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1
Reply to Martin and Grasemann: Is There a Role for Lumacaftor/Ivacaftor in Young Children with Cystic Fibrosis?回复马丁和格拉泽曼：鲁马卡托/依伐卡托对患有囊性纤维化的幼儿有作用吗？
Ann Am Thorac Soc. 2025 Mar;22(3):469-470. doi: 10.1513/AnnalsATS.202411-1237LE.
2
Long-term safety of lumacaftor-ivacaftor in children aged 2-5 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a multicentre, phase 3, open-label, extension study.在囊性纤维化跨膜传导调节因子（CFTR）基因 F508del 纯合突变的 2-5 岁儿童中， lumacaftor-ivacaftor 的长期安全性：一项多中心、3 期、开放性、扩展研究。
Lancet Respir Med. 2021 Sep;9(9):977-988. doi: 10.1016/S2213-2600(21)00069-2. Epub 2021 May 6.
3
Correctors (specific therapies for class II CFTR mutations) for cystic fibrosis.囊性纤维化的校正剂（针对II类CFTR突变的特异性疗法）
Cochrane Database Syst Rev. 2018 Aug 2;8(8):CD010966. doi: 10.1002/14651858.CD010966.pub2.
4
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.
5
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.
6
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).针对具有 II 类 CFTR 基因突变（最常见的是 F508del）的囊性纤维化患者的校正治疗（含或不含增效剂）。
Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
7
Lumacaftor/Ivacaftor in Patients Aged 6-11 Years with Cystic Fibrosis and Homozygous for F508del-CFTR.鲁马卡托/依伐卡托用于6至11岁患有囊性纤维化且F508del-CFTR基因纯合的患者。
Am J Respir Crit Care Med. 2017 Apr 1;195(7):912-920. doi: 10.1164/rccm.201608-1754OC.
8
A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for .一项评估 Lumacaftor/Ivacaftor 在 1 岁以下囊性纤维化纯合子患儿中的疗效和安全性的 3 期、开放标签研究。
Am J Respir Crit Care Med. 2022 Nov 15;206(10):1239-1247. doi: 10.1164/rccm.202204-0734OC.
9
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.
10
A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial.一种用于治疗携带 phe508del CFTR 突变的囊性纤维化患者的 CFTR 校正剂（芦卡帕替尼）和 CFTR 增强剂（依伐卡托）：一项 2 期随机对照试验。
Lancet Respir Med. 2014 Jul;2(7):527-38. doi: 10.1016/S2213-2600(14)70132-8. Epub 2014 Jun 24.

本文引用的文献

1
Long-Term Impact of Lumacaftor/Ivacaftor Treatment on Cystic Fibrosis Disease Progression in Children 2-5 Years of Age Homozygous for : A Phase 2, Open-Label Clinical Trial.鲁马卡托/依伐卡托治疗对2至5岁纯合子囊性纤维化患儿疾病进展的长期影响：一项2期开放标签临床试验。
Ann Am Thorac Soc. 2024 Nov;21(11):1550-1559. doi: 10.1513/AnnalsATS.202402-201OC.
2
Effects of lumacaftor-ivacaftor therapy on cystic fibrosis transmembrane conductance regulator function in F508del homozygous patients with cystic fibrosis aged 2-11 years.鲁马卡托-依伐卡托疗法对2至11岁F508del纯合子囊性纤维化患者囊性纤维化跨膜传导调节因子功能的影响。
Front Pharmacol. 2023 May 30;14:1188051. doi: 10.3389/fphar.2023.1188051. eCollection 2023.
3
Effects of Lumacaftor/Ivacaftor on Cystic Fibrosis Disease Progression in Children 2 through 5 Years of Age Homozygous for : A Phase 2 Placebo-controlled Clinical Trial.Lumacaftor/Ivacaftor 对 2 至 5 岁纯合子囊性纤维化患儿疾病进展的影响：一项 2 期安慰剂对照临床试验。
Ann Am Thorac Soc. 2023 Aug;20(8):1144-1155. doi: 10.1513/AnnalsATS.202208-684OC.
4
Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on CFTR Function in Patients with Cystic Fibrosis and One or Two Alleles.依伐卡托/泰它卡托/艾氟康唑治疗对具有一个或两个等位基因的囊性纤维化患者 CFTR 功能的影响。
Am J Respir Crit Care Med. 2022 Mar 1;205(5):540-549. doi: 10.1164/rccm.202110-2249OC.
5
Effects of Lumacaftor-Ivacaftor Therapy on Cystic Fibrosis Transmembrane Conductance Regulator Function in Phe508del Homozygous Patients with Cystic Fibrosis.利那洛肽治疗在囊性纤维化跨膜电导调节因子功能对囊性纤维化纯合子 Phe508del 患者的影响。
Am J Respir Crit Care Med. 2018 Jun 1;197(11):1433-1442. doi: 10.1164/rccm.201710-1983OC.
6
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.针对携带Phe508del CFTR纯合突变的囊性纤维化患者使用鲁马卡托-依伐卡托。
N Engl J Med. 2015 Oct 29;373(18):1783-4. doi: 10.1056/NEJMc1510466.

Reply to Martin and Grasemann: Is There a Role for Lumacaftor/Ivacaftor in Young Children with Cystic Fibrosis?

作者信息

Stahl Mirjam, Röhmel Jobst, Wielpütz Mark O, Mall Marcus A

机构信息

Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin Berlin, Germany.

German Center for Lung Research, associated partner site Berlin Berlin, Germany.

出版信息

Ann Am Thorac Soc. 2025 Mar;22(3):469-470. doi: 10.1513/AnnalsATS.202411-1237LE.

DOI:10.1513/AnnalsATS.202411-1237LE
PMID:39836962
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11892656/
Abstract
摘要