Evaluating preference effects in partially unblinded, randomized clinical trials
Evaluating preference effects in partially unblinded, randomized clinical trials
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DOI：
10.1016/s0895-4356(02)00598-x
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发表时间：
2003-02-01
影响因子：
7.2
通讯作者：
Halpern, SD
中科院分区：
文献类型：
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作者：
Halpern, SD
随机临床试验（RCT）对干预措施的特异性（即，先天药理学或生理学）作用依赖于至少两个假设。首先，假设其他预后效应（如疾病严重程度或治疗的心理效应）在治疗组之间均匀分布，其次，假设这些其他效应不与干预的具体效应相互作用。这篇文章展示了患者对治疗的偏好如何与干预措施的具体效果相互作用，从而使试验结果产生偏差。早期关于偏好效应如何在非盲法试验中产生偏倚的数学描述被扩展到（可能）盲法RCT的情况。在部分揭盲的安慰剂对照与活性对照药物试验中，考虑了偏好效应可能导致偏倚的程度。最后，提供了在部分揭盲随机对照试验中估计偏好效应的方法。结论是，研究者可以使用这些方法来量化潜在的偏好效应在部分揭盲的随机对照试验，从而更准确地估计治疗的具体效果。(C)2003年爱思唯尔科学公司版权所有
The ability of randomized, clinical trials (RCTs) to produce unbiased estimates of an intervention's specific (i.e., innate pharmacologic or physiologic) effect rests on at least two assumptions. First, other prognostic effects (such as disease severity or the psychologic effects of treatment) are assumed to be evenly distributed across treatment groups, and second, it is assumed that these other effects do not interact with the intervention's specific effect. This essay shows how the effect of patients' preferences for treatments may interact with an intervention's specific effect to bias the trial's results. Earlier mathematical descriptions of how preference effects may produce bias in nonblinded trials are extended to the case of (presumably) blinded RCTs. The extent to which preference effects may confer bias in partially unblinded placebo-controlled vs. active-controlled drug trials are considered. Finally, methods for estimating preference effects in partially unblinded RCTs are provided. It is concluded that investigators could use these methods to quantify potential preference effects in partially unblinded RCTs, and thereby more accurately estimate the specific effects of treatments. (C) 2003 Elsevier Science Inc. All-right reserved.