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

Andrew J. Swift, Frederick Wilson, Marcella Cogliano, Lindsay Kendall, Faisal Alandejani, Samer Alabed, Paul Hughes, Yousef Shahin, Laura Saunders, Charlotte Oram, David Capener, Alex Rothman, Pankaj Garg, Christopher Johns, Matthew Austin, Alistair Macdonald, Jo Pickworth, Peter Hickey, Robin Condliffe, Anthony CahnAllan Lawrie, Jim M Wild, David G Kiely

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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 (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.

Original languageEnglish
Pages (from-to)1032-1035
Number of pages4
JournalThorax
Volume76
Issue number10
Early online date25 Feb 2021
DOIs
Publication statusPublished - 17 Sep 2021

Keywords

  • imaging/CT MRI etc
  • primary pulmonary hypertension

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