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Allogeneic Stem Cell
Transplantation

Second Edition

Edited by

Hillard M. Lazarus
University Hospitals Case Medical Center
Cleveland, OH
USA

Mary J. Laughlin
Case Western Reserve University
Cleveland, OH
USA
Editors
Hillard M. Lazarus Mary J. Laughlin
University Hospitals Case Medical Center Case Western Reserve University
Cleveland, OH Cleveland, OH
USA USA
[email protected] [email protected]

ISBN 978-1-934115-33-6 e-ISBN 978-1-59745-478-0

DOI 10.1007/978-1-59745-478-0
Springer New York Dordrecht Heidelberg London

Library of Congress Control Number: 2009930362

© Springer Science+Business Media, LLC 2003, 2010

All rights reserved. This work may not be translated or copied in whole or in part without the written permission of the publisher
(Humana Press, c/o Springer Science+Business Media, LLC, 233 Spring Street, New York, NY 10013, USA), except for brief
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Dr. Hillard M. Lazarus dedicated his contributions
to his wife Joan and his sons Adam and Jeffrey
for their unwavering encouragement and support.
 Preface

Allogeneic hematopoietic stem cell (HSC) transplantation has undergone

fast-paced changes after our original publication of Allogeneic Stem Cell
Transplantation: Clinical Research and Practice, first published more than 5
years ago. In this second edition, the editors have focused on topics relevant to
evolving knowledge in the field in order to better guide clinicians in decision-
making and management of their patients, as well as help lead laboratory investi-
gators in new directions emanating from clinical observations. Some of the most
respected clinicians and scientists in this discipline have responded in this sec-
ond edition by providing state-of-the-art discussions addressing these topics.
Important advances have been recognized in HLA disparity between HSC
donor and recipient triggers for T-cell and NK-cell allorecognition; such
may induce the graft-versus-host disease (GVHD) and graft-versus-leukemia
(GVL) effects and may cause an engraftment failure. This text covers the scope
of human genomic variation, the methods of HLA typing, and interpretation of
high-resolution HLA results. Durable GVL responses may be the result of the
elimination of leukemia stem cells or the establishment of a durable immune
control on their progeny.
Alternative sources of donor HSC continue to be used for transplantation
at an increased frequency and include HLA-matched unrelated donor and
umbilical cord blood; overall patient outcome has improved steadily using
these diverse stem cell sources. The administration of reduced-intensity as
well as non-myeloablative conditioning has also brought forth new concepts
in the management of hematologic malignancies, thought to be of emerging
importance in patients with lower grade malignant disorders such as chronic
lymphocytic leukemia, multiple myeloma, and low-grade non-Hodgkin
lymphoma. The elderly or those with comorbid conditions who have acute
leukemia in complete remission also may benefit by using this lower-intensity
therapy. The reduced toxicity of these novel conditioning regimens has also
raised new possibilities in the application of allogeneic HSC transplantation
for patients with non-malignant hematologic disorders such as sickle cell ane-
mia and selected solid tumors such as renal cell carcinoma.
Allogeneic SCT remains the only available curative therapy for hematologic
malignancies and some inborn errors such as beta-thalassemia. Its application,
however, may result in significant morbidity and mortality, predominantly as a
consequence of opportunistic infections and GVHD. While differences in HLA
between donor and recipient make a crucial contribution to the alloreactivity

vii
viii Preface

driving the donor-mediated GVL response, the cytokine milieu both promotes
and regulates the allogeneic response after transplantation. As such, genetic
studies correlating donor, host, or the combination of cytokine polymorphisms
with disease outcomes have provided useful insight into disease pathogenesis,
often confirming effects that have been determined in pre-clinical studies. It
is now clear that the polymorphic expression of key cytokines (particularly
tumor necrosis factor and interleukin 10) has a demonstrable effect on disease
outcome and overall transplant-related mortality.
Many challenges in allogeneic SCT remain and include the risk of graft
failure, recurrent disease, acute GVHD, opportunistic infections and long-
term sequelae such as chronic GVHD, increased risk of second malignancies,
endocrinopathies, and iron overload. The editors hope that this new informa-
tion, well summarized by the authors in this text, will be of significant benefit
to clinicians and researchers in allogeneic HSC transplantation. We envision
that the generation of further knowledge and clinical studies to be of ultimate
benefit to our patients.
Cleveland, Ohio, USA Hillard M. Lazarus, MD
Mary J. Laughlin, MD
 Contents

1 Allogeneic Stem Cell Transplantation: The Last Century................ 1

John M. Goldman

2 Full Intensity and Reduced Intensity Allogeneic

Transplantation in AML.................................................................... 11
Charles Craddock

3 Allogeneic Stem Cell Transplantation for Adult Acute

Lymphoblastic Leukemia (ALL)...................................................... 29
Bella Patel, Anthony H. Goldstone, and Adele K. Fielding

4 Hematopoietic Progenitor Cell Transplantation

for Treatment of Chronic Lymphocytic Leukemia........................... 43
Leslie A. Andritsos, John C. Byrd, and Steven M. Devine

5 The Role of Allogeneic Hematopoietic Stem Cell

Transplantation for Chronic Myelogenous Leukemia
Patients in the Era of Tyrosine Kinase Inhibitors............................. 53
Richard T. Maziarz

6 Allogeneic Transplantation for Hodgkin’s Lymphoma.................... 75

William Broderick and Patrick Stiff

7 Myeloablative Allogeneic Stem Cell Transplantation

for Non-Hodgkin’s Lymphoma......................................................... 89
J. Kuruvilla, P. Mollee, and J.H. Lipton

8 Non-Hodgkin’s Lymphoma: Allogeneic Reduced Intensity

Conditioning..................................................................................... 109
Sonali M. Smith and Ginna G. Laport

9 The Role of Allogeneic Transplantation for Multiple

Myeloma in Older Adults................................................................. 127
Heidi D. Klepin and David D. Hurd

ix
x Contents

10 Single Versus Tandem Autologous Hematopoietic

Stem Cell Transplant in Multiple Myeloma..................................... 143
David H. Vesole

11 Treatment Strategies for Follicular Center Cell

Non-Hodgkin’s Lymphoma.............................................................. 159
Frank Heinzelmann, Hellmut Ottinge, and Claus Belka

12 The Role of Transplantation in Favorable-Risk Acute

Myeloid Leukemia............................................................................ 177
Mickey Liao and Gary J. Schiller

13 Allogeneic Stem Cell Transplantation for Acute

Lymphoblastic Leukaemia in Adults................................................ 193
David I. Marks

14 Allogeneic Transplantation for Myelodysplastic Syndromes........... 203

Geoffrey L. Uy and John F. DiPersio

15 Allogeneic Hematopoietic Stem Cell Transplantation

in Pediatric Acute Lymphoblastic Leukemia.................................... 219
Adriana Balduzzi, Lucia Di Maio, Mary Eapen,
and Vanderson Rocha

16 Allogeneic Transplantation for the Treatment

of Multiple Myeloma........................................................................ 261
Rebecca L. Olin, Dan T. Vogl, and Edward A. Stadtmauer

17 Blood Vs. Marrow Allogeneic Stem Cell Transplantation............... 281

Brian McClune and Daniel Weisdorf

18 Hematopoietic Cell Transplantation from Partially

HLA-Mismatched (HLA-Haploidentical) Related Donors.............. 299
Ephraim J. Fuchs and Heather J. Symons

19 Unrelated Donor Transplants............................................................ 345

Andrea Bacigalupo

20 Update on Umbilical Cord Blood Transplantation........................... 363

Karen Ballen

21 Selection of Cord Blood Unit(s) for Transplantation........................ 375

Donna A. Wall and Ka Wah Chan

22 Mobilization of Hematopoietic Cells Prior to Autologous

or Allogeneic Transplantation........................................................... 387
Steven M. Devine
 Contents xi

23 Natural Killer-Cell Based Treatment in Hematopoetic

Stem Cell Transplantation................................................................. 413
Martin Stern, Sandrine Meyer-Monard, Uwe Siegler,
and Jakob R. Passweg

24 Cryopreservation of Allogeneic Stem Cell Products........................ 427

Noelle V. Frey and Steven C. Goldstein

25 Concepts and Controversies in the Use of Novel Preparative

Regimens for Allogeneic Stem Cell Transplantation....................... 441
Steven C. Goldstein and Selina Luger

26 Allogeneic Haematopoietic Stem Cell Transplantation

and Natural Killer Cell Alloreactivity............................................... 459
Franco Aversa and Andrea Velardi

27 Therapeutic Potential of Mesenchymal Stem

Cells in Hematopoietic Stem Cell Transplantation........................... 477
Luis A. Solchaga and Hillard M. Lazarus

28 Hematopoietic Stem Cell Transplantation for Thalassemia............. 491

Javid Gaziev and Guido Lucarelli

29 Viral Infections in Hematopoietic Stem Cell Transplant

Recipients.......................................................................................... 505
Per Ljungman

30 Fungal Infections.............................................................................. 533

John R. Wingard

31 Immune Reconstitution and Implications for Immunotherapy

Following Hematopoeitic Stem Cell Transplantation....................... 545
Kirsten M. Williams and Ronald E. Gress

32 Acute Graft Versus Host Disease: Prophylaxis................................. 565

Corey Cutler, Vincent T. Ho, and Joseph H. Antin

33 Chronic Graft-Versus-Host Disease.................................................. 577

Madan Jagasia and Steven Pavletic

34 Post-transplant Lymphoproliferative Disorder.................................. 597

Ran Reshef, Alicia K. Morgans, and Donald E. Tsai

35 Psychological Care of Adult Allogeneic Transplant Patients........... 619

Flora Hoodin, Felicity W.K. Harper, and Donna M. Posluszny

36 Second Allogeneic Transplantation: Outcomes

and Indications.................................................................................. 657
Koen van Besien, Dan Pollyea, and Andrew Artz
xii Contents

37 Minimal Residual Disease................................................................ 667

Mehmet Uzunel

38 Functional Assessment Tools and Co-morbidity Scoring

in Hematopoietic Progenitor Cell Transplantation........................... 687
Sergio Giralt and Uday Popat

39 Unique Thrombotic and Hemostatic Complications

Associated with Allogeneic Hematopoietic
Stem Cell Transplantation................................................................. 695
Amber A. Petrolla, Hillard M. Lazarus, and Alvin H. Schmaier

40 How Much Isolation Is Enough for Allografts?................................ 717

Brandon Hayes-Lattin

41 Monoclonal Antibodies in Allogeneic Hematopoietic

Stem Cell Transplantation for Hematologic Malignancies............... 733
Maria Corinna Palanca-Wessels and Oliver Press

42 Treatment of Acute Graft-vs-Host Disease....................................... 747

Steven C. Goldstein, Sophie D. Stein, and David L. Porter

43 The Importance of Non-Human Primate Models

for Pre-clinical Studies in Hematopoiesis......................................... 767
Erzsebet Szilagyi, Nadim Mahmud, and Amelia Bartholomew

44 In Vivo Models of Allogeneic Hematopoietic Stem Cell

Transplantation.................................................................................. 789
Lisbeth A. Welniak and William J. Murphy

45 Dendritic Cells.................................................................................. 807

Jacalyn Rosenblatt and David Avigan

46 Augmentation of Hematopoietic Stem Cell Transplantation

with Anti-cancer Vaccines................................................................ 855
Edward D. Ball and Peter R. Holman

Erratum ...................................................................................................
. 869

Index......................................................................................................... 871
 Contributors

Leslie A. Andritsos, MD
Division of Hematology & Oncology, The Ohio State University Medical
Center, Columbus, OH, USA
Joseph H. Antin, MD
Harvard Medical School and Dana Farber Cancer Institute, Boston, MA,
USA
Andrew Artz, MS, MD
Section of Hematology and Oncology, Department of Medicine,
University of Chicago, Chicago, IL, USA
Franco Aversa, MD
Section of Haematology and Clinical Immunology, Department of Clinical
and Experimental Medicine, HSCT Unit, University of Perugia, Perugia,
Italy
David Avigan, MD
Division of Hematological Malignancies/Bone Marrow Transplantation,
Beth Israel Deaconess Medical Center, Boston, MA, USA
Andrea Bacigalupo
Ospedale San Martino, Genova, Italy
Adriana Balduzzi, MD
Hematopoeitic Transplant Unit, Clinica Pediatrica, Università degli
Studi di Milano, Bicocca Ospedale, San Gerardo, Italy
Edward D. Ball, MD
Division of Blood and Marrow Transplantation, Department of Medicine
and the Moores UCSD Cancer Center, University of California, San Diego,
La Jolla, CA, USA
Karen Ballen, MD
Division of Hematology/Oncology, Department of Medicine, Massachusetts
General Hospital, Boston, MA, USA
Amelia Bartholomew, MD
Division of Transplant Surgery, Department of Surgery, University of Illinois
at Chicago College of Medicine, Chicago, IL, USA

xiii
xiv Contributors

Claus Belka, MD
Department of Radiation Oncology, University of Tuebingen, Tuebingen,
Germany
William Broderick, MD
Division of Hematology-Oncology, Department of Medicine, Bone Marrow
Transplant Program, Loyola University Stritch School of Medicine,
Maywood, IL, USA
John C. Byrd, MD
Division of Hematology & Oncology, The Ohio State University Medical
Center, Columbus, OH, USA
Ka Wah Chan, MD
Pediatric Blood and Marrow Transplantation Program, Texas Transplant
Institute, San Antonio, TX, USA
Charles Craddock
Centre for Clinical Haematology, Queen Elizabeth Hospital, Edgbaston,
Birmingham, UK
Corey Cutler, MD, MPH, FRCP
Harvard Medical School and Dana Farber Cancer Institute, Boston, MA, USA
Steven M. Devine, MD
Division of Hematology & Oncology, The Ohio State University Medical
Center, Columbus, OH, USA
Lucia Di Maio, MD
Hematopoeitic Transplant Unit, Clinica Pediatrica, Università degli
Studi di Milano, Bicocca Ospedale, San Gerardo, Italy
John F. DiPersio, MD, PhD.
Section of BMT and Leukemia, Division of Oncology, Washington
University School of Medicine, St. Louis, MO, USA
Mary Eapen, MD
Center for International Blood and Marrow Transplant Research,
Medical College of Wisconsin, Milwaukee, WI, USA
Adele K. Fielding, MD
Department of Haematology, Royal Free and University College
Medical School, London, UK
Noelle V. Frey, MD
Division of Hematology-Oncology and Abramson Cancer Center,
University of Pennsylvania Medical Center, Philadelphia, PA, USA
Ephraim J. Fuchs, MD
Divisions of Pediatric Oncology, Cancer Immunology and Hematologic
Malignancies, Sidney Kimmel Comprehensive Cancer Center at Johns
Hopkins, Baltimore, MD, USA
Javid Gaziev, MD
International Centre for Transplantation in Thalassemia and Sickle Cell
Anemia, Mediterranean Institute of Hematology, Rome, Italy
 Contributors xv

Sergio Giralt, MD
Department of Stem Cell Transplant and Cellular Therapy,
University of Texas MD Anderson Cancer Center, Houston, TX, USA
John M. Goldman, MD
Department of Hematology, Imperial College Faculty of Medicine and World
Marrow Donor Association, London, UK
Steven C. Goldstein, MD
Division of Hematology-Oncology and Abramson Cancer Center,
University of Pennsylvania Medical Center, Philadelphia, PA, USA
Anthony H. Goldstone
Department of Haematology, University College London Hospitals,
London, UK
Ronald E. Gress, MD
Experimental Transplantation and Immunology Branch, National Cancer
Institute, National Institutes of Health, Bethesda, MD, USA
Felicity W.K. Harper, PhD
Communication and Behavioral Oncology Program, Barbara Ann Karmanos
Cancer Institute and Department of Family Medicine and Public Health
Sciences, Wayne State University School of Medicine, Detroit, MI, USA
Brandon Hayes-Lattin, MD
Center for Hematologic Malignancies, OHSU Cancer Institute,
Oregon Health and Science University, Portland, OR, USA
Frank Heinzelmann, MD
Department of Radiation Oncology, University of Tuebingen,
Tuebingen, Germany
Vincent T. Ho, MD
Harvard Medical School and Dana Farber Cancer Institute, Boston, MA, USA
Peter R. Holman, MD
Division of Blood and Marrow Transplantation, Department of Medicine
and The Moores UCSD Cancer Center, University of California, San Diego,
La Jolla, CA, USA
Flora Hoodin, PhD
Department of Psychology, Eastern Michigan University, Ypsilanti, MI, USA
David D. Hurd, MD
Section of Hematology-Oncology, Department of Internal Medicine,
School of Medicine, Wake Forest University, Winston-Salem, NC, USA
Madan Jagasia, MBBS, MS
Division of Hematology-Oncology, Department of Medicine, Vanderbilt
Ingram Cancer Center, Vanderbilt University Medical Center, Nashville,
TN, USA
Heidi D. Klepin, MD
Section of Hematology-Oncology, Department of Internal Medicine,
School of Medicine, Wake Forest University, Winston-Salem, NC, USA
xvi Contributors

John Kuruvilla, MD
Division of Medical Oncology and Hematology, Princess Margaret Hospital
and University of Toronto, Toronto, ON, Canada
Ginna G. Laport, MD
Division of Blood and Marrow Transplantation, Stanford University
Medical Center, Stanford, CA, USA
Hillard M. Lazarus, MD
Case Comprehensive Cancer Center, University Hospitals Case Medical
Center, Cleveland, OH, USA
Mickey Liao, MD
Hematologic Malignancies Unit/Stem Cell Transplant Unit,
University of California at Los Angeles, Los Angeles, CA, USA
Jeffrey H. Lipton, MD
Division of Medical Oncology and Hematology, Princess Margaret Hospital
and University of Toronto, Toronto, ON, Canada
Per Ljungman, MD
Department of Hematology, Karolinska University Hospital, Stockholm,
Sweden
Guido Lucarelli, MD
International Centre for Transplantation in Thalassemia and Sickle Cell
Anemia, Mediterranean Institute of Hematology, Rome, Italy
Selina Luger, MD
Division of Hematology-Oncology and Abramson Cancer Center,
University of Pennsylvania Medical Center, Philadelphia, PA, USA
Nadim Mahmud, MD, PhD
Division of Hematology-Oncology, University of Illinois at Chicago,
Chicago, IL, USA
David I. Marks, MD
University Hospitals of Bristol, Oncology Day Beds, Bristol Children’s
Hospital, Bristol, UK
Richard T. Maziarz, MD
Center for Hematologic Malignancies, Adult Bone Marrow Transplantation
Program, Oregon Health Science Cancer Institute, Oregon Health & Science
University, Portland, OR, USA
Brian McClune, DO
Blood and Marrow Transplantation Program, University of Minnesota,
Minneapolis, MN, USA
Keith McCrae, MD
Division of Hematology and Oncology, Case Western Reserve University
School of Medicine, Cleveland, OH, USA
Sandrine Meyer-Monard, MD
Division of Hematology, Basel University Hospital, Basel, Switzerland
 Contributors xvii

Peter Mollee, MD
Department of Haematology, Princess Alexandra Hospital
and University of Queensland, Brisbane, QLD, Australia
Alicia K. Morgans, MD
Abramson Cancer Center, Hematologic Malignancies Program,
University of Pennsylvania Medical Center, Philadelphia, PA, USA
William J. Murphy, MD
Department of Dermatology, University of California, Davis Sacramento,
CA 95817
Rebecca L. Olin, MD
Abramson Cancer Center, Bone Marrow and Stem Cell Transplant Program,
University of Pennsylvania School of Medicine, Philadelphia, PA, USA
Hellmut Ottinger, MD
Department of Bone Marrow Transplantation, University of Essen,
Essen, Germany
Maria Corinna Palanca-Wessels, MD
Fred Hutchinson Cancer Research Center and Department of Medicine,
University of Washington, Seattle, WA, USA
Jakob R. Passweg, MD
Division of Hematology, Geneva University Hospitals, Geneva, Switzerland
Bella Patel
Department of Haematology, Royal Free and University College
Medical School, London, UK
Steven Pavletic, MD
Graft-versus-Host and Autoimmunity Unit, Experimental Transplantation
and Autoimmunity Branch, Center for Cancer Research, National Cancer
Institute, National Institutes of Health, Bethesda, MD, USA
Amber A. Petrolla, MD
Department of Pathology, Case Western Reserve University and University
Hospitals Case Medical Group, Cleveland, OH, USA
Dan Pollyea, MD
Divisions of Hematology and Oncology, Stanford University
School of Medicine, Palo Alto, CA, USA
Uday Popat, MD
Department of Stem Cell Transplant and Cellular Therapy,
University of Texas MD Anderson Cancer Center, Houston, TX, USA
David L. Porter, MD
Allogeneic Stem Cell Transplantation, University of Pennsylvania Medical
Center, Philadelphia, PA, USA
Donna M. Posluszny, PhD
Department of Medicine, University of Pittsburgh School of Medicine
and Behavioral Medicine Clinical Service, University of Pittsburgh Cancer
Institute, Pittsburgh, PA, USA
xviii Contributors

Oliver W. Press, MD
Fred Hutchinson Cancer Research Center and University of Washington
School of Medicine, Seattle, WA, USA
Ran Reshef, MD
Abramson Cancer Center, Hematologic Malignancies Program,
University of Pennsylvania Medical Center, Philadelphia, PA, USA
Vanderson Rocha, MD, PhD
Acute Leukemia Working Party of the European Blood and Marrow
Transplant Group, Hopital Saint Antoine and Hematopoeitic Transplant
Unit and Eurocord Registry, Hopital Saint Louis, Assistance Publique des
Hopitaux de Paris, University of Paris, Paris, France
Jacalyn Rosenblatt, MD
Division of Hematology/Oncology, Department of Medicine,
Beth Israel Deaconess Medical Center, Boston, MA, USA
Gary J. Schiller, MD
Hematologic Malignancies Unit/Stem Cell Transplant Unit,
University of California at Los Angeles, Los Angeles, CA, USA
Alvin H. Schmaier, MD
Division of Hematology and Oncology, Case Western Reserve University
and University Hospital Case Medical Group, Cleveland, OH, USA
Uwe Siegler, MD
Division of Hematology, Basel University Hospital, Basel, Switzerland
Sonali M. Smith, MD
Section of Hematology/Oncology, The University of Chicago Medical
Center, Chicago, IL, USA
Luis A. Solchaga, PhD
Case Comprehensive Cancer Center, University Hospitals Case Medical
Center, Cleveland, OH, USA
Edward A. Stadtmauer, MD
Abramson Cancer Center, Bone Marrow and Stem Cell Transplant Program,
University of Pennsylvania School of Medicine, Philadelphia, PA, USA
Sophie D. Stein, MD
Department of Hematology-Oncology, University of Pennsylvania Medical
Center, Philadelphia, PA, USA
Martin Stern, MD
Division of Hematology, Basel University Hospital, Basel, Switzerland
Patrick Stiff, MD
Division of Hematology-Oncology, Department of Medicine, Bone Marrow
Transplant Program, Loyola University Stritch School of Medicine,
Maywood, IL, USA
Heather J. Symons, MD
Divisions of Pediatric Oncology, Cancer Immunology and Hematologic
Malignancies, Sidney Kimmel Comprehensive Cancer Center at Johns
Hopkins, Baltimore, MD, USA