Childhood Acute Lymphoblastic Leukemia

Childhood Acute Lymphoblastic Leukemia

Ajay Vora Editor Childhood Acute Lymphoblastic Leukemia

Editor Ajay Vora Department of Paediatric Haemotology Sheffield Children's Hospital NHS Trust University of Sheffield Sheffield South Yorkshire UK ISBN 978-3-319-39707-8 DOI 10.1007/978-3-319-39708-5 ISBN 978-3-319-39708-5 (ebook) Library of Congress Control Number: 2017937013 Springer International Publishing Switzerland 2017 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 Springer Nature The registered company is Springer International Publishing AG The registered company address is: Gewerbestrasse 11, 6330 Cham, Switzerland

Contents 1 Epidemiology and Etiology of Childhood ALL.................... 1 Morten Tulstrup, Ulrik Kristoffer Stoltze, Kjeld Schmiegelow, and Jun J. Yang 2 Clinical Presentation and Prognostic Factors.................... 29 John Moppett and Rachel Dommett 3 Diagnostic Flow Cytometry and Immunophenotypic Classification............................................... 49 Julie Irving 4 Cytogenetics and Molecular Genetics........................... 61 Christine J. Harrison, Anthony V. Moorman, Claire Schwab, Ilaria Iacobucci, and Charles Mullighan 5 Pharmacokinetics, Pharmacodynamics and Pharmacogenetics of Antileukemic Drugs....................................... 99 Kjeld Schmiegelow and Inge van der Sluis 6 Minimal Residual Disease (MRD) Diagnostics: Methodology and Prognostic Significance...................... 139 J.J.M. van Dongen, V.H.J. van der Velden, M. Brüggemann, and A. Orfao 7 First Line Treatment: Current Approach....................... 163 Ajay Vora 8 Targeted Therapy and Precision Medicine...................... 183 Sarah K. Tasian and Stephen P. Hunger 9 Monoclonal Antibodies in Pediatric Acute Lymphoblastic Leukemia.................................... 201 Christiane Chen-Santel v

vi Contents 10 Cellular Therapy........................................... 239 Sara Ghorashian and Persis Amrolia 11 Relapsed Acute Lymphoblastic Leukemia of Childhood.......... 255 Su Han Lum, Denise Bonney, and Vaskar Saha 12 Medical Supportive Care for Treatment- Related Toxicity in Childhood ALL................................... 299 Etan Orgel and Deepa Bhojwani 13 Developing World Perspective................................ 323 Allen Yeoh Index......................................................... 337

Contributors Persis Amrolia Department of Bone Marrow Transplant, Great Ormond St Children s Hospital, London, UK Molecular and Cellular Immunology Section, UCL Institute of Child Health, London, UK Deepa Bhojwani Children s Center for Cancer and Blood Diseases, Children s Hospital Los Angeles, Los Angeles, USA Keck School of Medicine, University of Southern California, Los Angeles, CA, USA Denise Bonney Paediatric Haematology, Royal Manchester Children s Hospital, Central Manchester University Hospitals NHS Trust, Manchester, UK Children s Cancer Group, Paterson Building, Institute of Cancer, University of Manchester, Manchester, UK M. Brüggemann Department of Hematology, University Hospital Schleswig Holstein, Campus Kiel (UNIKIEL), Kiel, DE, Germany Christiane Chen-Santel Department of Pediatric Oncology/Hematology/BMT, Charité University Medicine Berlin, Berlin, Germany Rachel Dommett Bristol Royal Hospital for Children, Bristol, UK Sara Ghorashian Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health, London, UK Christine Harrison Leukaemia Research Cytogenetics Group, Newcastle University, Newcastle upon Tyne, UK Stephen P. Hunger Department of Pediatrics and the Center for Childhood Cancer Research, Children s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA Ilaria Iacobucci St Jude s Hospital, Memphis, USA vii

viii Contributors Julie Irving Northern Institute for Cancer Research, Newcastle University, Newcastle upon Tyne, UK Su Han Lum Paediatric Haematology, Royal Manchester Children s Hospital, Central Manchester University Hospitals NHS Trust, Manchester, UK Anthony Moorman Leukaemia Research Cytogenetics Group, Newcastle University, Newcastle upon Tyne, UK John Moppett Bristol Royal Hospital for Children, Bristol, UK Charles Mullighan St Jude s Hospital, Memphis, USA A. Orfao Department of Medicine, Cancer Research Center (IBMCC-CSIC- USAL) and Cytometry Service (NUCLEUS), University of Salamanca (USAL) and IBSAL, Salamanca, ES, Spain Etan Orgel Children s Center for Cancer and Blood Diseases, Children s Hospital Los Angeles, Los Angeles, USA Keck School of Medicine, University of Southern California, Los Angeles, CA, USA Vaskar Saha Paediatric Haematology, Royal Manchester Children s Hospital, Central Manchester University Hospitals NHS Trust, Manchester, UK Children s Cancer Group, Paterson Building, Institute of Cancer, University of Manchester, Manchester, UK Tata Translational Cancer Research Centre, Tata Medical Center, Kolkata, India Kjeld Schmiegelow, MD, DrMedSci Department of Pediatrics and Adolescent Medicine, University Hospital Rigshospitalet, Copenhagen, Denmark Institute of Clinical Medicine, University of Copenhagen, Copenhagen, Denmark Claire Schwab Leukaemia Research Cytogenetics Group, Newcastle University, Newcastle upon Tyne, UK Ulrik Kristoffer Stoltze Department of Pediatrics and Adolescent Medicine, University Hospital Rigshospitalet, Copenhagen, Denmark Sarah K. Tasian Department of Pediatrics and the Center for Childhood Cancer Research, Children s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA Morten Tulstrup Department of Pediatrics and Adolescent Medicine, University Hospital Rigshospitalet, Copenhagen, Denmark Jun J. Yang, PhD Department of Pharmaceutical Sciences, Hematological Malignancies Program, St. Jude Children s Research Hospital, Memphis, TN, USA Inge van der Sluis, MD, PhD Department of Pediatric Hematology-Oncology, Erasmus Medical Center Sophia Children s Hospital, Rotterdam, The Netherlands V.H.J. van der Velden Department of Immunology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, Netherlands

Contributors ix J.J.M. van Dongen, MD, PhD Department of Immunohematology and Blood Transfusion, Room E-03-50, Leiden University Medical Center (LUMC), ZA, Leiden, The Netherlands Department of Immunology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands Ajay Vora University College London, London, UK Allen Yeoh Viva-University Children s Medical Institute, Singapore, Singapore Khoo Teck Puat-National University Children s Medical Centre, National University Hospital, Singapore, Singapore Yong Loo Lin School of Medicine, National University of Singapore, National University Health System, Singapore, Singapore