CFTR modulator therapies - Effect on life expectancy in people with cystic fibrosis

I M Balfour-Lynn; J A King

doi:10.1016/j.prrv.2020.05.002

CFTR modulator therapies - Effect on life expectancy in people with cystic fibrosis

Paediatr Respir Rev. 2022 Jun:42:3-8. doi: 10.1016/j.prrv.2020.05.002. Epub 2020 May 26.

Authors

I M Balfour-Lynn¹, J A King²

Affiliations

¹ Department of Paediatric Respiratory Medicine, Royal Brompton Hospital, London, UK. Electronic address: i.balfourlynn@ic.ac.uk.
² Department of Paediatric Respiratory Medicine, Royal Brompton Hospital, London, UK.

Abstract

CFTR modulators have dramatically changed the clinical course of CF in those fortunate enough to receive them. Inevitably, randomised controlled trials during the development of these drugs are too short to use mortality as an outcome. Evidence for their effect on life expectancy are best gained from real world registry studies specifically looking at mortality, but these are only available for ivacaftor to date. Therefore, indirect evidence must be obtained by looking at outcomes known to affect mortality and seeing the effect of these drugs on those outcomes.

Keywords: CFTR modulators; Cystic fibrosis; Mortality; Prognosis.

Publication types

Review

MeSH terms

Aminophenols / therapeutic use
Cystic Fibrosis Transmembrane Conductance Regulator / genetics
Cystic Fibrosis* / drug therapy
Humans
Life Expectancy
Mutation

Substances

Aminophenols
CFTR protein, human
Cystic Fibrosis Transmembrane Conductance Regulator