Observational studyJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2021
Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation.
Observational study in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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
47 citing papers in PubMed, 63 citations in OpenAlex.
- Impact of 2 years of treatment with elexacaftor/tezacaftor/ivacaftor on longitudinal changes in structural lung disease in people with cystic fibrosis: results from the RECOVER trial.Annals of the American Thoracic Society · 2026Trial
- Long-term effectiveness and safety of cystic fibrosis transmembrane conductance regulator modulators and their impact on the lives of people with cystic fibrosis.American journal of respiratory and critical care medicine · 2026Article
- Representation of minoritized people with cystic fibrosis in the Cystic Fibrosis Therapeutics Development Network clinical trials.Annals of the American Thoracic Society · 2026Article
- Shifting Nutrition Paradigms: Changing Attitudes Towards Diet and Body Perceptions Among People With CF in the Era of Highly Effective Modulators.Journal of human nutrition and dietetics : the official journal of the British Dietetic Association · 2026Article
- Cystic Fibrosis Mortality Trends 1999-2024-A CDC Wonder Study.Advances in respiratory medicine · 2026Article
- Assessing Dietary Patterns and Composition Among Adults With Cystic Fibrosis Taking Highly Effective Modulator Therapy.Pediatric pulmonology · 2026Article
- A pilot study of ultra-low-dose chest CT combined with co-production in cystic fibrosis care.Pediatric research · 2026Article
- ENHANCE: a community partnership charting the evolution of early-life cystic fibrosis disease manifestations in a new era of management.ERJ open research · 2026Article
- Evaluating Long-Term Effectiveness of Cystic Fibrosis Modulator Therapies after Rapid Adoption: A Dual-Approach Study.Annals of the American Thoracic Society · 2026Article
- The New Landscape of Cystic Fibrosis in the Era of Highly Effective Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy.Journal of mother and child · 2026Review
- Transcriptomic and functional responses of the cystic fibrosis airway epithelium to CFTR modulator therapy.JCI insight · 2025Article
- The State of Weight in Cystic Fibrosis: Understanding Nutritional Status and Individualizing Nutritional Care in the Modulator Era.Nutrients · 2025Review
- Elexacaftor-tezacaftor-ivacaftor pharmacokinetics with concurrent tacrolimus administration after lung transplant.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2025Observational
- Article
- Observational research in epidemic settings: a roadmap to reform.BMJ global health · 2025Article
- Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells.Nature biomedical engineering · 2025Article
- Article
- Glucagon-like peptide1 receptor agonist treatment of cystic fibrosis-related diabetes complicated by obesity: A cases series and literature review.Journal of clinical & translational endocrinology · 2024Review
- Therapeutic Interventions forJournal of clinical medicine · 2024Review
- Changing profile of bacterial infection and microbiome in cystic fibrosis: when to use antibiotics in the era of CFTR-modulator therapy.European respiratory review : an official journal of the European Respiratory Society · 2024Review
Corrections and comments
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Authors and funding
9 authors at 5 institutions in 1 country.
Funding
Abstract
backgroundThe cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, ivacaftor, was first approved for people with CF and the G551D CFTR mutation. This study describes the long-term clinical effectiveness of ivacaftor in this population.
methodsWe conducted a multicenter, prospective, longitudinal, observational study of people with CF ages ≥6 years with at least one copy of the G551D CFTR mutation. Measurements of lung function, growth, quality of life, and sweat chloride were performed after ivacaftor initiation (baseline, 1 month, 3 months, 6 months, and annually thereafter until 5.5 years).
resultsNinety-six participants were enrolled, with 81% completing all study measures through 5.5 years. This cohort experienced significant improvements in percent predicted forced expiratory volume in 1 second (ppFEV1) of 4.8 [2.6, 7.1] (p < 0.001) at 1.5 years, that diminished to 0.8 [-2.0, 3.6] (p = 0.57) at 5.5 years. Adults experienced larger improvements in ppFEV1 (7.4 [3.6, 11.3], p < 0.001 at 1.5 years and 4.3 [0.6, 8.1], p = 0.02 at 5.5 years) than children (2.8 [0.1, 5.6], p = 0.04 at 1.5 years and -2.0 [-5.9, 2.0], p = 0.32 at 5.5 years). Rate of lung function decline for the overall study cohort from 1 month after ivacaftor initiation through 5.5 years was estimated to be -1.22 pp/year [-1.70, -0.73]. Significant improvements in growth, quality of life measures, sweat chloride, Pseudomonas aeruginosa detection, and pulmonary exacerbation rates requiring antimicrobial therapy persisted through five years of therapy.
conclusionsThese findings demonstrate the long-term benefits and disease modifying effects of ivacaftor in children and adults with CF and the G551D mutation.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.