Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2022 Mar;21(2):316-322.
doi: 10.1016/j.jcf.2021.08.014. Epub 2021 Sep 6.

Treatment patterns in people with cystic fibrosis: have they changed since the introduction of ivacaftor?

Emily Granger  1 , Gwyneth Davies  2 , Ruth H Keogh  3
Affiliations

Treatment patterns in people with cystic fibrosis: have they changed since the introduction of ivacaftor?

Emily Granger et al. J Cyst Fibros. 2022 Mar.

Abstract

Background: In late 2012, ivacaftor became available in the UK for people with cystic fibrosis (CF) aged 6 years and over with a G551D mutation. Long-term changes in treatment patterns have not previously been reported. We investigated long-term treatment patterns in people with CF with a G551D mutation who took ivacaftor and compared these with non-ivacaftor-treated cohorts using the UK Cystic Fibrosis Registry.

Methods: Using 2007-2018 data we compared treatment patterns between four cohorts: 1: ivacaftor-treated; 2: ivacaftor era (2013-2018), ineligible genotype (no G551D mutation); 3: pre-ivacaftor era (2007-2012), eligible genotype (G551D mutation); 4: pre-ivacaftor era, ineligible genotype. Treatments included: inhaled antibiotics, dornase alfa, hypertonic saline, chronic oral antibiotics and supplementary feeding.

Results: Up to 2012 the percentages of people taking each treatment were similar between the two cohorts defined by genotype and tended to increase by year with a similar slope. Once ivacaftor was introduced, the use of other treatments tended to decrease or remain stable by year for the ivacaftor-treated cohort, whereas it remained stable or increased in the non-ivacaftor-treated cohort. This led to differences in treatment use between the two cohorts in the ivacaftor-era, which became more marked over time.

Conclusions: We have shown a clear divergence in treatment patterns since the introduction of ivacaftor in a number of key treatments widely used in CF. Further research is needed to investigate whether the differences in treatment patterns are associated with changes in health outcomes.

Keywords: cystic fibrosis; ivacaftor; registry data; treatment burden; treatment patterns.

PubMed Disclaimer

Conflict of interest statement

Conflict of interest statement GD has received personal fees from Chiesi Limited for lectures, unrelated to the current work. She is co-Chief Investigator for the CF STORM clinical trial. EG has no conflicts of interest to declare. RHK received funding from a Circle of Care Award from Vertex.

Figures

Figure 1
Fig. 1
The proportions and 95% confidence intervals in each cohort prescribed different treatments by year.
Figure 2
Fig. 2
The proportions and 95% confidence intervals in each cohort prescribed inhaled antibiotics, dornase alfa and hypertonic saline solution by year, stratified by FEV1% at baseline, sex and age.
See this image and copyright information in PMC

References

    1. Davies J., Sheridan H., Bell N., Cunningham S., Davis S.D., Elborn J.S. Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: a randomised controlled trial. Lancet Respir Med. 2013;1(8):630–638. - PubMed
    1. Ramsey B.W., Davies J., McElvaney N.G., Tullis E., Bell S.C., Drevinek P. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. New England J Med. 2011;365(18):1663–1672. - PMC - PubMed
    1. Sawicki G.S., McKone E.F., Pasta D.J., Millar S.J., Wagener J.S., Johnson C.A. Sustained Benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data. Am J Respir Crit Care Med. 2015;192(7):836–842. - PubMed
    1. Bessonova L., Volkova N., Higgins M., Bengtsson L., Tian S., Simard C. Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor. Thorax. 2018;73(8):731–740. - PMC - PubMed
    1. Higgins M., Volkova N., Moy K., Marshall B.C., Bilton D. Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012-2016 Experience. Pulm Ther. 2020;6(1):141–149. - PMC - PubMed

Publication types

MeSH terms