论文标题
使用源统计数据的摘要统计数据的风险预测通过源数据
Risk Projection for Time-to-event Outcome Leveraging Summary Statistics With Source Individual-level Data
论文作者
论文摘要
在临床实践中,预测慢性疾病的风险已变得越来越重要。当在给定的源组中开发预测模型时,将模型应用于其他同类群体通常会引起极大的兴趣。但是,由于不同队列与患者组成的基线疾病发生率的潜在差异,原始模型所预测的风险通常会低于或过高估计新队列中的风险。对于适当的医疗决策,需要进行这种校准的预测不佳的补救措施。在本文中,我们假设两个队列之间的预测因子的相对风险相同,并提出了一种新型的加权估计方程方法,以通过更新基线风险来重新校准目标人群的预计风险。重新校准利用有关感兴趣疾病和竞争事件的总体生存概率的知识,以及来自目标人群的危险因素的摘要信息。如果源和目标队列的风险因素分布相同,则建议的重新校准风险估计效率的效率相同,并且如果偏见有所不同,则很少有偏见。我们建立了所提出的估计量的一致性和渐近正态性。广泛的仿真研究表明,所提出的估计器在有限样品中的鲁棒性和效率方面表现出色。结直肠癌风险预测的实际数据应用还表明,该提出的方法可用于实践重新校准。
Predicting risks of chronic diseases has become increasingly important in clinical practice. When a prediction model is developed in a given source cohort, there is often a great interest to apply the model to other cohorts. However, due to potential discrepancy in baseline disease incidences between different cohorts and shifts in patient composition, the risk predicted by the original model often under- or over-estimates the risk in the new cohort. The remedy of such a poorly calibrated prediction is needed for proper medical decision-making. In this article, we assume the relative risks of predictors are the same between the two cohorts, and propose a novel weighted estimating equation approach to re-calibrating the projected risk for the targeted population through updating the baseline risk. The recalibration leverages the knowledge about the overall survival probabilities for the disease of interest and competing events, and the summary information of risk factors from the targeted population. The proposed re-calibrated risk estimators gain efficiency if the risk factor distributions are the same for both the source and target cohorts, and are robust with little bias if they differ. We establish the consistency and asymptotic normality of the proposed estimators. Extensive simulation studies demonstrate that the proposed estimators perform very well in terms of robustness and efficiency in finite samples. A real data application to colorectal cancer risk prediction also illustrates that the proposed method can be used in practice for model recalibration.