使用阴性对照人群来评估未测量的混杂因素和直接影响。

IF 4.7 2区 医学 Q1 PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH Epidemiology Pub Date : 2024-05-01 Epub Date: 2024-03-07 DOI:10.1097/EDE.0000000000001724
Marco Piccininni, Mats Julius Stensrud
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引用次数: 0

摘要

有时,治疗效果在人群中的某个子群体中是不存在的。例如,青霉素对感染耐药金黄色葡萄球菌的人的严重症状没有效果,可待因对 CYP2D6 酶中某些多态性的人的疼痛没有效果。治疗无效的亚组通常称为阴性对照组或安慰剂组。在不同学科中,它们被用来检测偏倚。在此,我们提出了正式的标准,证明使用阴性对照组来排除未测量的混杂因素和机理(直接)因果效应是合理的。我们进一步论证,满足我们的正式条件的阴性对照人群可用于多种场合,从传染病的临床研究到公共卫生干预的流行病学研究。阴性对照人群还可用于排除非盲随机实验中的安慰剂效应。作为一项案例研究,我们利用一项大型试验的数据评估了移动卒中单元派遣对疑似卒中患者出院时功能预后的影响。我们的分析支持移动卒中单元改善这些患者功能预后的假设。
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Using Negative Control Populations to Assess Unmeasured Confounding and Direct Effects.

Sometimes treatment effects are absent in a subgroup of the population. For example, penicillin has no effect on severe symptoms in individuals infected by resistant Staphylococcus aureus , and codeine has no effect on pain in individuals with certain polymorphisms in the CYP2D6 enzyme. Subgroups where a treatment is ineffective are often called negative control populations or placebo groups. They are leveraged to detect bias in different disciplines. Here we present formal criteria that justify the use of negative control populations to rule out unmeasured confounding and mechanistic (direct) causal effects. We further argue that negative control populations, satisfying our formal conditions, are available in many settings, spanning from clinical studies of infectious diseases to epidemiologic studies of public health interventions. Negative control populations can also be used to rule out placebo effects in unblinded randomized experiments. As a case study, we evaluate the effect of mobile stroke unit dispatches on functional outcomes at discharge in individuals with suspected stroke, using data from a large trial. Our analysis supports the hypothesis that mobile stroke units improve functional outcomes in these individuals.

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来源期刊
Epidemiology
Epidemiology 医学-公共卫生、环境卫生与职业卫生
CiteScore
6.70
自引率
3.70%
发文量
177
审稿时长
6-12 weeks
期刊介绍: Epidemiology publishes original research from all fields of epidemiology. The journal also welcomes review articles and meta-analyses, novel hypotheses, descriptions and applications of new methods, and discussions of research theory or public health policy. We give special consideration to papers from developing countries.
期刊最新文献
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