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纵向模型中缺失数据的多重填补与有效病例分析之间的偏差和效率比较

Bias and Efficiency Comparison between Multiple Imputation and Available-Case Analysis for Missing Data in Longitudinal Models.

作者信息

Zhang Panpan, Xie Sharon X

机构信息

Department of Biostatistics, Epidemiology and Informatics, University of Pennsylvania, 423 Gaurdian Drive, Philadelphia, 19104, PA, U.S.A.

出版信息

Stat Biosci. 2025 Jun 12. doi: 10.1007/s12561-025-09493-6.

DOI:10.1007/s12561-025-09493-6
PMID:40821499
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12356228/
Abstract

In this paper, we compare the performance of available-case analysis (ACA) and several multiple imputation (MI) approaches for handling missing data problems in longitudinal analysis through estimation bias and relative efficiency. When the missingness of covariates depends on observed responses, ACA produces estimation bias, but it is preferred when there are only missing values in longitudinal responses. Multilevel MI methods are not always a solution to longitudinal data analysis. Single-level MI methods, like fully conditional specification (FCS), provide unbiased estimates under a variety of missing data scenarios, and improve efficiency gain in certain scenarios. The general assumption of missing data mechanism is missing at random (MAR). We carry out a systematic synthetic data analysis where missing data exist in longitudinal outcomes or/and covariates under different kinds of missing data generation procedures. The analysis model is a linear mixed-effects model. For each of the missing data scenarios, we give our recommendation (between ACA and a specific MI method) based on theoretical justifications and extensive simulations. In addition, a longitudinal neurodegenerative disease dataset is used as a real case study.

摘要

在本文中,我们通过估计偏差和相对效率,比较了有效病例分析(ACA)和几种多重填补(MI)方法在纵向分析中处理缺失数据问题的性能。当协变量的缺失依赖于观测到的响应时,ACA会产生估计偏差,但当纵向响应中仅有缺失值时,它更受青睐。多级MI方法并不总是纵向数据分析的解决方案。单级MI方法,如完全条件设定(FCS),在各种缺失数据情形下都能提供无偏估计,并在某些情形下提高效率增益。缺失数据机制的一般假设是随机缺失(MAR)。我们进行了一项系统的合成数据分析,其中在不同类型的缺失数据生成过程下,纵向结果或/和协变量中存在缺失数据。分析模型是一个线性混合效应模型。对于每种缺失数据情形,我们基于理论依据和广泛的模拟给出建议(在ACA和一种特定的MI方法之间)。此外,一个纵向神经退行性疾病数据集被用作实际案例研究。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e9a1/12356228/a29714d62524/nihms-2099379-f0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e9a1/12356228/a29714d62524/nihms-2099379-f0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e9a1/12356228/a29714d62524/nihms-2099379-f0001.jpg

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Stat Med. 2022 Dec 30;41(30):5844-5876. doi: 10.1002/sim.9592. Epub 2022 Oct 11.
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Appropriateness of Applying Cerebrospinal Fluid Biomarker Cutoffs from Alzheimer's Disease to Parkinson's Disease.将阿尔茨海默病的脑脊液生物标志物界值应用于帕金森病的适宜性。
J Parkinsons Dis. 2022;12(4):1155-1167. doi: 10.3233/JPD-212989.
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Multiple Imputation via Generative Adversarial Network for High-dimensional Blockwise Missing Value Problems.
基于生成对抗网络的多重插补法解决高维分块缺失值问题
Proc Int Conf Mach Learn Appl. 2021 Dec;2021:791-798. doi: 10.1109/icmla52953.2021.00131.
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Biom J. 2021 Jun;63(5):915-947. doi: 10.1002/bimj.202000196. Epub 2021 Feb 24.
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Accounting for missing data in statistical analyses: multiple imputation is not always the answer.在统计分析中处理缺失数据:多重插补并不总是答案。
Int J Epidemiol. 2019 Aug 1;48(4):1294-1304. doi: 10.1093/ije/dyz032.
6
The proportion of missing data should not be used to guide decisions on multiple imputation.缺失数据的比例不应用于指导多重插补的决策。
J Clin Epidemiol. 2019 Jun;110:63-73. doi: 10.1016/j.jclinepi.2019.02.016. Epub 2019 Mar 13.
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Using simulation studies to evaluate statistical methods.运用模拟研究评估统计方法。
Stat Med. 2019 May 20;38(11):2074-2102. doi: 10.1002/sim.8086. Epub 2019 Jan 16.
8
The Parkinson's progression markers initiative (PPMI) - establishing a PD biomarker cohort.帕金森病进展标志物计划(PPMI)——建立帕金森病生物标志物队列。
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On the use of the not-at-random fully conditional specification (NARFCS) procedure in practice.关于在实践中使用非随机完全条件规范(NARFCS)程序。
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When and how should multiple imputation be used for handling missing data in randomised clinical trials - a practical guide with flowcharts.何时以及如何在随机临床试验中使用多重插补来处理缺失数据——附流程图的实用指南。
BMC Med Res Methodol. 2017 Dec 6;17(1):162. doi: 10.1186/s12874-017-0442-1.