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三联康(ELX/TEZ/IVA)和思合华(TEZ/IVA)治疗携带F508del等位基因的囊性纤维化的疗效:一项系统评价和荟萃分析。

Efficacy of Trikafta (ELX/TEZ/IVA) & Symdeko (TEZ/IVA) in Treating Cystic Fibrosis with F508del Allele: A Systematic Review and Meta-analysis.

作者信息

Hussain Kainat, Karhana Sonali, Garg Aakriti, Khan Mohd Ashif

机构信息

Department of Translational and Clinical Research, Jamia Hamdard School of Chemical and Life Sciences, New Delhi, India.

Department of Pharmacology, Jamia Hamdard School of Pharmaceutical Education and Research, New Delhi, India.

出版信息

Thorac Res Pract. 2025 Aug 15;26(5):248-259. doi: 10.4274/ThoracResPract.2025.2025-1-1. Epub 2025 May 8.

Abstract

The objective of the study was to assess and compare the efficacy of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) treatment with TEZ/IVA treatment in individuals diagnosed with cystic fibrosis (CF) and carrying the F508del allele. An extensive search of relevant literature was conducted using online resources, namely, PubMed, ScienceDirect, and Google Scholar. The initial search identified 248 articles, and after a careful examination of the full text of 18 articles, 7 met the inclusion and exclusion criteria. These selected reports were then thoroughly examined to perform a comparative analysis of the effectiveness of TEZ/IVA versus ELX/TEZ/IVA in CF patients with the F508del allele. The quality of the selected reports was evaluated using the Cochrane risk-of-bias tool for randomized studies, known as RoB 2. ELX/TEZ/IVA has shown significant improvements in key indicators of CF treatment. It has demonstrated a significant increase in forced expiratory volume in one second levels, indicating improved respiratory capacity and airflow. Additionally, ELX/TEZ/IVA successfully reduced sweat chloride levels and positively impacted Cystic Fibrosis Questionnaire-Revised Respiratory Domain scores, reflecting enhanced respiratory function and improved quality of life for patients. Overall, the study concluded that ELX/TEZ/IVA provided a clinically robust benefit compared to TEZ/IVA alone while maintaining a favourable safety profile.

摘要

本研究的目的是评估和比较依列卡福/替扎卡福/艾伐卡福(ELX/TEZ/IVA)治疗与替扎卡福/艾伐卡福(TEZ/IVA)治疗对诊断为囊性纤维化(CF)且携带F508del等位基因个体的疗效。使用在线资源,即PubMed、ScienceDirect和谷歌学术,对相关文献进行了广泛检索。初步检索识别出248篇文章,在仔细审查了18篇文章的全文后,7篇符合纳入和排除标准。然后对这些选定的报告进行了全面审查,以对携带F508del等位基因的CF患者中TEZ/IVA与ELX/TEZ/IVA的有效性进行比较分析。使用Cochrane随机研究偏倚风险工具(称为RoB 2)评估选定报告的质量。ELX/TEZ/IVA在CF治疗的关键指标上显示出显著改善。它已证明一秒用力呼气量水平显著增加,表明呼吸能力和气流得到改善。此外,ELX/TEZ/IVA成功降低了汗液氯化物水平,并对囊性纤维化问卷修订版呼吸领域得分产生了积极影响,反映出患者呼吸功能增强和生活质量提高。总体而言,该研究得出结论,与单独使用TEZ/IVA相比,ELX/TEZ/IVA在临床上提供了强大的益处,同时保持了良好的安全性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7538/12365565/4232616b61d0/TurkThoracJ-26-5-248-figure-10.jpg

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