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Since its inception in the mid-20th century, the field of hematopoietic cell transplantation (HCT) has evolved from experimental animal models of marrow transplantation to curative therapy for tens of thousands of people yearly who are affected by a wide variety of marrow failure states, myeloid and lymphoid malignancies, immune deficiencies, genetic disorders and inborn errors of metabolism. Advances in transplantation immunobiology and improvements in supportive care have made this evolution possible and have ushered in the modern era of HCT. This chapter discusses the biologic principles and clinical applications of HCT, along with its future applications. Selected results demonstrating important principles are highlighted.

Acronyms and Abbreviations

ALK, anaplastic lymphoma kinase; ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; ASBMT, American Society for Blood & Marrow Transplantation; ASTCT, American Society for Transplantation and Cellular Therapy; ATG, anti-thymocyte globulin; BCNU, 1,3-bis(2-choloroethyl)-1-nitrosurea, carmustine; BEAM, BCNU/etoposide/cytarabine/melphalan; BU, busulfan; CEBPA, CCAAT enhancer-binding protein alpha gene; CHOP, cyclophosphamide, doxorubicin, vincristine, prednisone; CI, comorbidity index; CLL, chronic lymphocytic leukemia; CML, chronic myelogenous leukemia; CMV, cytomegalovirus; CR1, first complete remission; CSP, cyclosporine; CY, cyclophosphamide; CXCL12, stromal cell-derived factor-1; CXCR4, chemokine-related receptor; DAH, diffuse alveolar hemorrhage; DLI, donor lymphocyte infusion; EBMT, European Society for Blood and Marrow Transplantation; FDG-PET, fluorodeoxyglucose-positron emission tomography; FLT3, Fms related receptor tyrosine kinase 3 gene; FLU, fludarabine; G-CSF, granulocyte colony-stimulating factor; GM-CSF, granulocyte-monocyte colony-stimulating factor; GVHD, graft-versus-host disease; GVT, graft-versus-tumor; HCT, hematopoietic cell transplantation; HL, Hodgkin lymphoma; HLA, human leukocyte antigen; hyperCVAD, hyperfractionated cyclophosphamide, vincristine, doxorubicin, dexamethasone; HSC, hematopoietic stem cells; IFN, interferon; IL, interleukin; IPS, idiopathic pneumonia syndrome; ITD, internal tandem duplication; KTLS, a composite phenotype of c-kit+, Thy-1.1lo, lineage marker–/lo, and Sca-1+; MHC, major histocompatibility complex; MMF, mycophenolate mofetil; MRD, minimal residual disease; MSC, mesenchymal stromal cell; MTX, methotrexate; NHL, non-Hodgkin lymphoma; NK, natural killer; NMDP, National Marrow Donor Program; NPM1, nucleophosmin 1 gene; PAM, pre-transplant assessment of mortality; PBPC, peripheral blood progenitor cells; Ph, Philadelphia chromosome; PSGL-1, P-selectin glycoprotein ligand-1; PTLD, post-transplant lymphoproliferative disorder; RIC, reduced-intensity conditioning; SCID, severe combined immunodeficiency; SOS, sinusoidal obstruction syndrome; SRL, sirolimus; TAC, tacrolimus; TBI, total body irradiation; Th1, T-cell helper type 1; Th2, T-cell helper type 2; TKI, tyrosine kinase inhibitors; TLI, total lymphoid irradiation; TRM, transplant-related mortality; Treg, regulatory T cell; UCB, umbilical cord blood; VOD, veno-occlusive disease; VCAM, vascular cell adhesion molecule.


The successful clinical application of hematopoietic cell transplantation (HCT) required a century of key developmental discoveries (Table 29–1). Between 1868 and 1906, European and American investigators established that marrow cells were the source of blood cell production. In 1939, the first documented human marrow transplantation was performed in a patient with gold-induced marrow aplasia.1 The patient was infused intravenously with marrow from a brother with an identical ABO blood type. The transplantation was not successful, and the patient died 5 days after the ...

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