Wintrobe’s Clinical Hematology
11th Edition

25
Hematopoietic Stem Cell Transplantation
Richard A. Nash
Pluripotent hematopoietic stem cells (HSCs) have the ability to reconstitute immunohematopoiesis after myeloablative therapy and transplantation (1). There are many potential sources for HSCs, which may be used for transplantation, including both the marrow and peripheral blood from the patient (autologous), family members, or volunteer unrelated donors. Umbilical cord blood has also been identified as a potential source of HSCs. HSC transplantation (HSCT) is done for a variety of therapeutic indications: (a) to support hematopoiesis after myeloablative doses of total body irradiation (TBI) and chemotherapy used in the treatment of hematologic malignancies, (b) to establish a graft-versus-leukemia (GVL) reaction, or (c) to replace diseased tissues of hematologic or immunologic origins. The advances in supportive care after transplantation have resulted in HSCT being a therapeutic modality that is highly successful in otherwise fatal diseases (2,3). The focus of this chapter is on the general indications for HSCT, sources of stem cells, conditioning regimens, transplant biology issues, and complications of HSCT; other chapters provide detailed discussions of the indications and results of HSCT in relation to specific diseases (Table 25.1).
TABLE 25.1. Disorders Treated by Bone Marrow or Stem Cell Transplantation

Disease Chapter

Nonmalignant  
  Aplastic anemia 44
  Fanconi anemia 45
  Diamond-Blackfan syndrome 45
  Sickle cell disease 40
  Thalassemia 42
  Paroxysmal nocturnal hemoglobinuria 37
  Myelofibrosis 86
  Congenital neutropenia 63
  Chédiak-Higashi syndrome 64
  Chronic granulomatous disease 64
  Glanzmann thrombasthenia 58
  Osteopetrosis
  Gaucher disease 65
  Mucopolysaccharidosis
  Mucolipidoses 65
  Immune deficiencies 68 and 69
Malignant  
  Acute nonlymphoblastic leukemia 80 and 81
  Acute lymphoblastic leukemia 78 and 79
  Hairy cell leukemia 93
  Myelodysplasia 83
  Chronic myelogenous leukemia 84
  Chronic lymphocytic leukemia 92
  Hodgkin disease 95
  Non-Hodgkin lymphoma 90
  Multiple myeloma 98
  Solid tumors