Pediatric Hematology, Third Edition

827 Pages·2006·9.38 MB·English

Pediatric Hematology
THIRD EDITION
Pediatric Hematology
EDITED BY
Robert J. Arceci
King Fahd Professor of Pediatric Oncology
Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore
Maryland
USA
Ian M. Hann
Professor of Paediatric Haematology and Oncology
Great Ormond Street Hospital for Children NHS Trust
London
UK
Owen P. Smith
Professor of Paediatric Haematology
Department of Paediatric Haematology/Oncology
Our Lady’s Hospital for Sick Children
Crumlin
Dublin
Ireland
THIRD EDITION
FOREWORD BY
A. Victor Hoffbrand
© 1992, 1999 Harcourt Publishers Ltd
© 2006 by Blackwell Publishing Ltd
Blackwell Publishing, Inc., 350 Main Street, Malden, Massachusetts 02148-5020, USA
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First published 1992
Second edition 1999
Third edition 2006
1 2006
Library of Congress Cataloging-in-Publication Data
Pediatric hematology / edited by Robert J. Arceci, Ian M. Hann, Owen
P. Smith ; foreword by A. Victor Hoffbrand. — 3rd ed.
p. ; cm.
Includes bibliographical references and index.
ISBN-13: 978-1-4051-3400-2 (alk. paper)
ISBN-10: 1-4051-3400-3 (alk. paper)
1. Pediatric hematology. I. Arceci, Robert. II. Hann, Ian M.
III. Smith, Owen P.
[DNLM: 1. Hematologic Diseases. 2. Child. WS 300 P126 2006]
RJ411.P35 2006
618.92′15—dc22
2006010107
ISBN-13: 978-1-4051-3400-2
ISBN-10: 1-4051-3400-3
A catalogue record for this title is available from the British Library
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any errors in the text or for the misuse or misapplication of material in this book.
Appreciation of Richard Frederick Stevens
Richard Stevens, better known to all his friends and col- Director, Chairman of the local Trust Staff Medical Com-
leagues as Dick, died in tragic circumstances in July 2003. mittee, Secretary to the National Childhood Leukaemia Work-
As John Lilleyman’s appreciation in the Journal of the Royal ing Party, and Secretary to the UK Haemophilia Doctors’
College of Pathologytestified, Dick radiated warmth of charac- Association and at the time of his disappearance he had just
ter and was the best of company. Those of us who travelled been elected President of the Manchester Paediatric Club.
with him to meetings were regaled with clinical conundrums, His lectures always contained fascinating anecdotes on the
insight into the workings of railway timetables and delays in topic in question. He became more and more respected as a
travel, and not a little in-depth knowledge of medical and historian. Those of us who knew him well and worked with him
musical history. This was frequently interspersed with clin- sadly miss him, his excellent company and his immensely
ical anecdotes and discussion on pediatric hemato-oncology friendly support. It can be said of very few of us that we make
problems. He was a national figure both in hemophilia and in friends but no enemies. He fought for his patients, particu-
acute leukemia management, particularly myeloid leukemia. larly at the difficult time which all hemophilia patients faced
With Ian Hann he oversaw the AML 10 study, which when recombinant factor VIII became available at a price that
produced the great leap forward in survival that we had all many in the Health Service were not willing to pay.
been waiting for since the 1970s. Dick wrote extremely well His death was an unpredictable tragedy. We are privileged
in his chosen subjects and in the previous editions of this to have known and worked with this very special man. He is
text on leukemia and in the last edition on disorders of truly a great loss to pediatric hematology in the UK and the
granulopoiesis and granulocyte function. His remarkable world.
enthusiasm led to far too many calls on his time: as Clinical
v
Appreciation of Anton H. Sutor
During the period of editing the current issue of this book, 100 articles in peer-reviewed journals and numerous text-
Professor Dr Anton H. Sutor, one of the leading scientists book chapters, edited 16 textbooks, and organized many
in pediatric hemostasis, passed away. We greatly appreciate international meetings. He also earned various awards,
his significant contributions to both clinical and laboratory including the Goedecke Research Award, the Alexander
research in pediatric hemostasis. Schmidt Award of the German Society of Thrombosis and
Born in Augsburg, Germany, Anton Sutor’s early years Hemostasis (GTH), and an award for excellence in teaching.
studying medicine were in Freiburg, Berlin and Worcester He served as chair for the section on pediatric hemostasis in
(MA). During a fellowship at the Mayo Clinic in Rochester, the GTH and as co-chairman of the International Society of
Minnesota, 1967–1970, he began working in the field of pedi- Thrombosis and Haemostasis (ISTH). In 2000, Professor
atric hemostasis. He returned to Freiburg, and from 1977 to 1982 Sutor was president of the International Conference of this
he held a position as an Associate Professor at the Depart- society in Freiburg, which was combined with a meeting of
ment of Pediatric Hematology and Oncology in Münster. the Japanese Society of Pediatric Hemostasis.
In 1982, he accepted the position of Professor of Pediatric Professor Sutor was very much honored by the inter-
Hematology and Hemostasis at the University of Freiburg. national scientific community in pediatric hematology and
Professor Sutor contributed significantly to pediatric oncology. He had excellent contacts with many international
hematology through his work on vitamin K, the genetics of groups, particularly in Japan. Besides medicine, he had a pas-
thrombosis, the toxicity of heparin, and the pathophysiology sion for flying, skiing, and chamber music. His tremendous
of procoagulant and anticoagulant factors. During his last clinical and scientific work, his skills for teaching students
years, he focused on the pathophysiology and treatment of and young fellows, and his lovely personality will be always
thrombocytosis and thrombocytopenia. He published over remembered by us.
vi
Contents
List of contributors, ix 10 Sickle cell disease, 213
Corrina McMahon
Foreword, xii
Preface, xiii 11 Hemoglobin variants and the rarer hemoglobin
disorders, 231
Andreas E. Kulozik
Section 1: The Scientific Basis 12 Red cell membrane abnormalities, 255
Patrick G. Gallagher
1 Hematopoiesis: an introduction, 3
13 Thalassemias, 281
Hugh J.M. Brady
Nancy F. Olivieri and David J. Weatherall
Section 2: Marrow Failure Syndromes
Section 4: Granulocyte Disorders
2 Failure of red cell production, 11
14 Disorders of granulopoiesis and granulocyte
Sarah E. Ball
function, 305
3 Inherited bone marrow failure syndromes, 30 Arian Laurence, Pratima Chowdary and Philip Ancliff
Yigal Dror
15 Histiocytic disorders, 340
4 Acquired aplastic anemia, 64 Amir H. Shahlaee and Robert J. Arceci
Juliana Teo and Yigal Dror
16 Acute myeloid leukemia, 360
Leslie S. Kean, Robert J. Arceci and William G. Woods
17 Chronic myeloid leukemia, 384
Section 3: Red Cell Disorders
Irene A.G. Roberts and Inderjeet S. Dokal
5 Disorders of iron metabolism: iron deficiency, iron 18 Myelodysplastic syndromes, 405
overload and the sideroblastic anemias, 79 David K.H. Webb
Andrew M. Will
6 Megaloblastic anemia and disorders of cobalamin and
folate metabolism, 105 Section 5: Lymphocyte Disorders
Eric J. Werner
19 Primary and acquired immunodeficiency, 425
7 Nonimmune neonatal anemias, 130 Andrew R. Gennery, Adam H.R. Finn and
Brenda E.S. Gibson and Christina Halsey Andrew J. Cant
8 Immune hemolytic anemias, 151 20 Clinical features and therapy of lymphoblastic
Bertil Glader leukemia, 450
Owen P. Smith and Ian M. Hann
9 Disorders of erythrocyte metabolism including
porphyria, 171 21 Lymphomas, 482
Lawrence Wolfe and Peter E. Manley O.B. Eden and Ross Pinkerton
vii
Contents
Section 6: Platelet Disorders Section 8: Supportive Therapy
22 Inherited and congenital thrombocytopenia, 507 32 Blood components and fractionated plasma
Owen P. Smith products: preparation, indications and
23 Idiopathic thrombocytopenic purpura, 526 administration, 693
Paul Imbach Nancy Robitaille and Heather A. Hume
24 Thrombocytosis, 548 33 Hazards of transfusion, 724
Christof Dame Naomi L.C. Luban and Edward C.C. Wong
25 Platelet function disorders, 562 34 Management of infection in children with bone
Alan D. Michelson marrow failure, 745
Subarna Chakravorty and Ian M. Hann
Section 7: Coagulation Disorders
Section 9: Secondary Problems
26 Hemophilia A and B, 585
Judith Smith and Owen P. Smith 35 Hematologic effects of systemic disease and
nonhematopoietic tumors, 757
27 von Willebrand disease, 598
Angela Thomas
David Lillicrap
36 Lysosomal storage disorders, 778
28 Rare congenital hemorrhagic disorders, 608
Brad T. Tinkle and Gregory A. Grabowski
Nigel S. Key and Margaret A. Heisel-Kurth
37 Reference values, 792
29 Acquired disorders of hemostasis, 624
Paula S. Simpkin and Roderick F. Hinchliffe
Elizabeth A. Chalmers, Michael D. Williams and
Angela Thomas
30 Bleeding in the neonate, 643 Index, 811
Elizabeth A. Chalmers
31 Thromboembolic complications in children, 672
Mary Bauman and M. Patricia Massicotte
viii
List of contributors
Philip Ancliff Pratima Chowdary
Consultant Haematologist, Camelia Botnar Laboratories, SpR Haematology, Department of Haematology, University
Great Ormond Street Hospital for Children, Great Ormond College Hospital, 235 Euston Road, London NW1 2BU, UK
Street, London WC1N 3JH, UK
Christof Dame
Robert J. Arceci Assistant Professor, Department of Neonatology, Charite –
King Fahd Profesor of Pediatric Oncology, Johns Hopkins University Medicine Berlin, Campus Virchow-Klinikum,
Oncology Center, 1650 Orleans Street, 2M51, Baltimore, Augustenburger Platz 1, D-13353 Berlin, Germany
MD 21231-1000, USA
Inderjeet S. Dokal
Sarah E. Ball Department of Haematology, Commonwealth Building,
Reader/Honorary Consultant in Paediatric Haematology, 4th Floor, Imperial College Hammersmith Campus,
St George’s Hospital Medical School, Cranmer Terrace, Du Cane Road, London W12 0NN, UK
London SW17 0RE, UK
Yigal Dror
Mary Bauman Director, Marrow Failure and Myelodysplasia Program,
Advanced Practice Nurse Intern, Pediatric Thrombosis, The Hospital for Sick Children, 555 University Avenue,
Stollery Children’s Hospital, University of Alberta, Toronto, Ontario, M5G 1X8, Canada
WMC 2C3, 8440 112 Street, Edmonton, Alberta, T6G 2B7,
O.B. Eden
Canada
Professor of Adolescent, Teenage and Young Adult Cancer,
Hugh J.M. Brady Paediatric, Adolescent and Young Adult Oncology Unit,
Head of Unit, Molecular Haematology and Cancer Biology Christie Hospital NHS Trust, University of Manchester,
Unit, Institute of Child Health, University College London, Manchester M20 4BX, UK
30 Guilford Street, London WC1N 1EH, UK
Adam H.R. Finn
Andrew J. Cant Institute of Child Life and Health Clinical Sciences at South
Consultant in Paediatric Immunology and Infectious Bristol, UBHT Education Centre, Upper Maudlin Street,
Diseases, Ward 23A, Paediatric Immunology and Infectious Bristol BS2 8AE, UK
Diseases Unit, Newcastle General Hospital, Westgate Road,
Patrick G. Gallagher
Newcastle Upon Tyne NE4 6BE, UK
Associate Professor, Department of Pediatrics, Yale
Subarna Chakravorty University School of Medicine, 333 Cedar Street, New
Specialist Registrar in Paediatric Haematology, Great Haven, LCI 402, New Haven, CT 06520-8064, USA
Ormond Street Hospital for Children, Great Ormond Street,
Andrew R. Gennery
London WC1N 3JH, UK
Watson Senior Lecturer in Paediatric Immunology and Bone
Elizabeth A. Chalmers Marrow Transplantation, Paediatric BMT Unit, Ward 23,
Consultant Paediatric Haematologist, Department of Newcastle General Hospital, Westgate Road, Newcastle
Haematology, Royal Hospital for Sick Children, Yorkhill, Upon Tyne NE4 6BE, UK
Glasgow G3 8SJ, UK
ix
List of contributors
Brenda E.S. Gibson Arian Laurence
Consultant Paediatric Haematologist, Department of Haematology Registrar, University College Hospital
Haematology, Royal Hospital for Sick Children, Yorkhill, London, Grafton Way, London WC1E 6DB, UK
Glasgow G3 8SJ, UK
David Lillicrap
Bertil Glader Professor, Department of Pathology and Molecular
Professor of Pediatrics (Hematology/Oncology), Stanford Medicine, Richardson Laboratory, Queen’s University,
University Medical Center, 300 Pasteur Drive, Stanford, 88 Stuart Street, Kingston, Ontario K7L 3N6,
CA 94305-5208, USA Canada
Gregory A. Grabowski
Naomi L.C. Luban
Professor and Director, Division and Program in Human
Chair, Laboratory Medicine and Pathology, and Director,
Genetics, Cincinnati Children’s Hospital Medical Center,
Transfusion Medicine/Donor Center, Children’s National
3333 Burnet Avenue, Cincinnati, OH 45229-3039, USA
Medical Center, 111 Michigan Avenue NW, Washington,
Christina Halsey DC 20010, USA
Leukaemia Research Fund (LRF) Clinical Research Fellow,
Peter E. Manley
Division of Immunology, Infection and Inflammation,
Division of Pediatric Hematology/Oncology, Hasbro
Glasgow Biomedical Research Centre NHS Trust, 120
Children’s Hospital, Brown University School of Medicine,
University Place, Glasgow G12 8TA, UK
593 Eddy Street, Providence, RI 02903, USA
Ian M. Hann
M. Patricia Massicotte
Professor of Haematology, Camelia Botnar Laboratories,
Professor of Pediatrics, University of Alberta Division
Great Ormond Street Hospital for Children NHS Trust,
of Hematology and Cardiology, Stollery Children’s
Great Ormond Street, London WC1N 3JH, UK
Hospital, 8440 112 Street, Edmonton, Alberta T6G 2B7
Margaret A. Heisel-Kurth
Canada
Pediatric Hematology-Oncology, Minneapolis Childrens’
Corrina McMahon
Hospitals and Clinics of Minnesota, and Hemophilia and
Consultant Haematologist, Department of Paediatric
Thrombosis Center, Fairview-University Medical Center,
Haematology/Oncology, Our Lady’s Hospital for Sick
Minneapolis, MN, USA
Children, Crumlin, Dublin 2, Ireland
Roderick F. Hinchliffe
Senior Chief Biomedical Scientist, Department of Paediatric Alan D. Michelson
Haematology, Sheffield Children’s NHS Trust, Western Director, Center for Platelet Function Studies, and Professor
Bank, Sheffield S10 2TH, UK of Pediatrics, Medicine and Pathology, Vice Chair for
Academic Affairs, Department of Pediatrics, University of
Heather A. Hume
Massachusetts Medical School, 55 Lake Avenue North,
Executive Medical Director, Transfusion Medicine Canadian
Worcester, MA 01655, USA
Blood Services, Associate Clinical Professor, Department of
Pediatrics, Université de Montréal, Montreal, Quebec, Canada Nancy F. Olivieri
Professor of Pediatrics and Medicine, Toronto General
Paul Imbach
Hospital, 200 Elizabeth Street, Toronto, Ontario, Canada
Paediatric Oncology/Haematology, University Children’s
Hospital Basel, and Children’s Hospital Aarau, Ross Pinkerton
Roemergasse 8, CH-4005 Basel, Switzerland Director of Cancer Services, Mater Hospitals, Raymond
Leslie S. Kean Terrace, Brisbane, Queensland 4101, Australia
AFLAC Cancer Center and Blood Disorders Service, Irene A.G. Roberts
Children’s Healthcare of Atlanta/Emory University, 1405 Professor of Paediatric Haematology, Departments
Clifton Road, Atlanta, GA 30322, USA of Paediatrics and Haematology, St Mary’s and
Nigel S. Key Hammersmith Hospitals, Imperial College London,
Professor of Medicine, University of Minnesota Medical Commonwealth Building, 4th Floor, Du Cane Road,
School, MMC 480 Mayo Building, 420 Delaware Street SE, London W12 0NN, UK
Minneapolis, MN 55455, USA
Nancy Robitaille
Andreas E. Kulozik Medical Director, Blood Bank, Centre Hospitalier
Professor of Pediatrics, Universitätsklinik fuer Kinder- und Universitaire Sainte-Justine and Clinical Instructor,
Jugendmedizin, Im Neuenheimer Feld 153, D-69120 Department of Pediatrics, Université de Montréal,
Heidelberg, Germany Montreal, Quebec, Canada
x
List of contributors
Amir H. Shahlaee David K.H. Webb
Assistant Professor of Pediatric Hematology and Oncology, Consultant Haematologist, Department of Haematology,
University of Florida Health Science Center, Box 100296, Great Ormond Street Hospital for Children, Great
Gainesville, FL 32610, USA Ormond Street, London WC1N 3JH, UK
Paula S. Simpkin Eric J. Werner
Senior Biomedical Scientist, Haematology Laboratory, Professor of Pediatrics, and Director, Division of Pediatric
Sheffield Children’s NHS Trust, Western Bank, Sheffield Hematology/Oncology, Eastern Virginia Medical School,
S10 2TH, UK and Co-Director, Division of Pediatric Hematology/
Oncology, Children’s Specialty Group, Children’s Hospital
Judith Smith
of the King’s Daughters, 601 Children’s Lane, Norfolk,
Nurse Manager, National Centre Hereditary Coagulation
VA 23507, USA
Disorders, Dublin, Ireland
Andrew M. Will
Owen P. Smith
Consultant Paediatric Haematologist, Royal Manchester
Professor of Paediatric Haematology and Oncology,
Children’s Hospital, Hospital Road, Pendlebury,
Department of Paediatric Haematology/Oncology, Our
Manchester M27 4HA, UK
Lady’s Hospital for Sick Children, Crumlin, Dublin 2,
Ireland Michael D. Williams
Juliana Teo Consultant Paediatric Haematologist, Birmingham
Children’s Hospital, Steelhouse Lane, Birmingham
Pediatric Hematologist, Division of Hematology/
B4 6NH, UK
Oncology, The Hospital for Sick Children, 555 University
Avenue, Toronto, Ontario, M5G 1X8, Canada
Lawrence Wolfe
Angela Thomas Division of Pediatric Hematology/Oncology, New England
Consultant Paediatric Haematologist, Department of Medical Center, 750 Washington Street, Boston, MA 02111,
Haematology, Royal Hospital for Sick Children, Sciennes USA
Road, Edinburgh EH9 1LF, UK
Edward C.C. Wong
Brad T. Tinkle Director of Hematology and Associate Director of
Clinical Geneticist, Division of Human Genetics, Cincinnati Transfusion Medicine, Children’s National Medical
Children’s Hospital Medical Center, 3333 Burnet Avenue, Center, 111 Michigan Avenue NW, Washington,
Cincinnati, OH 45229, USA DC 20010, USA
Sir David J. Weatherall William G. Woods
Weatherall Institute of Molecular Medicine, John Radcliffe Director, The Daniel P. Amos Children’s Chair for AFLAC,
Hospital, Headington, Oxford OX3 9DS, UK Cancer Center and Blood Disorders Service, Children’s
Healthcare of Atlanta/Emory University, 1405 Clifton
Road, Atlanta, GA 30322, USA
xi