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本文引用的文献

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Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial.依氟鸟氨酸苷单抗在全身性重症肌无力患者中的安全性、疗效和耐受性(ADAPT):一项多中心、随机、安慰剂对照、3 期临床试验。
Lancet Neurol. 2021 Jul;20(7):526-536. doi: 10.1016/S1474-4422(21)00159-9.
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Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension.REGAIN 研究及其开放标签扩展中,采用依库珠单抗治疗难治性重症肌无力患者的干预后状态。
Neurology. 2021 Jan 26;96(4):e610-e618. doi: 10.1212/WNL.0000000000011207. Epub 2020 Nov 23.
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International Consensus Guidance for Management of Myasthenia Gravis: 2020 Update.国际重症肌无力管理共识指南:2020 年更新版。
Neurology. 2021 Jan 19;96(3):114-122. doi: 10.1212/WNL.0000000000011124. Epub 2020 Nov 3.
4
'Minimal symptom expression' in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab.乙酰胆碱受体抗体阳性难治性全身型重症肌无力患者接受依库珠单抗治疗后的最小症状表现。
J Neurol. 2020 Jul;267(7):1991-2001. doi: 10.1007/s00415-020-09770-y. Epub 2020 Mar 18.
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Incidence, Epidemiology, and Transformation of Ocular Myasthenia Gravis: A Population-Based Study.眼肌型重症肌无力的发病情况、流行病学和转化:一项基于人群的研究。
Am J Ophthalmol. 2019 Sep;205:99-105. doi: 10.1016/j.ajo.2019.04.017. Epub 2019 May 9.
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Myasthenia Gravis.重症肌无力
N Engl J Med. 2016 Dec 29;375(26):2570-2581. doi: 10.1056/NEJMra1602678.
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Myasthenia gravis: subgroup classification and therapeutic strategies.重症肌无力:亚组分类与治疗策略。
Lancet Neurol. 2015 Oct;14(10):1023-36. doi: 10.1016/S1474-4422(15)00145-3.
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A systematic review of population based epidemiological studies in Myasthenia Gravis.一项基于人群的重症肌无力流行病学研究的系统综述。
BMC Neurol. 2010 Jun 18;10:46. doi: 10.1186/1471-2377-10-46.

依库珠单抗和依氟鸟氨酸治疗全身性重症肌无力的疗效和价值。

The effectiveness and value of eculizumab and efgartigimod for generalized myasthenia gravis.

机构信息

Division of General Internal Medicine, University of California San Francisco.

University of Illinois at Chicago College of Pharmacy, Chicago, IL.

出版信息

J Manag Care Spec Pharm. 2022 Jan;28(1):119-124. doi: 10.18553/jmcp.2022.28.1.119.

DOI:10.18553/jmcp.2022.28.1.119
PMID:34949112
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10373007/
Abstract

Funding for this summary was contributed by Arnold Ventures, The Donaghue Foundation, Harvard Pilgrim Health Care, and Kaiser Foundation Health Plan to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from AbbVie, America's Health Insurance Plans, Anthem, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Boehringer-Ingelheim, Cambia Health Services, CVS, Editas, Evolve Pharmacy, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, HealthFirst, Health Partners, Humana, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Pfizer, Premera, Prime Therapeutics, Regeneron, Sanofi, Sun Life Financial, uniQure, and United Healthcare. Agboola, Nikitin, and Pearson are employed by ICER. Through their affiliated institutions, Tice, Touchette, and Lien received funding from ICER for the work described in this summary.

摘要

本摘要的资助方为 Arnold Ventures、Donaghue 基金会、哈佛朝圣者健康保健和 Kaiser 基金会健康计划,他们为独立组织临床与经济评价研究所(ICER)提供了资金,该组织评估医疗干预措施的价值证据。ICER 的年度政策峰会由 AbbVie、美国健康保险计划协会、Anthem、Alnylam、阿斯利康、Biogen、加州蓝盾、勃林格殷格翰、Cambia 健康服务、CVS、Editas、Evolve 药房、Express Scripts、基因泰克/罗氏、葛兰素史克、哈佛朝圣者、医疗保健服务公司、HealthFirst、Health Partners、Humana、强生(杨森)、Kaiser 永久、利奥制药、美纳里克斯、默克、诺华、国家制药理事会、辉瑞、Premera、Prime 治疗学、再生元、赛诺菲、太阳生活金融、uniQure 和 United Healthcare 提供支持。Agboola、Nikitin 和 Pearson 受雇于 ICER。通过他们的附属机构,Tice、Touchette 和 Lien 从 ICER 获得了资金,用于开展本摘要中所描述的工作。