不同程度肌无力的重症肌无力患者的临床特征和日常生活障碍:一项针对美国和欧洲五国患者的真实世界研究。

IF 2.2 3区 医学 Q3 CLINICAL NEUROLOGY
Jacqueline Pesa, Zia Choudhry, Jonathan de Courcy, Sophie Barlow, Gregor Gibson, Emma Chatterton, Shiva Lauretta Birija, Bethan Hahn, Raghav Govindarajan
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引用次数: 0

摘要

背景:采用真实世界的数据来确定不同程度肌无力的重症肌无力(MG)患者的临床特征,这些特征是根据美国重症肌无力基金会(MGFA)的分类系统确定的:数据来自阿德尔菲重症肌无力疾病专项计划(Adelphi MG Disease Specific Programme,DSP)™,这是一项跨国(美国、法国、德国、意大利、西班牙、英国)调查,由 2020 年的医生及其重症肌无力患者共同完成。采用线性回归法检验了 MGFA 分级与日常生活活动(ADL)障碍之间的关系,并对性别和夏尔森合并症指数进行了调整。对每个单项进行了二元比较。此外,还根据 MGFA 分级探讨了一系列其他临床特征:结果:在 1232 名患者中,MGFA I 级患者的 ADL 损伤明显低于 II 级或 III/IV 级患者(根据性别和夏尔森合并症指数进行调整)(p 结论:虽然 MGFA I 级与 ADL 损伤相关性较高,但 MGFA II 级患者的 ADL 损伤明显低于 II 级或 III/IV 级患者:尽管MGFA分级与ADL功能障碍程度和其他临床特征相关,但每个分级的患者在症状、总体疾病负担和ADL功能障碍的确切性质方面存在差异。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Clinical characteristics and impairment of activities of daily living among patients with myasthenia gravis with differing degrees of muscle weakness: a real-world study of patients in the US and five European countries.

Background: Real-world data were employed to determine clinical characteristics of patients with myasthenia gravis (MG) with differing degrees of muscle weakness, as defined using the Myasthenia Gravis Foundation of America (MGFA) classification system.

Methods: Data were drawn from the Adelphi MG Disease Specific Programme (DSP)™, a multinational (United States, France, Germany, Italy, Spain, United Kingdom) survey completed by physicians and their patients with MG in 2020. The association between MGFA class and impairment in activities of daily living (ADL) was tested using linear regression adjusting for sex and Charlson Comorbidity Index. Bivariate comparisons were performed for each individual item. A range of other clinical characteristics were also explored according to MGFA class.

Results: Among 1232 patients, those in MGFA class I had significantly lower ADL impairment versus class II or III/IV (adjusted for sex and Charlson Comorbidity Index) (p < 0.01). However, heterogeneity occurred within each MGFA class. Bulbar symptoms (impaired speech, difficulty swallowing, and/or difficulty chewing/choking on food) were reported in some class I patients (mild in 1.1-1.9% and moderate in 0.3-1.1% of patients) and class II patients (mild in 8.5-16.4%, moderate in 4.7-7.4%, and severe in 0.3-0.9% of patients), and shortness of breath was reported in some class I (mild in 0.5% of patients) and class II patients (mild in 9.8%, moderate in 4.8%, and severe in 0.3% of patients). Conversely, in 11.2-19.2% of class III/IV patients, bulbar symptoms and shortness of breath reported were only mild in severity. In line with this finding, despite significant correlations between MGFA class and several clinical characteristics, patients across every class were at risk of experiencing myasthenic crisis or hospitalization, experiencing comorbidities including anxiety and depression, and not being in remission.

Conclusions: Although MGFA class correlates with greater ADL impairment and presence of other clinical characteristics, there is variability between patients in each class in terms of symptoms experienced, overall disease burden, and the precise nature of ADL impairment.

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来源期刊
BMC Neurology
BMC Neurology 医学-临床神经学
CiteScore
4.20
自引率
0.00%
发文量
428
审稿时长
3-8 weeks
期刊介绍: BMC Neurology is an open access, peer-reviewed journal that considers articles on all aspects of the prevention, diagnosis and management of neurological disorders, as well as related molecular genetics, pathophysiology, and epidemiology.
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