1. Longitudinal Trends in Real-World Outcomes after Initiation of Ivacaftor. A Cohort Study from the Cystic Fibrosis Registry of Ireland.
- Author
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Kirwan, Laura, Fletcher, Godfrey, Harrington, Mary, Jeleniewska, Paulina, Zhou, Shijun, Casserly, Brian, Gallagher, Charles G, Greally, Peter, Gunaratnam, Cedric, Herzig, Mary, Linnane, Barry, McElvaney, Noel Gerard, McKone, Edward F, McNally, Paul, Mullane, David, Ní Chróinín, Muireann, O'Mahony, Michael, Plant, Barry J, and Jackson, Abaigeal D
- Subjects
ANTIBIOTICS ,CYSTIC fibrosis ,HOSPITAL care ,LONGITUDINAL method ,LUNGS ,MEMBRANE proteins ,GENETIC mutation ,PHENOLS ,QUINOLONE antibacterial agents ,RESPIRATION ,GENETIC markers ,BODY mass index ,TREATMENT effectiveness ,ACQUISITION of data ,FORCED expiratory volume - Abstract
Rationale: Patient registries have the potential to collect and analyze high-quality postauthorization data on new medicines.Objectives: We used cystic fibrosis (CF) registry data to assess outcomes after the initiation of ivacaftor, a CF transmembrane conductance regulator (CFTR) potentiator approved for the treatment of CF with a defective gating CFTR mutation.Methods: Longitudinal trends were examined using mixed-effects regression analysis in 80 ivacaftor-treated patients with CF aged 6 to 56 years registered with the CF Registry of Ireland with at least 36 months of before and after commencement data. The effects of ivacaftor treatment on forced expiratory volume in 1 second (FEV1) % predicted, body mass index (BMI), hospitalization for pulmonary exacerbation, and oral and intravenous antibiotic use were assessed.Results: In the 36 months after ivacaftor initiation, FEV1% predicted improved by 2.26% per annum (95% confidence interval [CI], 0.2 to 4.3) for patients aged younger than 12 years, remained unchanged for 12- to younger than 18-year-olds (95% CI, -1.9 to 2.9), and declined in adults by 1.74% per annum (95% CI, -3.1 to -0.4). BMI in adults increased 0.28 kg/m2 per annum (95% CI, 0.03 to 0.5), and there was no significant change in BMI z-score in children (95% CI, -0.01 to 0.1). In the year after ivacaftor initiation, intravenous antibiotic treatment reduced by 46% (95% CI, -62.5% to -23.3%, oral antibiotic treatment reduced by 49% (95% CI, -61.1% to -32.1%), and there was no significant reduction in hospitalization (95% CI, -59.2% to 9.7%).Conclusions: In this study of real-world CF registry data, clinical outcomes improved and healthcare resource utilization decreased after commencing ivacaftor. [ABSTRACT FROM AUTHOR]- Published
- 2019
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