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重症肌无力临床治疗反应的测量

Measuring Clinical Treatment Response in Myasthenia Gravis.

作者信息

Barnett Carolina, Herbelin Laura, Dimachkie Mazen M, Barohn Richard J

机构信息

Neurology (Medicine), University of Toronto, University Health Network, Toronto, Ontario, Canada.

Department of Neurology, University of Kansas Medical Center, 3901 Rainbow Boulevard, Kansas City, KS 66160, USA.

出版信息

Neurol Clin. 2018 May;36(2):339-353. doi: 10.1016/j.ncl.2018.01.006.

DOI:10.1016/j.ncl.2018.01.006
PMID:29655453
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6697147/
Abstract

In this article we provide an overview of health-related outcome measurement-to better understand what different outcomes used in myasthenia actually measure-and to provide some guidance when choosing measures based on the clinical context and question. In myasthenia, the most commonly used outcome measures are aimed at assessing the signs and symptoms. In this review, we provide a summary of the most commonly used outcome measures. We discuss instruments that gauge disease overall health impact, such as on disability and quality of life. Finally, we discuss other relevant outcomes such as steroid-sparing effects and the role of surrogate markers.

摘要

在本文中,我们概述了与健康相关的结果测量,以便更好地理解重症肌无力中使用的不同结果实际测量的内容,并在根据临床背景和问题选择测量方法时提供一些指导。在重症肌无力中,最常用的结果测量旨在评估体征和症状。在本综述中,我们总结了最常用的结果测量方法。我们讨论了评估疾病对整体健康影响的工具,如对残疾和生活质量的影响。最后,我们讨论了其他相关结果,如类固醇节省效应和替代标志物的作用。

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本文引用的文献

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Myasthenia Gravis Impairment Index: Responsiveness, meaningful change, and relative efficiency.重症肌无力损伤指数:反应性、有意义的变化及相对效率。
Neurology. 2017 Dec 5;89(23):2357-2364. doi: 10.1212/WNL.0000000000004676. Epub 2017 Nov 3.
2
Rituximab as treatment for anti-MuSK myasthenia gravis: Multicenter blinded prospective review.利妥昔单抗治疗抗 MuSK 重症肌无力:多中心盲法前瞻性研究。
Neurology. 2017 Sep 5;89(10):1069-1077. doi: 10.1212/WNL.0000000000004341. Epub 2017 Aug 11.
3
Psychometric Properties of the Quantitative Myasthenia Gravis Score and the Myasthenia Gravis Composite Scale.
运用队列研究和个体化模型探讨重症肌无力患者日常活动、疲劳与症状严重程度之间的纵向关系。
Muscle Nerve. 2025 Jan;71(1):33-42. doi: 10.1002/mus.28282. Epub 2024 Oct 30.
4
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.在患有不同程度肌无力的重症肌无力患者中,临床特征和日常生活活动能力受损的情况:来自美国和五个欧洲国家的真实世界研究。
BMC Neurol. 2024 Oct 12;24(1):385. doi: 10.1186/s12883-024-03869-9.
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United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use.美国重症肌无力患者使用依库珠单抗的临床实践经验:症状、功能和免疫抑制剂治疗的应用。
J Neurol. 2024 Sep;271(9):6114-6126. doi: 10.1007/s00415-024-12569-w. Epub 2024 Jul 25.
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Innovative Therapeutic Approaches in Congenital Myasthenic Syndromes.先天性肌无力综合征的创新治疗方法
Neurol Clin Pract. 2024 Jun;14(3):e200277. doi: 10.1212/CPJ.0000000000200277. Epub 2024 May 7.
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Expert consensus recommendations for improving and standardising the assessment of patients with generalised myasthenia gravis.改善和规范全身性重症肌无力患者评估的专家共识建议。
Eur J Neurol. 2024 Jul;31(7):e16280. doi: 10.1111/ene.16280. Epub 2024 Mar 24.
8
Assessing the length of hospital stay for patients with myasthenia gravis based on the data mining MARS approach.基于数据挖掘MARS方法评估重症肌无力患者的住院时长。
Front Neurol. 2023 Dec 14;14:1283214. doi: 10.3389/fneur.2023.1283214. eCollection 2023.
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Biomedicines. 2023 Nov 29;11(12):3180. doi: 10.3390/biomedicines11123180.
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Validation of myasthenia gravis activity of daily living questionnaire: Persian version.重症肌无力日常生活问卷的验证:波斯语版本。
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J Neuromuscul Dis. 2015 Sep 2;2(3):301-311. doi: 10.3233/JND-150082.
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Translation, cross-cultural adaptation, and validation of the french version of the 15-item Myasthenia Gravis Quality Of life scale.15项重症肌无力生活质量量表法语版的翻译、跨文化调适及效度验证
Muscle Nerve. 2017 May;55(5):639-645. doi: 10.1002/mus.25381. Epub 2017 Jan 3.
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Randomized Trial of Thymectomy in Myasthenia Gravis.重症肌无力胸腺切除术的随机试验
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How Much Better Is Good Enough?: Patient-reported Outcomes, Minimal Clinically Important Differences, and Patient Acceptable Symptom States in Perioperative Research.做到多好才算足够好?:围手术期研究中的患者报告结局、最小临床重要差异及患者可接受症状状态
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J Neurol. 2016 May;263(5):871-882. doi: 10.1007/s00415-016-8072-9. Epub 2016 Mar 1.