Evidence for Early Cystic Fibrosis Transmembrane Conductance Regulator Modulator Treatment for Children with Cystic Fibrosis Keeps Growing.
作者信息
Hoppe Jordana E, Zemanick Edith T, Martiniano Stacey L
机构信息
Department of Pediatrics University of Colorado Anschutz Medical Campus Aurora, Colorado.
Breathing Institute Children's Hospital Colorado Aurora, Colorado.
出版信息
Am J Respir Crit Care Med. 2022 Dec 1;206(11):1308-1310. doi: 10.1164/rccm.202208-1507ED.
Abstract
摘要
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本文引用的文献
1
Disparities in first evaluation of infants with cystic fibrosis since implementation of newborn screening.自新生儿筛查实施以来，囊性纤维化婴儿首次评估中的差异。
J Cyst Fibros. 2023 Jan;22(1):89-97. doi: 10.1016/j.jcf.2022.07.010. Epub 2022 Jul 22.
2
Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study.依伐卡托钠/泰比卡托钠/埃他卡托钠在 和最小功能突变杂合子且年龄在 6 至 11 岁的囊性纤维化儿童中的疗效和安全性：一项 3b 期、随机、安慰剂对照研究。
Am J Respir Crit Care Med. 2022 Dec 1;206(11):1361-1369. doi: 10.1164/rccm.202202-0392OC.
3
Current state of CFTR modulators for treatment of Cystic Fibrosis.用于治疗囊性纤维化的 CFTR 调节剂的现状。
Curr Opin Pharmacol. 2022 Aug;65:102239. doi: 10.1016/j.coph.2022.102239. Epub 2022 May 21.
4
Left behind: The potential impact of CFTR modulators on racial and ethnic disparities in cystic fibrosis.遗留问题：CFTR 调节剂对囊性纤维化中种族和民族差异的潜在影响。
Paediatr Respir Rev. 2022 Jun;42:35-42. doi: 10.1016/j.prrv.2021.12.001. Epub 2021 Dec 22.
5
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.
6
Determinants of lung disease progression measured by lung clearance index in children with cystic fibrosis.肺清除指数测定囊性纤维化患儿肺部疾病进展的决定因素。
Eur Respir J. 2021 Jul 8;58(1). doi: 10.1183/13993003.03380-2020. Print 2021 Jul.
7
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.
8
A phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation.一项 3 期、双盲、平行分组研究，旨在评估 tezacaftor 联合 ivacaftor 在 6 至 11 岁囊性纤维化纯合子 F508del 或杂合子 F508del-CFTR 突变和残余功能突变的参与者中的疗效和安全性。
J Cyst Fibros. 2021 Jan;20(1):68-77. doi: 10.1016/j.jcf.2020.07.023. Epub 2020 Sep 21.
9
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.
10
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.
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