Minimal Clinically Important Differences in Pharmacological Trials
Minimal Clinically Important Differences in Pharmacological Trials
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DOI：
10.1164/rccm.201310-1863pp
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发表时间：
2014-02-01
影响因子：
24.7
通讯作者：
Wedzicha, Jadwiga A.
中科院分区：
文献类型：
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作者：
Jones, Paul W.;Beeh, Kai M.;Wedzicha, Jadwiga A.
最小临床重要差异（MCID）的概念已经确立。在这里，我们回顾了用于定义MCIDs的证据基础和方法，以及它们的优势和局限性。大多数慢性阻塞性肺疾病（COPD）的MCIDs是适用于患者群体的经验推导的估计值。经过验证的MCIDs可用于COPD的许多常用结果，包括肺功能（通过FEV1为100 ml），呼吸困难（改善>=过渡呼吸困难指数总分1个单位或加州大学圣地亚哥分校呼吸短促问卷5个单位），健康状况（圣乔治呼吸问卷总分减少4个单位）和运动能力（增加穿梭行走测试47.5 m）。耐力穿梭行走试验为45-85秒，恒载循环耐力试验为46-105秒)，但目前还没有证实的MCID对恶化的影响。在临床试验环境中，许多因素，包括研究持续时间、停药率、基线严重程度和霍桑效应，都可以影响所测量的治疗效果，并决定其是否达到MCID。我们还解决了近期临床试验提出的挑战，这些临床试验比较了积极的治疗方法，并建议MCIDs应用于确定受益的患者的额外比例，例如，当一种药物被另一种药物替代或当第二种药物添加到第一种药物中时。我们建议用“最小价值增量优势”来描述这个参数。
The concept of a minimal clinically important difference (MCID) is well established. Here, we review the evidence base and methods used to define MCIDs as well as their strengths and limitations. Most MCIDs in chronic obstructive pulmonary disease (COPD) are empirically derived estimates applying to populations of patients. Validated MCIDs are available for many commonly used outcomes in COPD, including lung function (100 ml for trough FEV1), dyspnea (improvement of >= 1 unit in the Transition Dyspnea Index total score or 5 units in the University of California, San Diego Shortness of Breath Questionnaire), health status (reduction of 4 units in the St George's Respiratory Questionnaire total score), and exercise capacity (47.5 m for the incremental shuttle walking test, 45-85 s for the endurance shuttle walking test, and 46-105 s for constant-load cycling endurance tests), but there is currently no validated MCID for exacerbations. In a clinical trial setting, many factors, including study duration, withdrawal rate, baseline severity, and Hawthorne effects, can influence the measured treatment effect and determine whether it reaches the MCID. We also address recent challenges presented by clinical trials that compare active treatments and suggest that MCIDs should be used to identify the additional proportion of patients who benefit, for example, when one drug is replaced by another or when a second drug is added to a first. We propose the term "minimum worthwhile incremental advantage" to describe this parameter.