在纵向多项患者报告结果中处理缺失数据的不同方法的比较:一个模拟研究
Minqian Yan1, Lizhi Zhou1, Chongye Zhao1
1State Key Laboratory of Organ Failure Research, Department of Biostatistics, Guangdong Provincial Key Laboratory of Tropical Disease Research, School of Public Health, Southern Medical University, Guangzhou, 510515, Guangdong, China.
Health and quality of life outcomes
|April 5, 2025
概括
处理缺失的患者报告结果 (PRO) 数据至关重要. 使用混合模型进行重复测量 (MMRM) 或通过链式方程 (MICE) 进行多重归算的项目级归算通常提供最佳性能,而模式混合模型 (PPMs) 则优先用于缺失的非随机数据.
科学领域:
- 临床研究方法论 临床研究方法论
- 生物统计学 生物统计学
- 健康 结果 研究 研究 结果
背景情况:
- 患者报告的结果 (PROs) 是重要的临床终点.
- 缺少PRO数据带来了重大的分析挑战.
- 本研究评估了处理缺少PRO数据的方法.
研究的目的:
- 为了比较各种统计方法处理缺失的PRO数据的性能.
- 根据缺少的数据特征,确定最佳的归算策略.
主要方法:
- 模拟的缺失数据场景使用真实的PRO数据集.
- 评估了八种方法:MMRM (有/没有归算),MICE (复合/项目级),PPM和LOCF.
- 基于不同缺失率和机制的偏差和统计能力的比较方法.
主要成果:
- 失踪率的增加导致了更高的偏差和更低的统计能力.
- 项目级别的归算表现优于复合级别的归算.
- 除了MNAR之外,MMRM和MICE (项目级) 显示出偏差最低和功率最高.
- 在失踪非随机 (MNAR) 条件下,PPM方法取得了卓越的成绩.
结论:
- 对于MNAR数据,建议使用项目级PPM.
- 项目级MMRM和MICE适用于其他缺失数据场景.
- 提供了为选择PROs适当的缺失数据处理技术的指导.
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