Evidence map›Paper›PMID 33465316›Full record

ArticleAnnals of the American Thoracic Society2021

Evaluating the Impact of Stopping Chronic Therapies after Modulator Drug Therapy in Cystic Fibrosis: The SIMPLIFY Clinical Trial Study Design.

Nicole Mayer-Hamblett, David P Nichols, Katherine Odem-Davis, Kristin A Riekert, Greg S Sawicki, Scott H Donaldson, Felix Ratjen, Michael W Konstan, Noah Simon, Daniel B Rosenbluth and 5 more

Open access · hybridAbstract readMulticenter Study
In one paragraph

Article 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 24 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed, 2 pooled it
7.5field-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

24 citing papers in PubMed, 2 syntheses or guidelines pooled it, 57 citations in OpenAlex.

  1. Nebulised hypertonic saline for cystic fibrosis.The Cochrane database of systematic reviews · 2023
    Pooled it
  2. Dornase alfa for cystic fibrosis.The Cochrane database of systematic reviews · 2021
    Pooled it
  3. Trial
  4. Trial
  5. Trial
  6. Trial
  7. Effects of CFTR Modulators onInfectious disease reports · 2025
    Review
  8. Article
  9. Article
  10. Article
  11. Article
  12. Article
  13. Review
  14. Review
  15. Role of inhaled antibiotics in the era of highly effective CFTR modulators.European respiratory review : an official journal of the European Respiratory Society · 2023
    Review
  16. Review
  17. Article
  18. Article
  19. Article
  20. Observational
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

15 authors at 10 institutions in 2 countries.

Nicole Mayer-HamblettCystic Fibrosis Therapeutics Development Network Coordinating Center, Seattle Children's Hospital, Seattle, Washington.ORCID 0000-0001-5538-9197
David P NicholsCystic Fibrosis Therapeutics Development Network Coordinating Center, Seattle Children's Hospital, Seattle, Washington.ORCID 0000-0001-8611-7960
Katherine Odem-DavisCystic Fibrosis Therapeutics Development Network Coordinating Center, Seattle Children's Hospital, Seattle, Washington.
Kristin A RiekertDivision of Pulmonary and Critical Care Medicine, Department of Medicine, School of Medicine, Johns Hopkins University, Baltimore, Maryland.
Greg S SawickiDivision of General Pediatrics and Respiratory Diseases, Boston Children's Hospital, Boston, Massachusetts.
Scott H DonaldsonDivision of Pulmonary and Critical Care Medicine and.
Felix RatjenDivision of Respiratory Translational Medicine Program, SickKids Research Institute, Medicine, The Hospital for Sick Children, Toronto, Ontario, Canada.
Michael W KonstanDepartment of Pediatrics, Rainbow Babies and Children's Hospital and School of Medicine, Case Western Reserve University, Cleveland, Ohio.
Noah SimonCystic Fibrosis Therapeutics Development Network Coordinating Center, Seattle Children's Hospital, Seattle, Washington.
Daniel B RosenbluthDivision of Pulmonary and Critical Care Medicine, Department of Medicine, School of Medicine, Washington University, St. Louis, Missouri.
George Retsch-BogartDivision of Pediatric Pulmonology, University of North Carolina, Chapel Hill, Chapel Hill, North Carolina.
John P ClancyDepartment of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Jill M VanDalfsenCystic Fibrosis Therapeutics Development Network Coordinating Center, Seattle Children's Hospital, Seattle, Washington.
Rachael BuckinghamCystic Fibrosis Therapeutics Development Network Coordinating Center, Seattle Children's Hospital, Seattle, Washington.
Alex H GiffordPulmonary and Critical Care Medicine, Dartmouth-Hitchcock Medical Center, Lebanon, New Hampshire; and.ORCID 0000-0002-6404-3793
Seattle Children's Hospital · USBoston Children's Hospital · USCincinnati Children's Hospital Medical Center · USDartmouth Institute for Health Policy and Clinical Practice · USJohns Hopkins University · USPulmonary and Critical Care Associates · USRainbow Babies & Children's Hospital · USUniversity of North Carolina at Chapel Hill · USUniversity of Toronto · CAWashington University in St. Louis · US

Funding

Transform Dissemination and Implementation Science in CTSA ProgramsUL1TR002319 · NCATS · UNIVERSITY OF WASHINGTON · PI John K. Amory · 2017 to 2026
$100.0M
Clinical and Translational Science Collaborative of ClevelandUL1TR002548 · NCATS · CASE WESTERN RESERVE UNIVERSITY · PI MCCOMSEY, GRACE A · 2018 to 2022
$35.5M
Translational Research Center to Expedite Novel Therapies in Cystic FibrosisP30DK089507 · NIDDK · SEATTLE CHILDREN'S HOSPITAL · PI Christopher Hooper Goss · 2010 to 2026
$21.4M
Translational Research CoreP30DK117469 · NIDDK · DARTMOUTH COLLEGE · PI DEAN R MADDEN · 2018 to 2026
$13.9M
Project 3 - Personalized cystic fibrosis treatment strategies with synergistic therapies Identified in vitro.P01HL128192 · NHLBI · INDIANA UNIVERSITY INDIANAPOLIS · PI GASTON, BENJAMIN · 2016 to 2020
$12.2M
A prospective trial of oral azithromycin when added to inhaled tobramycin in CFR01HL124053 · NHLBI · SEATTLE CHILDREN'S HOSPITAL · PI NICHOLS, DAVID PAUL · 2015 to 2019
$2.8M
NCATS NIH HHS UL1 TR002319NCATS NIH HHS UL1 TR002548NHLBI NIH HHS P01 HL128192NHLBI NIH HHS R01 HL124053NIDDK NIH HHS P30 DK089507NIDDK NIH HHS P30 DK117469
6 · The paper itself

Abstract

The care for individuals with cystic fibrosis (CF) with at least one F508del mutation will greatly change as a result of the unparalleled clinical benefits observed with the new triple-combination CFTR (CF transmembrane regulator)-modulator therapy elexacaftor/tezacaftor/ivacaftor (ETI). Incorporating ETI into the standard of care creates new motivation and opportunity to consider reductions in overall treatment burden and evaluate whether other chronic medications can now be safely discontinued without loss of clinical benefit. SIMPLIFY is a master protocol poised to test the impact of discontinuing versus continuing two commonly used chronic therapies in people with CF who are at least 12 years of age or older and stable on ETI therapy. The protocol is composed of two concurrent randomized controlled trials designed to evaluate the independent short-term effects of discontinuing hypertonic saline or dornase alfa, enabling individuals on both therapies to participate in one or both trials. The primary objective for each trial is to determine whether discontinuing treatment is

Indexed as

Cystic FibrosisQuinolonesAminophenolsBenzodioxolesCystic Fibrosis Transmembrane Conductance RegulatorHumansAminophenolsBenzodioxolesCystic Fibrosis Transmembrane Conductance RegulatorQuinolonesCFTR modulatorsnoninferiority trialtreatment burden

Identifiers

PMID33465316
PMCPMC8513667
OpenAlexW3125432050

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.