calcineurin inhibitor + methotrexate + mycophenolate mofetil

This combination regimen, consisting of a calcineurin inhibitor (CNI), methotrexate (MTX), and mycophenolate mofetil (MMF), is an immunosuppressive strategy used for the prophylaxis of graft-versus-host disease (GVHD) in patients undergoing hematopoietic stem cell transplantation (HSCT), particularly in the HLA-haploidentical setting. The regimen utilizes three distinct mechanisms to suppress the immune response: the CNI (such as tacrolimus or cyclosporine) inhibits the phosphatase activity of calcineurin, thereby blocking T-cell activation; methotrexate acts as a folate antagonist that inhibits dihydrofolate reductase, suppressing the proliferation of immune cells; and mycophenolate mofetil inhibits inosine monophosphate dehydrogenase, which is required for de novo purine synthesis in lymphocytes. This triple therapy is being evaluated as a standard-of-care comparator in clinical trials sponsored by Zhejiang University to assess the efficacy of adding or substituting agents like ruxolitinib for improved GVHD outcomes.

Other names

CNI + MTX + MMF