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Repeatability and sensitivity to change of non-invasive end points in PAH: the RESPIRE study
Thorax ( IF 9.0 ) Pub Date : 2021-10-01 , DOI: 10.1136/thoraxjnl-2020-216078
Andrew J Swift 1 , Frederick Wilson 2 , Marcella Cogliano 3 , Lindsay Kendall 2 , Faisal Alandejani 3 , Samer Alabed 3 , Paul Hughes 3 , Yousef Shahin 3 , Laura Saunders 3 , Charlotte Oram 3 , David Capener 3 , Alex Rothman 3 , Pankaj Garg 3 , Christopher Johns 3 , Matthew Austin 3 , Alistair Macdonald 3 , Jo Pickworth 3 , Peter Hickey 3 , Robin Condliffe 4 , Anthony Cahn 2, 5 , Allan Lawrie 3 , Jim M Wild 3 , David G Kiely 3, 4
Affiliation  

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][1]. [1]: /lookup/external-ref?link_type=CLINTRIALGOV&access_num=NCT03841344&atom=%2Fthoraxjnl%2F76%2F10%2F1032.atom

中文翻译:

PAH 中非侵入性终点变化的可重复性和敏感性:RESPIRE 研究

可重复且对变化敏感的终点对于肺动脉高压 (PAH) 的临床实践和新疗法试验非常重要。在 42 名 PAH 患者中,使用 Cohen's d 统计量使用组内相关系数和治疗效果大小评估重测重复性。组内相关系数证明了 MRI、6 分钟步行测试和对数到碱基 10 N 端脑利钠肽前体 (log10NT-proBNP) 的出色重复性。MRI 衍生的右心室射血分数的治疗效果很大(Cohen's d 0.81),而 6 分钟步行试验(Cohen's d 0.22)和 log10NT-proBNP(Cohen's d 0.20)的效果大小是公平的。本研究支持进一步评估 MRI 作为临床评估和 PAH 治疗试验的非侵入性终点。试用注册号 [NCT03841344][1]。[1]:/lookup/external-ref?link_type=CLINTRIALGOV&access_num=NCT03841344&atom=%2Fthoraxjnl%2F76%2F10%2F1032.atom
更新日期:2021-09-17
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