Elexacaftor-tezacaftor-ivacaftor for Cystic Fibrosis with Phe508del Mutation: Evidence from Randomized Controlled Trials

Overview

Journal SAGE Open Med

Publisher Sage Publications

Specialty General Medicine

Date 2024 Jan 22

PMID 38249954

Authors

Rong He

Fei Lin

Zehui Deng

Bin Yu

Affiliations

Soon will be listed here.

Abstract

Objective: This study aimed to conduct a systematic review and meta-analysis of randomized controlled trials to evaluate the effects of elexacaftor-tezacaftor-ivacaftor (ELX-TEZ-IVA) on patients with cystic fibrosis (CF).

Methods: A systematic search was performed in PubMed, Embase, and the Cochrane Library from inception to August 1, 2022. Meta-analysis was conducted using Review Manager 5.3 software.

Results: Six studies comprising seven reports involving a total of 1125 CF patients were included. The meta-analyses indicated that ELX-TEZ-IVA significantly improved the percentage predicted forced expiratory volume in 1 s (ppFEV1) by 10.29% (95% confidence interval (CI) (6.44, 14.14), < 0.00001) and the CF questionnaire-revised respiratory domain (CFQ-R RD) by 14.59 points (95% CI (9.25, 19.94), < 0.00001) compared to placebo, ivacaftor (IVA), or tezacaftor-ivacaftor (TEZ-IVA). In addition, the ELX-TEZ-IVA group showed significantly lower sweat chloride concentrations by 40.30 mmol/L (95% CI (-49.85, -30.74), < 0.00001). However, the incidence of adverse events in the ELX-TEZ-IVA group was slightly higher than that in the placebo, IVA, or TEZ-IVA groups.

Conclusion: ELX-TEZ-IVA demonstrated efficacy in improving ppFEV1, CFQ-R RD, and sweat chloride concentrations in patients with CF. However, caution should be exercised regarding the incidence of AEs, particularly mild and moderate ones.

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