INSERM U1138, Team 22, Centre de Recherche des Cordeliers, University Paris Descartes, University Pierre et Marie Curie, Paris, France.
Department of Biostatistics, MD Anderson Cancer Center, Houston, Texas.
Stat Med. 2019 May 30;38(12):2228-2247. doi: 10.1002/sim.8107. Epub 2019 Jan 22.
Using clinical data to model the medical decisions behind sequential treatment actions raises methodological challenges. Physicians often have access to many covariates that may be used when making sequential treatment decisions for individual patients. Statistical variable selection methods may help finding which of these variables are used for this decision in everyday practice. When the sample size is not large, Bayesian variable selection methods can address this setting and allow for expert information to be incorporated into prior distributions. Motivated by clinical practice data involving repeated dose adaptation for Irinotecan in colorectal metastatic cancer, we propose a modification of the stochastic search variable selection (SSVS) method, which we call weight-based SSVS (WBS). We use clinical relevance weights elicited from physician experts to construct prior distributions, with the goal to identify the most influential toxicities and other covariates used for dose adjustment. We evaluate and compare the WBS model performance to the Lasso and SSVS through an extensive simulation study. The simulations show that WBS has better performance and lower rates of false positives and false negatives than the other methods but depends strongly on the covariate weights.
利用临床数据来模拟序贯治疗决策背后的医学决策存在方法学上的挑战。医生在为个体患者做出序贯治疗决策时,通常可以获得许多可能会用到的协变量。统计变量选择方法可以帮助确定在日常实践中哪些变量用于做出该决策。当样本量较小时,贝叶斯变量选择方法可以解决此问题,并允许将专家信息纳入先验分布。受涉及结直肠转移性癌症伊立替康重复剂量调整的临床实践数据的启发,我们提出了对随机搜索变量选择(SSVS)方法的修改,我们称之为基于权重的 SSVS(WBS)。我们使用从医生专家那里获得的临床相关性权重来构建先验分布,目的是确定用于剂量调整的最有影响力的毒性和其他协变量。我们通过广泛的模拟研究来评估和比较 WBS 模型与 Lasso 和 SSVS 的性能。模拟结果表明,WBS 比其他方法具有更好的性能和更低的假阳性和假阴性率,但强烈依赖于协变量权重。