Abstract
BackgroundIn 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. MethodsUsing 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. ResultsUp 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. ConclusionsWe 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.
Highlights
Ivacaftor was the first CF transmembrane conductance regulator (CFTR) modulator therapy to be licensed for the treatment of people with cystic fibrosis (CF) with specific CF-causing genetic mutations
We investigated whether the trend in the proportions of treatment use over time differs by genotype group, in the preivacaftor era and the ivacaftor era, separately
We have shown a clear divergence in treatment use over time between individuals treated with ivacaftor and those untreated due to their genotype for a number of long-term treatments widely used in CF, including dornase alfa, hypertonic saline and inhaled antibiotics
Summary
Ivacaftor was the first CF transmembrane conductance regulator (CFTR) modulator therapy to be licensed for the treatment of people with cystic fibrosis (CF) with specific CF-causing genetic mutations. Phase III randomized controlled trials have found evidence that treatment with ivacaftor is associated with significant improvement in clinical outcomes including FEV1 for individuals with a Gly551Asp-CFTR (G551D) mutation [1,2]. 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. The use of other treatments tended to decrease or remain stable by year for the ivacaftortreated 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. Further research is needed to investigate whether the differences in treatment patterns are associated with changes in health outcomes
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