跨领域优化针对目标患者群体的个性化治疗。

IF 1.2 4区 数学
International Journal of Biostatistics Pub Date : 2024-09-26 eCollection Date: 2024-11-01 DOI:10.1515/ijb-2024-0068
Yuan Chen, Donglin Zeng, Yuanjia Wang
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引用次数: 0

摘要

针对目标精神障碍患者群体学习个性化治疗规则(ITR)面临着许多挑战。首先,目标人群可能不同于为学习 ITR 提供数据的训练人群。忽略训练患者数据与目标人群之间的差异,可能会导致针对目标人群的治疗策略达不到最佳效果。其次,对于精神障碍而言,患者的基本精神状态无法观察到,但可以通过症状的高维组合测量来推断。治疗机制是未知的,也可能是复杂的,因此治疗效果调节的形式也可能是复杂的。为了应对这些挑战,我们提出了一种新方法,通过潜变量将测量模型、高效加权方案和灵活的神经网络架构联系起来,为目标人群量身定制治疗方案。患者的基本心理状态由一组紧凑的潜在状态变量表示,同时保持可解释性。加权方案的设计基于低维潜在变量,以有效平衡人群差异,从而减轻学习潜在结构和治疗效果的偏差。广泛的模拟研究表明,所提出的方法和加权方法具有一致的优越性。在两项针对重度抑郁症患者的实际研究中的应用表明,所提出的方法在改善目标人群的治疗效果方面具有广泛的实用性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Optimizing personalized treatments for targeted patient populations across multiple domains.

Learning individualized treatment rules (ITRs) for a target patient population with mental disorders is confronted with many challenges. First, the target population may be different from the training population that provided data for learning ITRs. Ignoring differences between the training patient data and the target population can result in sub-optimal treatment strategies for the target population. Second, for mental disorders, a patient's underlying mental state is not observed but can be inferred from measures of high-dimensional combinations of symptomatology. Treatment mechanisms are unknown and can be complex, and thus treatment effect moderation can take complicated forms. To address these challenges, we propose a novel method that connects measurement models, efficient weighting schemes, and flexible neural network architecture through latent variables to tailor treatments for a target population. Patients' underlying mental states are represented by a compact set of latent state variables while preserving interpretability. Weighting schemes are designed based on lower-dimensional latent variables to efficiently balance population differences so that biases in learning the latent structure and treatment effects are mitigated. Extensive simulation studies demonstrated consistent superiority of the proposed method and the weighting approach. Applications to two real-world studies of patients with major depressive disorder have shown a broad utility of the proposed method in improving treatment outcomes in the target population.

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来源期刊
International Journal of Biostatistics
International Journal of Biostatistics Mathematics-Statistics and Probability
CiteScore
2.30
自引率
8.30%
发文量
28
期刊介绍: The International Journal of Biostatistics (IJB) seeks to publish new biostatistical models and methods, new statistical theory, as well as original applications of statistical methods, for important practical problems arising from the biological, medical, public health, and agricultural sciences with an emphasis on semiparametric methods. Given many alternatives to publish exist within biostatistics, IJB offers a place to publish for research in biostatistics focusing on modern methods, often based on machine-learning and other data-adaptive methodologies, as well as providing a unique reading experience that compels the author to be explicit about the statistical inference problem addressed by the paper. IJB is intended that the journal cover the entire range of biostatistics, from theoretical advances to relevant and sensible translations of a practical problem into a statistical framework. Electronic publication also allows for data and software code to be appended, and opens the door for reproducible research allowing readers to easily replicate analyses described in a paper. Both original research and review articles will be warmly received, as will articles applying sound statistical methods to practical problems.
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