Mycophenolate mofetil vs. methotrexate for the prevention of graft-versus-host-disease – Systematic review and meta-analysis

We performed a systematic review and meta-analysis of all trials comparing MMF and methotrexate as GVHD prophylaxis. Our search yielded 11 studies; 3 were randomized-control trials (RCTs). While the incidence of grades 2–4 acute GVHD was comparable, the incidence of grades 3 and 4 acute GVHD was higher in patients given MMF (RR 1.61; 95% CI 1.18–2.30). Incidence of mucositis was lower (RR 0.35; 95% CI 0.25–0.49) and time to engraftment was shorter (mean difference (−3.6); 95% CI −5.5 to −1.7) in patients given MMF. All other analyzed transplantation outcomes were comparable. We conclude that MMF, compared to methotrexate, is associated with increased severity of acute GVHD. Robustness of these results is hampered by the small number of RCTs.