
Thomas' Hematopoietic Cell Transplantation
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Contributors, xi
Preface to the First Edition, xvii
Preface to the Fifth Edition, xviii
Tribute, xix
List of Abbreviations, xxii
Section 1: History and Use of Hematopoietic Cell Transplantation
1 A History of Allogeneic and Autologous Hematopoietic Cell Transplantation, 1
Karl G. Blume and E. Donnall Thomas
2 Uses and Growth of Hematopoietic Cell Transplantation, 8
Mary M. Horowitz
Section 2: Scientific Basis or Hematopoietic Cell Transplantation
Section 2a: Hematopoiesis and Stem Biology Transplantation
3 Generation of Definitive Engraftable Hematopoietic Stem Cells from Human Pluripotent Stem Cells, 16
Laurence Dahéron and David T. Scadden
4 Hematopoietic Stem Cells, Regenerative Medicine, and Leukemogenesis, 25
Irving Weissman
5 Marrow Microenvironment and Biology of Mobilization of Stem Cells, 50
Michael P. Rettig, Mark A. Schroeder and John F. DiPersio
6 Expansion of Human Hematopoietic Stem Cells, 62
Colleen Delaney
7 Mesenchymal Stromal Cells and Hematopoietic Cell Transplantation, 69
Edwin M. Horwitz
8 Genetic Manipulation of Hematopoietic Stem Cells, 78
Jennifer E. Adair, Grant D. Trobridge and Hans-Peter Kiem
Section 2b: Immunology
9 Overview of Hematopoietic Cell Transplantation Immunology, 96
Paul J. Martin and Warren D. Shlomchik
10 Histocompatibility, 112
Effie W. Petersdorf
11 Natural Killer Cells and Allogeneic Hematopoietic Cell Transplantation, 126
Michael R. Verneris and Jeffrey S. Miller
12 Mechanisms of Tolerance, 136
Defu Zeng and Samuel Strober
13 The Pathophysiology of Graft-versus-Host Disease, 146
James L.M. Ferrara and Joseph H. Antin
14 Immune Regulation in Hematopoietic Cell Transplantation, 153
Robert S. Negrin and Jerome Ritz
15 Immune Reconstitution Following Hematopoietic Cell Transplantation, 160
Jarrod A. Dudakov, Miguel-Angel Perales and Marcel R.M. van den Brink
16 Biology of the Human Graft-versus-Tumor Response and How to Exploit It, 166
Edus H. Warren
17 Dendritic Cells in Hematopoietic Cell Transplantation, 178
Miriam Merad and Edgar G. Engleman
18 The Experimental Basis for Hematopoietic Cell Transplantation for Autoimmune Diseases, 191
Judith A. Shizuru
Section 2c: Technical Aspects
19 Pharmacologic Basis for High-dose Chemotherapy, 211
Borje S. Andersson, Benigno C. Valdez and Roy B. Jones
20 High-dose Preparatory Regimens, 223
William I. Bensinger
21 Reduced-intensity Allogeneic Transplantation Regimens, 232
Brenda M. Sandmaier and Rainer Storb
22 Radiotherapeutic Principles of Hematopoietic Cell Transplantation, 244
Jeffrey Y.C. Wong and Timothy Schultheiss
23 Radioimmunotherapy and Hematopoietic Cell Transplantation, 258
John M. Pagel and Ajay K. Gopal
24 Documentation of Engraftment and Characterization of Chimerism After Hematopoietic Cell Transplantation, 272
Paul J. Martin
25 The Detection and Significance of Minimal Residual Disease, 281
Merav Bar and Jerald P. Radich
26 Pathology of Hematopoietic Cell Transplantation, 292
Keith R. Loeb, Cecilia C. Yeung and Howard M. Shulman
27 Biostatistical Methods in Hematopoietic Cell Transplantation, 329
Joycelynne M. Palmer and Ted A. Gooley
28 Outcomes Research in Hematopoietic Cell Transplantation, 341
Stephanie J. Lee
Section 3: Patient-oriented Issues in Hematopoietic Cell Transplantation
29 The Evaluation and Counseling of Candidates for Hematopoietic Cell Transplantation, 349
Bart L. Scott and Brenda M. Sandmaier
30 Nursing Role in Hematopoietic Cell Transplantation, 362
Rosemary C. Ford, Mihkaila M. Wickline and Diane Heye
31 Ethical Issues in Hematopoietic Cell Transplantation, 374
David S. Snyder
32 Psychosocial Issues in Hematopoietic Cell Transplantation, 384
Richard P. McQuellon and Katharine E. Duckworth
33 Assessment of Quality of Life in Hematopoietic Cell Transplantation Recipients, 392
Karen L. Syrjala and Samantha Burns Artherholt
34 Sexuality Following Hematopoietic Cell Transplantation, 399
D. Kathryn Tierney
35 Hematopoietic Cell Transplantation: The Patient's Perspective, 407
Susan K. Stewart
Section 4: Sources of Hematopoietic Cell for Hematopoietic Cell Transplantation
36 Hematopoietic Cell Procurement, Processing, and Transplantation: Standards, Accreditation, and Regulation, 414
Phyllis I. Warkentin and Elizabeth J. Shpall
37 Bone Marrow and Peripheral Blood Cell Donors and Donor Registries, 423
Dennis L. Confer, John P. Miller and Jeffrey W. Chell
38 The Role of the Transplant Program in a Nuclear Accident or Terrorism, 431
Nelson J. Chao and Dennis L. Confer
39 Cord Blood Hematopoietic Cell Transplantation, 437
Hal E. Broxmeyer, Sherif S. Farag and Vanderson Rocha
40 Mobilization of Peripheral Blood Hematopoietic Cells for Autologous HCT, 452
Thomas C. Shea and John F. DiPersio
41 Peripheral Blood Hematopoietic Cells for Allogeneic Transplantation, 460
Norbert Schmitz and Peter Dreger
42 Cryopreservation of Hematopoietic Cells, 469
Scott D. Rowley
43 Use of Recombinant Growth Factors after Hematopoietic Cell Transplantation, 480
Jürgen Finke and Roland Mertelsmann
44 Hematopoietic Cell Transplantation from Human Leukocyte Antigen Partially Matched Related Donors, 489
Claudio Anasetti, Franco Aversa and Andrea Velardi
45 Hematopoietic Cell Transplantation from Unrelated Donors, 505
Effie W. Petersdorf
Section 5: Hematopoietic Cell Transplantation for Acquired Disease
46 Hematopoietic Cell Transplantation for Aplastic Anemia, 517
George E. Georges and Rainer Storb
47 Hematopoietic Cell Transplantation for Paroxysmal Nocturnal Hemoglobinuria, 537
Ryotaro Nakamura
48 Hematopoietic Stem Cell Transplantation for Chronic Myeloid Leukemia, 545
Jerald P. Radich and Ravi Bhatia
49 Hematopoietic Cell Transplantation for Juvenile Myelomonocytic Leukemia, 554
Charlotte M. Niemeyer and Franco Locatelli
50 Hematopoietic Cell Transplantation for Adult Acute Myeloid Leukemia, 563
Frederick R. Appelbaum
51 Hematopoietic Cell Transplantation for Childhood Acute Myeloid Leukemia, 576
Michael A. Pulsipher, Meghann McManus and Soheil Meshinchi
52 Hematopoietic Cell Transplantation for Acute Lymphoblastic Leukemia in Adults, 588
Stephen J. Forman
53 Hematopoietic Cell Transplantation for Acute Lymphoblastic Leukemia in Children, 600
Parinda A. Mehta and Stella M. Davies
54 Hematopoietic Cell Transplantation for Myelodysplastic Syndromes and Myeloproliferative Neoplasms, 613
H. Joachim Deeg
55 Hematopoietic Cell Transplantation for Multiple Myeloma, 625
Amrita Krishnan and Sergio A. Giralt
56 Hematopoietic Cell Transplantation for Hodgkin Disease, 636
Philip J. Bierman and Auayporn Nademanee
57 Hematopoietic Cell Transplantation for Non-Hodgkin Lymphoma (B Cell), 653
Leslie L. Popplewell and Ginna G. Laport
58 Hematopoietic Cell Transplantation for Non-Hodgkin Lymphoma (T Cell), 664
Jasmine Zain and Sandra H. Thomas
59 Hematopoietic Cell Transplantation for Chronic Lymphocytic Leukemia, 675
Aaron C. Logan and David B. Miklos
60 Autologous Hematopoietic Cell Transplantation for Systemic Light Chain (AL-) Amyloidosis, 685
Heather J. Landau, Morie A. Gertz and Raymond L. Comenzo
61 Autologous Hematopoietic Cell Transplantation for Breast Cancer and Germ-cell Tumors, 700
Yago Nieto and Elizabeth J. Shpall
62 Hematopoietic Cell Transplantation for Renal Cell Carcinoma and Other Solid Tumors, 709
Richard W. Childs and Ramaprasad Srinivasan
63 Hematopoietic Cell Transplantation for Neuroblastoma, 721
Justin T. Wahlstrom and Katherine K. Matthay
64 Hematopoietic Cell Transplantation for Other Pediatric Solid Tumors, 729
Masanori Hayashi, David M. Loeb and Allen R. Chen
65 Hematopoietic Cell Therapy for Human Immunodeficiency Virus Infection, 742
Amrita Krishnan, Joseph Alvarnas, John J. Rossi and John A. Zaia
66 Hematopoietic Cell Transplantation for Autoimmune Diseases, 753
Richard A. Nash
67 Hematopoietic Cell Transplantation for Rare Hematologic Malignancies, 765
Vinod Pullarkat and Stephen J. Forman
68 Adoptive T-cell Therapy for Viral Disease in the Setting of Hematopoietic Cell Transplantation, 777
Catherine M. Bollard and Helen E. Heslop
69 Adoptive T-cell Therapy for Malignancy in the Setting of Hematopoietic Cell Transplantation, 787
Stephen J. Forman, Michael Kalos and Carl H. June
70 Relapse of Hematologic Malignancy After Allogeneic Hematopoietic Transplantation, 797
Richard Champlin
Section 6: Hematopoietic Cell Transplantation for Inherited Disease
71 Hematopoietic Cell Transplantation for Thalassemia, 803
Guido Lucarelli and Javid Gaziev
72 Hematopoietic Cell Transplantation for Sickle Cell Disease, 814
Mark C. Walters
73 Hematopoietic Cell Transplantation for Immunodeficiency Diseases, 827
Richard J. O'Reilly and Wilhelm Friedrich
74 Hematopoietic Cell Transplantation for Storage Diseases, 846
Joseph Rosenthal
75 Hematopoietic Cell Transplantation for Macrophage, Granulocyte and Osteoclast, Disorders, 871
Rajni Agarwal
76 Hematopoietic Cell Transplantation for Fanconi Anemia, 884
John E. Wagner, Jakub Tolar, Arleen D. Auerbach and Margaret L. MacMillan
Section 7: Complications of Hematopoietic Cell Transplantation
77 Mechanisms and Treatment of Graft Failure, 905
Robert Lowsky and Hans Messner
78 Blood Group Incompatibilities and Hemolytic Complications of Hematopoietic Cell Transplantation, 916
Margaret R. O'Donnell
79 Principles of Transfusion Support Before and After Hematopoietic Cell Transplantation, 922
Jeffrey McCullough
80 Vascular Access and Complications, 937
I. Benjamin Paz
81 Pharmacologic Prevention of Acute Graft-versus-Host Disease, 951
Nelson J. Chao
82 T-cell Depletion to Prevent Graft-versus-Host Disease, 965
Robert J. Soiffer
83 Manifestations and Treatment of Acute Graft-versus-Host Disease, 973
Corey Cutler and Joseph H. Antin
84 Chronic Graft-versus-Host Disease - Clinical Manifestations and Therapy, 981
Paul A. Carpenter, Mary E.D. Flowers and Steven Z. Pavletic
85 Bacterial Infections, 999
Helen L. Leather and John R. Wingard
86 Fungal Infections After Hematopoietic Cell Transplantation, 1018
Kieren A. Marr
87 Cytomegalovirus Infection, 1030
John A. Zaia
88 Herpes Simplex Virus Infections, 1039
Sanjeet S. Dadwal and James I. Ito
89 Varicella Zoster Virus Infections, 1046
Dora Y. Ho and Ann M. Arvin
90 Epstein-Barr Virus Infection, 1066
Jennifer A. Kanakry and Richard F. Ambinder
91 Respiratory Viruses After Hematopoietic Cell Transplantation, 1073
Sachiko Seo and Michael Boeckh
92 HHV-6A, HHV-6B, HHV-7, and HHV-8 After Hematopoietic Cell Transplantation, 1083
Per Ljungman, Tetsushi Yoshikawa and Katherine N. Ward
93 Human Adenovirus, Polyomavirus, and Parvovirus Infections in Patients Undergoing Hematopoietic Stem Cell Transplantation, 1090
Hans H. Hirsch and Steven A. Pergam
94 Gastrointestinal and Hepatic Complications, 1101
David M. Hockenbery, Simone I. Strasser and George B. McDonald
95 Lung Injury Following Hematopoietic Cell Transplantation, 1117
Kenneth R. Cooke and Gregory A. Yanik
96 Kidney and Bladder Complications of Hematopoietic Cell Transplantation, 1131
Sangeeta Hingorani
97 Endocrine Complications Following Hematopoietic Cell Transplantation, 1142
Fouad R. Kandeel and Behrouz Salehian
98 Common Potential Drug Interactions Following Hematopoietic Cell Transplantation, 1167
Anne Poon and Lowan K. Ly
99 Nutrition Support of the Hematopoietic Cell Transplant Recipient, 1177
Paula Charuhas Macris and Kerry K. McMillen
100 Pain Management, 1186
Noelle V. Frey and Jonathan R. Gavrin
101 Oral Complications of Hematopoietic Cell Transplantation, 1203
Mark M. Schubert, Maria Elvira Pizzigatti Correa and Douglas E. Peterson
102 Growth and Development After Hematopoietic Cell Transplantation, 1218
K. Scott Baker and Anna Petryk
103 Delayed Non-malignant Complications After Hematopoietic Cell Transplantation, 1227
Smita Bhatia
104 Subsequent Malignant Neoplasms After Hematopoietic Cell Transplantation, 1236
Smita Bhatia and Ravi Bhatia
105 Neurologic Complications of Hematopoietic Cell Transplantation, 1248
Harry Openshaw and Bihong T. Chen
106 Vaccination of Allogeneic and Autologous Hematopoietic Cell Recipients, 1258
Trudy N. Small
Index, 1266
Contributors
Jennifer E. Adair, PhD
Assistant Member,
Clinical Research Division,
Fred Hutchinson Cancer Research Center;
University of Washington School of Medicine,
Seattle, WA, USA
Rajni Agarwal, MD
Associate Professor of Pediatrics,
Division of Stem Cell Transplantation
and Regenerative Medicine,
Department of Pediatrics,
Stanford University School of Medicine,
Stanford, CA, USA
Joseph Alvarnas, MD
Director of Medical Quality,
Department Hematology/HCT,
City of Hope Medical Center,
Duarte, CA, USA
Richard F. Ambinder, MD, PhD
Professor in the Departments of Oncology,
Pathology, Pharmacology, and Medicine,
Johns Hopkins School of Medicine,
Baltimore, MD, USA
Claudio Anasetti, MD
Senior Member and Chair,
Department of Blood and Marrow Transplantation,
Moffitt Cancer Center,
Tampa, FL, USA
Borje S. Andersson, MD, PhD
Professor of Medicine,
Department of Stem Cell Transplantation
and Cellular Therapy,
The University of Texas M.D.
Anderson Cancer Center, Houston, TX, USA
Joseph H. Antin, MD
Professor of Medicine,
Harvard Medical School;
Chief, Stem Cell Transplantation,
Dana-Farber Cancer Institute,
Brigham and Women's Hospital, Boston, MA, USA
Frederick R. Appelbaum, MD
Member and Director,
Clinical Research Division, Fred Hutchinson Cancer
Research Center;
Professor and Head,
Division of Medical Oncology,
University of Washington School of Medicine,
Seattle, WA, USA
Samantha Burns Artherholt, PhD
Acting Assistant Professor,
Department of Psychiatry and Behavioral Sciences,
University of Washington School of Medicine,
Seattle, WA, USA
Ann M. Arvin, MD
Professor of Pediatrics,
Microbiology, and Immunology,
Department of Pediatrics,
Division of Infectious Diseases,
Stanford University School of Medicine,
Stanford, CA, USA
Arleen D. Auerbach, PhD
Consultant,
Program in Human Genetics and Hematology,
The Rockefeller University,
New York, NY, USA
Franco Aversa, MD
Chair,
Hematology and BMT Unit,
Department of Clinical and Experimental Medicine,
University of Parma, Parma, Italy
K. Scott Baker, MD, MS
Director,
Pediatric Blood and Marrow Transplantation and
Survivorship Programs,
Clinical Research Division,
Fred Hutchinson Cancer Research Center,
Seattle, WA, USA
Merav Bar, MD
Assistant Member,
Clinical Research Division,
Fred Hutchinson Cancer Research Center,
Seattle, WA, USA
William I. Bensinger, MD
Member,
Fred Hutchinson Cancer Research Center;
Professor of Medicine, University of Washington
School of Medicine
Seattle, WA, USA
Ravi Bhatia, MD
Professor of Medicine,
Director, UAB Division of Hematology/Oncology,
Deputy Director, UAB Comprehensive Cancer Center,
University of Alabama at Birmingham,
Birmingham, AL, USA
Smita Bhatia, MD, MPH
Professor,
Department of Pediatrics;
Director of the Institute for Cancer Outcomes
and Survivorship,
School of Medicine;
Associate Director for Cancer Outcomes Research,
Comprehensive Cancer Center,
University of Alabama at Birmingham,
Birmingham, AL, USA
Philip J. Bierman, MD
Associate Professor,
University of Nebraska Medical Center,
Department of Internal Medicine,
Section of Oncology/Hematology,
Nebraska Medical Center, Omaha, NE, USA
Karl G. Blume, MD
Emeritus Professor of Medicine,
Stanford University School of Medicine,
Stanford, CA, USA
Michael Boeckh, MD
Member, Vaccine and Infectious Disease Division;
Member, Clinical Research Division;
Head, Infectious Disease Sciences Program,
Fred Hutchinson Cancer Research Center;
Professor, Department of Medicine,
University of Washington School of Medicine,
Seattle, WA, USA
Catherine M. Bollard, MBChB, MD
Professor of Pediatrics and Microbiology,
Immunology and Tropical Medicine,
The George Washington University,
School of Medicine and Health Sciences,
Children's National Health System,
Washington, DC, USA
Hal E. Broxmeyer, PhD
Distinguished Professor,
Department of Microbiology and Immunology,
Indiana University School of Medicine,
Indianapolis, IN, USA
Paul A. Carpenter, MB, BS
Member,
Clinical Research Division, Fred Hutchinson Cancer Research Center;
Professor of Pediatrics,
Department of Pediatrics,
University of Washington School of Medicine,
Seattle, WA, USA
Richard Champlin, MD
Chairman,
Department of Stem Cell Transplantation and Cellular Therapy;
Professor of Medicine,
Division of Cancer Medicine,
The University of Texas M.D.
Anderson Cancer Center, Houston, TX, USA
Nelson J. Chao, MD, MBA
Donald D. and Elizabeth G. Cooke Professor;
Chief, Division of Hematological Malignancies
and Cellular Therapy/BMT, Duke University,
Durham, NC, USA
Jeffrey W. Chell, MD
Chief Executive Officer,
National Marrow Donor Program,
Minneapolis, MN, USA
Allen R. Chen, MD, PhD, MHS
Associate Professor of Oncology and Pediatrics,
The Sidney Kimmel Comprehensive Cancer Center
at Johns Hopkins and Department of Pediatrics,
Johns Hopkins University School of Medicine,
Baltimore, MD, USA
Bihong T. Chen, MD, PhD
Associate Clinical Professor,
Department of Radiology,
City of Hope National Medical Center,
Duarte, CA, USA
Richard W. Childs, MD
Clinical Director,
NHLBI; Senior Investigator,
National Heart, Lung and Blood Institute,
National Institutes of Health, Bethesda, MD, USA
Raymond L. Comenzo, MD
Professor of Medicine and Pathology,
Tufts University School of Medicine; Attending
Physician and Director of the Transfusion Service
and the Cell Therapy Center,
Division of Hematology-Oncology,
Department of Medicine, Tufts Medical Center,
Boston, MA, USA
Dennis L. Confer, MD
Clinical Professor of Medicine,
Department of Medicine,
University of Minnesota; Chief Medical Officer,
National Marrow Donor Program,
Minneapolis, MN, USA
Kenneth R. Cooke, MD
Professor of Oncology and Pediatrics (PAR),
Division of Pediatric Hematology/Oncology,
Department of Oncology, Blood and Marrow
Transplant Program, Johns Hopkins University School of Medicine, Baltimore, MD, USA
Maria Elvira Pizzigatti Correa, DDS, PhD
Professor of Oral Diagnosis,
Piracicaba School of Dentistry;
Dentist,
Hematology and Blood Transfusion Center,
Hemocentro, University of Campinas, UNICAMP,
Campinas, SP, Brazil
Corey Cutler, MD, MPH, FRCP(C)
Associate Professor of Medicine,
Harvard Medical School, Dana-Farber Cancer Institute, Boston, MA, USA
Sanjeet S. Dadwal, MD
Associate Clinical Professor,
Division of Infectious Disease,
City of Hope National Medical Center,
Duarte, CA, USA
Laurence Dahéron, PhD
Head, iPS core facility,
Harvard Stem Cell Institute, Harvard University,
Boston, MA, USA
Stella M. Davies, MBBS, MRCP, PhD
Professor,
Division of Bone Marrow Transplantation and Immune
Deficiency, Cancer and Blood Diseases Institute,
Cincinnati Children's Hospital Medical Center,
Cincinnati, OH, USA
H. Joachim Deeg, MD
Professor of Medicine; Member,
Clinical Research Division, Fred Hutchinson Cancer
Research Center, University of Washington
Seattle, WA, USA
Colleen Delaney, MD, MSc
Director, Program in Cord Blood Transplantation
Associate Member, Clinical Research Division,
Fred Hutchinson Cancer Research Center
Associate Professor, Department of Pediatrics,
University of Washington School of Medicine,
Seattle, WA, USA
John F. DiPersio, MD, PhD
Professor of Medicine and Pathology and Immunology,
Deputy Director,
Siteman Cancer Center, Washington University School
of Medicine, St. Louis, MO, USA
Peter Dreger, MD
Professor of Medicine;
Head, Stem Cell Transplantation Unit,
Department of Internal Medicine V, University of
Heidelberg, Im Neuenheimer Feld 410,
Heidelberg, Germany
Katharine E. Duckworth, PhD
Assistant Professor,
Department of Medicine, Hematology, and Oncology,
Wake Forest Baptist Health, Comprehensive Cancer
Center of Wake Forest University,
Winston-Salem, NC, USA
Jarrod A. Dudakov, PhD
Senior Research Scientist,
Immunology Program, Memorial Sloan-Kettering
Cancer Center, New York, USA;
Department of Anatomy and Developmental Biology,
Monash University, Melbourne, Australia
Edgar G. Engleman, MD
Professor of Pathology and Medicine,
Stanford University School of Medicine;
Director, Stanford Blood Center,
Stanford, CA, USA
Sherif S. Farag, MD, PhD, MBBS
Lawrence H. Einhorn Professor of Oncology,
Department of Medicine, Indiana University School
of Medicine, Indianapolis, IN, USA
James L.M. Ferrara, MD, DSc...
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