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

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 ORCID:, Johns, Christopher, Austin, Matthew, Macdonald, Alistair, Pickworth, Jo, Hickey, Peter, Condliffe, Robin, Cahn, Anthony, Lawrie, Allan, Wild, Jim M and Kiely, David G (2021) Repeatability and sensitivity to change of non-invasive end points in PAH: the RESPIRE study. Thorax, 76 (10). pp. 1032-1035. ISSN 0040-6376

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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.

Item Type: Article
Additional Information: Funding Information: Contributors AJS, FW, JMW, AC, LK, DGK conceived the idea for the study. MC, MA, DGK, RC, AJS supported patient recruitment. AJS, JMW, DGK, AM, PH, LS devised the MRI protocol. AJC, CO, PH, LS analysed the MRI studies. FA, AM, CJ, PH, PG performed data quality control checks. MA performed the walk tests and FA, JP, AL performed the lab analyses. MC, LK, SA, AR, PG, AJS, YS, FS, PH, LS supported the data collation and analysis. Statistical analysis was performed by MC, SA, AJS, FA, LS, LK. All authors contributed to the drafting of the manuscript. All authors approved the final version of the manuscript. Funding This study was funded by GlaxoSmithKline (contract number COL100041816) and Wellcome Trust (205188/Z/16/Z and 206632/Z/17/Z). PH is funded by a research grant from GlaxoSmithKline (BIDS3000032592). Publisher Copyright: © Author(s) (or their employer(s)) 2021.
Uncontrolled Keywords: ct mri etc,primary pulmonary hypertension,pulmonary and respiratory medicine ,/dk/atira/pure/subjectarea/asjc/2700/2740
Faculty \ School: Faculty of Medicine and Health Sciences > Norwich Medical School
UEA Research Groups: Faculty of Medicine and Health Sciences > Research Centres > Metabolic Health
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Depositing User: LivePure Connector
Date Deposited: 13 Nov 2021 01:52
Last Modified: 19 Oct 2023 03:08
DOI: 10.1136/thoraxjnl-2020-216078

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