Studies identified in our initial database search
Study
Reason for exclusion
Adams 1998 [1] This paper described the rationale behind the design of a clinical trial
investigating stroke prevention in children with sickle cell anaemia. The
paper did not, however, develop or use methodology for selecting the
outcomes used in the trials.
Allen 1985[2]
This review article discussing design issues in clinical trials of children
with brain tumours is based on the author’s personal opinion. Although
features of some outcomes are discussed, this does not qualify as
methodology of selecting outcomes.
Andrasik
2005[3]
This review article discussing design issues in clinical trials of children
with headache is based on the author’s personal opinion. Although features
of some outcomes are discussed, methodology of selecting outcomes is not
used or developed.
Arnold 2003[4]
This study validated the use of parent-reported outcomes in clinical trials of
children with autism. The study was excluded, however, because it did not
relate to the actual methodology of selecting an outcome, but the validation
of an outcome which was already selected.
Ball 2003[5]
This study aimed to develop a definition of what constitutes a ‘responder’
in clinical trials of children with abdominal pain. Therefore the authors
used methodology to refine an existing outcome, rather than using
methodology for selecting outcomes for use in clinical trials of children
with abdominal pain.
Beghi 1993[6]
This paper describes the rationale behind the design of a clinical trial,
including the outcomes which were selected. The paper did not, however,
describe methodology for selecting outcomes for use in clinical trials.
Bower 1994[7]
The review article discussed in detail the potential ways of measuring
outcomes, but did not describe the selection of outcomes for use in clinical
trials of children with cerebral palsy.
Children’s
This paper described the rationale behind various aspects of a clinical trial
Amalgam Trial in children requiring dental restoration. The authors do not describe the
Study Group [8] methodology behind selecting the outcome.
Curley 2005[9]
This review article aims to review the performance characteristics of
outcomes used in clinical trials in children with sepsis. The study does not,
however, use methodology for selecting outcomes for use in these trials.
Curley 2006[10] This paper describes the rationale behind the design of a clinical trial,
including the outcomes which were selected. The paper did not, however,
describe methodology for selecting outcomes for use in clinical trials.
Furst 2005[11]
The review article does discuss scleroderma in paediatric trials, but
discusses how to measure an outcome, rather than select one.
Guilbert
2004[12]
This paper described the rationale behind the design of a clinical trial, but
did not explain the methodology behind selecting the outcomes measured in
the study.
Hargrave
2006[13]
In this review article the authors appraised the consistency between
different studies of children with brainstem glioma. The authors do not use
methodology for selecting outcome measures for use in clinical trials, nor
do they review the methodology that other authors use for this purpose.
Hollander
2004[14]
This review article related to study design in clinical trials of children with
autism. Because it was a commentary based on the personal opinion of the
author it was excluded, as it did not describe methodology for selecting
outcomes for use in clinical trials.
Juniper 2001[15] The study did not use methodology for selecting outcomes, and was not
relevant specifically to children
King 2004[16]
This systematic review did not use methodology for selecting outcomes in
clinical trials, but did suggest outcomes for use in future bronchiolitis
clinical trials.
Kirkham
2004[17]
This article reviews clinical trial design issues in children with arterial
stroke, and suggests strategies for future studies, but does not discuss
selection of outcomes or use methodology for selecting outcomes.
Law 1989[18]
This review article relates to several aspects of clinical trial design in the
field of occupational therapy. Although characteristics of instruments for
measuring outcomes are reviewed, the selection of outcomes or outcome
domains is not.
Mahoney
2006[19]
This article described the work of a research network for paediatric
cardiology clinical trials, including work on validating outcomes for use in
clinical trials. The article did not, however, describe studies which use
methodology for selecting which outcomes should be measured in clinical
trials of children with cardiology problems.
Marshall
2005[20]
Although the article does quote some paediatric trials, it does not
specifically state that the outcomes can be used in paediatric research.
Martin 2006[21] This consensus statement relates to clinical trial design in adults with graftversus-host disease (GVHD), but the authors do not specifically state that
the outcomes suggested are for use in paediatric trials.
McBride 1990
[22]
This review article is about outcomes in clinical trials of infants with
bronchiolitis. The article does not describe or develop a methodology for
selecting outcomes though.
McDaid
2005[23]
At the end of this systematic review of treatments for retinoblastoma, the
authors suggest outcomes for use in future clinical trials. These suggestions
are a result of the methodological assessment of previously published
studies, but not a result of methodology which the authors used to
determine the most appropriate outcomes to use.
Mehta 2003 [24] This study reviews outcomes in trials of adults with renal failure, but not
children.
Meltzer
2006[25]
Paper mentioned several outcome measures as part of guidelines for clinical
trials in rhinosinusitis, but did not discuss the process or methodology of
selecting them.
Mohanraj 2003
[26]
In this review article authors compare outcomes used to measure various
outcome domains in clinical trials of children with epilepsy. The article
does not, however, use methodology to select outcomes or outcome
domains.
Ozolins 2005
[27]
In the discussion section of this clinical trial of children with acne, the
author assesses the performance characteristics of some of the outcomes
which were used during the study, but he does not discuss outcome
domains which should be selected in future trials, or use methodology to
select outcomes.
Pauwels 2001
[28]
This paper discusses aspects of the design of a clinical trial in children with
asthma. The author does not describe the methodology used to select the
outcome domains or outcomes.
Ravelli 1997
[29]
This study assessed the collinearity between outcomes used in clinical trials
of children with rheumatoid arthritis. Although the authors, therefore,
assess performance characteristics of some outcomes, they do not use
methodology for selecting outcomes for use in clinical trials.
Romagnoli 2001 This clinical trial assessed the long-term effects of steroids on newborn
[30]
infants. The authors, when discussing design issues relevant to possible
future clinical trials, suggest outcomes for use in these trials. However, the
authors do not use methodology to select these outcomes.
Rosenfeld 2001 This study aimed to define ‘exacerbations’ as an outcome for use in clinical
[31]
trials in children with cystic fibrosis. The study did not, however, aim to
select outcomes for use in these clinical trials.
Rudorfer 1993
[32]
In this review article, the author reviews tools and instruments for
measuring outcomes in clinical trials of children with psychiatric illness,
but does not use methodology to select outcomes or outcomes that should
be used in these studies.
Ruperto 1996
[33]
This study assessed the redundancy of outcomes used in clinical trials of
children with rheumatoid arthritis. Although the authors, therefore, assess
performance characteristics of some outcomes, they do not use
methodology for selecting outcomes for use in clinical trials.
Schoenfeld 2002 The article discusses a study in which the participants were all adults.
[34]
Schonbeck 2005 This paper describes the rationale behind several aspects of a clinical trial
[35]
of influenza and pneumococcal vaccination, but the authors do not describe
methodology used to select the outcomes measured in the trial.
Shapiro 1999
[36]
The article describes several aspects of trial design in a clinical trial of
children with asthma, but the authors do not describe the methodology used
to select the outcomes.
Sharek 2002 [37] This study assesses the collinearity between five different measures of
asthma status, but does not use methodology to actually select outcomes for
clinical trials.
Spahn 2003 [38] This literature review assesses some of the performance characteristics of
outcomes used in clinical research in asthma, but the author does not use
methodology to identify which outcomes should be used in future clinical
trials.
Spencer 1997
[39]
The article does not use methodology for selecting outcome measures for
use in trials. It relates to the change in distribution of the DMF index, an
outcome used in clinical trials in dentistry. The author suggests outcomes
for use in future clinical trials, but does not use methodology for selecting
these outcomes.
Thase 1999 [40] This is a review article which does not use methodology for selecting
outcomes for use in clinical trials. The article does not relate to clinical
trials in children.
Theander 2005
[41]
The study does not relate to clinical trials in children.
Van Zanten
2006 [42]
The article discusses validation of an instrument to measure an outcome
(dyspepsia), rather than using methodology to select one. It is a study in
adults.
Walsh 2003 [43] In this study, the authors attempt to develop a physiological definition for
bronchopulmonary dysplasia, a widely used outcome in neonatal trials. In
other words, the authors are not using methodology for selecting outcomes,
but improving the performance characteristics of an existing outcome.
Walsh 2004[44] In this study, the authors assess the effect of a physiological definition on
the incidence of bronchopulmonary dysplasia. The study does not select
outcomes for use in future clinical trials.
Zhang 2000 [45] This study assesses the performance characteristics of outcomes used in
neonatal clinical trials, but is not about selection of outcome domains or
outcomes.
Studies suggested by MCRN CSG members
Study
Reason for exclusion
Aurora 2005[46] In this study the correlation of various tests of lung function in children
with CF was assessed. The study did not relate to the selection of which
outcomes should be measured in clinical trials.
Brody 2004[47]
In this study the authors assessed the correlation between high resolution
CT scanning and pulmonary function tests in children with CF. The
authors do not use methodology to determine which outcomes should
actually be measured in clinical trials of children with CF however.
Clayton 2002[48] Consensus about management of children with 21-hyroxylase deficiency,
not relating to clinical trial design
Clayton 2005[49] Consensus about management of children treated with Growth Hormone,
not relating to clinical trial design.
Dunger 2004[50] Consensus statement about management of children with Diabetic
Ketoacidosis, not relating to clinical trial design
Hughes 2006[51] Consensus statement about children suffering from intersex disorders, not
relating to clinical trial design.
Lum 2007[52]
In this study the authors assessed the extent at which multiple breath
washout parameters are able to detect airway dysfunction in children with
CF. The study did not relate to the selection of outcomes for use in clinical
trials.
Ordonez
2003[53]
This study assesses the performance characteristics of inflammatory and
microbiological markers in clinical trials of children with cystic fibrosis.
The authors do not use methodology to determine which outcomes should
actually be measured in these clinical trials however.
Ordonez
2004[54]
In this study the authors assess the reproducibility of inflammatory
markers as an outcome measure in children with CF. The authors do not
use methodology to determine which outcomes should actually be
measured in clinical trials of children with CF however.
Ratjen 2006[55]
In this review article the authors discuss some ways of measuring airway
inflammation in children with CF. They do not use methodology however
to determine which outcomes should be measured in clinical trials if
children with this condition.
Suri 2003[56]
In this study the authors assess the safety and usefulness of inducing
sputum from children with CF in order to measure various inflammatory
and microbiological markers that could be used as outcomes in clinical
trials. The study does not use methodology to determine which outcomes
should actually be measured in clinical trials of children with CF.
Whitehouse
2007[57]
In this study consumers were involved in the design of a clinical trial of
children with migraine. The consumers were not involved in the selection
of which outcomes should be measured in the trial (personal
communication from Dr Whitehouse)
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