对纵向二进制数据的基于参考的多重推算
Suzie Cro1, Matteo Quartagno2, Ian R White2
1Imperial Clinical Trials Unit, Imperial College London, London, UK.
基于参考的多重归算处理临床试验中缺失的数据,用于纵向二进制结果. 隐性正常模型的方法是首选的,因为它减少了偏差和信息定推理,特别是较罕见的结果.
科学领域:
- 生物统计学 生物统计学
- 临床试验方法论 临床试验方法论
- 纵向数据分析 纵向数据分析
背景情况:
- 治疗政策策略在临床试验中很常见,但由于治疗偏差后缺少的数据而复杂化.
- 对于连续的纵向结果,建立了基于参考的多重归算 (MI).
- 对于纵向二进制数据,基于参考的MI的最佳实现尚不清楚.
研究的目的:
- 为纵向二进制结果开发和比较基于参考的多重归算算法.
- 评估两种联合建模方法的性能:多变量正常分布与自适应圆和潜在的多变量正常模型.
- 评估拟议方法的偏见和信息定性质.
主要方法:
- 使用联合建模制定了基于参考的MI的两个算法.
- 算法1:多变量正常分布与自适应圆形化.
- 算法2:潜伏的多变量正常模型. 进行模拟研究来比较方法.
主要成果:
- 这两种方法在评估的场景中都提供了大约基于信息的推断.
- 隐性正常方法通常会产生较少的偏差,特别是对于较罕见的结果.
- 对于非常罕见的结果 (),表现可能不令人满意.
结论:
- 基于参考的多重归算是一种实用的,以信息为主导的工具,用于在治疗政策下估计治疗效果的纵向二进制结果.
- 潜伏多变量正常模型是其卓越性能的推实现.
- 对于非常罕见的结果,需要仔细考虑.
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