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Observational Study
. 2020 Jan;19(1):68-79.
doi: 10.1016/j.jcf.2019.05.015. Epub 2019 Jun 10.

Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries

Nataliya Volkova  1 , Kristin Moy  2 , Jennifer Evans  3 , Daniel Campbell  4 , Simon Tian  5 , Christopher Simard  6 , Mark Higgins  7 , Michael W Konstan  8 , Gregory S Sawicki  9 , Alexander Elbert  10 , Susan C Charman  11 , Bruce C Marshall  12 , Diana Bilton  13
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Free article
Observational Study

Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries

Nataliya Volkova et al. J Cyst Fibros. 2020 Jan.
Free article

Abstract

Background: Ivacaftor is the first in a class of drugs, CFTR modulators, that target the underlying defect in cystic fibrosis (CF). This long-term observational safety study evaluated CF disease progression in patients treated with ivacaftor in a real-world setting for up to 5 years.

Methods: Data from existing US and UK CF patient registries were used to assess longitudinal patterns in lung function, nutritional status, pulmonary exacerbations and hospitalizations, CF-related diabetes (CFRD), and Pseudomonas aeruginosa in ivacaftor-treated vs untreated comparator cohorts matched by age, sex, and disease severity.

Results: US analyses included 635 ivacaftor-treated patients and 1874 comparators followed for 5 years from year 1 of market availability (2012-2016). Evaluation of outcome patterns from pretreatment baseline (2011) through year 5 (2016), showed that relative to comparators, ivacaftor-treated patients had better preserved lung function (mean change in percent predicted FEV1, -0.7 percentage points with ivacaftor vs -8.3 percentage points in comparators) and improved nutritional status (mean body mass index change +2.4 kg/m2 with ivacaftor vs +1.6 kg/m2 in comparators). US patients treated with ivacaftor had significantly lower frequencies of exacerbations and hospitalizations in each of the 5 years of follow-up relative to pretreatment baseline and comparators. Favorable trends in CFRD and P. aeruginosa prevalence were also observed. Findings from the smaller UK registry were directionally similar to and consistent with US findings.

Conclusions: This observational study represents the largest longitudinal analysis of patients treated with ivacaftor in a real-world setting. The findings support disease modification by CFTR modulation with ivacaftor.

Keywords: CF complications; CF disease progression; CFTR; CFTR modulator; Ivacaftor.

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Conflict of interest statement

Conflict of interest statement All authors received nonfinancial support (assistance with manuscript preparation) from ArticulateScience LLC, which received funding from Vertex Pharmaceuticals. Additional disclosures are as follows: NV, KM, JE, DC, ST, CS, and MH: employee of Vertex Pharmaceuticals and may own stock or stock options in Vertex Pharmaceuticals. MWK: grants, personal fees, and nonfinancial support from Vertex Pharmaceuticals, during the course of the study; grants and nonfinancial support from the Cystic Fibrosis Foundation (CFF); grants, personal fees, and nonfinancial support from Corbus, Laurent, PTC, and Savara; personal fees and nonfinancial support from Chiesi, Celtaxsys, Genentech, and Merck; personal fees from Albumedix, Anthera, Ionis, Paranta, Protalix, and Santhera outside the submitted work. GSS: grants and personal fees from Vertex Pharmaceuticals outside the submitted work. AE and BCM: other support from several pharmaceutical companies during the conduct of the study (The CFF has entered into therapeutic development award agreements and licensing agreements to assist with the development of CFTR modulators that may result in intellectual property rights, royalties and other fees provided to CFF by various pharmaceutical companies). SCC: service agreement between Vertex Pharmaceuticals and Cystic Fibrosis Services Limited for statistical analysis. DB: member of the Steering Committee of the UK CF Registry, which provided data for this study.

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