使用大型医疗保健数据库对未测量的混进行调整的方法的评估:关于诱导早产的药物的实证研究
Chi-Hong Duong1, Sylvie Escolano1, Romain Demailly1,2
1High-Dimensional Biostatistics for Drug Safety and Genomics, CESP, Université Paris-Saclay, UVSQ, Université Paris-Sud, Inserm, Villejuif, France.
Clinical and translational science
|November 12, 2025
概括
诸如G计算 (GC) 和目标最大概率估计 (TMLE) 等因果推断方法有效地减少了大型医疗保健数据库中的混. GC在识别真正正面的药物早产症关联方面表现出卓越的表现.
科学领域:
- 药学流行病学 药学流行病学
- 因果推理因果推理
- 健康 数据科学 数据科学
背景情况:
- 大规模的医疗保健数据库为药物流行病学研究提供了机会.
- 缓解未测量的混对于这些数据集中有效的因果推断至关重要.
- 将因果推理方法与现实世界中的高维数据进行比较是必不可少的.
研究的目的:
- 为了比较G计算 (GC),目标最大概率估计 (TMLE) 和倾向得分方法在减少测量和未测量的混方面的性能.
- 在一个高维,现实世界的医疗保健数据库上使用机器学习 LASSO 算法来评估这些方法.
- 评估这些方法识别真假阳性药物早产症关联的能力.
主要方法:
- 利用法国国家医疗保健索赔数据库 (SNDS) 获得2,172,702例怀孕 (怀孕≥22周,2011-2014).
- 采用适用于高维数据的G计算 (GC),目标最大概率估计 (TMLE) 和倾向得分方法.
- 对早产风险评估了42种负效和13种阳性参考药物,估计了几率比率和置信区间.
主要成果:
- 所有因果推理方法在减少错误阳性结果方面都超过了原始模型.
- 目标最大概率估计 (TMLE) 产生了最低的假阳性率 (45.2%).
- G计算 (GC) 实现了最高的真实阳性率 (92.3%),表现出良好的性能和易于实施.
结论:
- 因果推理方法对于利用大型医疗保健数据库是有价值的.
- 由于其性能和实施简单,G计算 (GC) 对药物流行病学研究具有特别的希望.
- 这些发现支持使用先进的因果推断技术来解决现实世界健康数据研究中的混问题.
更多相关视频
相关概念视频
Strategies for Assessing and Addressing Confounding
345
Confounding is a critical issue in epidemiological studies, often leading to misleading conclusions about associations between exposures and outcomes. It occurs when the relationship between the exposure and the outcome is mixed with the effects of other factors that influence the outcome. Given that, addressing confounding is of high importance for drawing accurate inferences in research.
Confounding can be addressed at both the design phase of a study and through analytical methods after data...
Confounding can be addressed at both the design phase of a study and through analytical methods after data...
345
Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
392
Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
392
Factors Affecting Drug Response: Overview
2.9K
When it comes to infants and young children, they are typically administered smaller doses of medication in comparison to adults. This is primarily because their organ functions still need to fully develop, meaning their bodies are not as efficient at metabolizing or eliminating drugs. Additionally, their blood-brain barrier is more permeable than in adults. As a result, high concentrations of drugs can easily penetrate the central nervous system (CNS), potentially leading to neurological...
2.9K
Pharmacovigilance
1.6K
Post-marketing surveillance is a critical component of pharmaceutical regulation, often uncovering unanticipated adverse drug reactions (ADRs) once a drug is widely used over an extended period.
This process, termed pharmacovigilance, aims to detect, evaluate, and minimize harmful effects related to medication use. The data collection for pharmacovigilance depends on spontaneous reporting systems, where healthcare professionals or patients voluntarily report suspected ADRs.
In some cases, there...
This process, termed pharmacovigilance, aims to detect, evaluate, and minimize harmful effects related to medication use. The data collection for pharmacovigilance depends on spontaneous reporting systems, where healthcare professionals or patients voluntarily report suspected ADRs.
In some cases, there...
1.6K
Regression Toward the Mean
6.8K
Regression toward the mean (“RTM”) is a phenomenon in which extremely high or low values—for example, and individual’s blood pressure at a particular moment—appear closer to a group’s average upon remeasuring. Although this statistical peculiarity is the result of random error and chance, it has been problematic across various medical, scientific, financial and psychological applications. In particular, RTM, if not taken into account, can interfere when...
6.8K
Confounding in Epidemiological Studies
565
Confounding in statistical epidemiology represents a pivotal challenge, referring to the distortion in the perceived relationship between an exposure and an outcome due to the presence of a third variable, known as a confounder. This variable is associated with both the exposure and the outcome but is not a direct link in their causal chain. Its presence can lead to erroneous interpretations of the exposure's effect, either exaggerating or underestimating the true association. This...
565


