Aplastic Anemia: Pathophysiology and Treatment PDF
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APLASTIC ANEMIA: Pathophysiology and treatment Hubert Schrezenmeier Andrea Bacigalupo Cambridge University Press APLASTIC ANEMIA Pathophysiology and treatment This book takes account of the most recent Wndings in laboratory research and clinical trials to provide a comprehensive and up-to-date reference on the pathophysiology, epidemi- ology, diagnosis and treatment of acquired and inherited aplastic anemia. As well as a comprehensive and detailed overview of the pathophysiology of the disease, the international team of authors covers all aspects of management, including the well established approaches of bone marrow transplantation and immunosuppressive treat- ment, new approaches such as the use of hematopoietic growth factors and escalated immunosuppression, and controversial issues such as stem cell transplantation. The Wnal section concentrates on the inherited syndrome Fanconi’s anemia. Much of the recent work in this area has been coordinated by the European Group for Blood and Marrow Transplantation (EBMT). Included here is an important international consensus document with guidelines on treatment of aplastic anemia which combines the results and views of the EBMT with those of the international experts from America and Japan. Detailed treatment guidelines are given, making this the deWnitive resource for hematol- ogists and clinicians from other disciplines involved in the management and supportive care of patients with aplastic anemia. Scientists interested in mechanisms of bone marrow failure will also Wnd this an invaluable reference. Hubert Schrezenmeier is Professor of Medicine in the Free University of Berlin and Chairman of the EBMT Working Party on Severe Aplastic Anemia. Andrea Bacigalupois Director of the Bone Marrow Transplant Centre, Ospedale San Martino, Genoa, and President of the EBMT. This Page Intentionally Left Blank APL ASTIC ANEMIA Pathophysiology and treatment Edited by Hubert Schrezenmeier Free University of Berlin and Andrea Bacigalupo Bone Marrow Transplant Centre, Ospedale San Martino, Genoa published bythe press syndicate of the universityof cambridge The Pitt building, Trumpington Street, Cambridge, United Kingdom cambridge universitypress The Edinburgh Building, Cambridge CB2 2RU, UK www.cup.cam.ac.uk 40 West 20th Street, New York, NY 10011–4211, USA www.cup.org 10 Stamford Road, Oakleigh, Melbourne 3166, Australia Ruiz de Alarcón 13, 28014 Madrid, Spain © Cambridge University Press 2000 This book is in copyright. Subject to statutory exception and to the provisions of relevant collective licensing agreements, no reproduction of any part may take place without the written permission of Cambridge University Press. First published 2000 Printed in the United Kingdom at the University Press Cambridge TypefaceUtopia (Adobe) 9/13pt. SystemQuarkXPress® [se] A catalogue record for this book is available from the British Library Library of Congress Cataloguing in Publication data Aplastic anemia : pathophysiology and treatment / edited by Hubert Schrezenmeier and Andrea Bacigalupo. p. cm. ISBN 0 521 64101 2 (hardback) I. Schrezenmeier, H. (Hubert) II. Bacigalupo, A. [DNLM: 1. Anemia, Aplastic–physiopathology. 2. Anemia, Aplastic– therapy. WH 175 A6428 1999] RC641.7.A6A656 1999 616.1952–dc21 DNLM/DLC for Library of Congress 99-10964 CIP ISBN 0 521 64101 2 hardback Every eVort has been made in preparing this book to provide accurate and up-to-date information which is in accord with accepted standards and practice at the time of publication. Nevertheless, the authors, editors and publisher can make no warranties that the information contained herein is totally free from error, not least because clinical standards are constantly changing through research and regulation. The authors, editors and publisher therefore disclaim all liability for direct or consequential damages resulting from the use of material contained in this book. Readers are strongly advised to pay careful attention to information provided by the manufacturer of any drugs or equipment that they plan to use. Contents 12 List of contributors vii 12 Preface xi Part I Pathophysiology of acquired aplastic anemia 11 Stem cell defect in aplastic anemia 3 Judith C.W.Marsh and Nydia G.Testa 12 Cytokine abnormalities in aplastic anemia 21 Seiji Kojima 13 Role of T-lymphocytes in the pathophysiology of aplasticanemia 41 Shinji Nakao 14 Role of apoptosis in the pathophysiology of aplastic anemia 58 Frances M.Gibson,N.J.Philpott,Judith C.W.Marsh and E.C.Gordon-Smith 15 The interrelation between aplastic anemia and paroxysmal nocturnal hemoglobinuria 75 Gérard Socié,Jean-Yves Mary,Hubert Schrezenmeier and Eliane Gluckman 16 Aplastic anemia and other clonal disorders 88 Aruna Raghavachar Part II Epidemiology and clinical features of acquired aplasticanemia 17 Epidemiology and etiology of aplastic anemia 97 Hermann Heimpel 18 Clinical presentation, natural course, and prognostic factors 117 Pedro Marín-Fernandez Part III Treatment of acquired aplastic anemia 19 Supportive treatment of patients with severe aplastic anemia 137 Per Ljungman v vi Contents 10 Immunosuppressive treatment of aplastic anemia 154 André Tichelli,Hubert Schrezenmeier and Andrea Bacigalupo 11 Role of cytokines in the treatment of aplastic anemia 197 Hubert Schrezenmeier 12 HLA-identical sibling bone marrow transplantation to treat severe aplasticanemia 230 Shaun R.McCann,Jakob R.Passweg,Rainer Storb and H.Joachim Deeg 13 Alternative donor bone marrow transplantation for severe acquired aplastic anemia 258 Jill Hows,Judith Veum Stone and Bruce M.Camitta 14 Treatment of children with acquired aplastic anemia 275 Anna Locasciulli 15 Long-term follow-up of patients with aplastic anemia: clonal malignantand nonmalignant complications 288 André Tichelli and Gérard Socié 16 Guidelines for treating aplastic anemia Consensus Document of agroupof international experts 308 Part IV Fanconi’s anemia 17 Clinical features and diagnosis of Fanconi’s anemia 319 Blanche P.Alter 18 Genetic basis of Fanconi’s anemia 338 Manuel Buchwald and Madeleine Carreau 19 Treatment of Fanconi’s anemia 355 Eliane Gluckman,Gérard Socié,Philippe Guardiola for the European Blood and Marrow Transplant Group (EMBT) and European Fanconi’s Anemia Registry (EUFAR) 20 Genetic correction of Fanconi’s anemia 368 Johnson M.Liu 20 Index 380 Contributors Blanche P.Alter H.Joachim Deeg Division of Pediatric Clinical Research Division Hematology/Oncology Fred Hutchinson Cancer Research Children’s Hospital C3.270 Center University of Texas Medical Branch 1124 Columbia Street Galveston, TX 77555-0361 Seattle, WA 98104 USA USA Andrea Bacigalupo Frances M.Gibson IInd Division of Hematology Department of Haematology Ospedale San Martino St George’s Hospital Medical School Viale Benedetto XV Cranmer Terrace I-16132 Genova London SW17 0RE Italy UK Manuel Buchwald Department of Molecular and Medical Eliane Gluckman Genetics Service de Greffe de Moelle Osseuse The Hospital for Sick Children Hôpital Saint-Louis University of Toronto 1 Avenue Claude Vellefaux Toronto, Ontario F-75475 Paris, Cedex 10 Canada France Bruce M.Camitta E.C.Gordon-Smith Medical College ofWisconsin Department of Haematology PO Box 26509 St George’s Hospital Medical School 8701 Watertown Plank Road Cranmer Terrace Milwaukee, WI 53226 London SW17 0RE USA UK Madeleine Carreau Department of Molecular and Medical Philippe Guardiola Genetics Service de Greffe de Moelle Osseuse The Hospital for Sick Children Hôpital Saint-Louis University of Toronto 1 Avenue Claude Vellefaux Toronto, Ontario F-75475 Paris, Cedex 10 Canada France vii viii Contributors Hermann Heimpel Judith C.W.Marsh University of Ulm Department of Haematology Robert-Koch-Str aße8 St George’s Hospital Medical School D-89081 Ulm Cranmer Terrace Germany London SW17 0RE UK Jill M.Hows Division of Transplant Sciences Jean-Yves Mary Southmead Health Services Centre de Bioinformatique Bristol INSERM U444 BS10 5NB Université Paris VII UK Paris France Seiji Kojima Department of Developmental Paediatrics Shaun R.McCann Nagoya University School of Medicine Department of Haematology Nagoya 453 St James’s Hospital Japan Dublin 8 Ireland Johnson M.Liu Hematology Branch Shinji Nakao NHLBI, NIH Third Department of Medicine 10/ACRF/7C103 Kanazawa University School of Medicine Bethesda, MD 20892 13-1 Takaramachi, Kanazawa USA Ishikawa 920 Japan Per Ljungman Department of Hematology Jakob R.Passweg Karolinska Institute Department Innere Medizin Huddinge University Hospital Kantonsspital Basel SE-14186 Huddinge Petersgraben 4 Sweden CH-4031 Basel Switzerland Anna Locasciulli Division of Pediatric Hematology N.J.Philpott Ospedale San Gerardo Department of Haematology Via Donizetti 106 St George’s Hospital Medical School I-20052 Monza Cranmer Terrace Italy London SW17 0RE UK Pedro Marín-Fernandez Postgraduate School of Hematology Aruna Raghavachar (EHFC) Dept of Internal Medicine III Ferreras Valenti Hospital Clinic University of Ulm C/Villarroel 170 Robert-Koch-Straße8 E-08036 Barcelona D-89081 Ulm Spain Germany Contributors ix Hubert Schrezenmeier Rainer Storb Benjamin Franklin University Hospital Clinical Research Division Medical Clinic III Fred Hutchinson Cancer Research Center Free University of Berlin 1124 Columbia Street Hindenburgdamm 30 Seattle, WA 98104 D-12200Berlin USA Germany Nydia G.Testa GérardSocié Department of Experimental Haematology Bone Marrow Transplantation Unit Paterson Institute for Cancer Research Hôpital Saint-Louis Wilmslow Road 1 Avenue Claude Vellefaux Manchester M20 9BX 75475 Paris Cedex 10 UK France André Tichelli Judith Veum Stone Hematologie Statistical Center Kantonsspital Basel Medical College of Wisconsin Petersgraben 4-8 PO Box 26509, 8701 Watertown PlankRoad CH-4031 Basel Milwaukee, WI 53226 Switzerland USA
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