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
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
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DOI：
10.1093/rheumatology/keq261
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发表时间：
2011-01-01
期刊：
影响因子：
5.5
通讯作者：
Wedderburn, Lucy R.
中科院分区：
文献类型：
--
作者：
Martin, Neil;Krol, Petra;Wedderburn, Lucy R.
方法。英国建立了一个由中心和研究小组组成的网络来为这项研究做出贡献。就标准化患者评估、数据收集表格和样本方案达成一致。生物样本库包括外周血单核细胞、血清、基因组 DNA 和活检材料的收集。成立了一个独立的指导委员会来监督数据/样本的使用。中心提供患者评估、数据收集和输入方面的培训。结果。启动十年后，该研究已招募了 285 名儿童，其中 258 名患有 JDM 或青少年 PM； 86%的病例贡献了生物样本。直接链接到临床数据库的连续采样使其成为非常有价值的资源。该研究已成为 20 个子研究的平台，并吸引了大量资金支持。通过参与本研究，贡献中心对肌炎儿童的评估发生了变化。结论。多中心登记处和生物库的建立促进了研究，并有助于在一组复杂的罕见肌肉骨骼疾病的管理方面取得进展。
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.