Life expectancy for people with cystic fibrosis (CF) has steadily improved over the
past 2 decades, with a median survival of 48.4 (95% CI: 45.9-51.5) years in the United
States (US) according to the 2019 US Cystic Fibrosis Foundation Patient Registry Report.
This survival improvement is attributed to numerous advancements, including airway
clearance technology and antimicrobial therapies as well as the standardization of
clinical care guided by numerous multicenter clinical trials addressing key questions
in the management of CF. More recently, the advent of cystic fibrosis transmembrane
conductance regulator (CFTR) gene modulator therapy, which corrects the malfunctioning
protein, started with the approval of ivacaftor in 2012 and the triple combination
therapy (elexacaftor/tezacaftor/ivacaftor or Trikafta) in 2019. Trikafta greatly expanded
CTFR modulator use to a larger proportion of the CF population for those people with
at least one copy of the F508del mutation or one copy of 177 specified genes. These
new therapies that target the malfunctioning protein have significantly altered the
paradigm regarding the approach to clinical care for CF in the modern era.
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References
- Real-world outcomes of ivacaftor treatment in people with cystic fibrosis: a systematic review.J Clin Med. 2021; 10: 1527
- Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor.Thorax. 2018; 73: 731-740
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- To treat or not to treat: CFTR modulators after lung transplantation.Pediatr Transplant. 2021; 25: e14007
- CFTR modulator use in post lung transplant recipients.J Heart Lung Transplant. 2021; (Aug 26:S1053-2498(21)02482-7)
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Publication history
Published online: October 22, 2021
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