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与重症肌无力长达二十年的抗争:关于依库珠单抗和ravulizumab治疗长期成功的突破性病例报告

Two-decade battle with myasthenia gravis: A breakthrough case report on the long-term success with eculizumab and ravulizumab treatment.

作者信息

Quasthoff Stefan

机构信息

Department of Neurology Medical University of Graz Graz Austria.

出版信息

Clin Case Rep. 2024 Nov 4;12(11):e9547. doi: 10.1002/ccr3.9547. eCollection 2024 Nov.

DOI:10.1002/ccr3.9547
PMID:39502126
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11534635/
Abstract

This unique case of generalized myasthenia gravis shows sustained stability of a patient's condition for 3 years with eculizumab/ravulizumab treatment following 16 years of refractory disease. It highlights the long-term effectiveness of C5 inhibitors in a real-world setting, aiding physicians in their decision-making for refractory cases and treatment discontinuation scenarios.

摘要

这例独特的全身型重症肌无力病例显示,在经历16年难治性疾病后,患者接受依库珠单抗/ravulizumab治疗,病情持续稳定了3年。它凸显了C5抑制剂在现实环境中的长期有效性,有助于医生为难治性病例和治疗中断情况做出决策。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/74fe/11534635/1c78068d7b42/CCR3-12-e9547-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/74fe/11534635/9df046f606a1/CCR3-12-e9547-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/74fe/11534635/1c78068d7b42/CCR3-12-e9547-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/74fe/11534635/9df046f606a1/CCR3-12-e9547-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/74fe/11534635/1c78068d7b42/CCR3-12-e9547-g001.jpg

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引用本文的文献

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本文引用的文献

1
Cost-effectiveness of eculizumab and efgartigimod for the treatment of anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.依库珠单抗和依氟鸟氨酸治疗抗乙酰胆碱受体抗体阳性全身型重症肌无力的成本效果分析。
J Manag Care Spec Pharm. 2024 Jun;30(6):517-527. doi: 10.18553/jmcp.2024.30.6.517.
2
Economic burden of generalized myasthenia gravis (MG) in the United States and the impact of common comorbidities and acute MG-events.美国全身性重症肌无力(MG)的经济负担,以及常见合并症和急性 MG 事件的影响。
Curr Med Res Opin. 2024 Jul;40(7):1145-1153. doi: 10.1080/03007995.2024.2353381. Epub 2024 May 24.
3
Real-world experience with eculizumab and switching to ravulizumab for generalized myasthenia gravis.
真实世界中依库珠单抗和瑞伏鲁单抗用于治疗全身性重症肌无力的经验。
Ann Clin Transl Neurol. 2024 May;11(5):1338-1346. doi: 10.1002/acn3.52051. Epub 2024 Apr 4.
4
Efficacy of ravulizumab in patients with generalized myasthenia gravis by time from diagnosis: A post hoc subgroup analysis of the CHAMPION MG study.诊断后时间对瑞夫利珠单抗治疗全身性重症肌无力患者疗效的影响:CHAMPION MG 研究的事后亚组分析。
Muscle Nerve. 2024 May;69(5):556-565. doi: 10.1002/mus.28044. Epub 2024 Feb 21.
5
Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis.瑞昔珠单抗治疗全身性重症肌无力。
NEJM Evid. 2022 May;1(5):EVIDoa2100066. doi: 10.1056/EVIDoa2100066. Epub 2022 Apr 26.
6
Ravulizumab in Myasthenia Gravis: A Review of the Current Evidence.瑞武利珠单抗治疗重症肌无力:当前证据综述
Neuropsychiatr Dis Treat. 2023 Dec 1;19:2639-2655. doi: 10.2147/NDT.S374694. eCollection 2023.
7
Starting eculizumab as rescue therapy in refractory myasthenic crisis.开始使用依库珠单抗作为难治性肌无力危象的抢救治疗。
Neurol Sci. 2023 Oct;44(10):3707-3709. doi: 10.1007/s10072-023-06900-y. Epub 2023 Jun 12.
8
Long-term efficacy and safety of ravulizumab in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis: results from the phase 3 CHAMPION MG open-label extension.抗乙酰胆碱受体抗体阳性全身型重症肌无力成人患者使用拉那利尤单抗的长期疗效和安全性:III 期 CHAMPION MG 开放标签扩展研究结果。
J Neurol. 2023 Aug;270(8):3862-3875. doi: 10.1007/s00415-023-11699-x. Epub 2023 Apr 27.
9
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Eur J Health Econ. 2023 Dec;24(9):1455-1472. doi: 10.1007/s10198-022-01556-5. Epub 2023 Jan 12.