Trial reportJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2019
Lumacaftor/Ivacaftor reduces pulmonary exacerbations in patients irrespective of initial changes in FEV
Trial report in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 27 papers, 2 of them syntheses 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.
A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Who cites it
27 citing papers in PubMed, 2 syntheses or guidelines pooled it, 44 citations in OpenAlex.
- Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).The Cochrane database of systematic reviews · 2023Pooled it
- Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).The Cochrane database of systematic reviews · 2020Pooled it
- Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026Review
- Greenness, Whiteness, and Blueness Assessment With a Novel and Fully Validated HPLC Method for Simultaneous Analysis of Lumacaftor and Ivacaftor in Sweat.International journal of analytical chemistry · 2026Article
- The clinical, psychosocial, and economic burden of cystic fibrosis lung disease in the era of CFTR modulator therapy.Annals of the American Thoracic Society · 2026Review
- Allergic Bronchopulmonary Aspergillosis (ABPA) in the Era of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators.Journal of fungi (Basel, Switzerland) · 2024Review
- Advancing the pipeline of cystic fibrosis clinical trials: a new roadmap with a global trial network perspective.The Lancet. Respiratory medicine · 2023Review
- The clinical impact of Lumacaftor-Ivacaftor on structural lung disease and lung function in children aged 6-11 with cystic fibrosis in a real-world setting.Respiratory research · 2023Observational
- ROCK STUDY in CF: sustained anti-inflammatory effects of lumacaftor-ivacaftor in sputum and peripheral blood samples of adult patients with cystic fibrosis-an observational study.BMJ open respiratory research · 2023Observational
- Nutritional Status and Circulating Levels of Fat-Soluble Vitamins in Cystic Fibrosis Patients: A Cohort Study and Evaluation of the Effect of CFTR Modulators.Children (Basel, Switzerland) · 2023Article
- The effect of CFTR modulators on structural lung disease in cystic fibrosis.Frontiers in pharmacology · 2023Article
- Left behind: The potential impact of CFTR modulators on racial and ethnic disparities in cystic fibrosis.Paediatric respiratory reviews · 2022Review
- Review
- Elexacaftor/Tezacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for theJournal of clinical medicine · 2022Article
- Effect of lumacaftor-ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis: Results from the PROSPECT MCC sub-study.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2022Article
- Chronic rhinosinusitis in cystic fibrosis: a review of therapeutic options.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2022Review
- Current and Emerging Therapies to Combat Cystic Fibrosis Lung Infections.Microorganisms · 2021Review
- Reply to KuekAmerican journal of respiratory and critical care medicine · 2021Article
- Ex vivo model predicted in vivo efficacy of CFTR modulator therapy in a child with rare genotype.Molecular genetics & genomic medicine · 2021Article
- Sharing Patient-Controlled Real-World Data Through the Application of the Theory of Commons: Action Research Case Study.Journal of medical Internet research · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 7 institutions in 3 countries.
Funding
Abstract
backgroundImproved lung function and fewer pulmonary exacerbations (PEx) were observed with lumacaftor/ivacaftor (LUM/IVA) in patients with cystic fibrosis homozygous for F508del. It is unknown whether PEx reduction extends to patients without early lung function improvement.
methodsPost hoc analyses of pooled phase 3 data (NCT01807923, NCT01807949) categorized LUM/IVA-treated patients by percent predicted forced expiratory volume in 1 s (ppFEV
resultsLUM (400 mg q12h)/IVA (250 mg q12h)-treated patients (n = 369) experienced significantly fewer PEx vs placebo, regardless of threshold category. With LUM/IVA, PEx rate per patient per year was 0.60 for those with absolute change in ppFEV
conclusionsLUM/IVA significantly reduced PEx, even in patients without early lung function improvement.
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.