Approaches for estimating minimal clinically important differences in systemic lupus erythematosus.
Approaches for estimating minimal clinically important differences in systemic lupus erythematosus.
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DOI：
10.1186/s13075-015-0658-6
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发表时间：
2015-06-03
影响因子：
4.9
通讯作者：
Aviña-Zubieta JA
中科院分区：
文献类型：
--
作者：
Rai SK;Yazdany J;Fortin PR;Aviña-Zubieta JA
最小临床重要差异（MCID）是用于确定医疗干预是否改善患者感知结果的重要概念。在1989年引入这一概念之前，研究主要集中在统计意义上。由于系统性红斑狼疮（SLE）的最新临床试验未能显示出显着的效果，确定一个临床相关的阈值的结果评分（即MCID）的现有工具可能是至关重要的进行和解释有意义的临床试验，以及促进建立治疗建议的患者。为此，确定MCID的方法可以分为两个定义明确的类别：基于分布的方法和基于锚点的方法。基于分布的方法基于所获得的样本的统计特性。基于分布的方法中有各种方法，包括测量的标准误差、标准差、效应量、最小可检测变化、可靠变化指数和标准化响应均值。基于锚点的方法将患者报告结局的变化与作为锚的第二个外部变化指标（即，更清楚理解的指标，如全局评估）进行比较。最后，德尔菲技术可以作为一种辅助手段来定义临床上重要的差异。尽管文献中报道了大量的方法，但在SLE的背景下，MCID估计的工作很少。由于MCID可以帮助确定给定治疗对患者的影响，并为临床研究中的统计推断增加意义，我们认为应该重新关注这一领域。在这里，我们提供了一个最新的MCID在临床研究中的使用，审查在这方面的SLE的一些工作，并提出了未来的研究议程。
A minimal clinically important difference (MCID) is an important concept used to determine whether a medical intervention improves perceived outcomes in patients. Prior to the introduction of the concept in 1989, studies focused primarily on statistical significance. As most recent clinical trials in systemic lupus erythematosus (SLE) have failed to show significant effects, determining a clinically relevant threshold for outcome scores (that is, the MCID) of existing instruments may be critical for conducting and interpreting meaningful clinical trials as well as for facilitating the establishment of treatment recommendations for patients. To that effect, methods to determine the MCID can be divided into two well-defined categories: distribution-based and anchor-based approaches. Distribution-based approaches are based on statistical characteristics of the obtained samples. There are various methods within the distribution-based approach, including the standard error of measurement, the standard deviation, the effect size, the minimal detectable change, the reliable change index, and the standardized response mean. Anchor-based approaches compare the change in a patient-reported outcome to a second, external measure of change (that is, one that is more clearly understood, such as a global assessment), which serves as the anchor. Finally, the Delphi technique can be applied as an adjunct to defining a clinically important difference. Despite an abundance of methods reported in the literature, little work in MCID estimation has been done in the context of SLE. As the MCID can help determine the effect of a given therapy on a patient and add meaning to statistical inferences made in clinical research, we believe there ought to be renewed focus on this area. Here, we provide an update on the use of MCIDs in clinical research, review some of the work done in this area in SLE, and propose an agenda for future research.
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影响因子：
3
作者：
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通讯作者：
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