Evidence map›Paper›PMID 34083123›Full record

ArticleJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2022

Effect of lumacaftor-ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis: Results from the PROSPECT MCC sub-study.

Scott H Donaldson, Beth L Laube, Peter Mogayzel, Timothy E Corcoran, Joseph M Pilewski, Agathe Ceppe, Jihong Wu, Pradeep G Bhambhvani, Felix Ratjen, Scott D Sagel and 3 more

Open access · bronzeAbstract readMulticenter Study
In one paragraph

Article in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
2.8field-weighted citation impact, top 9% of its field
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

9 citing papers in PubMed, 20 citations in OpenAlex.

  1. Trial
  2. Trial
  3. New drugs, new challenges in cystic fibrosis care.European respiratory review : an official journal of the European Respiratory Society · 2024
    Review
  4. Article
  5. Effect of elexacaftor/tezacaftor/ivacaftor on mucus and mucociliary clearance in cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2024
    Observational
  6. Article
  7. QuantitativeERJ open research · 2023
    Article
  8. Review
  9. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors at 7 institutions in 2 countries.

Scott H DonaldsonDepartment of Medicine and the Marsico Lung Institute, University of North Carolina at Chapel Hill, 2204 Marsico Hall, CB#7248, 125 Mason Farm Road, Chapel Hill, NC 27514, United States. Electronic address: Scott_Donaldson@med.unc.edu.
Beth L LaubeDepartment of Pediatrics, Johns Hopkins University, Baltimore, MD, United States.
Peter MogayzelDepartment of Pediatrics, Johns Hopkins University, Baltimore, MD, United States.
Timothy E CorcoranDepartment of Medicine, University of Pittsburgh, Pittsburgh, PA, United States.
Joseph M PilewskiDepartment of Medicine, University of Pittsburgh, Pittsburgh, PA, United States.
Agathe CeppeDepartment of Medicine and the Marsico Lung Institute, University of North Carolina at Chapel Hill, 2204 Marsico Hall, CB#7248, 125 Mason Farm Road, Chapel Hill, NC 27514, United States.
Jihong WuDepartment of Medicine and the Marsico Lung Institute, University of North Carolina at Chapel Hill, 2204 Marsico Hall, CB#7248, 125 Mason Farm Road, Chapel Hill, NC 27514, United States.
Pradeep G BhambhvaniDepartment of Medicine and the Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, AL, United States.
Felix RatjenDepartment of Pediatrics, Hospital for Sick Children, Toronto, Ontario, Canada.
Scott D SagelDepartment of Pediatrics, Children's Hospital Colorado and University of Colorado Anschutz Medical Campus, Aurora, CO, United States.
J P ClancyCystic Fibrosis Foundation, Bethesda, MD, United States.
Steven M RoweDepartment of Medicine and the Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, AL, United States.
William D BennettDepartment of Medicine and the Marsico Lung Institute, University of North Carolina at Chapel Hill, 2204 Marsico Hall, CB#7248, 125 Mason Farm Road, Chapel Hill, NC 27514, United States.
University of North Carolina at Chapel Hill · USJohns Hopkins University · USUniversity of Alabama at Birmingham · USUniversity of Pittsburgh · USChildren's Hospital Colorado · USCystic Fibrosis Foundation · USHospital for Sick Children · CA

Funding

Vector CoreP30DK065988 · NIDDK · UNIV OF NORTH CAROLINA CHAPEL HILL · PI Martina Gentzsch · 2004 to 2026
$26.5M
UAB CF Research and Translation Core CenterP30DK072482 · NIDDK · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI Brian R. Davis · 2007 to 2026
$23.0M
NIDDK NIH HHS P30 DK065988NIDDK NIH HHS P30 DK072482
6 · The paper itself

Abstract

CFTR function is required for normal mucociliary clearance (MCC) and cough-assisted clearance (CC). Lumacaftor-ivacaftor is approved for use in people with cystic fibrosis (CF) carrying two copies of F508del-CFTR. In this observational study performed at four study sites, we characterized the effect of lumacaftor-ivacaftor on mucociliary and cough clearance and related this to other clinical and research endpoints after one month of treatment. Twenty-five adolescents and adults were enrolled. No effect on whole lung MCC was observed, but CC was significantly increased. Sweat chloride improved by 18 mEq/L in this group, indicating a modest restoration of CFTR activity, but no demonstrable change in FEV

Indexed as

AdolescentAdultAminophenolsAminopyridinesBenzodioxolesChildChloride Channel AgonistsCohort StudiesCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsFemaleHumansLongitudinal StudiesMaleMiddle AgedAminophenolsAminopyridinesBenzodioxolesChloride Channel AgonistsCystic Fibrosis Transmembrane Conductance RegulatorDrug Combinationslumacaftor, ivacaftor drug combinationQuinolones

Identifiers

PMID34083123
PMCPMC8630086
OpenAlexW3165786465

What OpenQuestion holds

Textmetadata
LicenceTDM
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.