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

Abstract

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 (log10NT-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 log10NT-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.

Metadata

Item Type:	Article
Authors/Creators:	Swift, A.J. https://orcid.org/0000-0002-8772-409X Wilson, F. Cogliano, M. Kendall, L. Alandejani, F. Alabed, S. https://orcid.org/0000-0002-9960-7587 Hughes, P. https://orcid.org/0000-0002-7979-5840 Shahin, Y. https://orcid.org/0000-0001-7425-7798 Saunders, L. https://orcid.org/0000-0002-1080-9861 Oram, C. Capener, D. Rothman, A. https://orcid.org/0000-0002-7847-4500 Garg, P. Johns, C. Austin, M. Macdonald, A. Pickworth, J. https://orcid.org/0000-0002-7199-364X Hickey, P. Condliffe, R. Cahn, A. Lawrie, A. https://orcid.org/0000-0003-4192-9505 Wild, J.M. https://orcid.org/0000-0002-7246-8660 Kiely, D.G. https://orcid.org/0000-0003-0184-6502
Copyright, Publisher and Additional Information:	© Author(s) (or their employer(s)) 2021. Re-use permitted under CC BY. Published by BMJ. https://creativecommons.org/licenses/by/4.0/ This is an open access article distributed in accordance with the Creative Commons Attribution 4.0 Unported (CC BY 4.0) license, which permits others to copy, redistribute, remix, transform and build upon this work for any purpose, provided the original work is properly cited, a link to the licence is given, and indication of whether changes were made. See: https://creativecommons.org/licenses/by/4.0/.
Dates:	Published: October 2021 Published (online): 25 February 2021 Accepted: 8 February 2021
Institution:	The University of Sheffield
Academic Units:	The University of Sheffield > Sheffield Teaching Hospitals
Funding Information:	Funder Grant number WELLCOME TRUST (THE) 206632/Z/17/Z WELLCOME TRUST (THE) 205188/Z/16/Z
Depositing User:	Symplectic Sheffield
Date Deposited:	24 Jan 2022 11:38
Last Modified:	24 Jan 2022 11:38
Status:	Published
Publisher:	BMJ
Refereed:	Yes
Identification Number:	10.1136/thoraxjnl-2020-216078
Open Archives Initiative ID (OAI ID):	oai:eprints.whiterose.ac.uk:182837

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

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