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多发性硬化症的临床结局测量:综述。

Clinical outcome measures in multiple sclerosis: A review.

机构信息

MS Center, Center of Clinical Neuroscience, Carl Gustav Carus University Clinic, Fetscherstr. 74, 01307 Dresden, Germany.

MS Center, Center of Clinical Neuroscience, Carl Gustav Carus University Clinic, Fetscherstr. 74, 01307 Dresden, Germany.

出版信息

Autoimmun Rev. 2020 May;19(5):102512. doi: 10.1016/j.autrev.2020.102512. Epub 2020 Mar 12.

DOI:10.1016/j.autrev.2020.102512
PMID:32173519
Abstract

A proper clinical evaluation of patients with Multiple Sclerosis (MS) is of great importance to detect MS progression. It provides important data for physicians on their daily practice as well as with scientific purpose, especially useful with the development of newer therapeutic options in MS. Clinical outcome measures (COMs) are instruments that enable a standardized characterization of the patient's clinical disease status. A proper COM should have - among other psychometric characteristics - a good validity, reliability and responsiveness. For MS disability, the Expanded Disability Status Scale (EDSS) is currently the most frequently used scale. However, different less known COMs are also available and could be implemented on clinical practice. We review the most frequently used COMs of MS progression including their operationalization and statistical considerations, as well as newer composite COMs and digital tools on development. We focus on their responsiveness or capacity to detect clinically relevant changes to identify progression or transition to progressive forms of the disease, as well as therapeutic response.

摘要

对多发性硬化症 (MS) 患者进行适当的临床评估对于发现 MS 进展非常重要。它为医生的日常实践提供了重要数据,具有科学目的,特别是在 MS 的新治疗选择不断发展的情况下非常有用。临床结局测量 (COM) 是一种能够对患者的临床疾病状况进行标准化描述的工具。一个合适的 COM 除了其他心理测量学特征外,还应该具有良好的有效性、可靠性和反应性。对于 MS 残疾,扩展残疾状况量表 (EDSS) 目前是最常用的量表。然而,也有一些不太知名的 COM 可用,并可以在临床实践中实施。我们回顾了最常用的 MS 进展 COM,包括它们的操作和统计考虑,以及新的综合 COM 和数字工具的开发。我们关注它们的反应能力或检测临床相关变化的能力,以识别疾病的进展或向进行性形式的转变,以及治疗反应。

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