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

RCT (n=309) found that ruxolitinib therapy led to significant improvements in overall response at day 28 compared to control group (62% vs. 39%; OR 2.64; 95% CI, 1.65 to 4.22; p<0.001) with a higher incidence of thrombocytopenia, the most frequent toxic effect.

SPS commentary:

According to an editorial, despite hundreds of approaches in murine models and dozens in patients, no progress has been made in determining which drug will be effective after glucocorticoid treatment has failed to control the disease. It applauds the authors for completing this successful randomised trial and it notes that given the effect of ruxolitinib in controlling glucocorticoid-refractory GVHD, it is interesting that the incidence of infectious complications or relapse was apparently not greater with ruxolitinib than with control therapy, though the total follow-up time was short. These observations raise important questions and set the stage for further work in this area.


New England Journal of Medicine

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