Abstract

The Japanese Society of Hematology 2011 Allogeneic hematopoietic stem cell transplantation (HSCT) has been established as an effective treatment for hematological disorders. However, graft-versus-host disease (GVHD) remains a major problem following allogeneic HSCT. The development of GVHD is strongly associated with an increased transplant-related mortality and impaired quality of life. The development of GVHD, however, may also decrease the incidence of relapse of hematological malignancies through the graft-versus-leukemia/lymphoma (GVL) effect. Nevertheless, the increased transplantedrelated mortality due to GVHD overweighs the benefit of the GVL effect in most patients, as shown in one Japanese study [1], and strategies for the prevention or treatment of GVHD remain a major issue in an effort to improve HSCT outcome. The ideal level of immunosuppression for the control of GVHD can be difficult to determine, as excessive immunosuppression may increase the incidence of disease relapse and infectious complications. In addition, each immunosuppressant has its own toxicities such as renal toxicity associated with the use of calcineurin inhibitors. The combination of cyclosporine (CSA) and methotrexate (MTX) has been established as a standard regimen for the prophylaxis against acute GVHD based on a randomized controlled trial performed by the Seattle group [2]. Subsequently, three randomized trials showed that tacrolimus (TAC) is more effective than CSA in preventing acute GVHD, although no survival benefit was observed, due to increased toxicity in the TAC group [3]. There was a

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