个性化治疗的稀疏2阶段贝叶斯荟萃分析。

IF 1.7 4区 数学 Q3 BIOLOGY
Biometrics Pub Date : 2025-07-03 DOI:10.1093/biomtc/ujaf082
Junwei Shen, Erica E M Moodie, Shirin Golchi
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引用次数: 0

摘要

个体化治疗规则根据临床、人口统计学和其他特征为患者量身定制治疗方案。估计个体化治疗规则需要确定从特定治疗中获益最多的个体,从而检测治疗效果的可变性。为了制定有效的个体化治疗规则,可能需要来自多地点研究的数据,因为较小的数据集用于检测通常较小的治疗-协变量相互作用的能力较低。然而,个人层面数据的共享有时受到限制。此外,稀疏性可能在两种意义上产生:不同的数据点可能从不同的种群中招募,使得在所有地点估计相同的模型或所有感兴趣的参数是不可行的,并且模型中用于处理规则的非零参数的数量可能很小。为了解决这些问题,我们采用两阶段贝叶斯荟萃分析方法来估计个性化治疗规则,该规则使用多站点数据优化患者预期结果,而不会泄露超出站点的个人水平数据。仿真结果表明,我们的方法可以提供一致的参数估计,充分表征了最优的个性化治疗规则。我们使用来自国际华法林药物遗传学协会的数据来估计最佳华法林剂量策略,其中数据稀疏和小治疗-协变量相互作用效应带来了额外的统计挑战。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Sparse 2-stage Bayesian meta-analysis for individualized treatments.

Individualized treatment rules tailor treatments to patients based on clinical, demographic, and other characteristics. Estimation of individualized treatment rules requires the identification of individuals who benefit most from the particular treatments and thus the detection of variability in treatment effects. To develop an effective individualized treatment rule, data from multisite studies may be required due to the low power provided by smaller datasets for detecting the often small treatment-covariate interactions. However, sharing of individual-level data is sometimes constrained. Furthermore, sparsity may arise in 2 senses: different data sites may recruit from different populations, making it infeasible to estimate identical models or all parameters of interest at all sites, and the number of non-zero parameters in the model for the treatment rule may be small. To address these issues, we adopt a 2-stage Bayesian meta-analysis approach to estimate individualized treatment rules which optimize expected patient outcomes using multisite data without disclosing individual-level data beyond the sites. Simulation results demonstrate that our approach can provide consistent estimates of the parameters which fully characterize the optimal individualized treatment rule. We estimate the optimal Warfarin dose strategy using data from the International Warfarin Pharmacogenetics Consortium, where data sparsity and small treatment-covariate interaction effects pose additional statistical challenges.

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来源期刊
Biometrics
Biometrics 生物-生物学
CiteScore
2.70
自引率
5.30%
发文量
178
审稿时长
4-8 weeks
期刊介绍: The International Biometric Society is an international society promoting the development and application of statistical and mathematical theory and methods in the biosciences, including agriculture, biomedical science and public health, ecology, environmental sciences, forestry, and allied disciplines. The Society welcomes as members statisticians, mathematicians, biological scientists, and others devoted to interdisciplinary efforts in advancing the collection and interpretation of information in the biosciences. The Society sponsors the biennial International Biometric Conference, held in sites throughout the world; through its National Groups and Regions, it also Society sponsors regional and local meetings.
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