Ruxolitinib Effective in Managing Graft-Versus-Host Disease

Ruxolitinib is both safe and effective at treating steroid-dependent/refractory acute and chronic graft-versus-host disease (GVHD), according to a study presented at the 2021 TCT Meetings of ASTCT & CIBMTR Digital Experience.

To assess the efficacy of ruxolitinib in a real-world setting, researchers analyzed 10 (four male, six female) consecutive patients with steroid-dependent/refractory GVHD post-hematopoietic cell transplantation (HCT). The average age at time of transplant was 19 years. Indicators of transplant, the researchers noted, included acute myeloid leukemia (n=3), thalassemia major (n=2), myelodysplastic syndromes (n=1), acute lymphocytic leukemia (n=1), aplastic anemia (n=1), Fanconi anemia (n=1), and severe combined immunodeficiency (n=1). The investigators noted that 80% of the study subjects received matched related donor transplant, while 20% received haploidentical HCT.

The GVHD treatments administered were cyclosporine plus methotrexate (80% of subjects), cyclosporine plus mycophenolate (n=1), and cyclosporine alone (n=1). One patient had steroid-refractory acute GVHD at day 63, five had overlap syndrome, and four had extensive chronic GVHD. Five patients (50%) had at least three prior lines of treatment, the researchers noted.

Ruxolitinib was commonly administered at 0.3 mg/kg or a flat dose of 20 mg daily, with an average treatment duration of 140 days.

The results showed the overall response rate for ruxolitinib was robust at 90% (complete response, 70%; partial response, 20%), and time to best response was 44 days (range, 10-95 days). Overall, ruxolitinib was well tolerated apart from “two patients developing tuberculosis and cytomegalovirus reactivation each and one developing pulmonary aspergillosis,” according to the researchers

Read more at:

Iftikhar R, et al. Ruxolitinib for Management of Graft Versus Host Disease: Real World Experience from a Developing Country. Published for the 2021 TCT Meetings of ASTCT & CIBMTR Digital Experience; February 8-12, 2021.