ReviewCells2022
CFTR Modulators in People with Cystic Fibrosis: Real-World Evidence in France.
Review in Cells, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 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
31 citing papers in PubMed, 1 synthesis or guideline pooled it, 55 citations in OpenAlex.
- Tools used to measure quality of life in adults with cystic fibrosis- a systematic review.Health and quality of life outcomes · 2025Pooled it
- Article
- SOLAR: a noninterventional study of outcomes over the long term of people with cystic fibrosis (CF) aged ≥6 years treated with elexacaftor/tezacaftor/ivacaftor using data from the French CF Registry.ERJ open research · 2026Article
- Review
- DICL: a manually curated database of ion channels and ligands as a useful platform for drug discovery targeting ion channels.Journal of cheminformatics · 2026Article
- Feasibility and Tolerance of High-Intensity Interval Training in Adults With Cystic Fibrosis.Archives of rehabilitation research and clinical translation · 2026Article
- Decoding liver injury in cystic fibrosis: How to tell drug-induced liver injury from cystic fibrosis liver disease.World journal of gastroenterology · 2026Review
- Restoration of Defective CFTR in Human Nasal Respiratory Epithelial Cells by CFTR Modulators and mRNA Transfection.International journal of molecular sciences · 2026Article
- Identity, functional consequences, and context effects of amino acids inserted during suppression of CFTR nonsense mutations.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2026Article
- Real-World Evaluation of Outcomes and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Pediatric Patients With Cystic Fibrosis: A Retrospective Study.Clinical and translational science · 2025Article
- Impact of CFTR Modulators on Longitudinal Cystic Fibrosis Survival and Mortality: Review and Secondary Analysis.Pulmonary therapy · 2025Review
- Decreased insulin dose-adjusted hemoglobin A1c in adults with cystic fibrosis-related diabetes treated with elexacaftor-tezacaftor-ivacaftor.Journal of clinical & translational endocrinology · 2025Article
- 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
- Evolution of Lung Disease Studied by Computed Tomography in Adults with Cystic Fibrosis Treated with Elexacaftor/Tezacaftor/Ivacaftor.Journal of imaging · 2025Article
- Improved Clinical Outcomes With Elexacaftor/Tezacaftor/Ivacaftor in Patients With Cystic Fibrosis and Advanced Lung Disease: Real-World Evidence From an Italian Single-Center Study.Pharmacology research & perspectives · 2025Article
- Unveiling the psychosocial impact of Elexacaftor/Tezacaftor/Ivacaftor therapy in Cystic Fibrosis patients.BMC pulmonary medicine · 2025Observational
- A Global Perspective on Transition Models for Pediatric to Adult Cystic Fibrosis Care: What Has Been Made So Far?Journal of clinical medicine · 2024Review
- One-Year Effect of Elexacaftor/Tezacaftor/Ivacaftor Therapy on HbA1c Levels and Insulin Requirement in Patients with Insulin-Dependent Cystic Fibrosis-Related Diabetes: A Retrospective Observational Study.Life (Basel, Switzerland) · 2024Article
- The ER Stress Induced in Human Neuroblastoma Cells Can Be Reverted by Lumacaftor, a CFTR Corrector.Current issues in molecular biology · 2024Article
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Cystic fibrosis (CF) is a rare genetic multisystemic disease, the manifestations of which are due to mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein and can lead to respiratory insufficiency and premature death. CFTR modulators, which were developed in the past decade, partially restore CFTR protein function. Their clinical efficacy has been demonstrated in phase 3 clinical trials, particularly in terms of lung function and pulmonary exacerbations, nutritional status, and quality of life in people with gating mutations (ivacaftor), homozygous for the F508del mutation (lumacaftor/ivacaftor and tezacaftor/ivacaftor), and in those with at least one F508del mutation (elexacaftor/tezacaftor/ivacaftor). However, many questions remain regarding their long-term safety and effectiveness, particularly in patients with advanced lung disease, liver disease, renal insufficiency, or problematic bacterial colonization. The impact of CFTR modulators on other important outcomes such as concurrent treatments, lung transplantation, chest imaging, or pregnancies also warrants further investigation. The French CF Reference Network includes 47 CF centers that contribute patient data to the comprehensive French CF Registry and have conducted nationwide real-world studies on CFTR modulators. This review seeks to summarize the results of these real-world studies and examine their findings against those of randomized control trials.
Indexed as
Identifiers
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.