LAUSR

Hematopoietic stem cell transplantation (bone marrow transplantation [BMT]) is the only curative treatment of severe aplastic anemia. BMT from an human leukocyte antigen (HLA)-matched sibling donor is the standard of care for young patients; immunosuppressive therapy is used for older patients or those lacking matched sibling donors. Patients with refractory or relapsed disease are increasingly treated with HLA haploidentical BMT. Historically, haploidentical BMT led to high rates of graft rejection and graft-versus-host disease. High-dose post transplant cyclophosphamide, which mitigates the risk of graft-versus-host disease, is a major advance. This article provides an overview of the haploidentical BMT approach in severe aplastic anemia.