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

荟萃分析 囊性纤维化 医学 等位基因 内科学 生物 遗传学 基因
作者
Kainat Hussain,Sonali Karhana,Aakriti Garg,Mohd Ashif Khan
标识
DOI:10.4274/thoracrespract.2025.2025-1-1
摘要

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.

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