This phase 3 multi-center randomized double-blinded trial examined whether mycophenolate mofetil (MMF) plus corticosteroids was superior to corticosteroids alone as initial therapy for acute GVHD. Eligible patients were those who had received HCT and required systemic therapy for newly diagnosed acute GVHD. The primary end point was acute or chronic GVHD-free survival at day 56 after initiation of therapy. In this Blood and Marrow Transplant Clinical Trials Network (BMT CTN) study, patients were randomized to receive prednisone with either MMF (n=116) or placebo (n=119). There were no significant differences in outcomes between the treatment arms for day 56 GVHD-free survival in patients who developed Grades III-IV acute GVHD (MMF, 54.1%; placebo, 51.2%; p=0.8) and those with liver/gut involvement (MMF, 51.6%; placebo, 60.3%, p=0.34). Complete response rates at day 56 were also comparable: MMF 60.3% vs. placebo 53.8% (p=0.31). The authors concluded that “the addition of MMF to corticosteroids as initial therapy for acute GVHD does not improve GVHD-free survival compared with corticosteroids alone.”
MMF Plus Corticosteroids Does Not Improve Acute GVHD Treatment Over Corticosteroids Alone
Nov 2014