ReviewPediatric pulmonology2015
Novel outcome measures for clinical trials in cystic fibrosis.
Review in Pediatric pulmonology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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
14 citing papers in PubMed, 42 citations in OpenAlex.
- Laboratory Tools to Predict CFTR Modulator Therapy Effectiveness and to Monitor Disease Severity in Cystic Fibrosis.Journal of personalized medicine · 2024Review
- Assays of CFTR Function In Vitro, Ex Vivo and In Vivo.International journal of molecular sciences · 2022Review
- Defect distribution index: A novel metric for functional lung MRI in cystic fibrosis.Magnetic resonance in medicine · 2021Article
- Methods Used to Evaluate the Immediate Effects of Airway Clearance Techniques in Adults with Cystic Fibrosis: A Systematic Review and Meta-Analysis.Journal of clinical medicine · 2021Review
- DNA Methylation atGenes · 2021Article
- Predictive value of the modified Bhalla score for assessment of pulmonary exacerbations in adults with cystic fibrosis.European radiology · 2021Article
- Designing Clinical Trials for Anti-Inflammatory Therapies in Cystic Fibrosis.Frontiers in pharmacology · 2020Review
- Article
- Semi-automatic Methods for Airway and Adjacent Vessel Measurement in Bronchiectasis Patterns in Lung HRCT Images of Cystic Fibrosis Patients.Journal of digital imaging · 2018Review
- Thirty Years of Sweat Chloride Testing at One Referral Center.Frontiers in pediatrics · 2017Article
- Ultrashort Echo-Time Magnetic Resonance Imaging Is a Sensitive Method for the Evaluation of Early Cystic Fibrosis Lung Disease.Annals of the American Thoracic Society · 2016Observational
- Quantification of heterogeneity in lung disease with image-based pulmonary function testing.Scientific reports · 2016Article
- Cardiorespiratory and sensory responses to exercise in adults with mild cystic fibrosis.Journal of applied physiology (Bethesda, Md. : 1985) · 2015Article
- The short-term effects of ORKAMBI (lumacaftor/ivacaftor) on regional and distal lung structures using functional respiratory imaging.Therapeutic advances in respiratory diseaseArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 10 institutions in 5 countries.
Funding
Abstract
Cystic fibrosis (CF) is a common inherited condition caused by mutations in the gene encoding the CF transmembrane regulator protein. With increased understanding of the molecular mechanisms underlying CF and the development of new therapies there comes the need to develop new outcome measures to assess the disease, its progression and response to treatment. As there are limitations to the current endpoints accepted for regulatory purposes, a workshop to discuss novel endpoints for clinical trials in CF was held in Anaheim, California in November 2011. The pros and cons of novel outcome measures with potential utility for evaluation of novel treatments in CF were critically evaluated. The highlights of the 2011 workshop and subsequent advances in technologies and techniques that could be used to inform the development of clinical trial endpoints are summarized in this review. Pediatr Pulmonol. © 2014 The Authors. Pediatric Pulmonology published by Wiley Periodicals, Inc.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
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.