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Hematopoietic Stem Cell Transplantation This is the best therapy for the young patient with a fully histocompatible sibling donor (Chap. 108). Human leukocyte antigen (HLA) typing should be ordered as soon as the diagnosis of aplastic anemia is established in a child or younger adult. In transplant candidates, transfusion of blood from family members should be avoided so as to prevent sensitization to histocompatibility antigens; while transfusions in general should be minimized, limited numbers of blood products probably do not seriously affect outcome. For allogeneic transplant from fully matched siblings, long-term survival rates for children are 80–90%. . | Chapter 102. Aplastic Anemia Myelodysplasia and Related Bone Marrow Failure Syndromes Part 8 Hematopoietic Stem Cell Transplantation This is the best therapy for the young patient with a fully histocompatible sibling donor Chap. 108 . Human leukocyte antigen HLA typing should be ordered as soon as the diagnosis of aplastic anemia is established in a child or younger adult. In transplant candidates transfusion of blood from family members should be avoided so as to prevent sensitization to histocompatibility antigens while transfusions in general should be minimized limited numbers of blood products probably do not seriously affect outcome. For allogeneic transplant from fully matched siblings long-term survival rates for children are 80-90 . Transplant morbidity and mortality are increased among adults due mainly to the higher risk of chronic GVHD and serious infections. Graft rejection was historically a major determinant of outcome in transplant for aplastic anemia perhaps related to the underlying pathophysiology as well as to alloimmunization from transfusions the latter now much improved by leukocyte depletion before blood product administration . Most patients do not have a suitable sibling donor. Occasionally a full phenotypic match can be found within the family and serve as well. Far more available are other alternative donors either unrelated but histocompatible volunteers or closely but not perfectly matched family members. Survival using alternative donors is about half that of conventional sibling transplants but improving with higher-resolution HLA matching and more effective conditioning regimens and GVHD prophylaxis. Patients will be at risk for late complications especially a higher rate of cancer if radiation is used as a component of conditioning. Immunosuppression Used alone ALG or antithymocyte globulin ATG induces hematologic recovery independence from transfusion and a leukocyte count adequate to prevent infection in about 50 of patients. The
