Zeiser Robert, Polverelli Nicola, Ram Ron, et al. Ruxolitinib for Glucocorticoid-Refractory Chronic Graft-versus-Host Disease. New England Journal of Medicine. 2021; 385 (3): 228 (doi: 10.1056/NEJMoa2033122).
A Phase III study shows that ruxolitinib, a Janus kinase inhibitor, has clinical benefit for patients with glucocorticoid-refractory or -dependent manifestations of chronic graft-versus-host disease. The REACH3 study included 329 patients aged 12 years and older, half of whom were randomly assigned to take 10 mg of ruxolitinib twice per day. The remainder underwent control therapy — the investigator's choice among 10 commonly used treatment options considered best available care. Overall response, measured at week 24, was 49.7% in the group treated with ruxolitinib and 25.6% in the group that received standard best-available care. In addition to greater overall response, ruxolitinib was associated with increased failure-free survival and better symptom response than control. However, the study drug was also associated with a greater prevalence of thrombocytopenia and anemia.