Boosting CNI and PTCy with MMF to prevent extensive chronic GVHD in allo-HSCT for AML
Battipaglia G, Labopin M, Kulagin A, et al. Addition of Mycophenolate Mofetil to a Calcineurin Inhibitor and Post-Transplant Cyclophosphamide Results in Lower Incidence of Extensive Chronic Graft-Versus-Host Disease in HLA-Matched Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia in Complete Remission: A Matched-Pair Analysis on Behalf of the Acute Leukemia Working Party of the EBMT. Bone Marrow Transplantation. 2025; (doi: 10.1038/s41409-025-02610-5).
Evidence suggests that adding both a calcineurin inhibitor (CNI) and mycophenolate mofetil (MMF) to post-transplant cyclophosphamide (PTCy) may help prevent a common complication after HLA-matched allogeneic hematopoietic stem cell transplantation (allo-HSCT). Researchers conducted a retrospective investigation of 292 adults with acute myeloid leukemia (AML), all of whom underwent allo-HSCT with peripheral blood stem cells between 2010 and 2020. A total of 146 participants were treated with PTCy plus CNI, while 146 received PTCy plus CNI and MMF. At 2-year follow-up, extensive chronic GVHD had occurred in 16% of the PTCy+CNI recipients versus 6% of the PTCy+CNI+MMF group, with all other transplant outcomes comparable.