A phase 3 Blood and Marrow Transplant Clinical Trials Network (BMT CTN) study of 304 randomized patients demonstrated that tacrolimus and sirolimus (Tac/Sir) was comparable to tacrolimus and methotrexate (Tac/Mtx) in preventing acute GVHD and early mortality after related donor hematopoietic cell transplantation (HCT). The primary end point of the trial was grades 2-4 acute GVHD-free survival at day 114. Results from the two arms revealed similar outcomes: 67% (Tac/Sir) vs. 62% (Tac/Mtx); p=0.38. However, neutrophil and platelet engraftment were more rapid in the Tac/Sir arm and oropharyngeal mucositis was less severe in the Tac/Sir arm. The results revealed a trend toward a reduction in severe, grades 3-4 acute GVHD in the Tac/Sir arm, and the authors therefore suggested that “the benefit of the Tac/Sir regimen might be more prominent in higher-risk transplantations, such as mismatched or unrelated donor HCT.”
Tacrolimus-Sirolimus Comparable to Tacrolimus-Methotrexate as GVHD Prophylaxis
Aug 2014