Ravelli Angelo, Ruperto Nicolino, Trail Lucia, Felici Enrico, Sala Elena, Martini Alberto
Istituto di Ricovero e Cura a Carattere Scientifico G. Gaslini, Università di Genova, Dipartimento di Pediatria, Italy.
Autoimmunity. 2006 May;39(3):197-203. doi: 10.1080/08916930600622553.
Juvenile dermatomyositis (JDM) is a multisystem inflammatory disease of unknown etiology that affects primarily the skin and muscles. Although the prognosis of JDM has improved considerably in the last three decades, a number of patients may develop irreversible damage due to the disease activity or its treatment. This damage may cause permanent disability and affect the quality of life of patients and their families. In the clinical management of patients with JDM, there is, therefore, the need of monitoring the level of disease activity, the accrual of organ damage, and the impact of the illness on patients' daily living. A reliable assessment of these different aspects of disease requires the availability of well-designed and standardized clinical tools. In the recent years, there has been increasing collaborative effort to devise new assessment measures and these measures have been included into disease activity and damage core sets of outcome variables that have been developed through international consensus. In addition, preliminary definitions of clinical improvement for patients with JDM and other idiopathic inflammatory myopathies have been created. In this review, the latest advances in the development of standardized instruments for the clinical assessment of JDM patients are illustrated and the recent international efforts that have led to the development of core sets of outcome measures and to preliminary definitions of improvement for JDM clinical trials are summarized.
青少年皮肌炎（JDM）是一种病因不明的多系统炎症性疾病，主要影响皮肤和肌肉。尽管在过去三十年中JDM的预后有了显著改善，但仍有一些患者可能因疾病活动或其治疗而出现不可逆的损害。这种损害可能导致永久性残疾，并影响患者及其家人的生活质量。因此，在JDM患者的临床管理中，需要监测疾病活动水平、器官损害的累积以及疾病对患者日常生活的影响。对疾病这些不同方面进行可靠评估需要有精心设计和标准化的临床工具。近年来，各方越来越多地共同努力设计新的评估措施，这些措施已被纳入通过国际共识制定的疾病活动和损害核心结局变量集。此外，还制定了JDM和其他特发性炎性肌病患者临床改善的初步定义。在本综述中，阐述了用于JDM患者临床评估的标准化工具开发的最新进展，并总结了近期国际上为制定核心结局指标集和JDM临床试验改善的初步定义所做的努力。
