Hematopoietic Cell Transplantation for Aplastic Anemia

Aplastic anemia (AA) is a rare disease characterized by pancytopenia and marrow parenchyma that is lacking hematopoietic elements. This chapter summarizes the results of marrow hematopoietic cell transplantation (HCT) for severe AA over the past 40 years. Early reports of marrow HCT for SAA identified three major transplant-related problems: graft rejection; acute graft-versus-host disease (GVHD); and chronic GVHD. The largest number of transplants for SAA performed to date have been from human leukocyte antigen (HLA)-matched sibling donors. There are at least two reasons for the improved survival of patients with AA who were treated with HLA-identical HCT. One is the decreased incidence of graft rejection. The second reason for improved survival has been a decrease in the incidence and severity of acute GVHD through the introduction of better GVHD prevention regimens, such as combined MTX-CSP.