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2015 ; 16
(ä): 268
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Development of an internationally agreed minimal dataset for juvenile
dermatomyositis (JDM) for clinical and research use
#MMPMID26063230
McCann LJ
; Kirkham JJ
; Wedderburn LR
; Pilkington C
; Huber AM
; Ravelli A
; Appelbe D
; Williamson PR
; Beresford MW
Trials
2015[Jun]; 16
(ä): 268
PMID26063230
show ga
BACKGROUND: Juvenile dermatomyositis (JDM) is a rare autoimmune inflammatory
disorder associated with significant morbidity and mortality. International
collaboration is necessary to better understand the pathogenesis of the disease,
response to treatment and long-term outcome. To aid international collaboration,
it is essential to have a core set of data that all researchers and clinicians
collect in a standardised way for clinical purposes and for research. This should
include demographic details, diagnostic data and measures of disease activity,
investigations and treatment. Variables in existing clinical registries have been
compared to produce a provisional data set for JDM. We now aim to develop this
into a consensus-approved minimum core dataset, tested in a wider setting, with
the objective of achieving international agreement. METHODS/DESIGN: A two-stage
bespoke Delphi-process will engage the opinion of a large number of key
stakeholders through Email distribution via established international paediatric
rheumatology and myositis organisations. This, together with a formalised
patient/parent participation process will help inform a consensus meeting of
international experts that will utilise a nominal group technique (NGT). The
resulting proposed minimal dataset will be tested for feasibility within existing
database infrastructures. The developed minimal dataset will be sent to all
internationally representative collaborators for final comment. The participants
of the expert consensus group will be asked to draw together these comments,
ratify and 'sign off' the final minimal dataset. DISCUSSION: An internationally
agreed minimal dataset has the potential to significantly enhance collaboration,
allow effective communication between groups, provide a minimal standard of care
and enable analysis of the largest possible number of JDM patients to provide a
greater understanding of this disease. The final approved minimum core dataset
could be rapidly incorporated into national and international collaborative
efforts, including existing prospective databases, and be available for use in
randomised controlled trials and for treatment/protocol comparisons in cohort
studies.