ArticleThorax2024
Long-term impact of ivacaftor on mortality rate and health outcomes in people with cystic fibrosis.
Article in Thorax, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
15 citing papers in PubMed.
- Trial
- An Observational Cohort Study of Targeted Antibiotic Treatments of Cystic Fibrosis Exacerbations.Open forum infectious diseases · 2026Trial
- Exploring Exercise as Airway Clearance in Cystic Fibrosis: A Qualitative Study From the ExACT-CF Feasibility Trial.Pediatric pulmonology · 2026Trial
- Article
- Cystic Fibrosis Mortality Trends 1999-2024-A CDC Wonder Study.Advances in respiratory medicine · 2026Article
- Structural and Functional Pulmonary MRI to Predict Pulmonary Exacerbations in Cystic Fibrosis.Chest · 2026Article
- Ceragenins in Combination with Ivacaftor Prevent the Formation of Biofilm by Bacteria That Cause Rhinosinusitis.Pharmaceutics · 2025Article
- Maximising opportunity for therapeutic success: sequential participation in cystic fibrosis nucleic acid-based therapy trials.The Lancet. Respiratory medicine · 2025Review
- Transcriptomic and functional responses of the cystic fibrosis airway epithelium to CFTR modulator therapy.JCI insight · 2025Article
- Challenges and opportunities for drug development in rare pulmonary diseases like cystic fibrosis: an industry perspective.Current opinion in pulmonary medicine · 2025Review
- Impact of CFTR Modulators on Longitudinal Cystic Fibrosis Survival and Mortality: Review and Secondary Analysis.Pulmonary therapy · 2025Review
- Evolving nutrition therapy in cystic fibrosis: Adapting to the CFTR modulator era.Nutrition in clinical practice : official publication of the American Society for Parenteral and Enteral Nutrition · 2025Review
- An integrated multimodal approach to drug repurposing in endometriosis, using ROR1 as a target.Frontiers in pharmacology · 2025Article
- A Retrospective, Longitudinal Registry Study on the Long-Term Durability of Ivacaftor Treatment in People with Cystic Fibrosis.Pulmonary therapy · 2024Article
- Impact of age at ivacaftor initiation on pulmonary outcomes among people with cystic fibrosis.Thorax · 2024Observational
Corrections and comments
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Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundIvacaftor (IVA) has been shown to improve lung function and other clinical outcomes in people with cystic fibrosis (CF). A decade of real-world IVA availability has enabled the examination of long-term outcomes with this treatment. This retrospective, longitudinal cohort study investigated the impact of IVA on mortality rate and health outcomes among people with CF in the US.
methodsData from the US CF Foundation Patient Registry from January 2010 to December 2019 were analysed. The IVA-treated cohort included people with a CF transmembrane conductance regulator (
findingsOver a maximum follow-up of 7.9 years, the IVA-treated cohort (N=736) had lower rates of mortality (hazard ratio [HR] (95% CI): 0.22 (0.09 to 0.45)), lung transplant (HR: 0.11 (95% CI 0.02 to 0.28)), PEx (rate ratio: 0.49 (95% CI 0.42 to 0.55)) and all-cause hospitalisations (rate ratio: 0.50 (95% CI 0.43 to 0.56)) as well as better lung function (mean difference in ppFEV
interpretationOur analysis suggests that IVA provides sustained clinical benefits in people with CF over a follow-up period of approximately 8 years. These findings reinforce the existing real-world evidence that IVA can slow disease progression and decrease the healthcare burden of CF over the long term.
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