Validation of Minnesota acute graft-versus-host disease Risk Score

Using multicenter data, we developed a novel acute graft- versus-host disease Risk Score which more accurately predicts response to steroid treatment, survival and transplant related mortality than other published risk scores based upon clinical grading criteria. 1 To validate this Risk Score in a contemporary cohort, we examined 355 recent University of Minnesota patients (2007-2016) diagnosed with acute graft- versus-host disease and treated with prednisone 60 mg/m 2/day for 14 days, followed by an 8-week taper. Overall response [complete response + partial response] was higher in the 276 standard risk versus 79 high risk graft- versus-host disease patients at day 14 (71% versus 56%, P<0.01), day 28 (74% versus 59%, P=0.02) and day 56 (68% versus 49%, P<0.01) after steroid initiation. Day 28 response did not differ by the initial graft- versus-host disease grade. In multiple regression analysis, patients with high risk graft- versus-host disease were less likely to respond at day 28 (odds ratio 0.5, 95% CI 0.3-0.9, P<0.01) and had higher risks of 2 year transplant related mortality (Hazard Ratio 1.8, 95% CI, 1.0-2.1, P=0.03) and overall mortality (Hazard Ratio 1.7, 95% CI, 1.2-2.4, P<0.01) than patients with a standard risk graft- versus-host disease. This analysis confirms the Minnesota graft- versus-host disease Risk Score as a valuable bedside tool to define risk in patients with acute graft- versus-host disease. A tailored approach to upfront acute graft- versus-host disease therapy based upon the Minnesota Risk Score may improve outcomes and facilitate testing of novel treatments in these patients.