Childhood Leukemia: A Practical Handbook PDF
Preview Childhood Leukemia: A Practical Handbook
Pediatric Oncology Gregory H. Reaman • Franklin O. Smith (Editors) Childhood Leukemia A Practical Handbook Editors Gregory H. Reaman Franklin O. Smith, III George Washington University School Cincinnati Children’s Hospital Medical of Medicine & Health Sciences Center, University of Cincinnati The Children’s National Medical Center Collage of Medicine III Michigan Ave. NW, 3333 Burnett Avenue Washington, D.C. 20010 Cincinnati, OH, USA [email protected] [email protected] ISBN 978-3-642-13780-8 e-ISBN 978-3-642-13781-5 DOI 10.1007/978-3-642-13781-5 Springer Heidelberg Dordrecht London New York Library of Congress Control Number: 2010930092 © Springer-Verlag Berlin Heidelberg 2011 This work is subject to copyright. All rights are reserved, whether the whole or part of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broadcasting, reproduction on microfilm or in any other way, and storage in data banks. 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Cover design: eStudioCalamar, Figueres/Berlin Printed on acid-free paper Springer is part of Springer Science+Business Media (www.springer.com) Preface Among the truly great success stories of modern medicine are the striking improvements in survival and cure rates for children with acute leukemia during the past 4–5 decades. This is particularly, but no longer exclusively, true for acute lymphoblastic leukemia (ALL). Although improvements in supportive care have clearly contributed to this success in both ALL and acute myeloid leukemia (AML), the improvements have largely resulted from specific refinements in anti-leukemia therapy and enhanced biological insights into these diseases. Although definitive mechanisms of causation remain elusive, they are far less so. Genomic interrogation of the leukemias, both the leukemia cell and the affected host, has and will continue to yield much useful insight as to molecular etiology as the technology improves. Such findings have enormous potential for therapeutic exploitation and hopefully, through investigation of gene- environment relationships, provide important information for the generation of risk prevention strategies. The final common pathway for translating scientific discovery to clinical applica- tion is through clinical research and the conduct of controlled clinical trials, ultimately resulting in continually improved standards of care. It has been an honor and immea- surable source of gratification to have played a small role in this success. Despite the success, we remain resolute in advancing science to fight this battle for as long as we are able and until such time that cure is a reality for every child with leukemia. We are enormously indebted to our legendary teachers and mentors as well as to numerous wonderful colleagues in basic, clinical, statistical, and epidemiologic research, many of whom have contributed to this project. In addition to our scientific colleagues as collaborators, clinical research would be impossible without the effec- tive and essential partnership of patients and families impacted by leukemia who are willing participants in clinical trials. Many have directly benefitted as a result of their participation, and many more have unselfishly contributed to knowledge which has only helped those who have followed them. It is to all of these children and families that we humbly and thankfully dedicate this book. Gregory H. Reaman Franklin O. Smith III v Acknowledgments We would like to express our sincere appreciation for the enormous editorial assis- tance provided to us by Dr. Judy Racadio, to the untiring efforts of our assistants, Ms. Vaishali Revollo and Ms. Holly Ward, and to the staff at Springer-Verlag. We are also indebted to our wives, Susan and Phyllis, and to our families for their patience and support. vii Contents Part I: Genetics and the Epidemiology of Leukemia in Children 1 Epidemiology of Acute Childhood Leukemia .................... 3 Julie A. Ross, Kimberly J. Johnson, Logan G. Spector, and John H. Kersey Part II: Biology of Pediatric Leukemia 2 The Biology of Acute Lymphoblastic Leukemia.................. 29 William L. Carroll, Mignon Loh, Andrea Biondi, and Cheryl Willman 3 Biology of Acute Myeloid Leukemia ........................... 63 Robert J. Arceci and Soheil Meshinchi Part III: Treatment Considerations in Childhood Leukemia 4 Classification and Treatment of Acute Lymphoblastic Leukemia.... 79 Stephen P. Hunger, Valentino Conter, Elizabeth A. Raetz, Maria Grazia Valsecchi, and Guenter Henze 5 Treatment of Acute Myeloid Leukemia......................... 121 Brenda Gibson, John Perentesis, Todd A. Alonzo, and Gertjan J.L. Kaspers Part IV: The Impact of Pharmacogenetics and Pharmacogenomics on Childhood Leukemia 6 Pharmacogenetic and Pharmacogenomic Considerations in the Biology and Treatment of Childhood Leukemia ............ 163 Jun J. Yang, Parinda A. Mehta, Mary V. Relling, and Stella M. Davies Part V: The Potential Role of Biologically Targeted Therapy for Childhood Leukemia 7 Promising Targeted Agents................................... 193 Patrick Brown, Gregory H. Reaman, Nita L. Seibel, and Pamela Kearns ix x Contents 8 Strategies for New Agent Development and Clinical Trial Considerations ........................................ 215 Malcolm Smith, Meenakshi Devidas, Keith Wheatley, Richard B. Lock, and Sally Hunsberger Part VI: Late Effects of Leukemia and Its Therapy 9 Late Sequelae in Children with Acute Lymphoblastic Leukemia: Impact on Long-Term Survival and Quality of Life .................................. 245 Joseph P. Neglia, Maura O’Leary, and Smita Bhatia 10 Acute Toxicities, Late Sequelae, and Quality of Survivorship in Children with Acute Myeloid Leukemia: The Impact of Allogeneic Stem Cell Transplant.................. 265 Julianne Byrne, John Horan, and H. Stacy Nicholson Part VII: Psychosocial Implications of Acute Leukemia Diagnosis and Treatment for Children and Families 11 Appreciation and the Interdisciplinary Management of the Psychosocial Impact of Leukemia on Children and Their Families.......................................... 285 Anne L. Angiolillo, Momcilo Jankovic, Riccardo Haupt, Kathleen Ruccione, E. Anne Lown, and Robert B. Noll Part VIII: Global Strategies to Improve Leukemia Care and Outcome for Children 12 Improved Outcome for Children with Acute Leukemia: How to Address Global Disparities ............................ 305 Yaddanapudi Ravindranath, Hans Peter Wagner, Giuseppe Masera, Fulgencio Baez, Anjo.J.P. Veerman, Jacqueline Cloos, Raul Ribeiro, and Gregory H. Reaman Part IX: Perspectives and Future Direction 13 Future Challenges and Opportunities to Improve Outcomes for Children with Leukemia . . . . . . . . . . . . . . . . . . . . . . . . . 325 Gregory H. Reaman and Franklin O. Smith Index . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 331 Part I Genetics and the Epidemiology of Leukemia in Children Epidemiology of Acute 1 Childhood Leukemia Julie A. Ross, Kimberly J. Johnson, Logan G. Spector, and John H. Kersey Contents 1.4.3 Comprehensive Use of Animal Models to Assess Exposure/Cancer Relationships ....................19 1.5 Conclusion ...................................................................20 1.1 Classification and Natural History ..............................3 1.1.1 Classification: Immunophenotype, Morphology ............3 References ..............................................................................21 1.1.2 Classification: Molecular and Cytogenetics ....................4 1.1.3 Natural History of Leukemia ..........................................6 1.2 Incidence, Survival, and Trends ..................................7 1.2.1 International Incidence Rates ..........................................7 1.1 Classification and Natural History 1.2.2 U.S. Incidence Rates and Trends ....................................7 1.2.3 Survival .........................................................................10 1.1.1 Classification: Immunophenotype, 1.3 Risk Factors for Childhood Leukemia ......................11 1.3.1 Genetic Syndromes .......................................................11 Morphology 1.3.2 Environmental and Other Risk Factors for Childhood Leukemia ...............................................11 1.3.3 Lifestyle Factors ............................................................13 All cells that comprise distinct entities in the blood 1.3.4 Genetic Susceptibility ...................................................16 (e.g., platelets, T-cells, etc.) arise from a pluripotent 1.4 Challenges and Future Directions .............................17 stem cell, which can form lymphoid stem cells or trilin- 1.4.1 Improvements in Identification of Cases eage myeloid stem cells. In the classical model of and Controls for Studies in the United States ...............17 hematopoiesis, lymphoid stem cells differentiate fur- 1.4.2 Opportunities for Validation .........................................18 ther into T-cell progenitor and B-cell progenitor cells, which eventually can mature into T- and B-cells, respec- J.A. Ross (*) tively (McKenzie 1996). Myeloid stem cells form Clinical Research, Department of Pediatrics, myeloid cell progenitors, granulocyte/macrophage pro- University of Minnesota Cancer Center, MMC 422, 420 genitors, and eosinophil progenitors, from which Delaware St. S.E., Minneapolis, MN, 55455 USA e-mail: [email protected] mature cells such as erythrocytes, platelets, polymor- phonuclear leukocytes, monocytes/macrophages, and K.J. Johnson Department of Pediatrics, MMC 715, 420 Delaware St. S.E., eosinophils arise. However, recent data support a more Minneapolis, MN, 55455 USA complex model for hematopoiesis, whereby intermedi- email: [email protected] ate progenitor cells retain both lymphoid and myeloid L.G. Spector potential; thus the developmental potential of cells at Department of Pediatrics, various stages is less clear (Ceredig et al. 2009; University of Minnesota Cancer Center, MMC 715, 420 Delaware St. S.E., Minneapolis, Kawamoto and Katsura 2009). Nevertheless, acute leu- MN, 55455 USA kemias that arise in children often occur in the earlier e-mail: [email protected] stages of lymphoid or myeloid cell maturation. J.H. Kersey Childhood ALL was initially classified using the Department of Pediatrics, French-American-British (FAB) Cooperative Group University of Minnesota Cancer Center, MMC 86, criteria using morphological features (Bennett et al. 420 Delaware St. S.E., Minneapolis, MN, 55455 USA e-mail: [email protected] 1976) and consisted of three subgroups: L1, L2, and G.H. Reaman and F.O. Smith (eds.), Childhood Leukemia, 3 DOI: 10.1007/978-3-642-13781-5_1, © Springer-Verlag Berlin Heidelberg 2011
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