD. Prior lines of therapy (LOTs) included tacrolimus (58%), sirolimus (46%), extracorporeal photopheresis (42%), mycophenolate mofetil (27%), ibrutinib (27%), and ruxolitinib (21%).
Researchers observed a median FFS of 14 months, with estimated overall FFS rates of 75% (95% confidence interval [CI], 68–81%), 54% (95% CI, 47–61%), and 38% (95% CI, 29–47%) at six, 12 and 24 months, respectively. The reasons for failure included recurrent malignancy (6%), non-relapse mortality (NRM; 7%), and the addition of a new systemic cGVHD therapy (43%). Finally, the factors linked to increased risk of failure included “progressive onset of cGVHD (multivariate hazard ratio [HR] = 2.1 [1.2–3.4]), absence of glucocorticoids in upfront therapy for cGVHD (HR = 2.2 [1.2–4.0]) and ?2 prior LOTs (HR=3.7 [1.2–12.2]).”
Lazaryan and colleagues summarized that that belumosudil yielded high FFS when compared to historic refractory cGVHD benchmarks, with low NRM and relapse rates, and suggested that the risk factors they identified “can inform risk stratification and prognostication of patients being treated with belumosudil.”