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Acute Promyelocytic Leukemia Oussama Abla • Francesco Lo Coco Miguel A

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Acute Promyelocytic Leukemia Oussama Abla • Francesco Lo Coco Miguel A Acute Promyelocytic Leukemia Oussama Abla • Francesco Lo Coco Miguel A. Sanz Editors Acute Promyelocytic Leukemia A Clinical Guide Editors Oussama Abla M.D. Francesco Lo Coco M.D. Division of Hematology/Oncology Department of Hematology The Hospital for Sick Children and University of Tor Vergata University of Toronto Rome, Italy Toronto, Ontario, Canada Miguel A. Sanz M.D. Department of Medicine, Hospital Universitari i Politecnic La Fe, University of Valencia Valencia, Spain Centro de Investigacion Biomedica en Red de Cancer Instituto Carlos III, Madrid, Spain ISBN 978-3-319-64256-7 ISBN 978-3-319-64257-4 (eBook) https://doi.org/10.1007/978-3-319-64257-4 Library of Congress Control Number: 2017962620 © Springer International Publishing AG, part of Springer Nature 2018 This work is subject to copyright. All rights are reserved by the Publisher, whether the whole or part of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broadcasting, reproduction on microfilms or in any other physical way, and transmission or information storage and retrieval, electronic adaptation, computer software, or by similar or dissimilar methodology now known or hereafter developed. The use of general descriptive names, registered names, trademarks, service marks, etc. in this publication does not imply, even in the absence of a specific statement, that such names are exempt from the relevant protective laws and regulations and therefore free for general use. The publisher, the authors and the editors are safe to assume that the advice and information in this book are believed to be true and accurate at the date of publication. Neither the publisher nor the authors or the editors give a warranty, express or implied, with respect to the material contained herein or for any errors or omissions that may have been made. The publisher remains neutral with regard to jurisdictional claims in published maps and institutional affiliations. Printed on acid-free paper This Springer imprint is published by the registered company Springer International Publishing AG part of Springer Nature The registered company address is: Gewerbestrasse 11, 6330 Cham, Switzerland Preface Since its first identification by Leif Hillestad 60 years ago, acute promyelo- cytic leukemia (APL) has evolved from being the most fatal to the most cur- able type of acute myeloid leukemia. Acute Promyelocytic Leukemia: A Clinical Guide is the first book entirely dedicated to APL, a disease with a unique and fascinating biology, whose treatment has become a paradigm of modern targeted therapy. Every chapter in this volume is written by a renowned expert in the field. The first three chapters address the historical aspects, origins, and molecular mechanisms of this leukemia. The following three chapters focus on the patho- physiology of bleeding and thrombosis, early death, and prognostic factors. The next three cover the evolution of therapy from chemotherapy-based to all-trans retinoic acid-chemotherapy, arsenic trioxide, and finally chemother- apy-free regimens. Subsequently, the book discusses minimal residual disease monitoring, treatment of refractory/relapsed disease, and the role of hemato- poietic stem cell transplant. Special situations such as APL in children, in the elderly, and in pregnancy, therapy-related APL, and rare variants are also addressed. The final three chapters focus on therapy-related complications and management of APL in developing countries. We would like to thank all the authors for their generous contributions to this volume. Toronto, ON, Canada Oussama Abla Rome, Italy Francesco Lo Coco Madrid, Spain Miguel A. Sanz v Contents 1 History of Acute Promyelocytic Leukemia ���������������������������������� 1 Laurent Degos 2 Molecular Targets of Treatment in Acute Promyelocytic Leukemia ���������������������������������������������������� 17 Ramy Rahmé, Cécile Esnault, and Hugues de Thé 3 The Leukemic Stem Cell �������������������������������������������������������������� 29 Thalia Vlachou†, Giulia De Conti†, Anna Giulia Sanarico, and Pier Giuseppe Pelicci 4 Molecular Genetics of Acute Promyelocytic Leukemia �������������� 41 Lourdes Mendez, Ming Chen, and Pier Paolo Pandolfi 5 Acute Promyelocytic Leukemia Coagulopathy �������������������������� 55 Anna Falanga, Laura Russo, and Pau Montesinos 6 Early Death in Acute Promyelocytic Leukemia �������������������������� 71 Sören Lehmann 7 Prognostic Factors in Acute Promyelocytic Leukemia �������������� 87 Javier de la Serna, Pau Montesinos, and Miguel A. Sanz 8 First-Line Therapy for Acute Promyelocytic Leukemia: Chemotherapy-Based Approach �������������������������������������������������� 99 Aaron D. Goldberg and Martin S. Tallman 9 First-Line Therapy: ATRA-ATO/Reduced Chemotherapy Approach �������������������������������������������������������������� 113 Harry Iland 10 Cure of Acute Promyelocytic Leukemia Without Chemotherapy �������������������������������������������������������������������������������� 133 Mary-Elizabeth M. Percival and Elihu H. Estey 11 Minimal Residual Disease in Acute Promyelocytic Leukemia ���������������������������������������������������������������������������������������� 153 Laura Cicconi and Eva Barragàn 12 Treatment of Refractory and Relapsed Acute Promyelocytic Leukemia �������������������������������������������������������������� 163 Eva Lengfelder vii viii Contents 13 Hematopoietic Stem Cell Transplantation in Acute Promyelocytic Leukemia ������������������������������������������������ 171 Jaime Sanz and Miguel A. Sanz 14 Acute Promyelocytic Leukemia in Children �������������������������������� 179 Matthew A. Kutny and Anna Maria Testi 15 Management of Acute Promyelocytic Leukemia in the Elderly ���������������������������������������������������������������������������������� 197 Ramy Rahmé, Lionel Adès, and Pierre Fenaux 16 Special Situations in Acute Promyelocytic Leukemia ���������������� 203 Massimo Breccia, Gloria Iacoboni, and Miguel A. Sanz 17 Acute Promyelocytic Leukemia in Developing Countries: A Chemotherapy-Based Approach ���������������������������������������������� 211 Luisa Corrêa de Araújo Koury, Raul Ribeiro, and Eduardo M. Rego 18 Acute Promyelocytic Leukemia in Developing Countries: ATO-Based Approach ������������������������ 217 Vikram Mathews 19 Therapy-Related Acute Promyelocytic Leukemia ���������������������� 231 Kristen Pettit and Richard A. Larson 20 Rare Acute Leukemia Variants Involving Retinoic Acid Receptor Genes ������������������������������������������������������ 243 Laura Cicconi and Oussama Abla 21 Management of Treatment-­Related Complications in Acute Promyelocytic Leukemia ������������������������������������������������ 257 Ombretta Annibali and Giuseppe Avvisati Index �������������������������������������������������������������������������������������������������������� 269 Contributors Oussama Abla, M.D. Division of Hematology/Oncology, Department of Pediatrics, Hospital for Sick Children, Toronto, ON, Canada Lionel Adès, M.D., Ph.D Service Hématologie Séniors, Hôpital Saint- Louis, Assistance Publique—Hôpitaux de Paris, Paris, France Université Paris Diderot, Paris, France Ombretta Annibali, M.D., Ph.D. UOC Hematology, Stem Cell Transplantation, University Hospital Campus Bio Medico, Rome, Italy Luisa Corrêa de Araújo Koury, M.D. Department of Internal Medicine, Medical School of Ribeirão Preto and Centre for Cell-Based Therapy, University of São Paulo, São Paulo, SP, Brazil Giuseppe Avvisati, M.D., Ph.D. UOC Hematology, Stem Cell Transplantation, University Hospital Campus Bio Medico, Rome, Italy Eva Barragàn, Ph.D. Department of Medical Biopathology, Hospital Universitario La Fe, Valencia, Spain Massimo Breccia, M.D., Ph.D. Department of Cellular Biotechnologies and Hematology, Sapienza University, Rome, Italy Ming Chen, M.D. Department of Medicine and Pathology, Cancer Research Institute, Beth Israel Deaconess Cancer Center, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, USA Laura Cicconi, M.D. Department of Biomedicine and Prevention, University Tor Vergata, Rome, Italy Giulia De Conti, Ph.D. Department of Experimental Oncology, European Institute of Oncology, Milan, Italy Laurent Degos, M.D., Ph.D. Paris Diderot University—Academy of Medicine, Paris, France Cécile Esnault, Ph.D. Université Paris Diderot, Sorbonne Paris Cité, Institut Universitaire d’Hématologie, Hôpital St. Louis, Paris, France INSERM UMR 944, Equipe Labellisée par la Ligue Nationale contre le Cancer, Hôpital St. Louis, Paris, France CNRS UMR 7212, Hôpital St. Louis, Paris, France ix x Contributors Elihu H. Estey, M.D. Department of Medicine, University of Washington, Seattle, WA, USA Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA Anna Falanga, M.D. Department of Immunohematology and Transfusion Medicine and the Hemostasis and Thrombosis Center, Hospital Papa Giovanni XXIII, Bergamo, Italy Pierre Fenaux, M.D., Ph.D. Service Hématologie Séniors, Hôpital Saint- Louis, Assistance Publique—Hôpitaux de Paris, Paris, France Université Paris Diderot, Paris, France Aaron D. Goldberg, M.D., Ph.D. Leukemia Service, Memorial Sloan Kettering Cancer Center, Weill Cornell Medical College, New York, NY, USA Gloria Iacoboni, M.D. Department of Hematology, Hospital Universitari i Politècnic La Fe, Valencia, Spain Harry Iland, M.B.B.S., F.R.A.C.P., F.R.C.P.A. Institute of Haematology, Royal Prince Alfred Hospital, Camperdown, NSW, Australia Matthew
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