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第 272 届 ENMC 国际研讨会:10 年进展——重新修订 ENMC 2013 包涵体肌炎诊断标准和临床试验准备。2023 年 6 月 16-18 日,荷兰霍夫多普。

272nd ENMC international workshop: 10 Years of progress - revision of the ENMC 2013 diagnostic criteria for inclusion body myositis and clinical trial readiness. 16-18 June 2023, Hoofddorp, The Netherlands.

机构信息

Manchester Centre for Clinical Neurosciences, Northern Care Alliance NHS Foundation Trust, Manchester Academic Health Science Centre, Salford, UK; Division of Musculoskeletal and Dermatological Sciences, The University of Manchester, Manchester, UK.

Department of Neurology, Mayo Clinic, Rochester, Minnesota, USA.

出版信息

Neuromuscul Disord. 2024 Apr;37:36-51. doi: 10.1016/j.nmd.2024.03.001. Epub 2024 Mar 7.

Abstract

Since the publication of the 2013 European Neuromuscular Center (ENMC) diagnostic criteria for Inclusion Body Myositis (IBM), several advances have been made regarding IBM epidemiology, pathogenesis, diagnostic tools, and clinical trial readiness. Novel diagnostic tools include muscle imaging techniques such as MRI and ultrasound, and serological testing for cytosolic 5'-nucleotidase-1A antibodies. The 272nd ENMC workshop aimed to develop new diagnostic criteria, discuss clinical outcome measures and clinical trial readiness. The workshop started with patient representatives highlighting several understudied symptoms and the urge for a timely diagnosis. This was followed by presentations from IBM experts highlighting the new developments in the field. This report is composed of two parts, the first part providing new diagnostic criteria on which consensus was achieved. The second part focuses on the use of outcome measures in clinical practice and clinical trials, highlighting current limitations and outlining the goals for future studies.

摘要

自从 2013 年欧洲神经肌肉中心(ENMC)发布包涵体肌炎(IBM)的诊断标准以来,在 IBM 的流行病学、发病机制、诊断工具以及临床试验准备方面取得了多项进展。新的诊断工具包括肌肉成像技术,如 MRI 和超声,以及细胞质 5'-核苷酸酶-1A 抗体的血清学检测。第 272 届 ENMC 研讨会旨在制定新的诊断标准,讨论临床结果测量和临床试验准备。研讨会开始时,患者代表强调了几个研究不足的症状,并迫切需要及时诊断。随后,IBM 专家介绍了该领域的新进展。本报告由两部分组成,第一部分提供了达成共识的新诊断标准。第二部分重点介绍了临床实践和临床试验中使用的结果测量方法,强调了当前的局限性,并概述了未来研究的目标。

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