A Guide to Blood and Marrow Transplantation PDF
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A Guide to Blood and Marrow Transplantation Springer-Verlag Berlin Heidelberg GmbH H.J. DEEG H.-G. KLINGEMANN G.L. PHILLIPS G. VAN ZANT AG uide to Blood and Marrow Transplantation Third Completely Revised Edition With 12 Figures and 32 Tables t Springer H. JOACHIM DEEG, M.D. GORDON 1. PHILLIPS, M.D. Fred Hutchinson Cancer Research Center Director, BMT Program 1100 Fairview Ave. North Markey Cancer Center P.O. Box 19024 University of Kentucky Seattle, WA 98109-1024 800 Rose St. U.S.A. Lexington, KY 40536-0093 U.S.A. HANS-GEORG KLINGEMANN, M.D., PH.D. Coleman Foundation Professor of Medicine GARY VAN ZANT, PH.D. Director, Section of Bone Marrow Director, Stem Cell Laboratories Transplantation Blood and Marrow Transplant Program Rush-Presbyterian-St. Luke's Medical Center University of Kentucky Medical Center 1653 West Congress Parkway 800 Rose St. Chicago, IL 60612 Lexington, KY 40536-0093 U.S.A. U.S.A. ISBN 978-3-642-62122-2 CIP Data applied for Die Deutsche Bibliothek - CIP-Einheitsaufnahme A Guide to blood and marrow transplantation / H.J. Deeg ... (ed.). -3., completely rev. and en!. ed. Berlin; Heidelberg; New York; Barcelona; Budapest; Hong Kong; London; Milan; Paris; Singapore; Tokyo: Springer, 1998. 2. Aufl. u.d. T.: A Guide to bone marrow transplantation ISBN 978-3-642-62122-2 ISBN 978-3-642-18248-8 (eBook) DOI 10.1007/978-3-642-18248-8 This work is subject to copyright. All rights are reserved, whether the whole or part of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broad casting, reproduction on microfilms or in any other way, and storage in data banks. Duplication of this publication or parts thereof is permitted only under the provisions of the German Copyright Law of September 9, 1965, in its current version, and permission for use must always be obtained from Springer-Verlag. Violations are liable for prosecution under the German Copyright Law. © Springer-Verlag Berlin Heidelberg 1999 Originally published by Springer-Verlag Berlin Heidelberg in 1999 Softcover repriot of the hardcover 3rd edition 1999 The use of general descriptive names, registered names, trademarks, etc. in this publication does not imply, even in the absence of a specific statement, that such names are exempt from the relevant pro tective laws and regulations and therefore free for general use. Product liability: The publishers cannot guarantee the accuracy of any information about dosage and application contained in this book. In every individual case the user must check such information by consulting the relevant literature. Cover Design: Design & Production GmbH, Heidelberg Typesetting: Data conversion by Springer-Verlag, Quark XPress 4.0 SPIN: 10568767 9/3134-543210 - Printed on acid-free paper Preface to the Third Edition The first edition to A Guide to Bone Marrow Transplantation was published 10 years ago. At that time we perceived a need for an introductory text to the area of marrow transplantation, a rapidly developing field with few general texts avail able. Since then the situation has changed dramatically. Several introductory texts, therapeutic manuals and comprehensive volumes have been published. There is a heightened awareness of the availability, utility and potential of bone marrow transplantation, and of peripheral blood stem cell transplantation in particular. In many cases stem cell transplantation is being practiced by physi cians as a standard therapy. For those reasons we weighed carefully the question whether the Guide was still useful or even had a place. The feedback we received from physicians in practice or in training (including both hematologists and other physicians), as well as from nurses, physician's assistants, nurse practitioners, and other personnel in volved in one way or another in hematopoietic stem cell transplantation indicat ed that such a text was still desirable as an introduction that focused on princi ples rather than the latest up-to-date results. As before, we have aimed at maintaining a concise text. We decided to change the title to cover more broadly the field of hematopoietic cell (rather than only mar row) transplantation and, because of numerous new developments, a fourth au thor (GVZ), who comes with considerable expertise in stem cell biology, was add ed. We have restructured the contents to emphasize various sources of stem cells as well as their collection, processing and potential expansion. We also have ex panded our discussion on the prevention and treatment of disease recurrence af ter transplantation in view of the progress that has been made with immunother apy, in particular, and with attempts that are currently being made with gene manipulation. Furthermore, longer observation in larger cohorts of patients now allows for more comprehensive studies on long-term results as well as delayed ef fects, and this has been emphasized. Finally, we have added a more detailed index to allow readers to orient themselves more quickly and easily. As in previous editions, the Guide was not written as a manual on how to carry out stem cell transplants. The focus has remained on principles guiding our ap proach to the management of patients who undergo transplantation. We thank all those who have given generously of their time and expertise to make this book possible, Drs. J. Wingard and C. Pomeroy in particular. We give special thanks to Ms. Monika Schrimpf from Springer-Verlag for her understanding and for accommodating many last-minute changes, as well as to other staff members VI Preface to the Third Edition of Springer-Verlag for their support. We would like to thank B. Larson and H. Childs for their help with manuscript preparation and editing. Seattle, Chicago, Lexington H. JOACHIM DEEG HANS-GEORG KLINGEMANN GORDON L. PHILLIPS GARY VAN ZANT Preface to the Second Edition In 1988 we presented out Guide to Bone Marrow Transplantation. The reception has been enthusiastic and we have received a flood of critical comments, sugges tions and requests to provide an update in due time. Although several books on marrow transplantation have recently been published, their scope and goal have generally been different. Hence, we have decided to prepare a second edition of the Guide. Our aim was to maintain a short, concise text which nevertheless would incorpo rate changes that have occurred over the past four or five years. We have stream lined the description of pretransplant considerations, by condensing two sections into one (Treatment Planning and Timing of Transplantation). This also facilitat ed the review of controversial indications for marrow transplantation, for exam ple in patients with acute myelogenous leukemia in forst chemotherapy-induced remission. We have updated the chapter dealing with conditioning regimens and have expanded the section on domor selection, in particular in regard to the cur rent level of tissue typing and the identification of unrelated volunteer donors. In the chapter on collection, processing, and infusion of marrow, we have incorpo rated recent developments, for example, the use of closed systems for marrow harvesting and processind and the use of solid phase separation of stem cells. In the section dealing with acute transplant-related complications, we have added recent results on graft-versus-host disease prevention, and we have included up to-date information on the prevention of cytomegalovirus (-related disease by the use of CMV-negative blood products and treatment with ganciclovir. We have reworked thoroughly the section on delayed transplant-related compli cations since a considerable amount of data has accumulated. We have added a section on complications not previously addressed (for example, dental abnor malities) and added a chapter on rehabilitation. Finally, to facilitate retrieval of information, we have generated a small index. Despite all efforts it is likely that already at the time of publication of this book new results and insights not available at the time of this writing will have changed our approach to certain problems in the field. We emphasize again that the book was not conceived as a manual on how to do marrow transplants nor as a textbook giving countless statistics. The goal remains that of an overview of principles and concepts of marrow transplanta tion for those who are not necessarily confronting these issues on a daily basis. We thank all those who have given generously of their time and effort to make this book possible. We thank expecially Dr. V. Gebhardt and the staff of Springer- VIII Preface to the Second Edition Verlag for their responsiveness and support, Drs. T. Nevill and J. Wingard for reading the manuscript and for their suggestions, and Ms. 1. Williams, B. Larson, and D. Gayle for their help with manuscript preparation and editing. We also wish to acknowledge the continued support of our teachers, especially Rainer Storb and E. Donnall Thomas. Seattle and Vancouver H. JOACHIM DEEG HANS-GEORG KLINGEMANN GORDON 1. PHILLIPS Preface to the First Edition In the late 1940s investigators observed that mice given supralethal doses of total body irradiation were protected by infusion of viable spleen or marrow cells fol lowing irradiation, and that this was accomplished by hemopoietic reconstitution with donor cells as proven using genetic markers. If a similar approach could be applied to humans, it should be possible to treat leukemia patients with any dose of chemoradiotherapy as far as nonmarrow toxicity permitted, and then rescue them by marrow transplantation. Early clinical attempts were generally unsuc cessful, mostly due to a lack of knowledge of histocompatibility antigens and appropriate supportive care. These areas developed rather quickly during the 1960s, and for almost two decades now clinical marrow transplantation has been carried out with increasing success. After initially using only bone marrow from HLA indentical siblings, the field has expanded rapidly to incorporate HLA non identical related donors, and recently even marrow from unrelated volunteer donors. Furthermore, since for numerous patients who otherwise could benefit from transplantation a donor cannot be identified, there has been a growing interest in using the patient's own (autologous) bone marrow. Our understanding of the principles of transplantation and our knowledge of the potential risks and benefits have quickly grown. At times it is difficult, however, to decide what is the best option for a given patient. This problem is further accen tuated by the fact that the nontransplant management of the patients under con sideration (e.g., patients with severe aplastic anemia, acute or chronic leukemia, lymphomas) has improved concurrently. Rather than being used in a comple mentary fashion, they have often been presented as competing options. Therefore, we felt that there was a need for a text that would address these issues. Conceivably, if all treatment options including bone marrow transplantation are included in treatment planning early after a patient's diagnosis is established, they can be applied more intelligently, hopefully with reducted toxicity, and hence at less risk for the patient. Consequently long-term results should improve. From a psychological point of view this may also allow a patient more time to weigh the various options and adjust to potential problems. Although this book is directed primarily at internists and pediatricians, in par ticular hematologists and oncologists treating patients who might benefit bone marrow transplantation, we believe that it will also be of interest to other physi cians, students and physicians in training, nurses, technologists involved in mar row transplantations, patients, and possibly those involved in insurance ques tions and other administrative aspects. x Preface to the First Edition Our aim was to present each achapter and section in this book as a closed entity. Of necessity this has led to some overlap and duplication of sections of the dis cussion which appear to be pertinent in more than one place. We believe this may actually be an advantage since the readers will find any topic of interest under one heading rather than having to go through the entire book. We are indebted to our teachers who introduced us to the exciting field of bone marrow transplantation, to our patients who gave us the gratifying experience of success, and to our colleagues and students who continue to challenge our views. We would like to thank our secretaries for their never-fatiguing support during the preparation of the manuscript, and the staff of Springer-Verlag, especially Dr. J. Wieczorek, for their responsiveness to our requests and the ability to accommo date last-minute modifications. We hope that we have not neglected any of our other duties during the preparation of the manuscript, but if we have done so we hope that the benefits of this book will allow us to make good for it. H. JOACHIM DEEG HANS-GEORG KLINGEMANN GORDON L. PHILLIPS
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