Department of Infection, Immunity and Cardiovascular Disease, The University of Sheffield, Sheffield, UK
Research and Development, GlaxoSmithKline, Stevenage, Hertfordshire, UK.
Thorax. 2021 Oct;76(10):1032-1035. doi: 10.1136/thoraxjnl-2020-216078. Epub 2021 Feb 25.
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 (logNT-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 logNT-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.
在肺动脉高压（PAH）中，可重复且对变化敏感的终点对于临床实践和新疗法的试验很重要。在 42 名 PAH 患者中，使用组内相关系数评估了 MRI、6 分钟步行试验和基于对数基数 10 的 N 端脑利钠肽前体（logNT-proBNP）的测试-再测试可重复性，并用 Cohen's d 统计量评估了治疗效果大小。MRI、6 分钟步行试验和 logNT-proBNP 的组内相关系数均显示出极好的可重复性。MRI 衍生的右心室射血分数的治疗效果大小较大（Cohen's d 0.81），而 6 分钟步行试验（Cohen's d 0.22）和 logNT-proBNP（Cohen's d 0.20）的效果中等。这项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。试验注册号：NCT03841344。
