Ruxolitinib is an oral inhibitor of Janus kinase 1 (JAK1) and JAK2 that is approved for patients with steroid-refractory or steroid-dependent chronic graft-versus-host disease (cGVHD) who have failed at least 2 prior lines of therapy. Researchers, led by Jennifer White, sought to expand available data on real-world ruxolitinib treatment.
They reviewed outcomes of heavily pretreated patients with cGVHD who received ruxolitinib and found that roughly half of patients responded at months 6 and 12, and many patients were able to completely taper off prednisone at 1 year. Their findings were published in Transplantation and Cellular Therapy.
Real-world Evidence Supports Efficacy of Ruxolitinib
This retrospective study included 115 patients with steroid-refractory cGVHD who previously failed systemic therapies from 5 transplant centers. Authors noted 60% of the population had severe cGVHD and 82% (n=96) had received 3 or more prior lines of therapy before ruxolitinib. Clinical benefit was defined by a combination of objective response rate (ORR) and steroid use reduction.
According to the authors, the ORR at 3, 6, and 12 months was 48.6%, 54.9%, and 48.5%, respectively, and a clinical benefit was identified in 58.7%, 64.8%, and 60.6% of patients at the same time points. In addition, 37.9% of the patients were able to taper prednisone to a daily dose <0.1 mg/kg at 1 year. Failure-free survival at 12 months was 64.6% (95% CI, 54.1-73.2).
The researchers also reported that patients with severe cGVHD had greater risk of failure due to therapy switch, and patients with a pretransplant hematopoietic cell transplantation comorbidity index score ≥3 exhibited high failure due to increasing nonrelapse mortality risk.
“This study demonstrates the therapeutic efficacy of ruxolitinib for cGVHD in a heavily pretreated real-world population,” the authors summarized.