Department of Paediatric Rheumatology, Alder Hey Children's NHS Foundation Trust, Liverpool, UK.
Department of Paediatric Rheumatology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Ann Rheum Dis. 2018 Feb;77(2):241-250. doi: 10.1136/annrheumdis-2017-212141. Epub 2017 Oct 30.
This study aimed to develop consensus on an internationally agreed dataset for juvenile dermatomyositis (JDM), designed for clinical use, to enhance collaborative research and allow integration of data between centres.
A prototype dataset was developed through a formal process that included analysing items within existing databases of patients with idiopathic inflammatory myopathies. This template was used to aid a structured multistage consensus process. Exploiting Delphi methodology, two web-based questionnaires were distributed to healthcare professionals caring for patients with JDM identified through email distribution lists of international paediatric rheumatology and myositis research groups. A separate questionnaire was sent to parents of children with JDM and patients with JDM, identified through established research networks and patient support groups. The results of these parallel processes informed a face-to-face nominal group consensus meeting of international myositis experts, tasked with defining the content of the dataset. This developed dataset was tested in routine clinical practice before review and finalisation.
A dataset containing 123 items was formulated with an accompanying glossary. Demographic and diagnostic data are contained within form A collected at baseline visit only, disease activity measures are included within form B collected at every visit and disease damage items within form C collected at baseline and annual visits thereafter.
Through a robust international process, a consensus dataset for JDM has been formulated that can capture disease activity and damage over time. This dataset can be incorporated into national and international collaborative efforts, including existing clinical research databases.
本研究旨在就一个为临床使用而设计的、针对幼年型皮肌炎 (JDM) 的国际公认数据集达成共识，以促进合作研究并允许中心之间的数据整合。
通过包括分析特发性炎症性肌病患者现有数据库中的项目在内的正式流程开发了原型数据集。该模板用于辅助结构化多阶段共识流程。利用 Delphi 方法，通过国际儿科风湿病学和肌炎研究小组的电子邮件分发列表向 JDM 患者的医护人员分发了两份基于网络的调查问卷。向 JDM 患儿的家长和 JDM 患者单独发送了一份调查问卷，这些患者通过既定的研究网络和患者支持团体确定。这些平行流程的结果为国际肌炎专家的面对面名义小组共识会议提供了信息，会议的任务是定义数据集的内容。在进行审查和最终确定之前，对该开发数据集在常规临床实践中进行了测试。
制定了一个包含 123 个项目的数据集，并附有词汇表。仅在基线访视时收集表单 A 中的人口统计学和诊断数据，在每次访视时收集表单 B 中的疾病活动测量数据，并在基线和此后的年度访视时收集表单 C 中的疾病损害项目。
通过一个强大的国际流程，制定了一个针对 JDM 的共识数据集，该数据集可以随时间捕捉疾病活动和损害。该数据集可纳入国家和国际合作努力，包括现有临床研究数据库。
