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. 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.

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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

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