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The Future of Highly Effective Modulator Therapy in Cystic Fibrosis.

作者信息

Daines Cori L, Morgan Wayne J

机构信息

Department of Pediatrics University of Arizona Tucson, Arizona.

出版信息

Am J Respir Crit Care Med. 2021 Jun 15;203(12):1453-1455. doi: 10.1164/rccm.202104-0850ED.

DOI:10.1164/rccm.202104-0850ED
PMID:33901406
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8483216/
Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3536/8483216/90a4c9af0708/rccm.202104-0850EDf1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3536/8483216/90a4c9af0708/rccm.202104-0850EDf1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3536/8483216/90a4c9af0708/rccm.202104-0850EDf1.jpg

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4
Therapy for cystic fibrosis--the end of the beginning?囊性纤维化的治疗——开端的结束？
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COUNTERPOINT: In the Era of Cystic Fibrosis Transmembrane Regulator Protein Modulator Therapy, Are the Treatment Goals for Adults Now Different From Those for Children With Cystic Fibrosis? No.反驳观点：在囊性纤维化跨膜调节蛋白调节剂治疗时代，成人囊性纤维化的治疗目标与儿童现在有所不同吗？没有。
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Targeting the basic defect in cystic fibrosis.针对囊性纤维化的基本缺陷。
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JCI Insight. 2024 Jun 18;9(14):e181836. doi: 10.1172/jci.insight.181836.
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Elexacaftor-Tezacaftor-Ivacaftor as a Final Frontier in the Treatment of Cystic Fibrosis: Definition of the Clinical and Microbiological Implications in a Case-Control Study.

本文引用的文献

1
A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One Allele.一项评估 elexacaftor/tezacaftor/ivacaftor 在至少携带一个突变等位基因的囊性纤维化 6 至 11 岁儿童中的疗效和安全性的 3 期、开放性标签研究。
Am J Respir Crit Care Med. 2021 Jun 15;203(12):1522-1532. doi: 10.1164/rccm.202102-0509OC.
2
Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study.针对携带F508del-CFTR突变纯合子的6至11岁囊性纤维化儿童，鲁马卡托-依伐卡托治疗的长期安全性和有效性：一项3期开放标签扩展研究。
Lancet Respir Med. 2021 Jul;9(7):721-732. doi: 10.1016/S2213-2600(20)30517-8. Epub 2021 Jan 28.
3
依列卡福妥-替扎卡福妥-依伐卡托作为囊性纤维化治疗的最后前沿：病例对照研究中临床和微生物学意义的界定
Pharmaceuticals (Basel). 2022 May 14;15(5):606. doi: 10.3390/ph15050606.
4
Increasing life expectancy in cystic fibrosis: Advances and challenges.提高囊性纤维化患者的预期寿命：进展与挑战。
Pediatr Pulmonol. 2022 Feb;57 Suppl 1(Suppl 1):S5-S12. doi: 10.1002/ppul.25733. Epub 2021 Nov 11.
Cystic fibrosis patients of minority race and ethnicity less likely eligible for CFTR modulators based on CFTR genotype.囊性纤维化患者中，少数族裔和少数民族群体基于 CFTR 基因型，获得 CFTR 调节剂治疗的可能性较低。
Pediatr Pulmonol. 2021 Jun;56(6):1496-1503. doi: 10.1002/ppul.25285. Epub 2021 Feb 1.
4
Ivacaftor in Infants Aged 4 to <12 Months with Cystic Fibrosis and a Gating Mutation. Results of a Two-Part Phase 3 Clinical Trial.依伐卡托特在 4 至<12 个月患有囊性纤维化且存在门控突变婴儿中的应用。两项 3 期临床试验的结果。
Am J Respir Crit Care Med. 2021 Mar 1;203(5):585-593. doi: 10.1164/rccm.202008-3177OC.
5
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.
6
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.
7
A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis.一项评估三联疗法（tezacaftor 联合 ivacaftor）在 6 至 11 岁儿童囊性纤维化患者中的 III 期临床研究。
J Cyst Fibros. 2019 Sep;18(5):708-713. doi: 10.1016/j.jcf.2019.06.009. Epub 2019 Jun 26.
8
Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries.依伐卡托治疗囊性纤维化患者的疾病进展：来自美国和英国国家注册中心的数据。
J Cyst Fibros. 2020 Jan;19(1):68-79. doi: 10.1016/j.jcf.2019.05.015. Epub 2019 Jun 10.
9
An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2-5 years (KLIMB).KLIMB 研究：在 2-5 岁有 CFTR 门控突变的 CF 患儿中开展 ivacaftor 的开放标签扩展研究，以启动治疗。
J Cyst Fibros. 2019 Nov;18(6):838-843. doi: 10.1016/j.jcf.2019.03.009. Epub 2019 Apr 30.
10
Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2-5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study.在囊性纤维化跨膜电导调节因子纯合子 F508del-CFTR 的 2-5 岁儿童中， lumacaftor 和 ivacaftor 联合治疗的安全性、药代动力学和药效学：一项开放标签的 3 期研究。
Lancet Respir Med. 2019 Apr;7(4):325-335. doi: 10.1016/S2213-2600(18)30460-0. Epub 2019 Jan 24.