Efficacy and Safety of Elexacaftor-Tezacaftor-Ivacaftor in the Treatment of Cystic Fibrosis: A Systematic Review

被引:33
作者
Kapouni, Nikoletta [1 ]
Moustaki, Maria [2 ]
Douros, Konstantinos [1 ]
Loukou, Ioanna [2 ]
机构
[1] Natl & Kapodistrian Univ Athens, Attikon Univ Hosp, Sch Med, Dept Pediat 3,Pediat Allergy & Resp Unit, Athens 11527, Greece
[2] Agia Sofia Childrens Hosp, Cyst Fibrosis Dept, Athens 11527, Greece
来源
CHILDREN-BASEL | 2023年 / 10卷 / 03期
关键词
cystic fibrosis; cystic fibrosis transmembrane conductance regulator (CFTR) protein; CFTR modulators; elexacaftor; ivacaftor; tezacaftor; kaftrio; trikafta; ELEXACAFTOR/TEZACAFTOR/IVACAFTOR; PEOPLE; ADULTS;
D O I
10.3390/children10030554
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) is a new CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator treatment, used over the last few years, which has shown an improvement in different clinical outcomes in patients with cystic fibrosis (CF). The objective of this study was a systematic research of the literature on the efficacy and safety of this CFTR modulator on patients with CF. A search of Pubmed was conducted for randomized clinical trials and observational studies published from 2012 to September 2022. The included full manuscripts comprised nine clinical trials and 16 observational studies, whose participants were aged >= 12 years or were children 6-11 years old with at least one Phe508del mutation and/or advanced lung disease (ALD). These studies reported that ELX/TEZ/IVA has a significant positive effect on the lung function of patients with CF, by ameliorating parameters such as FEV1, LCI, pulmonary exacerbations or sweat chloride concentration, increasing BMI and improving quality of their life. Its role in cystic fibrosis-related diabetes (CFRD) is not yet clear. It was found that this new CFTR modulator has an overall favorable safety profile, with mild to moderate adverse events. Further studies are needed for a deeper understanding of the impact of CFTR modulators on other CF manifestations, or the possibility of treating with ELX/TEZ/IVA CF patients with rare CFTR mutations.
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页数:23
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