Study
Condition
Type of Study
Sepsis
Consensus statement
De Rouen
2002 [2]
Dental restoration
Griffiths 2005 [3]
Crohn’s disease
Paper describes the design
of specific trial– the
authors of the trial
consulted with a
multidisciplinary team of
experts to select the
outcomes measured in the
trial.
(No of participants not
reported)
Consensus statement
Goldstein 2005
[1]
People involved in selecting
outcomes
Paediatric critical care
specialists; physicians and
scientists with research
experience in paediatric
sepsis, members of past
consensus conferences on
adult sepsis
Physicians, epidemiologists,
biostatisticians. Number of
participants not stated
Patient
involvement
No
Methods used to select outcome
No
The consensus was reached by
discussion within the group.
Clinicians with expertise in
paediatric inflammatory
bowel disease; clinical trial
design specialists
(Number of participants not
reported)
No
Semi-structured discussion was used to
select the most appropriate outcome
domains and outcomes to measure
The group arrived at consensus by way
of semi-structured discussion.
Study
Condition
Type of Study
Pavletic 2006 [4]
Graft Versus Host
disease (GVHD)
Consensus statement
regarding trials of children
with GVHD, including
formulation of a
preliminary core set of
outcomes.
Finer 2006 [5]
Neonatal apnoea
On behalf of
NDDI
Short 2006 [6]
On behalf of
NDDI
Neonatal
cardiovascular
instability
People involved in selecting
outcomes
Clinical experts in GVHD;
Experts from other fields
(gastroenterology and
rheumatology) who were
experienced in selecting
outcomes for use in clinical
trials within those fields.
(Number of participants not
stated)
Consensus statement
Physicians, research experts
(Number of participants not
reported)
Consensus conference
regarding trials of neonates
with cardiovascular
instability
Physicians, research experts
(Number of participants not
reported)
Patient
involvement
Yes – at the end
of the report,
the authors
acknowledge
“patients and
patient and
research
advocacy
groups”. The
level of their
involvement
was not
reported by the
authors.
No
Methods used to select outcome
No
The group arrived at consensus by
using semi-structured discussion.
The group arrived at consensus by way
of semi-structured discussion.
The consensus was reached by semistructured discussions by the group.
Study
Anand 2005
Anand 2006 [7,8]
Condition
Type of Study
Neonatal pain
1. Systematic review of
literature relating to trial
design in studies of
neonatal pain
2. Consensus statement
regarding trial design
considerations relevant to
clinical trials of pain relief
for neonates
Neonatal
Postoperative
Cardiac
Dysfunction
Neonatal seizures
Consensus statement
regarding trials of neonates
with Postoperative Cardiac
Dysfunction
Consensus statement
on behalf of the
NDDI
Roth 2006[9]
On behalf of
NDDI
Clancy 2006 [10]
On behalf of
NDDI
People involved in selecting Patient
outcomes
involvement
Experts on neonatal pain,
No
clinical researchers
(Number of participants not
reported)
Physicians, research experts.
(number of participants not
reported)
No
Physicians, research experts
(Number of participants not
reported)
No
Methods used to select outcome
A systematic review of literature
relating to trial design in studies of
neonatal pain was performed, and
specific articles relating to outcome
measurement were reviewed. These
findings were discussed amongst a
group of experts, using a semistructured discussion approach, and a
consensus statement regarding the
outcomes that should be measured was
developed. Although a lot of the
discussion centred around tools to
measure outcomes, there was also
discussion about composite outcomes,
and also about outcome domains, such
as long-term neurodevelopmental
outcome.
The group arrived at consensus by
using semi-structured discussion.
The consensus was reached by semistructured discussions by the group.
Study
Lux 2004
[11]Osborne 2001
[12]On behalf of
the West Delphi
collaboration
Condition
Type of Study
Infantile spasms
Consensus statement
regarding trials of children
with IS, including
formulation of a
preliminary core set of
outcomes.
People involved in selecting Patient
outcomes
involvement
Authors who had published
No
articles on infantile spasms
during the previous 10 years
were invited to join the
group, and were asked to
invite others who may be
interested in participating.
(Questionnaires were sent to
133 people, and there were
eventually 31 participants)
Methods used to select outcome
Delphi consensus performed over 6
‘rounds’ of questions:
1) Multiple Choice Questions (MCQ)
covering various aspects of clinical
trial design, including outcomes.
2) Qualitative comments and MCQ
questions regarding the same questions,
having fed the results of round 1 back
to the group
3) Formulation of statements from
rounds 1 and 2 which represented
majority opinion. Participants were
invited to respond as to whether they
agreed or disagreed with these.
4) These statements were modified,
and participants commented on their
suitability and content.
5) and 6) consisted of formulation of a
draft and subsequently a final paper
which were altered according to the
group’s comments.
Study
Condition
Type of Study
Carlson 2003 [13]
Bipolar affective
disorder
Consensus workshop
regarding trial design
considerations relevant to
clinical trials of
adolescents with bipolar
disorder.
LaFrance (2006)
[14]
Non-Epileptic
Seizures (NES)
Consensus statement about
clinical trials of patients
suffering from NES
People involved in selecting
outcomes
Clinical researchers with
expertise in bipolar illness,
pharmaceutical industry
sponsors, staff of the Food
and Drug Administration,
representatives of families
with affected children.
(Total number of
participants: 53)
Patient
involvement
Yes. The group
included parents
of children who
were affected
by bipolar
affective
disorder.
Multidisciplinary group of
neurologists, psychiatrists,
neuropsychiatrists,
psychologists, statisticians,
nurses and other researchers
familiar with NES (44
participants)
No
Methods used to select outcome
Several separate groups used semistructured discussion to come to
consensus on several issues relating to
clinical trial design, and one group was
specifically given the remit of
discussing outcomes. This included
discussion on which primary and
secondary outcomes should be
measured, and which aspects of mania
or depression best represent change in
a child’s condition.
Structured and focussed discussion.
The group discussed the question
“which variables or domains should be
regarded as reflecting outcome?” The
group identified areas of outcomes
relevant to NES that could potentially
be used as outcomes reflecting the
effects of an intervention. These
included psychosocial outcomes,
clinical outcome, psychiatric status,
health-related quality of life, medical
resource utilisation, and
psychophysiological markers.
Study
Smith 1996 [15]
Ramsey 1994 [16]
Condition
Type of Study
Asthma
Questionnaire-based
survey of health
professionals and
researchers.
Cystic Fibrosis
Consensus statement
regarding trials of children
with Cystic Fibrosis,
including formulation of a
preliminary core set of
outcomes.
People involved in selecting Patient
outcomes
involvement
Researchers and clinical
No
experts with experience in
treating asthma. (14
participants)
Clinicians with expertise in
CF, laboratory and clinical
researchers in the field of
CF, representatives from the
U.S. Food and Drug
Administration (Number of
participants not reported)
No
Methods used to select outcome
Questionnaires to health professionals
and researchers asking which outcomes
they would use in various scenarios,
including clinical trials, to assess
whether a patient’s asthma had been
improved by an intervention. The
participants were also asked to provide
an estimate of how valid they thought
the indicators were.
The group arrived at consensus by
using semi-structured discussion.
Study
Miller 2001
Rider 2002
Rider 2003
Rider 2004 [17–
20]
On behalf of the
IMACS
collaboration
Oddis 2005 [21]
Describing the
work of the
IMACS
collaboration
On behalf of the
PRINTO
collaboration
Ruperto 2003
Ruperto 2004
Ruperto 2006 [22–
24]
Condition
Type of Study
Juvenile idiopathic
inflammatory
myopathies (IIM)
Development of core set of
outcomes for use in trials
of children with IIM, by
formulation of a consensus
statement
Systemic lupus
erythematosus
(SLE) and
dermatomyositis
(DM)
Development of a core set
of outcomes for use in
trials of children with SLE
and DM
People involved in selecting
outcomes
Adult and paediatric
specialists, patient support
group leaders with expertise
in IIM. This group was
called The International
Myositis Outcomes
Assessment Collaborative
Study Group (IMACS).
(Number of participants not
reported)
Patient
involvement
Patient support
group leaders
were the parents
of children with
IIM.
Specialists in paediatric
rheumatology
267 people were initially
involved, followed by a
meeting of 40 participants
No
Methods used to select outcome
It was decided that in order to fully
understand the totality of effects of
interventions on patients with IIM, the
outcomes should measure disease
activity, disease damage and quality of
life. A literature review was performed
to review the performance
characteristics of outcome measures
used in IIM clinical trials, and Delphi
technique was used to determine which
outcomes best represented these
domains. A definition of improvement
was then developed, and this was
subsequently validated.
1) Development of a core set of
outcomes
Phase 1: (Delphi Technique) A
questionnaire was sent to the group,
and participants were asked to rank the
10 variables they judged as clinically
most important when determining
whether a patient with SLE or DM has
responded to therapy. Variables ranked
by at least 10 responders were then
listed alphabetically and participants
were asked again to rank their top 10
choices.
Phase 2: (Nominal group technique) A
four-day international consensus
conference was attended by 40
participants. Using 5 exercises, the core
set of outcomes was determined: 1)
Classify all variables into one of 2
domains (disease activity and disease
damage).The participants were invited
to suggest any other domains. 2)
Classify variables into ‘concepts’ of
disease activity and damage 3) Select
and rank the domains that should be
included in the core set 4) Select the
variables that should be used to
measure these domains
2) Validation of core outcomes
This was done in clinical practice, on
patients who were starting a new
medication. Validation of the following
characteristics was conducted:
feasibility; face and content validity;
responsiveness, discriminative ability;
convergent construct validity; internal
consistency
3) Development of a definition of
improvement
Condition
Giannini 1997 [25] Juvenile Arthritis
Type of Study
Development and
validation of a set of core
outcomes using consensus
formation techniques, and
development of a
definition of improvement
for individual patients.
People involved in selecting Patient
outcomes
involvement
Paediatric rheumatologists,
No
OMERACT participants,
and academic practitioners.
(16 participants in initial
‘Advisory Council’;
dissemination of the results
to 198 paediatric
rheumatologists for their
opinions)
Methods used to select outcome
1) 25 variables which had been used in
juvenile arthritis (JIA) trials were listed
in a questionnaire.
2) This questionnaire was sent to a 16member ‘advisory council’ who were
asked to rank their top 6 choices, and
list other variables which were not
included on the list. 16 variables
received votes, and these became the
‘candidate variables’.
3) The performance characteristics
(validity, reliability, sensitivity to
change, redundancy) of the candidate
variables were reviewed using existing
literature.
4) Using nominal group technique, the
group selected a preliminary core set of
6 outcomes. These were physician
global assessment of disease activity,
parent or patient reported assessment of
overall well being, functional ability,
number of joints with active arthritis,
number of joints with limited range of
movement, and erythrocyte
sedimentation rate.
5) International consensus on the
acceptability of this core set of
variables was ascertained using a
questionnaire.
6) The multicollinearity of these
outcomes was assessed using real
patients.
7) A definition of improvement was
developed
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