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本文引用的文献

1
Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele.依伐卡托与泰比卡托和艾克卡托三联复方药物治疗携带单个 F508del 突变的囊性纤维化
N Engl J Med. 2019 Nov 7;381(19):1809-1819. doi: 10.1056/NEJMoa1908639. Epub 2019 Oct 31.
2
Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial.在纯合子 F508del 突变的囊性纤维化患者中,elexacaftor 加 tezacaftor 加 ivacaftor 联合治疗方案的疗效和安全性:一项双盲、随机、3 期临床试验。
Lancet. 2019 Nov 23;394(10212):1940-1948. doi: 10.1016/S0140-6736(19)32597-8. Epub 2019 Oct 31.
3
VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles.VX-445-泰泽卡托维瓦卡托联合治疗伴有一个或两个 Phe508del 等位基因的囊性纤维化患者。
N Engl J Med. 2018 Oct 25;379(17):1612-1620. doi: 10.1056/NEJMoa1807120. Epub 2018 Oct 18.
4
Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR.Lumacaftor/ivacaftor 治疗 F508del-CFTR 纯合子的囊性纤维化和晚期肺病患者。
J Cyst Fibros. 2018 Mar;17(2):228-235. doi: 10.1016/j.jcf.2017.09.012. Epub 2017 Nov 8.
5
Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis.用于囊性纤维化残余功能杂合子的泰扎卡托-依伐卡托
N Engl J Med. 2017 Nov 23;377(21):2024-2035. doi: 10.1056/NEJMoa1709847. Epub 2017 Nov 3.
6
Nationwide trends of hospitalizations for cystic fibrosis in the United States from 2003 to 2013.2003年至2013年美国全国范围内囊性纤维化住院情况的趋势。
Intractable Rare Dis Res. 2017 Aug;6(3):191-198. doi: 10.5582/irdr.2017.01043.
7
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.鲁马卡托-依伐卡托用于携带苯丙氨酸508位缺失CFTR基因纯合突变的囊性纤维化患者。
N Engl J Med. 2015 Jul 16;373(3):220-31. doi: 10.1056/NEJMoa1409547. Epub 2015 May 17.
8
Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis.囊性纤维化的增效剂(针对III类和IV类突变的特定疗法)。
Cochrane Database Syst Rev. 2015 Mar 26(3):CD009841. doi: 10.1002/14651858.CD009841.pub2.

新型囊性纤维化治疗方法的效果和价值。

The effectiveness and value of novel treatments for cystic fibrosis.

机构信息

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

School of Public Health, University of Minnesota.

出版信息

J Manag Care Spec Pharm. 2021 Feb;27(2):276-280. doi: 10.18553/jmcp.2021.27.2.276.

DOI:10.18553/jmcp.2021.27.2.276
PMID:33506736
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10391049/
Abstract

Funding for this summary was contributed by Arnold Ventures, California Health Care 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 Aetna, America's Health Insurance Plans, Anthem, Allergan, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Boehringer-Ingelheim, Cambia Health Services, CVS, Editas, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, HealthFirst, Health Partners, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Pfizer, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, and United Healthcare. Seidner, Rind, and Pearson are employed by ICER. Tice reports contracts to his institution, University of California, San Francisco, from ICER during the conduct of this study. Wherry has nothing to disclose.

摘要

本摘要的资助由 Arnold Ventures、加州医疗保健基金会、哈佛朝圣者健康保健、Kaiser 基金会健康计划提供给临床和经济评估研究所(ICER),这是一个独立评估医疗保健干预措施价值证据的组织。ICER 的年度政策峰会由 Aetna、美国健康保险计划、Anthem、Allergan、Alnylam、阿斯利康、Biogen、加州蓝盾、勃林格殷格翰、Cambia 健康服务、CVS、Editas、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、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 Therapeutics、Regeneron、赛诺菲、Spark Therapeutics 和联合健康保险公司赞助。Seidner、Rind 和 Pearson 受雇于 ICER。Tice 在进行这项研究期间向他的机构加州大学旧金山分校报告了来自 ICER 的合同。Wherry 没有需要披露的信息。