Ruxolitinib in acute and chronic graft-versus-host disease: real life long-term experience in a multi-center study for adult and pediatric patients, on behalf of the GETH-TC.

Abstract

Ruxolitinib has been approved for the treatment of adults and pediatric patients ≥12 years with steroid refractory graft-versus-host disease (GvHD). However, real-life studies are needed to confirm the results of clinical trials and further assess its efficacy in special populations. We performed a descriptive, retrospective, multi-center study of 352 adults and 42 pediatric patients treated with ruxolitinib for steroid-refractory acute or chronic GvHD. Among 119 and 233 adult patients with acute and chronic GvHD, overall response rate (ORR) was 58.8% (CR 33.6%) and 65.7% (CR 18.5%), respectively. Corticosteroids were withdrawn in 59.2% and 40.1%, and ruxolitinib in 47.2% and 34.8% in the acute and chronic groups of responders. Among 29 and 13 pediatric patients with acute and chronic GvHD, ORR was 82.7% (CR 51.7%) and 100% (CR 23%), respectively. Among responder patients, corticosteroids were withdrawn in 72.7% and 50%, and ruxolitinib in 75% and 30.7% in both groups respectively. Ruxolitinib in the real world setting, showed similar results as compared to clinical trials. Its efficacy is maintained in subsequent lines of treatment. In the pediatric population, the data are more favorable. In the long-term follow-up, corticosteroids, ruxolitinib and other inmunosuppressive drugs could be eliminated in a remarkably proportion of patients.