Evidence-Based Pediatric Oncology

576 Pages·2007·7.857 MB·English

by  Editor:A. G. Shankar, Edi Matthay, Editor:Ross Pinkerton

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Evidence-based
Pediatric Oncology
Second Edition
Edited by
Ross Pinkerton
Childrens Cancer Unit, Mater Childrens Hospital, Raymond Terrace, Brisbane, Australia
AG Shankar
Department of Paediatric and Adolescent Oncology, University College of
London NHS Trust, London, UK
Katherine Matthay
Department of Pediatric Oncology, University of California at San Francisco,
San Francisco, CA, USA
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© 2007 by Blackwell Publishing
BMJ Books is an imprint ofthe BMJ Publishing Group Limited,used under licence
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First edition published 2002
Second edition published 2007
1 2007
Library ofCongress Cataloging-in-Publication Data
Evidence-based pediatric oncology / edited by Ross Pinkerton,A.G.
Shankar,Katherine Matthay.— 2nd ed.
p.;cm.
Rev.ed.of:Evidence-based paediatric oncology / edited by Ross
Pinkerton,Thierry Philip,Beatrice Fervers.2002.
Includes bibliographical references and index.
ISBN-13:978-1-4051-4268-7 (hardback :alk.paper)
ISBN-10:1-4051-4268-5 (hardback :alk.paper)
1. Tumors in children.2. Evidence-based pediatrics. I. Pinkerton,
C.R.(C.Ross),1950- II.Shankar,A.G.(Ananth Gouri),1962- III.
Matthay,Katherine.IV.Evidence-based paediatric oncology.
[DNLM:1. Neoplasms.2. Child.3. Evidence-Based Medicine. QZ 275
E93 2007]
RC281.C4E95 2007
618.92'994—dc22
2006027487
ISBN:978-1-4051-42687
A catalogue record for this title is available from the British Library
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Contents
Contributors,v Part 2: Leukemia, 221
AG Shankar
Acknowledgments,vi
Acute myeloid leukemia,223
Introduction,vii
Commentaries by Judith Chessells and Vaskar Saha
Ross Pinkerton
11 Induction regimens in acute myeloid
Part 1: Solid Tumors, 1 leukemia,227
Ross Pinkerton
12 Role ofautologous BMT in children with acute
1 Rhabdomyosarcoma,3 myeloid leukemia in first remission,240
Commentary by Michael Stevens and
Meriel Jenney 13 Role ofmaintenance treatment in childhood
acute myeloid leukemia,250
2 Osteosarcoma,26
Commentary by Alan Craft Childhood lymphoblastic leukemia,267
Commentaries by Vaskar Saha and Judith Chessells
3 Ewing’s sarcoma,49
Commentary by Alan Craft
14 Steroids and asparaginases during remission
4 Wilms’tumor,66 induction therapy in childhood lymphoblastic
Commentary by Daniel Green leukemia,279
5 Neuroblastoma,93 15 CNS prophylaxis in childhood lymphoblastic
Commentary by Katherine Matthay leukemia,327
6 Hepatoblastoma and malignant germ-cell
16 Continuing therapy in childhood lymphoblastic
tumors,115
leukemia – duration ofcontinuing therapy,400
Commentary by Ross Pinkerton
7 Medulloblastoma,125 Part 3: Supportive Care in Pediatric
Commentary by Joann Ater Oncology, 453
8 Glioma,150 Katherine Matthay and AG Shankar
Commentary by Joann Ater
17 Use ofhemopoietic colony stimulating factors
9 Non-Hodgkin’s lymphoma,162 (G-CSF,G-MCSF and erythropoietin),459
Commentary by Tim Eden and Ross Pinkerton
18 Cardioprotection in pediatric oncology,538
10 Hodgkin’s disease,199
Commentary by Tim Eden and Ross Pinkerton Index,553
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Contributors
Joann Ater Katherine Matthay
MD Anderson Cancer Center,University ofTexas,1515 Department ofPediatric Oncology,University ofCalifornia
Holcombe Boulevard,Houston,TX,USA at San Francisco,San Francisco,CA,USA
Judith Chessells Ross Pinkerton
Department ofHaematology,Institute ofChild Health, Director ofCancer Services,Childrens Cancer Unit,
London,UK Mater Childrens Hospital,Raymond Terrace,Brisbane,
QLD,Australia
Alan Craft
Vaskar Saha
School ofChild Medical Sciences,University ofNewcastle
upon Tyne,Royal Victoria Infirmary,Queen Victoria Road, Academic Head ofPaediatric Haematology and Oncology,
Newcastle upon Tyne,UK 3rd Floor Medical Oncology,John Vane Science Building,
Charterhouse Square,West Smithfield,London,UK
Tim Eden
AG Shankar
Academic Unit ofPaediatric Oncology,Christie Hospital
NHS Trust,Wilmslow Road,Withington,Manchester,UK Department ofPaediatric and Adolescent Oncology,
University College ofLondon NHS Trust,London,UK
Daniel Green
Michael Stevens
Department ofPediatrics,Roswell Park Cancer Institute,
Elm and Carlton Street,Buffalo,NY,USA Institute ofChild Life and Health,University ofBristol,
Bristol,UK
Meriel Jenney
Department ofPaediatric Oncology,Children’s Hospital for
Wales,Heath Park,Cardiff,South Glamorgan,UK
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Acknowledgments
We would sincerely like to thank the following societies Massachusetts Medical Society
for generously allowing the reproduction of a large www.massmed.org
number offigures originally published in their journals. 860 Winter Street,Waltham Woods Corporate Center
Waltham,MA 02451-1411
American Society of Clinical Oncology
The physician members of the Massachusetts Medical
www.asco.org
Society share a common goal – to make a difference in
1900 Duke Street,Suite 200
the lives ofour patients and in the practice ofmedicine.
Alexandria,VA 22314
For permission to reproduce images from the
The American Society ofClinical Oncology (ASCO) is
New England Journal ofMedicine.
a non-profit organization,founded in 1964,with over-
www.nejm.org
arching goals ofimproving cancer care and prevention
and ensuring that all patients with cancer receive care
ofthe highest quality.
For permission to reproduce images from the Journal
ofClinical Oncology.
www.jco.org
American Society of Hematology
www.hematology.org
1900 M Street,NW,Suite 200
Washington,DC 20036
The mission of the American Society of Hematology
(ASH) is to further the understanding, diagnosis,
treatment and prevention of disorders affecting the
blood,bone marrow,and the immunologic,hemostatic
and vascular systems, by promoting research, clinical
care,education,training and advocacy in hematology.
For permission to reproduce images from Blood.
www.bloodjournal.org
vi
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Introduction
Ross Pinkerton
The introduction of effective chemotherapy in the Similarly,there is a temptation in patients with poor
early 1960s led to a dramatic improvement in the out- prognoses to apply investigational regimens in the
come of childhood leukemia and solid tumors.Cure hope that ifthere is an improvement this will become
rates have been further improved by the judicious use evident when compared with historical controls.Such
of surgery and radiotherapy and the application of an approach has in many ways delayed progress.
appropriate staging systems based on sophisticated Reluctance to run large randomized trials has
imaging techniques. resulted in the overuse of inappropriate strategies
In recent years,the rate ofimprovement has tended to and the slow application ofeffective ones.Differences
reach a plateau and it has become increasingly impor- in outcome not only between continents, but even
tant to design trials that ask explicit questions, are within Europe – highlighted by the Eurocare project –
powered to be reliable and will provide answers in a emphasize the need for standardized,evidence-based
reasonable time.Excellent examples where this has been treatments.1
the case include the series oftrials in Wilms’tumor run The aim ofthis book is to summarize the informa-
by the National Wilms’Tumour Study Group (NWTS) tion that is available for randomized trials in child-
and International Society of Paediatric Oncology hood cancer. These data should not only provide a
(SIOP) Groups and the IRS trials in soft tissue sar- rational evidence base for current practice, but also
coma.Much has also been learned in acute leukemias indicate where there are gaps in our knowledge and
and lymphoma from the American Children’s Cancer new studies are a priority.
Group (CCG),the French Society ofPediatric Oncology The inspiration for this book was the standards,
(SFOP), Pediatric Oncology Group (POG) and UK options and recommendations (SOR) project of the
Medical Research Council (MRC) trials. National Federation of French Cancer Centres. This
Trial design is a complex procedure starting with an ambitious project set out to review clinical trials –
individual idea and ultimately brought through a multi- both randomized and non-randomized – in adult and
disciplinary group to a formal study protocol.This is a childhood cancer and provide evidence-based guide-
time-consuming process often involving contentious lines for clinical practice.2–5In the absence ofrandom-
issues and compromise on the part ofparticipants who ized trials the presentation of“best available evidence”
may have their own ideas about priorities.Moreover, helps to guide practice (Tables 1 and 2).
because of concerns over late sequelae, long-term Guidelines are ideally based on systematic reviews
follow-up is required in many studies.It is easier often that follow the Cochrane methodology.These are very
to design simpler, limited center studies which are labour intensive, requiring exhaustive searches for
under-powered and fail to address clear questions. both published and unpublished data. The recent
Consequently, the pediatric oncology literature is initiative from the University of Amsterdam group
littered with small single arm “studies”and reports of has lead the way in initiating Cochrane Reviews in
what is essentially “best standard practice”, which, childhood cancer. To date most have related to sup-
whilst of interest, often fail to take things forward. portive care and toxicity.6–8 The small number of
vii
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Introduction
Table 1 Definition oflevel ofevidence (SOR).
Level A There exists a meta-analysis of high standard or several randomized therapeutic trials of high
standard which give consistent results
Level B There exist studies, therapeutic trials, quasi-experimental trials or comparisons of populations, of
which the results are consistent when considered together
Level C There exist studies, therapeutic trials, quasi-experimental trials or comparisons of populations, of
which the results are not consistent when considered together
Level D Either the scientific data does not exist or there is only a series of cases
Expert agreement The data does not exist for the method concerned but the experts are unanimous in their judgment
Table 2 Levels ofevidence (Scottish Intercollegiate Guideline Network).
Level Type of evidence
1(cid:1)(cid:1) Evidence from high-quality meta-analyses, systematic reviews of RCTs or RCTs with a very low risk of bias
1(cid:1) Evidence from well-conducted meta-analyses, systematic reviews of RCTs or RCTs with a low risk of bias
1(cid:2) Evidence from meta-analyses, systematic reviews of RCTs or RCTs with a high risk of bias
2(cid:1)(cid:1) Evidence from high-quality systematic reviews of case-control or cohort studies or high-quality case-control
or cohort studies with a very low risk of confounding, bias or chance and a high probability that the
relationship is causal
2(cid:1) Evidence from well-conducted case-control or cohort studies with a low risk of confounding, bias or chance
and a moderate probability that the relationship is causal
2(cid:2) Evidence from case-control or cohort studies with a high risk of confounding, bias or chance and a
significant risk that the relationship is not causal
3 Evidence from non-analytic studies, e.g. case reports, case series
4 Evidence from expert opinion
randomized trials for individual tumors restricts this evaluation proves to have been unjustified.It is,there-
type ofanalysis. fore, of importance that all novel strategies are ade-
Similarly,because ofthe small number ofrandom- quately evaluated before they become accepted as
ized trials in most childhood solid tumors, formal standard practice.
meta-analysis is often not possible.Only in acute lym- It is hoped that the data in this book will provide
phoblastic leukemia are there sufficient studies asking ready access to background information for those
comparable questions for this approach to be fol- involved in trial design and also be of value to those
lowed.9,10 There are, however, solid tumors, such as early in their oncology careers who should be aware
Wilms’ tumor and rhabdomyosarcoma where meta- of what studies have been done but find that current
analysis should be attempted.Meta-analyses in child- textbooks provide only minimal details of these
hood cancer have often focused on studies on trials.From short summary tables it is impossible to
potential etiologies.11,12There may be a place for pool- assess the quality of the study or the strength of the
ing data from single arm studies to learn more about conclusions.
prognostic factors.13–15 We have been fortunate to have persuaded many
Much current practice is based on protocols that well-known figures in children’s cancer to add short
appear to produce the most favorable results in single commentaries to each section. These are aimed to
arm studies.Many are associated with significant early focus on the major conclusions from the studies pre-
and late morbidity which subsequent randomized sented and also on future research priorities.
viii
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Introduction
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