Photo Credit: Meletios Verras

The following is a summary of “Methotrexate Versus Mycophenolate Mofetil Prophylaxis in Allogeneic Hematopoietic Cell Transplantation for Chronic Myeloid Malignancies: A Retrospective Analysis on Behalf of the Chronic Malignancies Working Party of the EBMT” published in the November 2024 issue of Hematology by Luft et al. 

Prophylaxis strategies for Graft vs Host Disease (GVHD) in allogeneic hematopoietic cell transplantation (allo-HCT) include combining calcineurin inhibitors (CNI) with methotrexate (MTX) or mycophenolate mofetil (MMF). 

Researchers conducted a retrospective study to compare outcomes in chronic myeloid malignancies and secondary acute myeloid leukemia (sAML) between MTX- and MMF-based prophylaxis. 

They performed a retrospective study using the EBMT registry, which included 13,699 patients from 321 centers transplanted between 2007 and 2017 with MTX- or MMF-based prophylaxis alongside a CNI. The study assessed overall survival, relapse-free survival (RFS), relapse incidence, non-relapse mortality (NRM), and Grades 2–4 acute GVHD (aGVHD), with a median follow-up of 42.8 months. 

The results showed that MTX prophylaxis reduced overall mortality (HR 0.87, P = 0.001) and NRM (HR 0.86, P = 0.006) compared to MMF. No differences were found in relapse risk (HR 1.03, P = 0.53) or RFS (HR 0.95, P = 0.12). MTX lowered Grades 2–4 aGVHD risk and improved survival after aGVHD but showed no impact on outcomes in patients without aGVHD at 3 months. 

Investigators found that MTX-complemented CNI prophylaxis improved survival and reduced GVHD compared to MMF. 

Source: onlinelibrary.wiley.com/doi/10.1002/ajh.27531