Fowler Erin E, Berglund Anders, Schell Michael J, Sellers Thomas A, Eschrich Steven, Heine John
Cancer Epidemiology Department, MCC, Moffitt Cancer Center & Research Institute, 12901 Bruce B. Downs Blvd, Tampa, FL 33612, United States.
Department of Biostatistics and Bioinformatics, MCC, Moffitt Cancer Center & Research Institute, 12901 Bruce B. Downs Blvd, Tampa, FL 33612, United States.
J Biomed Inform. 2020 May;105:103408. doi: 10.1016/j.jbi.2020.103408. Epub 2020 Mar 12.
Limited sample sizes can lead to spurious modeling findings in biomedical research. The objective of this work is to present a new method to generate synthetic populations (SPs) from limited samples using matched case-control data (n = 180 pairs), considered as two separate limited samples. SPs were generated with multivariate kernel density estimations (KDEs) with unconstrained bandwidth matrices. We included four continuous variables and one categorical variable for each individual. Bandwidth matrices were determined with Differential Evolution (DE) optimization by covariance comparisons. Four synthetic samples (n = 180) were derived from their respective SPs. Similarity between observed samples with synthetic samples was compared assuming their empirical probability density functions (EPDFs) were similar. EPDFs were compared with the maximum mean discrepancy (MMD) test statistic based on the Kernel Two-Sample Test. To evaluate similarity within a modeling context, EPDFs derived from the Principal Component Analysis (PCA) scores and residuals were summarized with the distance to the model in X-space (DModX) as additional comparisons. Four SPs were generated from each sample. The probability of selecting a replicate when randomly constructing synthetic samples (n = 180) was infinitesimally small. MMD tests indicated that the observed sample EPDFs were similar to the respective synthetic EPDFs. For the samples, PCA scores and residuals did not deviate significantly when compared with their respective synthetic samples. The feasibility of this approach was demonstrated by producing synthetic data at the individual level, statistically similar to the observed samples. The methodology coupled KDE with DE optimization and deployed novel similarity metrics derived from PCA. This approach could be used to generate larger-sized synthetic samples. To develop this approach into a research tool for data exploration purposes, additional evaluation with increased dimensionality is required. Moreover, given a fully specified population, the degree to which individuals can be discarded while synthesizing the respective population accurately will be investigated. When these objectives are addressed, comparisons with other techniques such as bootstrapping will be required for a complete evaluation.
在生物医学研究中,有限的样本量可能会导致虚假的建模结果。这项工作的目的是提出一种新方法,使用匹配的病例对照数据(n = 180对)从有限样本中生成合成总体(SPs),这些数据被视为两个独立的有限样本。使用具有无约束带宽矩阵的多元核密度估计(KDEs)生成SPs。我们为每个个体纳入了四个连续变量和一个分类变量。通过协方差比较,利用差分进化(DE)优化确定带宽矩阵。从各自的SPs中导出了四个合成样本(n = 180)。假设观察样本和合成样本的经验概率密度函数(EPDFs)相似,比较了它们之间的相似性。基于核双样本检验,通过最大均值差异(MMD)检验统计量比较EPDFs。为了在建模背景下评估相似性,作为额外的比较,用X空间中到模型的距离(DModX)总结了从主成分分析(PCA)得分和残差导出的EPDFs。从每个样本中生成了四个SPs。在随机构建合成样本(n = 180)时选择重复样本的概率极小。MMD检验表明,观察样本的EPDFs与各自的合成EPDFs相似。对于这些样本,与各自的合成样本相比,PCA得分和残差没有显著偏差。通过在个体水平上生成与观察样本在统计上相似的合成数据,证明了该方法的可行性。该方法将KDE与DE优化相结合,并采用了从PCA导出的新颖相似性度量。这种方法可用于生成更大规模的合成样本。为了将这种方法发展成为一种用于数据探索目的的研究工具,需要增加维度进行额外评估。此外,在给定完全指定的总体的情况下,将研究在准确合成各自总体时可以丢弃个体的程度。当这些目标实现后,为了进行全面评估,将需要与其他技术(如自助法)进行比较。