School of Economics and Statistics, Guangzhou University, Guangzhou, China.
Department of Statistics, University of Missouri, Columbia, Missouri, USA.
BMC Med Res Methodol. 2023 Apr 4;23(1):82. doi: 10.1186/s12874-023-01903-x.
Failure time data frequently occur in many medical studies and often accompany with various types of censoring. In some applications, left truncation may occur and can induce biased sampling, which makes the practical data analysis become more complicated. The existing analysis methods for left-truncated data have some limitations in that they either focus only on a special type of censored data or fail to flexibly utilize the distribution information of the truncation times for inference. Therefore, it is essential to develop a reliable and efficient method for the analysis of left-truncated failure time data with various types of censoring.
This paper concerns regression analysis of left-truncated failure time data with the proportional hazards model under various types of censoring mechanisms, including right censoring, interval censoring and a mixture of them. The proposed pairwise pseudo-likelihood estimation method is essentially built on a combination of the conditional likelihood and the pairwise likelihood that eliminates the nuisance truncation distribution function or avoids its estimation. To implement the presented method, a flexible EM algorithm is developed by utilizing the idea of self-consistent estimating equation. A main feature of the algorithm is that it involves closed-form estimators of the large-dimensional nuisance parameters and is thus computationally stable and reliable. In addition, an R package LTsurv is developed.
The numerical results obtained from extensive simulation studies suggest that the proposed pairwise pseudo-likelihood method performs reasonably well in practical situations and is obviously more efficient than the conditional likelihood approach as expected. The analysis results of the MHCPS data with the proposed pairwise pseudo-likelihood method indicate that males have significantly higher risk of losing active life than females. In contrast, the conditional likelihood method recognizes this effect as non-significant, which is because the conditional likelihood method often loses some estimation efficiency compared with the proposed method.
The proposed method provides a general and helpful tool to conduct the Cox's regression analysis of left-truncated failure time data under various types of censoring.
失效时间数据经常出现在许多医学研究中,并且经常伴有各种类型的删失。在某些应用中,可能会发生左截断,这会导致抽样偏差,从而使实际数据分析变得更加复杂。现有的左截断数据分析方法存在一些局限性,要么只关注特殊类型的删失数据,要么无法灵活利用截断时间的分布信息进行推断。因此,开发一种用于分析具有各种删失类型的左截断失效时间数据的可靠且高效的方法至关重要。
本文涉及在各种删失机制下,包括右删失、区间删失和两者混合的情况下,使用比例风险模型对左截断失效时间数据进行回归分析。所提出的成对伪似然估计方法本质上是基于条件似然和成对似然的组合构建的,该方法消除了麻烦的截断分布函数或避免了其估计。为了实现所提出的方法,通过利用自洽估计方程的思想,开发了一种灵活的 EM 算法。该算法的一个主要特点是,它涉及到大型维数的 nuisance 参数的闭式估计器,因此计算上是稳定和可靠的。此外,还开发了一个 R 包 LTsurv。
广泛的模拟研究得到的数值结果表明,所提出的成对伪似然方法在实际情况下表现相当不错,并且如预期的那样,明显比条件似然方法更有效。使用所提出的成对伪似然方法对 MHCPS 数据的分析结果表明,男性失去活跃生命的风险明显高于女性。相比之下,条件似然方法认为这种影响不显著,这是因为条件似然方法通常比所提出的方法损失一些估计效率。
所提出的方法为在各种删失类型下进行左截断失效时间数据的 Cox 回归分析提供了一种通用且有用的工具。