使用伪观测对间隔审查数据的结构嵌套平均值模型的G估计.
Shiro Tanaka1,2, M Alan Brookhart2, Jason Fine3
1Department of Clinical Biostatistics, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Statistics in medicine
|July 4, 2023
概括
在2型糖尿病患者中使用费诺纤维酸可显著降低糖尿病视网膜病变的进展在最初的4年. 然而,超过这一时期的长期疗效并未得到这种因果分析的支持.
科学领域:
- 糖尿病学 糖尿病学
- 眼科医生 眼科 眼科
- 生物统计学 生物统计学
背景情况:
- 随机试验 (FIELD,ACCORD) 显示,纤维酸可减少糖尿病视网膜病变的进展.
- 之前的分析因治疗转换和间隔审查数据而复杂化.
- 在2型糖尿病中长期使用纤维酸的准确因果效应估计是具有挑战性的.
研究的目的:
- 解决长期纤维化物使用对估计因果影响的挑战.
- 建议使用间隔审查数据对时间变化的治疗效应提出新的统计方法.
- 通过一项队列研究,重新评估费诺纤维酸在糖尿病视网膜病变进展中的疗效.
主要方法:
- 使用结构嵌套平均值模型 (SNMMs) 进行时间变化的处理效应.
- 开发了间隔审查数据的伪观测估计器.
- 使用非参数最大概率估计器 (MLE) 和参数断片指数分布进行估计.
主要成果:
- 伪观测估计器,特别是非参数的Wellner-Zhan估计器,即使在依赖间隔审查的情况下也表现良好.
- 对糖尿病队列 (8年随访) 的应用表明,纤维酸在前4年内降低了糖尿病视网膜病变的风险.
- 费诺纤维酸在减少糖尿病视网膜病变进展方面的有效性在4年后没有观察到.
结论:
- 新的统计方法有效地估计了间隔审查和时间变化的治疗存在的因果关系.
- 费诺纤维酸在减少2型糖尿病糖尿病的糖尿病视网膜病变进展方面显示出短期 (4年) 的益处.
- 在这个队列中,长期使用费诺纤维酸盐超过4年并没有显示对糖尿病视网膜病变的持续疗效.
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