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

被引:69
作者
Martin, Neil [1 ,2 ]
Krol, Petra [3 ]
Smith, Sally [1 ]
Murray, Kevin [4 ]
Pilkington, Clarissa A. [1 ,2 ]
Davidson, Joyce E. [5 ]
Wedderburn, Lucy R. [1 ,2 ]
机构
[1] UCL, UCL Inst Child Hlth, Rheumatol Unit, London WC1N 1EH, England
[2] Great Ormond St Hosp Sick Children, London WC1N 3JH, England
[3] Charles Univ Prague, Sch Med 1, Dept Paediat & Adolescent Med, Prague, Czech Republic
[4] Princess Margaret Hosp, Perth, WA, Australia
[5] Royal Hosp Sick Children, Dept Rheumatol, Glasgow G3 8SJ, Lanark, Scotland
基金
英国惠康基金;
关键词
Juvenile myositis; Dermatomyositis; Idiopathic inflammatory myopathy; Paediatric; Registry; Biobank; Multicentre; MYOSITIS ASSESSMENT SCALE; MHC CLASS-I; CLINICAL CHARACTERISTICS; INTERNATIONAL CONSENSUS; MUSCLE FUNCTION; DAMAGE INDEXES; DISEASE; ADULT; VALIDATION; CHILDREN;
D O I
10.1093/rheumatology/keq261
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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.
引用
收藏
页码:137 / 145
页数:9
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