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Repeatability and sensitivity to change of non-invasive end points in PAH: the RESPIRE study

Title: Repeatability and sensitivity to change of non-invasive end points in PAH: the RESPIRE study
Authors: Swift, Andrew J; Wilson, Frederick; Cogliano, Marcella; Kendall, Lindsay; Alandejani, Faisal; Alabed, Samer; Hughes, Paul; Shahin, Yousef; Saunders, Laura; Oram, Charlotte; Capener, David; Rothman, Alex; Garg, Pankaj; Johns, Christopher; Austin, Matthew; Macdonald, Alistair; Pickworth, Jo; Hickey, Peter; Condliffe, Robin; Cahn, Anthony; Lawrie, Allan; Wild, Jim M; Kiely, David G
Contributors: Wellcome Trust; GlaxoSmithKline
Source: Thorax ; volume 76, issue 10, page 1032-1035 ; ISSN 0040-6376 1468-3296
Publisher Information: BMJ
Publication Year: 2021
Description: End points that are repeatable and sensitive to change are important in pulmonary arterial hypertension (PAH) for clinical practice and trials of new therapies. In 42 patients with PAH, test–retest repeatability was assessed using the intraclass correlation coefficient and treatment effect size using Cohen’s d statistic. Intraclass correlation coefficients demonstrated excellent repeatability for MRI, 6 min walk test and log to base 10 N-terminal pro-brain natriuretic peptide (log 10 NT-proBNP). The treatment effect size for MRI-derived right ventricular ejection fraction was large (Cohen’s d 0.81), whereas the effect size for the 6 min walk test (Cohen’s d 0.22) and log 10 NT-proBNP (Cohen’s d 0.20) were fair. This study supports further evaluation of MRI as a non-invasive end point for clinical assessment and PAH therapy trials. Trial registration number NCT03841344 .
Document Type: article in journal/newspaper
Language: English
DOI: 10.1136/thoraxjnl-2020-216078
Availability: https://doi.org/10.1136/thoraxjnl-2020-216078; https://syndication.highwire.org/content/doi/10.1136/thoraxjnl-2020-216078
Rights: https://creativecommons.org/licenses/by/4.0/
Accession Number: edsbas.8B99E42F
Database: BASE