Prophylaxis of acute graft-versus-host disease

Abstract

Graft-versus-host disease (GVHD) is an important complication in allogeneic hematopoietic stem cell transplantation. The standard regimen for GVHD prophylaxis in bone marrow transplantation and peripheral blood stem cell transplantation from human leukocyte antigen-matched donors is a combination of a calcineurin inhibitor, such as cyclosporin or tacrolimus, and short-term methotrexate. This regimen is well researched and has been nearly optimized in terms of infusion methods and the target blood concentration. The use of mycophenolate mofetil instead of methotrexate has the advantage of a lower incidence and severity of oropharyngeal mucositis. The addition of anti-thymocyte globulin significantly decreases the incidences of severe acute GVHD and extensive chronic GVHD, but does not lead to a significant improvement in overall survival. The GVHD prophylaxis regimens in cord blood transplantation and HLA-haploidentical donor transplantation have not been standardized. The efficacy of other immunosuppressive drugs, regulatory T lymphocytes, and mesenchymal stem cells are currently under investigation. Establishment of new evidence for GVHD prophylaxis from Japan is expected.