title: A national registry for juvenile dermatomyositis and other paediatric idiopathic inflammatory myopathies: 10 years' experience; the Juvenile Dermatomyositis National (UK and Ireland) Cohort Biomarker Study and Repository for Idiopathic Inflammatory Myopathies.
abstract: OBJECTIVE
  The paediatric idiopathic inflammatory myopathies (IIMs) are a group of rare chronic inflammatory disorders of childhood, affecting muscle, skin and other organs. There is a severe lack of evidence base for current treatment protocols in juvenile myositis. The rarity of these conditions means that multicentre collaboration is vital to facilitate studies of pathogenesis, treatment and disease outcomes. We have established a national registry and repository for childhood IIM, which aims to improve knowledge, facilitate research and clinical trials, and ultimately to improve outcomes for these patients.
  
  
  METHODS
  A UK-wide network of centres and research group was established to contribute to the study. Standardized patient assessment, data collection forms and sample protocols were agreed. The Biobank includes collection of peripheral blood mononuclear cells, serum, genomic DNA and biopsy material. An independent steering committee was established to oversee the use of data/samples. Centre training was provided for patient assessment, data collection and entry.
  
  
  RESULTS
  Ten years after inception, the study has recruited 285 children, of which 258 have JDM or juvenile PM; 86% of the cases have contributed the biological samples. Serial sampling linked directly to the clinical database makes this a highly valuable resource. The study has been a platform for 20 sub-studies and attracted considerable funding support. Assessment of children with myositis in contributing centres has changed through participation in this study.
  
  
  CONCLUSIONS
  This establishment of a multicentre registry and Biobank has facilitated research and contributed to progress in the management of a complex group of rare muscloskeletal conditions.
authors.affiliation: Rheumatology Unit, UCL Institute of Child Health, Great Ormond Street Hospital, 30 Guilford Street, University College London, London WC1N 1EH, UK.
authors.forename: Neil
authors.initials: N
authors.lastname: Martin
authors.forename: Petra
authors.initials: P
authors.lastname: Krol
authors.forename: Sally
authors.initials: S
authors.lastname: Smith
authors.forename: Kevin
authors.initials: K
authors.lastname: Murray
authors.forename: Clarissa A
authors.initials: CA
authors.lastname: Pilkington
authors.forename: Joyce E
authors.initials: JE
authors.lastname: Davidson
authors.forename: Lucy R
authors.initials: LR
authors.lastname: Wedderburn
mesh_terms: D015415:Biomarkers; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D003882:Dermatomyositis / Q000503:physiopathology; D018450:Disease Progression; D005260:Female; D006801:Humans; D007494:Ireland; D008297:Male; D009220:Myositis / Q000503:physiopathology*; D012042:Registries / Q000592:standards*; D012720:Severity of Illness Index; D006113:United Kingdom
chemical_list: D015415:Biomarkers
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