Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis.

ABSTRACT:

Background and aim

Cystic fibrosis (CF) is a genetic disease that is difficult to treat and caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function. However, the data on children with CF are scarce. This meta-analysis aimed to evaluate the effectiveness and safety of this therapy in children diagnosed with CF.

Materials and methods

Relevant studies were identified through searching medical databases before April 1, 2022. The primary outcomes of ppFEV₁, lung clearance index_2.5 (LCI_2.5), sweat chloride concentration (SwCI), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) score were pooled and

SUBMITTER: Li Q 

PROVIDER: S-EPMC9276987 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature