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Over the past 60 years 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 malignant diseases, immune deficiencies, and inborn errors of metabolism. Advances in transplantation immune biology combined with 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

Acronyms and abbreviations that appear in this chapter include: ALL, acute lymphoblastic leukemia; AML, acute myelogenous leukemia; BCNU, carmustine; BuCy, busulfan and cyclophosphamide; c-GVHD, chronic graft-versus-host disease; CIBMTR, Center for International Blood and Marrow Transplantation Research; CML, chronic myelogenous leukemia; CMV, cytomegalovirus; CR1, first complete remission; CXCR4, chemokine-related receptor; DLI, donor leukocyte infusion; FK506, tacrolimus; G-CSF, granulocyte colony-stimulating factor; GVHD, graft-versus-host disease; GVT, graft-versus-tumor; HCT, hematopoietic cell transplantation; HL, Hodgkin lymphoma; HLA, human leukocyte antigen; HSC, hematopoietic stem cell; HSV, herpes simplex virus; 131I, radioactive iodine; Ig, immunoglobulin; IL, interleukin; MMF, mycophenolate mofetil; MSC, mesenchymal stromal cell; NHL, non-Hodgkin lymphoma; NK, natural killer; NRM, nonrelapse mortality; PBMC, blood mononuclear cells; PCR, polymerase chain reaction; RIC, reduced-intensity conditioning; SOS, sinusoidal obstructive syndrome; TBI, total-body irradiation; Th, T-cell helper; TREC, T-cell–receptor excision circle; Treg, T-regulatory cell; UCB, umbilical cord blood; VCAM-1, vascular cell adhesion molecule; VP-16, etoposide; VZV, varicella-zoster virus.

The successful clinical application of hematopoietic cell transplantation (HCT) to the therapeutic armamentarium of the hematologist required a century of key developmental discoveries (Table 21–1).

Table 21–1. Key Historical Periods in Hematopoietic Cell Transplantation

Between the years of 1868 and 1906 European and American investigators established that marrow cells ...

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