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Review

Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review

1
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough, Cardiff CF64 2XX, UK
2
Pharmerit—An OPEN Health Company, 4350 East-West Highway, Suite 1100, Bethesda, MD 20814, USA
3
Vertex Pharmaceuticals Incorporated, Boston, MA 02210, USA
4
Vertex Pharmaceuticals (Europe) Limited, London W2 6BD, UK
*
Author to whom correspondence should be addressed.
J. Clin. Med. 2021, 10(7), 1527; https://doi.org/10.3390/jcm10071527
Submission received: 3 February 2021 / Revised: 24 March 2021 / Accepted: 26 March 2021 / Published: 6 April 2021

Abstract

Cystic fibrosis (CF) is a rare, progressive, multi-organ genetic disease. Ivacaftor, a small-molecule CF transmembrane conductance regulator modulator, was the first medication to treat the underlying cause of CF. Since its approval, real-world clinical experience on the use of ivacaftor has been documented in large registries and smaller studies. Here, we systematically review data from real-world observational studies of ivacaftor treatment in people with CF (pwCF). Searches of MEDLINE and Embase identified 368 publications reporting real-world studies that enrolled six or more pwCF treated with ivacaftor published between January 2012 and September 2019. Overall, 75 publications providing data from 57 unique studies met inclusion criteria and were reviewed. Studies reporting within-group change for pwCF treated with ivacaftor consistently showed improvements in lung function, nutritional parameters, and patient-reported respiratory and sino-nasal symptoms. Benefits were evident as early as 1 month following ivacaftor initiation and were sustained over long-term follow-up. Decreases in pulmonary exacerbations, Pseudomonas aeruginosa prevalence, and healthcare resource utilization also were reported for up to 66 months following ivacaftor initiation. In studies comparing ivacaftor treatment to modulator untreated comparator groups, clinical benefits similarly were reported as were decreases in mortality, organ-transplantation, and CF-related complications. The safety profile of ivacaftor observed in these real-world studies was consistent with the well-established safety profile based on clinical trial data. Our systematic review of real-world studies shows ivacaftor treatment in pwCF results in highly consistent and sustained clinical benefit in both pulmonary and non-pulmonary outcomes across various geographies, study designs, patient characteristics, and follow-up durations, confirming and expanding upon evidence from clinical trials.
Keywords: cystic fibrosis; cystic fibrosis transmembrane conductance regulator (CFTR); ivacaftor; real-world evidence; systematic literature review cystic fibrosis; cystic fibrosis transmembrane conductance regulator (CFTR); ivacaftor; real-world evidence; systematic literature review

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MDPI and ACS Style

Duckers, J.; Lesher, B.; Thorat, T.; Lucas, E.; McGarry, L.J.; Chandarana, K.; De Iorio, F. Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review. J. Clin. Med. 2021, 10, 1527. https://doi.org/10.3390/jcm10071527

AMA Style

Duckers J, Lesher B, Thorat T, Lucas E, McGarry LJ, Chandarana K, De Iorio F. Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review. Journal of Clinical Medicine. 2021; 10(7):1527. https://doi.org/10.3390/jcm10071527

Chicago/Turabian Style

Duckers, Jamie, Beth Lesher, Teja Thorat, Eleanor Lucas, Lisa J. McGarry, Keval Chandarana, and Fosca De Iorio. 2021. "Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review" Journal of Clinical Medicine 10, no. 7: 1527. https://doi.org/10.3390/jcm10071527

APA Style

Duckers, J., Lesher, B., Thorat, T., Lucas, E., McGarry, L. J., Chandarana, K., & De Iorio, F. (2021). Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review. Journal of Clinical Medicine, 10(7), 1527. https://doi.org/10.3390/jcm10071527

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