Evidence map›Paper›PMID 38355350›Full record

Trial reportJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2024

The effect of discontinuing hypertonic saline or dornase alfa on mucociliary clearance in elexacaftor/tezacaftor/ivacaftor treated people with cystic fibrosis: The SIMPLIFY-MCC Study.

Scott H Donaldson, Timothy E Corcoran, Joseph M Pilewski, Beth L Laube, Peter Mogayzel, Agathe Ceppe, Jihong Wu, Kirby Zeman, Steven M Rowe, David P Nichols and 4 more

Open access · greenAbstract readRandomized Controlled TrialMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
7.1field-weighted citation impact, top 2% 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

10 citing papers in PubMed, 17 citations in OpenAlex.

  1. Trial
  2. Article
  3. Review
  4. Article
  5. Cystic Fibrosis Year in Review 2024.Pediatric pulmonology · 2025
    Review
  6. Observational
  7. Review
  8. Article
  9. Cystic fibrosis.Nature reviews. Disease primers · 2024
    Review
  10. Review
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

14 authors at 6 institutions in 1 country.

Scott H DonaldsonDepartment of Medicine and the Marsico Lung Institute, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA. Electronic address: Scott_Donaldson@med.unc.edu.
Timothy E CorcoranDepartment of Medicine, University of Pittsburgh, Pittsburgh, PA, USA.
Joseph M PilewskiDepartment of Medicine, University of Pittsburgh, Pittsburgh, PA, USA.
Beth L LaubeDepartment of Pediatrics, Johns Hopkins University, Baltimore, MD, USA.
Peter MogayzelDepartment of Pediatrics, Johns Hopkins University, Baltimore, MD, USA.
Agathe CeppeDepartment of Medicine and the Marsico Lung Institute, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Jihong WuDepartment of Medicine and the Marsico Lung Institute, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Kirby ZemanDepartment of Medicine and the Marsico Lung Institute, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Steven M RoweDepartment of Medicine and the Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, AL, USA.
David P NicholsSeattle Children's Research Institute, Department of Pediatrics, University of Washington, Seattle, WA, USA.
Alex H GiffordUniversity Hospitals Cleveland Medical Center and University Hospitals Rainbow Babies and Children's Hospital, Cleveland, OH, USA.
William D BennettDepartment of Medicine and the Marsico Lung Institute, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Nicole Mayer-HamblettSeattle Children's Research Institute, Department of Pediatrics, University of Washington, Seattle, WA, USA.
SIMPLIFY MCC Study teamsDepartment of Medicine and the Marsico Lung Institute, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
University of North Carolina at Chapel Hill · USJohns Hopkins University · USSeattle Children's Hospital · USUniversity of Pittsburgh · USRainbow Babies & Children's Hospital · USUniversity of Alabama at Birmingham · US

Funding

Transform Dissemination and Implementation Science in CTSA ProgramsUL1TR002319 · NCATS · UNIVERSITY OF WASHINGTON · PI John K. Amory · 2017 to 2026
$100.0M
Vector CoreP30DK065988 · NIDDK · UNIV OF NORTH CAROLINA CHAPEL HILL · PI Scott H Randell · 2004 to 2026
$26.5M
UAB CF Research and Translation Core CenterP30DK072482 · NIDDK · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI AMIT GAGGAR · 2007 to 2026
$23.0M
Translational Research Center to Expedite Novel Therapies in Cystic FibrosisP30DK089507 · NIDDK · SEATTLE CHILDREN'S HOSPITAL · PI Christopher Hooper Goss, Lucas R Hoffman · 2010 to 2026
$21.4M
NCATS NIH HHS UL1 TR002319NIDDK NIH HHS P30 DK065988NIDDK NIH HHS P30 DK072482NIDDK NIH HHS P30 DK089507
6 · The paper itself

Abstract

Many people with CF (pwCF) desire a reduction in inhaled treatment burden after initiation of elexacaftor/tezacaftor/ivacaftor. The randomized, open-label SIMPLIFY study showed that discontinuing hypertonic saline (HS) or dornase alfa (DA) was non-inferior to continuation of each treatment with respect to change in lung function over a 6-week period. In this SIMPLIFY substudy, we used gamma scintigraphy to determine whether discontinuation of either HS or DA was associated with deterioration in the rate of in vivo mucociliary clearance (MCC) in participants ≥12 years of age. While no significant differences in MCC endpoints were associated with HS discontinuation, significant improvement in whole and peripheral lung MCC was observed after discontinuing DA. These results suggest that pwCF on ETI with mild lung disease do not experience a subclinical deterioration in MCC that could later impact health outcomes after discontinuing HS, and in fact may benefit from improved MCC after stopping DA treatment.

Indexed as

AminophenolsBenzodioxolesCystic FibrosisDeoxyribonuclease IIndolesMucociliary ClearancePyrazolesQuinolonesAdolescentAdultChildChloride Channel AgonistsDrug CombinationsFemaleHumansMaleAminophenolsBenzodioxolesChloride Channel AgonistsDeoxyribonuclease Idornase alfaDrug CombinationselexacaftorIndolesivacaftorPyrazolesPyridinesPyrrolesPyrrolidinesQuinolonesRecombinant ProteinsSaline Solution, HypertonictezacaftorClinical trialCystic fibrosisDornase alfaHypertonic salineMucociliary clearanceWithdrawal

Identifiers

PMID38355350
PMCPMC11182703
OpenAlexW4391788073

What Socratic 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 Socratic graph.