UCLA Medical Center, David Geffen School of Medicine at UCLA, Medicine, Los Angeles, California, United States ;
UCLA, Radiological Science, Los Angeles, California, United States ;
Am J Respir Crit Care Med. 2018 Mar 1;197(5):644-652. doi: 10.1164/rccm.201709-1845OC. Epub 2017 Nov 3.
To assess the reliability and the minimal clinically important differences (MCID) for FVC% predicted in the Scleroderma Lung Study I and II.
Using data from SLS I and II (N=300), we evaluated the test-retest reliability for FVC% predicted (FVC%; screening vs. baseline) using intra-class correlation (ICC). MCID estimates at 12 months were calculated in the pooled cohort (SLS-I and II) using 2 anchors: Transition Dyspnea Index (≥change of 1.5 units for improvement and worsening, respectively) and the SF-36 Health Transition question: "Compared to one year ago, how would you rate your health in general now?", where "somewhat better" or "somewhat worse" were defined as the MCID estimates. We next assessed the association of MCID estimates for improvement and worsening of FVC% with patient reported outcomes (PROs) and computer-assisted quantitation of extent of fibrosis (QLF) and of total ILD (QILD) on HRCT.
Reliability of FVC%, assessed at a mean of 34 days, was 0.93 for the pooled cohort. The MCID estimates for the pooled cohort at 12 months for FVC% improvement ranged from 3.0 % to 5.3% and for worsening from -3.0% to -3.3%. FVC% improvement by ≥MCID was associated with either statistically significant or numerical improvements in some PROs, QILD, and QLF, while FVC% worsening ≥MCID was associated with statistically significant or numerical worsening of PROs, QILD, and QLF.
FVC% has acceptable test-retest reliability, and we have provided the MCID estimates for FVC% in SSc-ILD based changes at 12 months from baseline in two clinical trials. Clinical trial registration available at www.clinicaltrials.gov, IDs NCT00004563 and NCT00883129.
评估 Scleroderma Lung Study I 和 II 中预测的 FVC%的可靠性和最小临床重要差异(MCID)。
使用来自 SLS I 和 II 的数据(N=300),我们使用组内相关系数(ICC)评估了预测的 FVC%(FVC%;筛查与基线)的测试-重测可靠性。在合并队列(SLS-I 和 II)中,使用 2 个锚点计算了 12 个月时的 MCID 估计值:过渡呼吸困难指数(改善分别为变化 1.5 个单位,恶化分别为变化 1.5 个单位)和 SF-36 健康过渡问题:“与一年前相比,您现在如何评价自己的整体健康状况?”,其中“稍有改善”或“稍有恶化”被定义为 MCID 估计值。接下来,我们评估了 FVC%改善和恶化的 MCID 估计值与患者报告的结局(PROs)以及 HRCT 上纤维化程度的计算机辅助定量(QLF)和总ILD(QILD)之间的相关性。
在平均 34 天的时间内,合并队列中 FVC%的可靠性为 0.93。在 12 个月时,合并队列的 FVC%改善的 MCID 估计值范围为 3.0%至 5.3%,恶化的 MCID 估计值范围为-3.0%至-3.3%。FVC%改善≥MCID 与某些 PROs、QILD 和 QLF 的统计学显著或数值改善相关,而 FVC%恶化≥MCID 与 PROs、QILD 和 QLF 的统计学显著或数值恶化相关。
FVC%具有可接受的测试-重测可靠性,我们已经为 SSc-ILD 基于基线的 12 个月内的变化提供了 FVC%的 MCID 估计值,这些变化来自两项临床试验。临床试验注册可在 www.clinicaltrials.gov 上获得,ID 为 NCT00004563 和 NCT00883129。