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Review
. 2021 Dec;56(12):3595-3606.
doi: 10.1002/ppul.25627. Epub 2021 Aug 18.

Review of CFTR modulators 2020

Danielle M Goetz  1 , Adrienne P Savant  2
Affiliations
Review

Review of CFTR modulators 2020

Danielle M Goetz et al. Pediatr Pulmonol. 2021 Dec.

Abstract

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are small molecules that directly impact the CFTR protein, improving the function of the CFTR chloride and bicarbonate channel. Beginning in 2012 with the Food and Drug Administration approval of the first CFTR modulator, ivacaftor, this class of medications has had largely positive effects on many outcomes in people with cystic fibrosis (PwCF), including lung function, growth, and other clinical parameters. There have been continued exciting developments in the current research on CFTR modulators, expanding beyond original studies. This first part of a three-part cystic fibrosis (CF) year in review 2020 will focus on research on CFTR modulators. In addition to reviewing new clinical insights, we describe work done on novel outcomes, adverse effects, issues related to cost, and next steps for clinical trials. The review focuses on articles from Pediatric Pulmonology published in 2020, but it includes articles from other journals that are of particular interest to clinicians. New developments in CF research continue to be brought forth to the CF community, deepening the understanding of this disease and improving clinical care.

Keywords: cystic fibrosis; cystic fibrosis transmembrane regulator (CFTR) modulators; pulmonology; treatment.

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References

REFERENCES

    1. Solomon GMMS, Ramsey BW, Rowe SM. Breakthrough therapies: cystic fibrosis (CF) potentiators and correctors. Pediatr Pulmonol. 2015;50(Suppl 40):S3-S13.
    1. Davies JC, Wainwright CE, Sawicki GS, et al. Ivacaftor in infants aged 4 to <12 months with cystic fibrosis and a gating mutation: results of a 2-part phase 3 clinical trial. Am J Respir Crit Care Med. 2021;203:585-593.
    1. Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018;6(7):545-553.
    1. Davies JC, Cunningham S, Harris WT, et al. Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study. Lancet Respir Med. 2016;4:107-115.
    1. Aalbers BL, de Winter-de Groot KM, Arets HGM, et al. Clinical effect of lumacaftor/ivacaftor in F508del homozygous CF patients with FEV1 >/= 90% predicted at baseline. J Cyst Fibros. 2020;19(4):654-658.

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