在数据缺失的情况下,将次要结果信息可靠地纳入主要结果分析。

IF 1.6 3区 医学 Q3 HEALTH CARE SCIENCES & SERVICES
Statistical Methods in Medical Research Pub Date : 2024-07-01 Epub Date: 2024-05-20 DOI:10.1177/09622802241254195
Daxuan Deng, Vernon M Chinchilli, Hao Feng, Chixiang Chen, Ming Wang
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引用次数: 0

摘要

在临床和观察性研究中,在收集每个受试者的主要结果的同时,经常会收集次要结果,但它们在提高分析效率方面的潜力仍未得到充分利用。此外,在实践中经常会遇到数据缺失的情况,如果处理不当,会给估计结果带来偏差。本文提出了一种创新方法,通过整合缺失数据技术来增强基于经验似然法的信息借用方法,确保稳健的数据整合。我们引入了一种插件式反概率加权估计器来处理主要分析中的缺失,并证明了在温和条件下它与标准联合估计器的等效性。为了解决次要结果缺失可能造成的偏差,我们提出了一种统一映射策略,将不完整的次要结果归入一个统一的空间。大量的模拟突出了我们方法的有效性,显示了在涉及缺失数据和/或次级模型失当的各种情况下,我们的估计方法是一致、高效和稳健的。最后,我们将我们的建议应用于国家阿尔茨海默氏症协调中心的统一数据集,举例说明其实际应用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Robust integration of secondary outcomes information into primary outcome analysis in the presence of missing data.

In clinical and observational studies, secondary outcomes are frequently collected alongside the primary outcome for each subject, yet their potential to improve the analysis efficiency remains underutilized. Moreover, missing data, commonly encountered in practice, can introduce bias to estimates if not appropriately addressed. This article presents an innovative approach that enhances the empirical likelihood-based information borrowing method by integrating missing-data techniques, ensuring robust data integration. We introduce a plug-in inverse probability weighting estimator to handle missingness in the primary analysis, demonstrating its equivalence to the standard joint estimator under mild conditions. To address potential bias from missing secondary outcomes, we propose a uniform mapping strategy, imputing incomplete secondary outcomes into a unified space. Extensive simulations highlight the effectiveness of our method, showing consistent, efficient, and robust estimators under various scenarios involving missing data and/or misspecified secondary models. Finally, we apply our proposal to the Uniform Data Set from the National Alzheimer's Coordinating Center, exemplifying its practical application.

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来源期刊
Statistical Methods in Medical Research
Statistical Methods in Medical Research 医学-数学与计算生物学
CiteScore
4.10
自引率
4.30%
发文量
127
审稿时长
>12 weeks
期刊介绍: Statistical Methods in Medical Research is a peer reviewed scholarly journal and is the leading vehicle for articles in all the main areas of medical statistics and an essential reference for all medical statisticians. This unique journal is devoted solely to statistics and medicine and aims to keep professionals abreast of the many powerful statistical techniques now available to the medical profession. This journal is a member of the Committee on Publication Ethics (COPE)
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