Chen Xiaofei, Heitjan Daniel F, Greil Gerald, Jeon-Slaughter Haekyung
Department of Statistical Science, Southern Methodist University, Dallas, Texas.
Department of Population & Data Sciences, UT Southwestern Medical Center, Dallas, Texas.
Biometrics. 2021 Jun;77(2):729-739. doi: 10.1111/biom.13311. Epub 2020 Jun 15.
Infants with hypoplastic left heart syndrome require an initial Norwood operation, followed some months later by a stage 2 palliation (S2P). The timing of S2P is critical for the operation's success and the infant's survival, but the optimal timing, if one exists, is unknown. We attempt to identify the optimal timing of S2P by analyzing data from the Single Ventricle Reconstruction Trial (SVRT), which randomized patients between two different types of Norwood procedure. In the SVRT, the timing of the S2P was chosen by the medical team; thus with respect to this exposure, the trial constitutes an observational study, and the analysis must adjust for potential confounding. To accomplish this, we propose an extended propensity score analysis that describes the time to surgery as a function of confounders in a discrete competing-risk model. We then apply inverse probability weighting to estimate a spline hazard model for predicting survival from the time of S2P. Our analysis suggests that S2P conducted at 6 months after the Norwood gives the patient the best post-S2P survival. Thus, we place the optimal time slightly later than a previous analysis in the medical literature that did not account for competing risks of death and heart transplantation.
患有左心发育不全综合征的婴儿需要先进行诺伍德手术,数月后再进行二期姑息治疗(S2P)。S2P的时机对于手术成功和婴儿存活至关重要,但最佳时机(如果存在的话)尚不清楚。我们试图通过分析单心室重建试验(SVRT)的数据来确定S2P的最佳时机,该试验将患者随机分为两种不同类型的诺伍德手术。在SVRT中,S2P的时机由医疗团队选择;因此,就这种暴露而言,该试验构成一项观察性研究,分析必须对潜在的混杂因素进行调整。为了实现这一点,我们提出了一种扩展倾向评分分析,该分析在离散竞争风险模型中将手术时间描述为混杂因素的函数。然后,我们应用逆概率加权来估计一个样条风险模型,以预测从S2P时间起的存活率。我们的分析表明,在诺伍德手术后6个月进行S2P能使患者在S2P后的存活率最佳。因此,我们确定的最佳时间比医学文献中之前未考虑死亡和心脏移植竞争风险的分析稍晚。