McCann Liza J, Arnold Katie, Pilkington Clarissa A, Huber Adam M, Ravelli Angelo, Beard Laura, Beresford Michael W, Wedderburn Lucy R
Alder Hey Children's NHS Foundation Trust, Eaton Road, Liverpool L12 2AP, UK.
Rheumatology Unit, UCL Institute of Child Health, University College London, London, UK.
Pediatr Rheumatol Online J. 2014 Jul 21;12:31. doi: 10.1186/1546-0096-12-31. eCollection 2014.
Juvenile dermatomyositis (JDM) is a rare but severe autoimmune inflammatory myositis of childhood. International collaboration is essential in order to undertake clinical trials, understand the disease and improve long-term outcome. The aim of this study was to propose from existing collaborative initiatives a preliminary minimal dataset for JDM. This will form the basis of the future development of an international consensus-approved minimum core dataset to be used both in clinical care and inform research, allowing integration of data between centres.
A working group of internationally-representative JDM experts was formed to develop a provisional minimal dataset. Clinical and laboratory variables contained within current national and international collaborative databases of patients with idiopathic inflammatory myopathies were scrutinised. Judgements were informed by published literature and a more detailed analysis of the Juvenile Dermatomyositis Cohort Biomarker Study and Repository, UK and Ireland.
A provisional minimal JDM dataset has been produced, with an associated glossary of definitions. The provisional minimal dataset will request information at time of patient diagnosis and during on-going prospective follow up. At time of patient diagnosis, information will be requested on patient demographics, diagnostic criteria and treatments given prior to diagnosis. During on-going prospective follow-up, variables will include the presence of active muscle or skin disease, major organ involvement or constitutional symptoms, investigations, treatment, physician global assessments and patient reported outcome measures.
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. This preliminary dataset can now be developed into a consensus-approved minimum core dataset and tested in a wider setting with the aim of achieving international agreement.
幼年皮肌炎（JDM）是一种罕见但严重的儿童自身免疫性炎性肌病。开展临床试验、了解该疾病并改善长期预后，国际合作至关重要。本研究的目的是从现有的合作项目中提出一个JDM的初步最小数据集。这将构成未来国际共识认可的最小核心数据集发展的基础，该数据集将用于临床护理和为研究提供信息，从而实现各中心之间的数据整合。
成立了一个具有国际代表性的JDM专家工作组，以制定一个临时最小数据集。对目前特发性炎性肌病患者的国家和国际合作数据库中包含的临床和实验室变量进行了审查。判断依据是已发表的文献以及对英国和爱尔兰幼年皮肌炎队列生物标志物研究与储存库的更详细分析。
已生成一个临时最小JDM数据集，并附有相关的定义词汇表。临时最小数据集将在患者诊断时以及进行前瞻性随访期间要求提供信息。在患者诊断时，将要求提供患者人口统计学信息、诊断标准以及诊断前给予的治疗。在进行前瞻性随访期间，变量将包括活动性肌肉或皮肤疾病的存在、主要器官受累情况或全身症状、检查、治疗、医生整体评估以及患者报告的结局指标。
一个国际认可的最小数据集有可能显著加强合作，使各团队之间能够进行有效的沟通，提供最低护理标准，并能够分析尽可能多的JDM患者，以便更好地了解这种疾病。这个初步数据集现在可以发展成为一个经共识认可的最小核心数据集，并在更广泛的环境中进行测试，以期达成国际共识。
