Chronic myeloid leukaemia : biology and treatment PDF
Preview Chronic myeloid leukaemia : biology and treatment
436_Chronic_prelims 24/05/2001 3:41 pm Page i RUNNING HEADLINE i Chronic Myeloid Leukaemia 436_Chronic_prelims 24/05/2001 3:41 pm Page ii 436_Chronic_prelims 24/05/2001 3:41 pm Page iii Chronic Myeloid Leukaemia Biology and Treatment Edited by ANGELO M CARELLA,MD Division of Hematology and Stem Cell Transplantation Unit Ospedale ‘Casa Sollievo della Sofferenza’-IRCCS San Giovanni Rotondo, Italy GEORGE Q DALEY,MD, PhD Whitehead Institute Cambridge, MA, USA CONNIE J EAVES,PhD Terry Fox Laboratory BC Cancer Agency Vancouver, BC, Canada JOHN M GOLDMAN,DM, FRCP, FRCPath Hammersmith Hospital Department of Haematology Imperial College School of Medicine London, UK RÜDIGER HEHLMANN,MD III Medizinische Klinik Mannheim Universität Heidelberg Mannheim, Germany MARTIN DUNITZ © 2001, Martin Dunitz Ltd, a member of the Taylor & Francis group First published in the United Kingdom in 2001 by Martin Dunitz Ltd The Livery House 7–9 Pratt Street London NW1 0AE Tel: +44-(0)20-7482-2202 Fax: +44-(0)20-7267-0159 E-mail: [email protected] Website: http://www.dunitz.co.uk This edition published in the Taylor & Francis e-Library, 2004. All rights reserved. No part of this publication may be reproduced, stored in a retrieval system, or transmitted, in any form or by any means, electronic, mechanical, photocopying, recording or otherwise, without the prior permission of the publisher or in accordance with the provisions of the Copyright, Designs and Patents Act 1988, or under the terms of any licence permitting limited copying issued by the Copyright Licensing Agency, 90 Tottenham Court Road, London W1P 0LP. Although every effort has been made to ensure that drug doses and other information are presented accurately in this publication, the ultimate responsibility rests with the prescribing physician. Neither the publishers nor the authors can be held responsible for errors or for any consequences arising from the use of information contained herein. For detailed prescribing information or instructions on the use of any product or procedure discussed herein, please consult the prescribing information or instructional material issued by the manufacturer. A CIP catalogue record for this book is available from the British Library ISBN 0-203-21301-7 Master e-book ISBN ISBN 0-203-27010-X (Adobe eReader Format) ISBN 1-85317-890-X (Print Edition) Distributed in the USA by Fulfilment Center Taylor & Francis 7625 Empire Drive Florence, KY 41042, USA Toll Free Tel: 1-800-634-7064 Email: cserve@routledge nv.com Distributed in Canada by Taylor & Francis 74 Rolark Drive Scarborough Ontario M1R 4G2, Canada Toll Free Tel: 1-877-226-2237 Email: tal [email protected] Distributed in the rest of the world by ITPS Limited Cheriton House North Way, Andover Hampshire SP10 5BE, UK Tel: +44 (0)1264 332424 Email: [email protected] 436_Chronic_prelims 24/05/2001 3:41 pm Page v Contents Preface......................................................................................................................................................... vii Contributors............................................................................................................................................... ix Part 1 Molecular biology of chronic myeloid leukaemia 1. BCR/ABLgene structure and BCR function Nora Heisterkamp, John Groffen ........................ 3 2. BCR/ABL protein domain function and signaling Ann Marie Pendergast .......................... 19 3. Abnormalities in hematopoietic progenitor adhesion Ravi Bhatia, Catherine Verfaillie ...... 41 4. Functional complementation of cytokine receptor signaling by BCR/ABL Eugene Y Koh, George Q Daley ........................................................................................................ 55 5. Progenitor cell dynamics Connie J Eaves, Allen C Eaves ........................................................... 73 6. Animal models of Philadelphia-positive leukemia Richard A Van Etten .............................. 101 Part 2 Conventional treatment for chronic myeloid leukaemia 7. Biology of interferon Thomas Fischer, Moshe Talpaz .................................................................. 135 8. Interferon-αdosage regimens Patricia CA Shepherd ................................................................ 149 9. Chemotherapy versus interferon: Long-term effects Andreas Hochhaus, Rüdiger Hehlmann ............................................................................................................................. 165 10. Chemotherapy Bengt Simonsson ................................................................................................. 181 11. Interferon-αand Ara-C Gianantonio Rosti, Elena Trabacchi, Francesca Bonifazi, Antonio de Vivo, Simona Bassi, Sante Tura ...................................................................................... 191 12. Prognostic factors Joerg Hasford, Markus Pfirrmann, Rüdiger Hehlmann, Patricia CA Shepherd, François Guilhot, François X Mahon, Josef Thaler, Juan L Steegmann, Hanneke C Kluin-Nelemans, Andries Louwagie, Kazunori Ohnishi, Otto Kloke ............................................................................ 205 13. Evidence-based guidelines for the treatment of chronic-phase chronic myeloid leukemia Richard T Silver ............................................................................................................. 225 Part 3 Allogeneic haematopoietic stem cell transplantation for chronic myeloid leukaemia 14. Donor selection in allogeneic bone marrow transplantation Andrea L Pay, Ann-Margaret Little, J Alejandro Madrigal ...................................................................................... 241 15. Risk assessmant for allogeneic transplantation Alois Gratwohl ................................................. 251 16. The decision whether to allograft a patient with CML John M Goldman ................................ 263 436_Chronic_prelims 24/05/2001 3:41 pm Page vi vi CONTENTS 17. Conditioning regimens and T-cell depletion Charles Craddock ............................................... 273 18. Blood versus marrow stem cells Peter Dreger, Norbert Schmitz .............................................. 287 19. HLA-identical sibling transplantation David G Savage ............................................................. 303 20. Results with alternative donors Jane F Apperley ........................................................................ 323 21. Patient monitoring after allogeneic stem cell transplantation or interferon-αtherapy Andreas Hochhaus ............................................................................................................................ 339 22. Recommendations for assessment and definitions of response and relapse in CML: A report from the Chronic Leukemia Working Committee of the IBMTR Sergio A Giralt, John M Goldman, Claudio Anasetti, Francisco Cervantes, Richard Champlin, Nicholas Cross, Andreas Hochhaus, John P Klein, Esperanza Papadopoulos, Kathleen Sobocinski, Daniel Weisdorf, Mary Horowitz on behalf of the Chronic Leukemia Working Committee of the IBMTR ...................................................................................................................................... 357 23. Basis of GVL John M Barrett ......................................................................................................... 369 24. Donor lymphocyte infusions Francesco Dazzi, Hans J Stauss .................................................. 385 25. Late complications, including late relapse Gérard Socié, Rochelle E Curtis, John P Klein ..... 401 Part 4 Autografting in chronic myeloid leukaemia 26. Autografting with unmanipulated stem cells: The European experience Arnaud Pigneux, Eduardo Olavarria, François X Mahon, Josy Reiffers ....................................................................... 421 27. Autografting with cultured marrow Michael J Barnett,Connie J Eaves, Allen C Eaves ......... 431 28. Autografting with Ph-negative haematopoietic progenitor cells Angelo M Carella ............ 445 29. Autologous hematopoietic stem cell transplantation: Experience at the University of Minnesota Philip McGlave ............................................................................................................ 457 30. What role for autografting? A personal view Stephen G O’Brien............................................ 465 Part 5 Target-directed therapies: Chronic myeloid leukaemia as a paradigm for oncology 31. Gene therapy Alan M Gewirtz ..................................................................................................... 475 32. Target-directed therapies Mikhail L Gishizky ............................................................................ 485 33. Development of kinase inhibitors Enrica Lerma, Toa Wang, Takuma Fujii, Victor Chang, David Austin, Albert A Deisseroth ................................................................................................... 495 34. STI571 as a therapeutic agent Michael E O’Dwyer, Michael J Mauro, Brian J Druker ............ 501 35. Immunotherapeutic strategies Kathleen NS Cathcart, Javier Pinilla, David A Scheinberg ..... 507 Index ........................................................................................................................................................... 521 436_Chronic_prelims 24/05/2001 3:41 pm Page vii Preface Much has changed in the field of chronic agreed to contribute to this work; without their myeloid leukaemia (CML), particularly during willingness and enthusiasm, it would not have the past decade. New advances in therapy and been possible to put together up-to-date great achievements in cellular biology, molecu- reviews of the variety of biologic and therapeu- lar biology, and chemotherapy, coupled with tic methods available today. We hope that the the development of tyrosine kinase inhibitors, reader will benefit from this book, which have all radically changed the clinical and basic should be suitable for specialists and postgrad- approaches to the therapy of this disease. The uate physicians in training as well as for under- chapters in this book reflect these changes, and graduate teaching in haematology. each includes new data made available via the great advances in research into these topics, which have added so much to our understand- AM Carella ing of this complex disease. GQ Daley It is a pleasure to acknowledge the many CJ Eaves obligations incurred in the preparation of this JM Goldman book. We are grateful to all the scientists who R Hehlmann 436_Chronic_prelims 24/05/2001 3:41 pm Page viii 436_Chronic_prelims 24/05/2001 3:41 pm Page ix Contributors Claudio Anasetti, MD John M Barrett, MD Division of Clinical Research National Institutes of Health National Heart Fred Hutchinson Cancer Research Center Lung & Blood Institute 1100 Fairview Avenue North BMT Unit – RM 7N248 Seattle, WA 98109-1024 9000 Rockville Pike USA Bethesda, MD 20892 USA Jane F Apperley, MD Department of Haematology Simona Bassi, MD Hammersmith Hospital Istituto di Ematologia e Oncologia Medica Imperial College School of Medicine “Lorenzo e Ariosto Seràgnoli” Du Cane Road Via G. Massarenti 9 London W12 0NN 40138 Bologna UK Italy David Austin, PhD Ravi Bhatia, MD Department of Chemistry Department of Hematology & BMT Yale University City of Hope National Medical Center New Haven, CT 06115 1500 East Duarte Road USA Duarte, CA 91010 USA Michael J Barnett, BM Leukemia/Bone Marrow Transplantation Francesca Bonifazi, MD Program of British Columbia Istituto di Ematologia e Oncologia Medica 910 West 10th Avenue “Lorenzo e Ariosto Seràgnoli” Vancouver, BC V5Z 4E3 Via G. Massarenti 9 Canada 40138 Bologna Italy
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