Thomsen Jan Lykke Scheel, Andersen Henning
Department of Neurology, Aarhus University Hospital, Aarhus, Denmark.
Front Neurol. 2020 Dec 23;11:596382. doi: 10.3389/fneur.2020.596382. eCollection 2020.
Myasthenia gravis (MG) is a heterogeneous disorder whose clinical presentation ranges from mild ocular deficits to severe widespread weakness. This variance poses a challenge when quantifying clinical deficits. Deficits and symptoms are quantified using standardized clinical scales and questionnaires which are often used as outcome measures. The past decades have seen the development of several validated outcome measures in MG, which are used in clinical trials to obtain regulatory approval. In recent years, emphasis has moved from objective assessments to patient-reported outcomes. Despite a growing body of literature on the validity of the MG-specific outcome measures, several unresolved factors remain. As several novel therapeutics are currently in clinical development, knowledge about capabilities and limitations of outcome measures is needed. In the present paper, we describe the most widely used clinical classifications and scales in MG. We highlight the choice of outcome measures in published and ongoing trials, and we denote whether trial efficacy was reached on these outcomes. We discuss advantages and limitations of the individual scales, and discuss some of the unresolved factors relating to outcome assessments in MG.
重症肌无力（MG）是一种异质性疾病，其临床表现从轻度眼部功能缺陷到严重的全身广泛性肌无力不等。这种差异在量化临床缺陷时构成了挑战。使用标准化临床量表和问卷对缺陷和症状进行量化，这些量表和问卷常被用作疗效指标。在过去几十年中，重症肌无力领域已开发出多种经过验证的疗效指标，用于临床试验以获得监管批准。近年来，重点已从客观评估转向患者报告的结局。尽管关于重症肌无力特异性疗效指标有效性的文献越来越多，但仍有几个未解决的因素。由于目前有几种新型疗法正在进行临床开发，因此需要了解疗效指标的能力和局限性。在本文中，我们描述了重症肌无力中最广泛使用的临床分类和量表。我们强调已发表和正在进行的试验中疗效指标的选择，并指出这些结局是否达到了试验疗效。我们讨论了各个量表的优缺点，并讨论了与重症肌无力结局评估相关的一些未解决因素。
