Evidence map›Paper›PMID 28215711›Full record

ReviewJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2016

Biomarkers for cystic fibrosis drug development.

Marianne S Muhlebach, J P Clancy, Sonya L Heltshe, Assem Ziady, Tom Kelley, Frank Accurso, Joseph Pilewski, Nicole Mayer-Hamblett, Elizabeth Joseloff, Scott D Sagel

Abstract readReview
In one paragraph

Review in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers.

0numbers the graph read from it
0cells of the map it votes in
35citing papers in PubMed
–field-weighted citation impact
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

35 citing papers in PubMed.

  1. Trial
  2. Trial
  3. Review
  4. Observational
  5. Article
  6. Article
  7. Article
  8. Review
  9. Ceftobiprole in cystic fibrosis: a case series.JAC-antimicrobial resistance · 2025
    Article
  10. Article
  11. Review
  12. Measuring the impact of CFTR modulation on sweat chloride in cystic fibrosis: Rationale and design of the CHEC-SC study.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2021
    Observational
  13. Article
  14. Article
  15. Article
  16. DNA Methylation atGenes · 2021
    Article
  17. Safety and efficacy of the cystic fibrosis transmembrane conductance regulator potentiator icenticaftor (QBW251).Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2021
    Article
  18. Calprotectin in Lung Diseases.International journal of molecular sciences · 2021
    Review
  19. Article
  20. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors.

Marianne S MuhlebachDivision of Pulmonology, Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, NC, United States. Electronic address: marianne_muhlebach@med.unc.edu.
J P ClancyDivision of Pulmonary Medicine, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, United States.
Sonya L HeltsheDivision of Pulmonary, Department of Pediatrics, University of Washington, Seattle, WA, United States.
Assem ZiadyDivision of Pulmonary Medicine, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, United States.
Tom KelleyDepartment of Biostatistics, University of Washington, Seattle, WA, United States.
Frank AccursoDepartment of Pediatrics, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO, United States.
Joseph PilewskiDivision of Pulmonary, Allergy, and Critical Care Medicine, Department of Medicine, University of Pittsburgh, Pittsburgh, PA, United States.
Nicole Mayer-HamblettDivision of Pulmonary, Department of Pediatrics, University of Washington, Seattle, WA, United States; Division of Pulmonology, Department of Pediatrics, Case Western Reserve University, Cleveland, OH, United States.
Elizabeth JoseloffCystic Fibrosis Foundation, Bethesda, MD, United States.
Scott D SagelDepartment of Pediatrics, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO, United States.

Funding

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
Translational StudiesP30DK072506 · NIDDK · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI PILEWSKI, JOSEPH MARK · 2005 to 2017
$12.5M
MR predictors of infection, inflammation, and structural lung damage in CFR01HL116226 · NHLBI · CINCINNATI CHILDRENS HOSP MED CTR · PI CLANCY, JOHN PAUL · 2012 to 2015
$2.1M
Mucus and hypoxia in heterogeneous and progressive CF lung diseaseR01HL116228 · NHLBI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI ESTHER, CHARLES RICHARD, MUHLEBACH, MARIANNE S · 2012 to 2015
$2.1M
NHLBI NIH HHS R01 HL116226NHLBI NIH HHS R01 HL116228NIDDK NIH HHS P30 DK072506NIDDK NIH HHS P30 DK089507
6 · The paper itself

Abstract

purposeTo provide a review of the status of biomarkers in cystic fibrosis drug development, including regulatory definitions and considerations, a summary of biomarkers in current use with supportive data, current gaps, and future needs.

methodsBiomarkers are considered across several areas of CF drug development, including cystic fibrosis transmembrane conductance regulator modulation, infection, and inflammation.

resultsSweat chloride, nasal potential difference, and intestinal current measurements have been standardized and examined in the context of multicenter trials to quantify CFTR function. Detection and quantification of pathogenic bacteria in CF respiratory cultures (e.g.: Pseudomonas aeruginosa) are commonly used in early phase antimicrobial clinical trials, and to monitor safety of therapeutic interventions. Sputum (e.g.: neutrophil elastase, myeloperoxidase, calprotectin) and blood biomarkers (e.g.: C reactive protein, calprotectin, serum amyloid A) have had variable success in detecting response to inflammatory treatments.

conclusionsBiomarkers are used throughout the drug development process in CF, and many have been used in early phase clinical trials to provide proof of concept, detect drug bioactivity, and inform dosing for later-phase studies. Advances in the precision of current biomarkers, and the identification of new biomarkers with 'omics-based technologies, are needed to accelerate CF drug development.

Indexed as

Cystic FibrosisBiomarkersCystic Fibrosis Transmembrane Conductance RegulatorDrug DiscoveryHumansBiomarkersCFTR protein, humanCystic Fibrosis Transmembrane Conductance RegulatorAdvanced technologiesBiomarkerElectrophysiologyInfectionInflammationMicrobiome

Identifiers

PMID28215711
PMCPMC5321565

What OpenQuestion holds

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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.