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.
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。
相似文献
1
Measuring Clinical Treatment Response in Myasthenia Gravis.重症肌无力临床治疗反应的测量
Neurol Clin. 2018 May;36(2):339-353. doi: 10.1016/j.ncl.2018.01.006.
2
Quantitative myasthenia gravis score: assessment of responsiveness and longitudinal validity.重症肌无力定量评分：反应性评估与纵向效度
Neurology. 2005 Jun 14;64(11):1968-70. doi: 10.1212/01.WNL.0000163988.28892.79.
3
History of outcome measures for myasthenia gravis.重症肌无力结局评估指标的历史。
Muscle Nerve. 2010 Jul;42(1):5-13. doi: 10.1002/mus.21713.
4
The utility of a single simple question in the evaluation of patients with myasthenia gravis.单一简单问题在重症肌无力患者评估中的效用。
Muscle Nerve. 2018 Feb;57(2):240-244. doi: 10.1002/mus.25720. Epub 2017 Jun 27.
5
[Myasthenia gravis: current status of antibody diagnostics and aspects on refractory myasthenia gravis].重症肌无力：抗体诊断的现状及难治性重症肌无力的相关方面
Fortschr Neurol Psychiatr. 2018 Sep;86(9):551-558. doi: 10.1055/a-0624-9397. Epub 2018 Sep 24.
6
The quantitative myasthenia gravis score: comparison with clinical, electrophysiological, and laboratory markers.重症肌无力定量评分：与临床、电生理及实验室指标的比较
J Clin Neuromuscul Dis. 2012 Jun;13(4):201-5. doi: 10.1097/CND.0b013e31824619d5.
7
Ocular myasthenia gravis.眼肌型重症肌无力。
Curr Opin Ophthalmol. 2012 Nov;23(6):537-42. doi: 10.1097/ICU.0b013e328358b94a.
8
Validation of the MG-DIS: a disability assessment for myasthenia gravis.重症肌无力残疾评定量表（MG-DIS）的验证：一项针对重症肌无力的残疾评估
J Neurol. 2016 May;263(5):871-882. doi: 10.1007/s00415-016-8072-9. Epub 2016 Mar 1.
9
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.
10
Myasthenia gravis: a quantitative evaluation system. Disability status scale (DSS) applied for myasthenia gravis.
Eur Neurol. 1976;14(6):439-46. doi: 10.1159/000114771.
引用本文的文献
1
Improvement in Patient-Reported Symptoms of Generalised Myasthenia Gravis With Rozanolixizumab in the Randomised Phase 3 MycarinG Study Using the MG Symptoms PRO.在使用MG症状患者报告结局（PRO）的随机3期MycarinG研究中，rozanolixizumab治疗使全身型重症肌无力患者报告的症状得到改善。
Eur J Neurol. 2025 Aug;32(8):e70231. doi: 10.1111/ene.70231.
2
Real-World Case Series of Ravulizumab Use in Patients with Myasthenia Gravis in Romania.罗马尼亚使用ravulizumab治疗重症肌无力患者的真实世界病例系列
Brain Sci. 2025 Mar 28;15(4):350. doi: 10.3390/brainsci15040350.
3
Longitudinal relationships between free-living activities, fatigue, and symptom severity in myasthenia gravis using cohort and individualized models.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.
5
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.
6
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.
7
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.
9
Plasma Exchange versus Intravenous Immunoglobulin in Worsening Myasthenia Gravis: A Systematic Review and Meta-Analysis with Special Attention to Faster Relapse Control.血浆置换与静脉注射免疫球蛋白治疗重症肌无力病情恶化的比较：一项系统评价和荟萃分析，特别关注更快的复发控制
Biomedicines. 2023 Nov 29;11(12):3180. doi: 10.3390/biomedicines11123180.
10
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。
Curr J Neurol. 2022 Jan 5;21(1):35-39. doi: 10.18502/cjn.v21i1.9360.
本文引用的文献
1
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.重症肌无力定量评分及重症肌无力综合量表的心理测量特性。
J Neuromuscul Dis. 2015 Sep 2;2(3):301-311. doi: 10.3233/JND-150082.
4
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.
5
Randomized Trial of Thymectomy in Myasthenia Gravis.重症肌无力胸腺切除术的随机试验
N Engl J Med. 2016 Aug 11;375(6):511-22. doi: 10.1056/NEJMoa1602489.
6
Development and validation of the Myasthenia Gravis Impairment Index.重症肌无力损伤指数的开发与验证
Neurology. 2016 Aug 30;87(9):879-86. doi: 10.1212/WNL.0000000000002971. Epub 2016 Jul 8.
7
A randomized controlled trial of methotrexate for patients with generalized myasthenia gravis.甲氨蝶呤治疗全身型重症肌无力患者的一项随机对照试验。
Neurology. 2016 Jul 5;87(1):57-64. doi: 10.1212/WNL.0000000000002795. Epub 2016 Jun 15.
8
International clinimetric evaluation of the MG-QOL15, resulting in slight revision and subsequent validation of the MG-QOL15r.对MG-QOL15进行国际临床计量学评估，结果对MG-QOL15进行了轻微修订并随后对MG-QOL15r进行了验证。
Muscle Nerve. 2016 Dec;54(6):1015-1022. doi: 10.1002/mus.25198. Epub 2016 Nov 7.
9
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
Anesthesiology. 2016 Jul;125(1):7-10. doi: 10.1097/ALN.0000000000001159.
10
Validation of the MG-DIS: a disability assessment for myasthenia gravis.重症肌无力残疾评定量表（MG-DIS）的验证：一项针对重症肌无力的残疾评估
J Neurol. 2016 May;263(5):871-882. doi: 10.1007/s00415-016-8072-9. Epub 2016 Mar 1.
Zotero 插件