药物使用和剂量的动态治疗策略的比较有效性:使用观察数据模拟目标试验
Kate Birnie1, Charles Tomson2, Fergus J Caskey1,3
1From the Population Health Sciences, Bristol Medical School, University of Bristol, Bristol, United Kingdom.
电子健康记录 (EHR) 能够对动态治疗策略进行比较有效性研究. 方法C,使用多项回归来进行剂量建模,在用达比波治疗的血液透析患者中提供了可靠的死亡率估计.
科学领域:
- 医疗信息学 医疗信息学
- 观察数据分析 观察数据分析
- 药物经济学 药物经济学
背景情况:
- 电子健康记录 (EHR) 提供了丰富的数据,用于比较有效性研究.
- 动态治疗策略可以使用观察数据进行评估,并以反向概率 (IP) 权重进行权衡.
- 连续治疗的IP权重可能对所选择的统计模型敏感.
研究的目的:
- 模拟一个使用EHR数据的目标试验,以比较血液透析患者的贫血治疗策略.
- 评估四种不同的方法来建模时间变化的治疗决策.
主要方法:
- 利用英国脏注册局 (2004-2016) 血液透析患者的EHR数据.
- 我们比较了四种用于建模时间变化的达比波埃丁剂量的方法:逻辑,标准线性,异种线性和多项回归.
- 评估死亡危险比率估计的可靠性和IP权重行为.
主要成果:
- 方法C,包括低/高剂量的多项回归,是唯一产生可靠的死亡风险比率估计的方法.
- 方法C在重量截减后显示了较少的极端重量和稳定的估计.
- 在将IP权重缩减到第95个百分点后,估计在各方法中变得相似.
结论:
- 电子健康记录数据可以模拟动态治疗策略的目标试验.
- 在EHR数据分析中处理连续治疗变量时,模型检查,体重监测和适应策略至关重要.
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