Ruxolitinib for chronic steroid-refractory graft versus host disease: a single center experience

Researchers at the University of Milan and the IRCSS San Rafaelle Scientific Institute in Italy have investigated the use of the JAK1/2 inhibitor Ruxolitinib for treating patients suffering from acute Graft Versus Host Disease (GVHD). Their study's cohort consisted of 36 patients with moderate or severe steroid refractory or steroid dependent chronic GVHD who were given Ruxolitinib.

Following hematopoietic stem cell transplantation (HSCT), patients can develop GVHD and require immunosuppressant (IST) steroid treatment in severe cases. However, repeated IST treatment and enduring GVHD can both result in tissue damage, thereby leading to poor patient prognosis. As such, researchers sought to determine the efficacy of Ruxolitinib for improving patient outcomes.

In this study, 36 patients received Ruxolitinib for a median duration of 8.6 months. An overall response of 59% at three months and 62% at six months was observed. Post-treatment outcomes at the one- and two-year mark were positive, with an overall survival (OS) rate of 81% and 74% respectively, and a relapse rate of 0% and 11%. To further improve the OS rate, researchers note that additional focus should be given toward the optimal timing of treatment, so that patients suffering from GVHD can receive the fastest clinical response possible. These findings indicate that Ruxolitinib is a safe and effective treatment for advanced stage GVHD.


Ferreira AM, Pontes da Silva CA, Pereira AD, et al. Ruxolitinib in steroid-refractory chronic graft-versus-host disease: experience of a single center. Bone Marrow Transplant. 53(4):503-506 (2018).


Tags: GVHD, transplantation, cell therapy, chronic, single center, experience, ruxolitinib, steroid

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