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A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis
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A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis
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Journal Article
A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis
2019
Overview
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 FEV
1
% predicted (ppFEV
1
). Fourteen RCTs met our eligibility criteria. The largest improvement in ppFEV
1
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 ppFEV
1
in F508del homozygous individuals but there was increased reporting of respiratory adverse events with LUM-IVA compared to placebo. IVA also significantly improved ppFEV
1
in a sub-group of individuals ≥18 years old with an R117H mutation. No significant improvements in ppFEV
1
were observed for IVA, LUM, or TEZ in F508del homozygous individuals, LUM or LUM-IVA in F508del heterozygous individuals, or ataluren in individuals with a nonsense mutation. Significant improvements in ppFEV
1
and other clinical outcomes were observed for IVA in G551D individuals, TEV-IVA and LUM-IVA in F508del homozygous individuals, and IVA in adults with a R117H mutation.
Publisher
Nature Publishing Group UK,Nature Publishing Group
Subject
/ Aminophenols - adverse effects
/ Aminophenols - therapeutic use
/ Benzodioxoles - therapeutic use
/ Chloride Channel Agonists - adverse effects
/ Chloride Channel Agonists - therapeutic use
/ Cystic Fibrosis - drug therapy
/ Cystic fibrosis transmembrane conductance regulator
/ Cystic Fibrosis Transmembrane Conductance Regulator - chemistry
/ Cystic Fibrosis Transmembrane Conductance Regulator - genetics
/ Cystic Fibrosis Transmembrane Conductance Regulator - metabolism
/ Humanities and Social Sciences
/ Humans
/ Mutation
/ Polymorphism, Single Nucleotide
/ Quinolones - adverse effects
/ Quinolones - therapeutic use
/ Science
