title: Development of an internationally agreed minimal dataset for juvenile dermatomyositis (JDM) for clinical and research use.
abstract: 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
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Alder Hey Children's NHS Foundation Trust, Eaton Road, Liverpool, UK. liza.mccann@alderhey.nhs.uk.
authors.forename: Liza J
authors.initials: LJ
authors.lastname: McCann
authors.affiliation: MRC North West Hub for Trials Methodology Research, Department of Biostatistics, University of Liverpool, Liverpool, UK. j.j.kirkham@liverpool.ac.uk.
authors.forename: Jamie J
authors.initials: JJ
authors.lastname: Kirkham
authors.affiliation: Infection, Immunology, and Rheumatology Section UCL Institute of Child Health, University College London, London, UK. l.wedderburn@ucl.ac.uk.|Great Ormond Street Hospital NHS Foundation Trust, London, UK. l.wedderburn@ucl.ac.uk.|Centre for Adolescent Rheumatology at University College London, University College London Hospital, London, UK. l.wedderburn@ucl.ac.uk.
authors.forename: Lucy R
authors.initials: LR
authors.lastname: Wedderburn
authors.affiliation: Great Ormond Street Hospital NHS Foundation Trust, London, UK. clarissa.pilkington@gosh.nhs.uk.|Centre for Adolescent Rheumatology at University College London, University College London Hospital, London, UK. clarissa.pilkington@gosh.nhs.uk.
authors.forename: Clarissa
authors.initials: C
authors.lastname: Pilkington
authors.affiliation: IWK Health Centre and Dalhousie University, 5850 University Avenue, Halifax, NS, B3K 6R8, Canada. Adam.Huber@iwk.nshealth.ca.
authors.forename: Adam M
authors.initials: AM
authors.lastname: Huber
authors.affiliation: Università degli Studi di Genova and Istituto Giannina Gaslini, Via G. Gaslini 5, 16147, Genoa, Italy. Angeloravelli@ospedale-gaslini.ge.it.
authors.forename: Angelo
authors.initials: A
authors.lastname: Ravelli
authors.affiliation: MRC North West Hub for Trials Methodology Research, Department of Biostatistics, University of Liverpool, Liverpool, UK. D.E.Appelbe@liverpool.ac.uk.
authors.forename: Duncan
authors.initials: D
authors.lastname: Appelbe
authors.affiliation: MRC North West Hub for Trials Methodology Research, Department of Biostatistics, University of Liverpool, Liverpool, UK. P.R.Williamson@liverpool.ac.uk.
authors.forename: Paula R
authors.initials: PR
authors.lastname: Williamson
authors.affiliation: Alder Hey Children's NHS Foundation Trust, Eaton Road, Liverpool, UK. M.W.Beresford@liverpool.ac.uk.|Department of Women's and Children's Health, Institute of Translational Medicine, University of Liverpool, Liverpool, UK. M.W.Beresford@liverpool.ac.uk.
authors.forename: Michael W
authors.initials: MW
authors.lastname: Beresford
mesh_terms: D035843:Biomedical Research / Q000379:methods*; D032921:Consensus; D003245:Consensus Statements as Topic; D003299:Cooperative Behavior; D066264:Datasets as Topic; D003697:Delphi Technique; D003882:Dermatomyositis / Q000175:diagnosis / Q000503:physiopathology / Q000523:psychology / Q000628:therapy; D034742:Electronic Mail; D006801:Humans; D033183:Interdisciplinary Communication; D007391:International Cooperation; D020407:Internet; D010290:Parents / Q000523:psychology; D010361:Patients / Q000523:psychology; D016730:Program Development; D012107:Research Design; D011795:Surveys and Questionnaires