ReviewJournal of clinical medicine2021
Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review.
Review in Journal of clinical medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
23 citing papers in PubMed, 1 synthesis or guideline pooled it, 36 citations in OpenAlex.
- Ivacaftor-tezacaftor-elexacaftor, tezacaftor-ivacaftor and lumacaftor-ivacaftor for treating cystic fibrosis: a systematic review and economic evaluation.Health technology assessment (Winchester, England) · 2025Pooled it
- Article
- Repurposing of ivacaftor shows potential to treat ROR1 expressing high-grade serous ovarian cancer.Therapeutic advances in medical oncology · 2026Article
- Impact of CFTR Modulators on Longitudinal Cystic Fibrosis Survival and Mortality: Review and Secondary Analysis.Pulmonary therapy · 2025Review
- Interrelationships between diet quality and health-related quality of life in Irish adults living with cystic fibrosis.European journal of nutrition · 2025Article
- Gene expression responses of CF airway epithelial cells exposed to elexacaftor/tezacaftor/ivacaftor suggest benefits beyond improved CFTR channel function.American journal of physiology. Lung cellular and molecular physiology · 2024Article
- Long-term impact of ivacaftor on mortality rate and health outcomes in people with cystic fibrosis.Thorax · 2024Article
- Impact of age at ivacaftor initiation on pulmonary outcomes among people with cystic fibrosis.Thorax · 2024Observational
- Cystic fibrosis patient characteristics and healthcare resource utilization in Finland using linked registries.Heliyon · 2024Article
- Impact of lumacaftor/ivacaftor on nutrition and growth in modulator-naïve children over 24 weeks.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2024Article
- Levelling the playing field through the London Network of the UK clinical trials accelerator platform.Contemporary clinical trials communications · 2024Article
- CFTR Modulators: From Mechanism to Targeted Therapeutics.Handbook of experimental pharmacology · 2024Article
- Neutrophil bactericidal activity and host defenses in cystic fibrosis: a narrative review.Journal of thoracic disease · 2023Review
- Understanding and addressing the needs of people with cystic fibrosis in the era of CFTR modulator therapy.The Lancet. Respiratory medicine · 2023Review
- Qualitative and quantitative evaluation of computed tomography changes in adults with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor: a retrospective observational study.Frontiers in pharmacology · 2023Article
- Article
- The CFHealthHub Learning Health System: Using Real-Time Adherence Data to Support a Community of Practice to Deliver Continuous Improvement in an Archetypal Long-Term Condition.Healthcare (Basel, Switzerland) · 2022Review
- Therapeutic Drug Monitoring of Ivacaftor, Lumacaftor, Tezacaftor, and Elexacaftor in Cystic Fibrosis: Where Are We Now?Pharmaceutics · 2022Review
- An Update on CFTR Modulators as New Therapies for Cystic Fibrosis.Paediatric drugs · 2022Review
- Real-World Outcomes in Cystic Fibrosis Telemedicine Clinical Care in a Time of a Global Pandemic.Chest · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 4 institutions in 2 countries.
Funding
Abstract
Cystic fibrosis (CF) is a rare, progressive, multi-organ genetic disease. Ivacaftor, a small-molecule CF transmembrane conductance regulator modulator, was the first medication to treat the underlying cause of CF. Since its approval, real-world clinical experience on the use of ivacaftor has been documented in large registries and smaller studies. Here, we systematically review data from real-world observational studies of ivacaftor treatment in people with CF (pwCF). Searches of MEDLINE and Embase identified 368 publications reporting real-world studies that enrolled six or more pwCF treated with ivacaftor published between January 2012 and September 2019. Overall, 75 publications providing data from 57 unique studies met inclusion criteria and were reviewed. Studies reporting within-group change for pwCF treated with ivacaftor consistently showed improvements in lung function, nutritional parameters, and patient-reported respiratory and sino-nasal symptoms. Benefits were evident as early as 1 month following ivacaftor initiation and were sustained over long-term follow-up. Decreases in pulmonary exacerbations,
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What OpenQuestion holds
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.