估计囊性纤维化中胃肠道症状的临床重要差异最小值 (MCID)
MinJae Lee1, Meghana Sathe2, Baha Moshiree3
1Peter O'Donnell Jr. School of Public Health, University of Texas Southwestern Medical Center, Dallas, TX.
概括
这项研究确定了囊性纤维化 (CF) 患者关键胃肠道症状措施的临床重要的最小差异 (MCID). 这些发现有助于评估影响CF患者的治疗方法.
科学领域:
- 胃肠病学 胃肠病学
- 肺部病理学 肺部病理学
- 儿科医学 儿科医学
背景情况:
- 确定最小临床重要差异 (MCID) 对于在研究中解释患者报告的结果指标 (PROM) 至关重要.
- 银河研究是一项前性观察性研究,专注于囊性纤维化 (CF) 患者的胃肠道 (GI) 症状.
研究的目的:
- 确定GALAXY研究中使用的三个关键PROM的MCID:患者对便秘症状的评估 (PAC-SYM),患者对上部胃肠道症状的评估 (PAGI-SYM) 和患者对便秘-生活质量的评估 (PAC-QOL).
- 使用基于分布的方法建立初始的MCID,以帮助评估针对CF (PwCF) 患者的GI症状的干预研究.
主要方法:
- 四百二名患有囊性纤维化 (PwCF) 的个人参与了调查,所有问卷都有可用的基线数据.
- 评估了PAC-SYM,PAGI-SYM和PAC-QOL的MCID,包括它们的子尺度.
- 采用了两种基于分布的方法:乘以标准偏差 (SD) 和平均值的标准误差 (SEM) 来估计MCID.
主要成果:
- 这两种基于分布的方法都在MCID估计中产生了可比的趋势,跨越子尺度和总分数.
- 总体而言,MCID对子尺度的估计通常高于所有三种测量的总分数.
- 总分数的具体MCID估计是:PAC-SYM (0.26 SD,0.14 SEM),PAGI-SYM (0.32 SD,0.15 SEM) 和PAC-QOL (0.27 SD,0.18 SEM). 总分数的具体MCID估计是:PAC-SYM (0.26 SD,0.14 SEM),PAGI-SYM (0.32 SD,0.15 SEM) 和PAC-QOL (0.27 SD,0.18 SEM) 总分数的估计是:PAC-SYM (0.26 SD,0.14 SEM),PAGI-SYM (0.32 SD,0.15 SEM) 和PAC-QOL (0.27 SD,0.18 SEM) 总分数的估计是:
结论:
- 本研究为PAC-SYM,PAGI-SYM和PAC-QOL提供了基于初始分布的MCID估计.
- 这些已建立的MCID可用于评估干预措施对PwCF的GI症状的影响.
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