A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis.

ABSTRACT: Several placebo-controlled trials have been recently published evaluating novel therapies targeting the defective CFTR protein. This systematic review examines the clinical efficacy and safety of CFTR modulators in individuals with cystic fibrosis (CF) with specific genetic mutations. Online sources were searched for placebo-controlled, parallel-design clinical trials investigating CFTR modulators from January 1, 2005 to March 31, 2018. The primary outcome of interest was FEV₁% predicted (ppFEV₁). Fourteen RCTs met our eligibility criteria. The largest improvement in ppFEV₁ favouring treatment was observed for ivacaftor (IVA) in G551D individuals (≥6 years old). Both tezacaftor-ivacaftor (TEZ-IVA) and lumacaftor-ivacaftor (LUM-IVA) also improved ppFEV₁

SUBMITTER: Habib AR 

PROVIDER: S-EPMC6510767 | biostudies-literature | 2019 May

REPOSITORIES: biostudies-literature