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
Open Access
- 7 September 2010
- journal article
- research article
- Published by Oxford University Press (OUP) in Rheumatology
- Vol. 50 (1), 137-145
- https://doi.org/10.1093/rheumatology/keq261
Abstract
Objectives. 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.Keywords
This publication has 44 references indexed in Scilit:
- Long‐term outcome and prognostic factors of juvenile dermatomyositis: A multinational, multicenter study of 490 patientsArthritis Care & Research, 2009
- Juvenile dermatomyositis: extramuscular manifestations and their managementCurrent Opinion in Rheumatology, 2009
- Juvenile dermatomyositis: new developments in pathogenesis, assessment and treatmentBest Practice & Research Clinical Rheumatology, 2009
- Damage extent and predictors in adult and juvenile dermatomyositis and polymyositis as determined with the myositis damage indexArthritis & Rheumatism, 2009
- HLA-DPB1 associations differ between DRB1*03 positive anti-Jo-1 and anti-PM-Scl antibody positive idiopathic inflammatory myopathyRheumatology, 2009
- Predicting the course of juvenile dermatomyositis: Significance of early clinical and laboratory featuresArthritis & Rheumatism, 2008
- Age-dependent inhibition of ectopic calcification: a possible role for fetuin-A and osteopontin in patients with juvenile dermatomyositis with calcinosisRheumatology, 2008
- Effectiveness of infliximab in the treatment of refractory juvenile dermatomyositis with calcinosisRheumatology, 2008
- Persistent association of nailfold capillaroscopy changes and skin involvement over thirty‐six months with duration of untreated disease in patients with juvenile dermatomyositisArthritis & Rheumatism, 2008
- Clinical associations of autoantibodies to a p155/140 kDa doublet protein in juvenile dermatomyositisRheumatology, 2007