Graft-versus-Host Disease Treatment: Predictors of Survival

Acute graft versus-host disease (aGVHD) following allogeneic hematopoietic cell transplant (HCT) is the major reason for nonrelapse mortality (NRM) and thus is a major determinant of long-term survival Clinical trials of new aGVHD treatments are needed to identify approaches that will ultimately improve upon HCT survival At present it is not clear how quickly response to GVHD treatment needs to be established to reliably categorize patients at high risk for death or to promptly identify those who might benefit from alternate treatment Therefore we analyzed time to response from onset of aGVHD treatment in 180 patients who were enrolled on a national randomized phase 11 aGVHD treatment clinical trial whose initial treatment of GVHD consisted of high dose steroids plus a second immunosuppressive agent The aim of this analysis was to determine whether time to aGVHD treatment response predicts patient outcomes especially survival We used response at 14 28 and 56 days from initiation of aGVHD treatment to categorize patients for NRM and survival Multivariate analyses and specificity/sensitivity analyses identified that day 28 response (complete or partial response) best categorized patients by NRM and survival at 9 months from start of aGVHD treatment If verified as a reliable predictor of late outcomes following other aGVHD treatment approaches day 28 response should serve as a standard early endpoint for future trials of aGVHD therapy Biol Blood Marrow Transplant 16 1693-1699 (2010) (C) 2010 American Society for Blood and Marrow Transplantation