实验性研究方法设计及实例.ppt

Thank You * * * * * * * * 数据分析 Excluding randomized participants or observed outcomes from analysis and sub-grouping on the basis of outcome or response variables (including non-adherence) can lead to biased results of unknown magnitude or direction. Including all randomized participants in the analysis, in the group they were assigned, is the intent-to-treat principle. The intent-to-treat analysis is viewed as the most valid approach (least susceptible to bias). 意向性分析(Intent-to-Treat Principle) Once randomized . . . . . . Always analyzed! 意向性分析定义 参与随机化分组的对象，无论其是否接受该组的治疗，最终应该纳入所分配的组中进行疗效的统计分析。 只对“资料完整”者进行分析就会破坏组间的均衡性。 观察性研究结果显示，退出试验病例的疗效谱表比完成治疗病例的疗效差和/或毒副反应多。 意向性分析的方法 结局指标的数据类型不同，分析方法不同： 计数资料：将治疗组和对照组中退出或失访的病例作为治疗失败处理（无效）或有毒副作用；或将治疗组退出或失访的病例作为无效或有毒副作用，而将对照退出或失访的病例作为有效或无毒副作用—“最差情况演示分析”。 计量资料：通常采用“推依”法，即对退出或失访病例的结局采用治疗前或最后一次随访测点的水平作为最后分析的测定值。 小样本的研究中，失访或退出病例数超过20%，结果的评价都可能不准确。 混杂因素的校正 While randomization eliminates bias, it does not guarantee comparable baseline characteristics of patients in different treatment groups in a particular trial. Baseline balance is not a requirement for obtaining valid variables. Imbalance will matter only if characteristic is related to patient outcome, ie., it is prognostic. When randomization leads to chance baseline imbalance, estimates of treatment effects will be biased when using unadjusted analysis. Validity and Reliability Validity Represents the degree to which a measurement represents a true value. Reliability A measure of the reproducibility of a result or observation. ie., How closely do repeated measurements on the same object agree? Validity and Reliability Errors can be caused by a lack of either validity or reliability Validity and reliability are related. If a measure is unreliable, it is not capable of producing valid results. Better reliability is necessary, but not sufficient for validity. Threats to Study Validity Bias Selection Information Confounding Regression to the Mean 这是临床上见到的一种现象，即一些极端的临床症状或体征