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Ruxolitinib: a potential treatment for corticosteroid refractory acute graft-versus-host disease. Expert Opin Investig Drugs 2020 May;29(5):423-427



Pubmed ID




Scopus ID

2-s2.0-85083651597   5 Citations


INTRODUCTION: Allogeneic transplantation represents a potentially curative procedure for various lethal conditions; however, it is associated with serious immune mediated complications. The most serious complication is graft-versus-host disease (GVHD). Acute GVHD (aGVHD) is unresponsive to initial corticosteroid therapy in ~40% of cases. Corticosteroid-refractory aGVHD (CR-aGVHD) represents a significant unmet need with nearly 60% mortality in patients developing this state.

AREAS COVERED: We review landmark trials which led to the US FDA approval of Ruxolitinb for the treatment of CR-aGVHD. Ruxolitinib phosphate is a Janus Kinase 1 and 2 inhibitor which inhibits pro-inflammatory signaling, and modulates T-cell subsets to an anti-inflammatory phenotype. Ruxolitinib was recently prospectively studied for the treatment of CR-aGVHD in the REACH1 trial, which added Ruxolitinib to corticosteroid therapy. This trial demonstrated favorable results with 73% of patients ultimately responding. Among responders, over 70% remained alive at 6 months. These results led to the US FDA to approve ruxolitinib as treatment for CR-aGVHD, the first approval of its kind.

EXPERT OPINION: Ruxolitinib represents the standard option for CR-aGVHD. Future studies should identify patients less likely to respond to ruxolitinib and/or focus on a more targeted approaches to reduce infectious complications and cytopenias seen with this drug.

Author List

Abedin SM, Hamadani M


Sameem Abedin MD Assistant Professor in the Medicine department at Medical College of Wisconsin
Mehdi H. Hamadani MD Professor in the Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adrenal Cortex Hormones
Graft vs Host Disease
Hematopoietic Stem Cell Transplantation
Janus Kinase 1
Janus Kinase 2