Journal article

Ruxolitinib for Glucocorticoid-Refractory Acute Graft-versus-Host Disease

Robert Zeiser, Nikolas von Bubnoff, Jason Butler, Mohamad Mohty, Dietger Niederwieser, Reuven Or, Jeff Szer, Eva M Wagner, Tsila Zuckerman, Bruyere Mahuzier, Judith Xu, Celine Wilke, Kunal K Gandhi, Gerard Socie

New England Journal of Medicine | MASSACHUSETTS MEDICAL SOC | Published : 2020

Abstract

BACKGROUND: Acute graft-versus-host disease (GVHD) remains a major limitation of allogeneic stem-cell transplantation; not all patients have a response to standard glucocorticoid treatment. In a phase 2 trial, ruxolitinib, a selective Janus kinase (JAK1 and JAK2) inhibitor, showed potential efficacy in patients with glucocorticoid-refractory acute GVHD. METHODS: We conducted a multicenter, randomized, open-label, phase 3 trial comparing the efficacy and safety of oral ruxolitinib (10 mg twice daily) with the investigator's choice of therapy from a list of nine commonly used options (control) in patients 12 years of age or older who had glucocorticoid-refractory acute GVHD after allogeneic st..

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