Multiple myeloma and related disorders Edited by Gösta Gahrton MD PhD Professor, Department of Medicine Karolinska Institute, Huddinge University Hospital Stockholm, Sweden Brian G.M. Durie MD Departments of Hematology and Oncology Cedars-Sinai Medical Centre, Los Angeles, CA, USA and Diana M. Samson MD Senior Lecturer in Haematology Hammersmith Hospital, London, UK A member ofthe Hodder Headline Group LONDON First published in Great Britain in 2004 by Arnold, a member of the Hodder Headline Group, 338 Euston Road, London NW1 3BH http://www.arnoldpublishers.com Distributed in the United States of America by Oxford University Press Inc., 198 Madison Avenue, New York, NY10016 Oxford is a registered trademark of Oxford University Press ©2004 Arnold All rights reserved. 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Please send your comments to [email protected] Contents List of contributors v Preface ix List of abbreviations x Notes on reference annotation xiv The colour plate section appears between pages 338 and 339 PART 1 HISTORY AND EPIDEMIOLOGY 1 1 History of multiple myeloma 3 Robert A. Kyle and David P. Steensma 2 Epidemiology and etiology of plasma cell neoplasms 15 Daniel E. Bergsagel and P. Leif Bergsagel PART 2 BIOLOGY AND PATHOPHYSIOLOGY 27 3 The immune system in myeloma 29 Douglas E. Joshua, P. Joy Ho, John Gibson and Ross D. Brown 4 Molecular biology and cytogenetics 46 Johannes Drach, Jutta Ackermann, Sonja Seidl and Hannes Kaufmann 5 Biology of the malignant plasma cell 58 Federico Caligaris-Cappio 6 Pathophysiology of myeloma bone disease 74 Babatunde O. Oyajobi and Gregory R. Mundy PART 3 CLINICAL FEATURES, DIAGNOSIS, AND INVESTIGATION 89 7 Clinical features 91 Dietrich Peest 8 Hematologic investigations: morphologic and phenotypic features of myeloma marrow diagnosis 102 Thomas M. Grogan, Deborah Gelbspan and Lisa Rimsza 9 Biochemical and immunological investigations 125 Joanna M. Sheldon and Pamela G. Riches 10 The role of imaging in multiple myeloma 155 Brian G.M. Durie 11 Innovative approaches for diagnosis and monitoring 164 Faith E. Davies and Gareth J. Morgan iv Contents 12 Multiple myeloma: differential diagnosis and prognosis 179 Jesús F. San Miguel and Ramón García-Sanz PART 4 TREATMENT OF MYELOMA 201 13 Principles of management 203 Diana M. Samson 14 Chemotherapy, steroids, and interferon 220 Diana M. Samson 15 Radiotherapy Deborah A. Frassica and Maria C. Jacobs 241 16 Autologous stem cell transplantation 250 Jean-Luc Harousseau and Michel Attal 17 Allogeneic transplantation and treatment with donor lymphocytes 260 Gösta Gahrton and Per Ljungman 18 Supportive care 275 Heinz Ludwig and Johannes Drach 19 Biological basis of therapy: new molecular targets 292 Terry H. Landowski and William S. Dalton 20 Immunotherapeutic approaches 305 Håkan Mellstedt PART 5 COMPLICATIONS 317 21 Management of bone disease 319 Eugene V. McCloskey 22 Management of myeloma patients with renal failure 339 Joan Bladé and Laura Rosiñol 23 Neurologic aspects of monoclonal gammopathy of undetermined significance, 350 multiple myeloma, and related disorders Robert A. Kyle and Angela Dispenzieri PART 6 RELATED DISORDERS 367 24 Monoclonal gammopathies of undetermined significance: the transition from MGUS to myeloma 369 John A. Lust, Kathleen A. Donovan and Philip R. Greipp 25 Solitary bone and extramedullary plasmacytoma 379 Meletios A. Dimopoulos and Lia A. Moulopoulos 26 Plasma cell leukaemia 388 Xavier Leleu, Ibrahim Yakoub-Agha and Thierry Facon 27 AL amyloidosis 400 Raymond L. Comenzo 28 Heavy-chain diseases 420 Jean-Paul Fermand and Jean-Claude Brouet 29 Waldenström’s disease 429 Giampaolo Merlini and Steven P. Treon Index 453 Contributors Jutta Ackermann Meletios A. Dimopoulos MD Department of Medicine I, Clinical Division of Oncology, Professor of Therapeutics, Department of Clinical Therapeutics, University Hospital, Vienna, Austria University of Athens School of Medicine, Athens, Greece Michel Attal Angela Dispenzieri MD Head of Hematological Department, Hôpital Purpan, Toulouse, Consultant, Division of Hematology and Internal Medicine, France Mayo Clinic; and Assistant Professor of Medicine, Mayo Medical School, Rochester, MN, USA Daniel E. Bergsagel CMMDDPhil Professor of Medicine, University of Toronto, Department of Kathleen A. Donovan PhD Medicine, Ontario Cancer Institute/Princess Margaret Hospital, Professional Associate in Research, Division of Hematology and Toronto, Ontario, Canada Internal Medicine, Mayo Clinic, Rochester, MN, USA P. Leif Bergsagel MDFRCP(C) Johannes Drach MD Associate Professor of Medicine, Weill Medical College of Department of Medicine I, Clinical Division of Oncology, Cornell University, Department of Medicine, New York University Hospital, Vienna, Austria Presbyterian Hospital, New York, NY, USA Joan Bladé MD Brian G.M. Durie MD Department of Hematology, Institute of Hematology and Departments of Hematology and Oncology, Cedars-Sinai Oncology, Postgraduate School of Hematology ‘Farreras Valentí’, Medical Centre, Los Angeles, CA, USA Institut d’Investigacions Biomèdiques ‘August Pi i Sunyer’, Thierry Facon MD Hospital Clínic, University of Barcelona, Barcelona, Spain Service des Maladies du Sang, Hôpital Claude Huriez, CHU, Jean-Claude Brouet MDPhD Lille, France Service d’Immunologie Clinique, Hôpital Saint-Louis, Paris, France Jean-Paul Fermand MD Ross D. Brown PhDMBAFAIMS Service d’Immunologie Clinique, Hôpital Saint-Louis, Paris, Institute of Haematology, University of Sydney, Royal Prince France Alfred Hospital, Camperdown, NSW, Australia Deborah A. Frassica MD Federico Caligaris-Cappio MD Assistant Professor of Radiation Oncology, Department of Professor of Internal Medicine, Università Vita Salute, Istituto Radiation Oncology and Molecular Radiation Sciences, Sidney Scientifico San Raffaele, Milan, Italy Kimmel Comprehensive Cancer Center, Johns Hopkins Raymond L. Comenzo MD University, Baltimore, MD, USA Division of Hematologic Oncology, Department of Medicine, Memorial Sloan-Kettering Cancer Center, New York, NY, USA Gösta Gahrton MDPhD Professor, Department of Medicine, Karolinska Institute, William S. Dalton PhDMD Huddinge University Hospital, Stockholm, Sweden H Lee Moffitt Cancer Center and Research Institute, University of South Florida, Tampa, FL, USA Ramón García-Sanz MDPhD Department of Hematology, University Hospital of Salamanca, Faith E. Davies MBBChMRCPMD Salamanca, Spain Department of Health Clinician Scientist and Academic SpR, Epidemiology and Genetics Unit, Academic Department of Deborah Gelbspan MD Haematology and Oncology, School of Medicine, University of UMC, Department of Pathology, University of Arizona, Tucson, Leeds, Leeds, UK AZ, USA vi Contributors John Gibson MBBSPhDFRACPFRCPA Håkan Mellstedt MDPhD Institute of Haematology, University of Sydney, Royal Prince Professor of Oncologic Biotherapy, Karolinska Institute, Alfred Hospital, Camperdown, NSW, Australia Managing Director, Cancer Centre Karolinska, Department of Oncology and Hematology, Karolinska Hospital, Stockholm, Philip R. Greipp MD Sweden Consultant, Division of Hematology and Internal Medicine, Mayo Clinic, and Professor of Medicine, Mayo Medical School, Giampaolo Merlini MD Rochester, MN, USA Professor of Clinical Biochemistry, Department of Biochemistry, University of Pavia, and Biotechnology Research Laboratories, Thomas M. Grogan MD University Hospital Policlinico San Matteo, Pavia, Italy UMC, Department of Pathology, University of Arizona, Tucson, AZ, USA Gareth J. Morgan PhDFRCPFRCPath Professor of Haematology, Epidemiology and Genetics Unit, Jean-Luc Harousseau Academic Unit of Haematology and Oncology, School of Professor, Université de Nantes, Centre Hospitalier Régional, Medicine, University of Leeds, Leeds, UK Hôtel-Dieu, Département d’Hématologie, Nantes, France P. Joy Ho MBBSDPhilFRACPFRCPA Lia A. Moulopoulos MD Institute of Haematology, University of Sydney, Royal Prince Assistant Professor, Department of Radiology, University of Alfred Hospital, Camperdown, NSW, Australia Athens School of Medicine, Athens, Greece Maria C. Jacobs MD Gregory R. Mundy MD Director, Department of Radiation Oncology, Mercy Medical Departments of Cellular and Structural Biology and Center, Baltimore, MD, USA Orthopedics, University of Texas Health Science Center at San Antonio, San Antonio Cancer Institute, San Antonio, TX, USA Douglas E. Joshua BScMBBSDPhilFRACPFRCPA Professor and Head of Institute of Haematology, University of Babatunde O. Oyajobi MDChBPhD Sydney, Royal Prince Alfred Hospital, Camperdown, NSW, Australia Departments of Cellular and Structural Biology and Molecular Medicine, University of Texas Health Science Center at San Hannes Kaufmann MD Antonio, San Antonio, TX, USA Department of Medicine I, Clinical Division of Oncology, University Hospital, Vienna, Austria Dietrich Peest MD Professor of Medicine and Clinical Immunology, Department Robert A. Kyle MD of Hematology and Oncology, Hannover Medical School, Consultant, Division of Hematology and Internal Medicine, Hannover, Germany Mayo Clinic; and Professor of Medicine and of Laboratory Medicine, Mayo Medical School, Rochester, MN, USA Pamela G. Riches PhDFRCPath Professor, Protein Reference Unit, Department of Biochemistry Terry H. Landowski PhD and Immunology, St George’s Hospital Medical School, Research Assistant Professor, Department of Medicine, Arizona London, UK Cancer Center, University of Arizona, Tucson, AZ, USA Xavier Leleu MD Lisa Rimsza MD UMC, Department of Pathology, University of Arizona, Tucson, Service des Maladies du Sang, Hôpital Claude Huriez, CHU, AZ, USA Lille, France Per Ljungman MDPhD Laura Rosiñol MD Department of Medicine, Karolinska Institute, Huddinge Department of Hematology, Institute of Hematology and University Hospital, Stockholm, Sweden Oncology, University of Barcelona, Barcelona, Spain Heinz Ludwig MD Diana M. Samson MD Professor,Department of Medicine and Medical Oncology, Senior Lecturer in Haematology, Hammersmith Hospital, Wilhelminenspital, Vienna, Austria London, UK John A. Lust MDPhD Jesús F. San Miguel MDPhD Consultant, Division of Hematology and Internal Medicine, Professor of Hematology, Department of Mayo Clinic, and Associate Professor of Medicine, Mayo Hematology, University Hospital of Salamanca, Medical School, Rochester, MN, USA Salamanca, Spain Eugene V. McCloskey MBBChMRCPMD Sonja Seidl MD WHO Collaborating Centre for Metabolic Bone Diseases, Department of Medicine I, Clinical Division of Oncology, University of Sheffield Medical School, Sheffield, UK University Hospital, Vienna, Austria Contributors vii Joanna M. Sheldon PhDMRCPath Steven P. Treon MDMAPhD Protein Reference Unit, Department of Biochemistry Director, Waldenstrom’s Macroglobulinemia Program, Dana and Immunology, St George’s Hospital Medical School, Farber Cancer Institute, Instructor of Medicine, Harvard London, UK University, Boston, MA, USA David P. Steensma MD Ibrahim Yakoub-Agha MD Fellow in Hematology, Mayo Graduate School of Medicine, Service des Maladies du Sang, Hôpital Claude Huriez, CHU, Mayo Clinic, Rochester, MN, USA Lille, France This page intentionally left blank Preface Since the publication of our original book on multiple Very important progress has been made in stem-cell myeloma in 1996,there has been an enormous increase transplantation and it is now confirmed in more than one in knowledge about the disease:more than 5000 articles prospective study that high-dose therapy with autologous have been published in scientific journals.The develop- transplantation is superior to conventional chemother- ment has been impressive both in the basic knowledge apy for patients under 65 years of age. It also seems about the disease and in its treatment. that tandem transplantation may be superior to single New technologies have made it possible to refine stud- autologous transplantation, at least for subgroups of ies on chromosomes and genes and to gain information patients.Interesting attempts are being made to decrease about gene expression.Microarray is in its early stage,but the intensity of the conditioning regimen for allogeneic already there have been pattern descriptions that may transplantation,and to utilize the immune effect of the have prognostic implications. donor marrow and donor lymphocytes. We have learned not only that deletions of chromo- These and other new developments, not only in some 13 are important but also that chromosome 14 multiple myeloma, but also in related disorders, such as translocations involving candidate oncogenes on other amyloid light-chain amyloidosis, heavy-chain disease, chromosomes,such as Cyclin D1 or FGFR3,may be even and Waldenström’s macroglobulinemia, are described more important in the pathogenesis of the disease.The in this new,expanded book.Each chapter area is able to prognostic impact ofchromosomal abnormalities is now stand alone;thus,there is some deliberate overlap between clearer than before, and the complicated interaction chapters. between the stroma cells,the myeloma cells,and numer- With this approach,our hope is that the book will be ous cytokines and their receptors has been better of interest not only for scientists and specialists in the explained. Most importantly, attempts have been made field,but also for practitioners and non-specialists with to utilize this new knowledge to develop targeted ther- an interest in basic aspects oftreatment ofpatients with apy and,although results are still modest,improvements multiple myeloma. have been obtained with thalidomide and analogs, as Gösta Gahrton well as with proteasome inhibitors,such as bortezomib. Brian G.M.Durie Many other approaches to targeted therapy are being Diana M.Samson attempted and are reviewed.
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