Trial reportAnnals of the American Thoracic Society2021
Clinical Effectiveness of Lumacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for F508del-CFTR. A Clinical Trial.
Open access · greenAbstract readClinical TrialMulticenter Study
In one paragraph
Trial report in Annals of the American Thoracic Society, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
0numbers the graph read from it
0cells of the map it votes in
26citing papers in PubMed
4.7field-weighted citation impact, top 4% of its field
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
26 citing papers in PubMed, 45 citations in OpenAlex.
- Review
- Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Cystic Fibrosis: A Review of Registry-Based Evidence.Journal of clinical medicine · 2025Review
- Endoplasmic reticulum stress as a driver and therapeutic target for kidney disease.Nature reviews. Nephrology · 2025Review
- Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data.Scientific reports · 2025Observational
- The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol.ERJ open research · 2025Article
- Targeting ubiquitination machinery in cystic fibrosis: Where do we stand?Cellular and molecular life sciences : CMLS · 2024Review
- 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
- Anti-Microorganisms · 2023Review
- Characterizing CFTR modulated sweat chloride response across the cf population: Initial results from the CHEC-SC study.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2023Article
- Screening by high-throughput sequencing for pathogenic variants in cystic fibrosis: Benefit of introducing personalized therapies.Journal of cellular and molecular medicine · 2022Article
- CGM patterns in adults with cystic fibrosis-related diabetes before and after elexacaftor-tezacaftor-ivacaftor therapy.Journal of clinical & translational endocrinology · 2022Article
- Profiling the response to lumacaftor-ivacaftor in children with cystic between fibrosis and new insight from a French-Italian real-life cohort.Pediatric pulmonology · 2022Article
- Real life evaluation of the multi-organ effects of Lumacaftor/Ivacaftor on F508del homozygous cystic fibrosis patients.BMC pharmacology & toxicology · 2022Article
- Long-term effectiveness of dual CFTR modulator treatment of cystic fibrosis.ERJ open research · 2022Article
- Left behind: The potential impact of CFTR modulators on racial and ethnic disparities in cystic fibrosis.Paediatric respiratory reviews · 2022Review
- Genetic evidence supports the development of SLC26A9 targeting therapies for the treatment of lung disease.NPJ genomic medicine · 2022Article
- Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial.American journal of respiratory and critical care medicine · 2022Observational
- A year in review: Real world evidence, functional monitoring and emerging therapeutics in 2021.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2022Review
- Assays of CFTR Function In Vitro, Ex Vivo and In Vivo.International journal of molecular sciences · 2022Review
- 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
Corrections and comments
- Commented on by
Authors and funding
11 authors at 10 institutions in 2 countries.
Scott D SagelDepartment of Pediatrics, Children's Hospital Colorado and University of Colorado Anschutz Medical Campus, Aurora, Colorado.ORCID 0000-0001-6172-4465
Umer KhanCystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington.
Sonya L HeltsheCystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington.
John P ClancyDepartment of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Drucy BorowitzDepartment of Pediatrics, Jacobs School of Medicine and.
Daniel GelfondWestern New York Pediatric Gastroenterology, University at Buffalo, Buffalo, New York.
Scott H DonaldsonDepartment of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.
Antoinette MoranDepartment of Pediatrics, University of Minnesota, Minneapolis, Minnesota.
Felix RatjenDepartment of Pediatrics, Hospital for Sick Children, Toronto, Ontario, Canada; and.
Jill M VanDalfsenCystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington.
Steven M RoweDepartment of Medicine and the Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama.
Cystic Fibrosis Foundation · USChildren's Hospital Colorado · USCincinnati Children's Hospital Medical Center · USHospital for Sick Children · CAJacobs (United States) · USUniversity at Buffalo, State University of New York · USUniversity of Alabama at Birmingham · USUniversity of Minnesota · USUniversity of North Carolina at Chapel Hill · USUniversity of Washington · US
Funding
NIH Prior Approval Process ProfessionalUL1TR002535 · NCATS · UNIVERSITY OF COLORADO DENVER · PI SOKOL, RONALD J. · 2018 to 2022
$51.1MUAB CF Research and Translation Core CenterP30DK072482 · NIDDK · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI Brian R. Davis · 2007 to 2026
$23.0MTranslational Research Center to Expedite Novel Therapies in Cystic FibrosisP30DK089507 · NIDDK · SEATTLE CHILDREN'S HOSPITAL · PI Christopher Hooper Goss · 2010 to 2026
$21.4MTranslational Program in CFTR-Related Airway DiseasesR35HL135816 · NHLBI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI ROWE, STEVEN MARK · 2017 to 2022
$6.3MNCATS NIH HHS UL1 TR002535NHLBI NIH HHS R35 HL135816NIDDK NIH HHS P30 DK072482NIDDK NIH HHS P30 DK089507
Abstract
PubMed holds no abstract for this paper.
Indexed as
AminophenolsAminopyridinesBenzodioxolesCystic FibrosisQuinolonesAdolescentAdultChildCohort StudiesCystic Fibrosis Transmembrane Conductance RegulatorFemaleForced Expiratory VolumeHumansLongitudinal StudiesMaleMutationAminophenolsAminopyridinesBenzodioxolesCFTR protein, humanCystic Fibrosis Transmembrane Conductance RegulatorivacaftorlumacaftorQuinolonesclinical effectivenesscystic fibrosisivacaftorlumacaftorpostapproval study
Identifiers
What OpenQuestion holds
Textmetadata
Read underepoch 390
Registered trials
None linked
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.