Personalized Treatment Selection for Multivariate Ordinal Scale Outcomes and Multiple Treatments.

IF 1.5 4区 医学 Q4 PHARMACOLOGY & PHARMACY
Chathura Siriwardhana, Bakeerathan Gunaratnam, K B Kulasekera
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引用次数: 0

Abstract

In this study, we present an innovative approach for tailoring treatment selection on an individualized basis in the presence of correlated multiple responses, particularly those measured on ordinal scales, including binary responses. Our methodology involves the utilization of rank lists for treatments, generated from probabilities of observing responses of higher order than each level of the ordinal outcome, conditional on patient covariate measurements. We introduce a rank aggregation technique designed to amalgamate multiple lists of ranks, allowing for correlations both within these lists and among elements within each list. Our approach is versatile, accommodating any number of treatments and responses, and is applicable across a wide range of models. Our method offers flexibility by allowing the integration of response weights, enabling customization based on patient and clinician preferences on an individual case basis for optimal treatment decisions. To evaluate the performance of our proposed method in finite samples, we conducted a simulation study. Furthermore, we provide two illustrative examples using data from clinical trials on Cystic Fibrosis and Alzheimer's Disease, demonstrating the application of our proposed procedure in real-world scenarios.

多元有序量表结果和多种治疗的个性化治疗选择。
在这项研究中,我们提出了一种创新的方法,在存在相关多重反应的情况下,在个性化的基础上定制治疗选择,特别是那些在有序量表上测量的治疗选择,包括二元反应。我们的方法包括对治疗的排序列表的使用,由观察到的反应的高阶概率产生,高于顺序结果的每个水平,以患者协变量测量为条件。我们引入了一种级别聚合技术,用于合并多个级别列表,允许在这些列表中以及每个列表中的元素之间建立相关性。我们的方法是通用的,适应任何数量的治疗和反应,并适用于广泛的模型。我们的方法提供了灵活性,允许响应权重的整合,使定制基于患者和临床医生的偏好,在个案的基础上,最佳的治疗决策。为了评估我们提出的方法在有限样本中的性能,我们进行了模拟研究。此外,我们提供了两个说明性的例子,使用囊性纤维化和阿尔茨海默病的临床试验数据,展示了我们提出的程序在现实世界中的应用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Pharmaceutical Statistics
Pharmaceutical Statistics 医学-统计学与概率论
CiteScore
2.70
自引率
6.70%
发文量
90
审稿时长
6-12 weeks
期刊介绍: Pharmaceutical Statistics is an industry-led initiative, tackling real problems in statistical applications. The Journal publishes papers that share experiences in the practical application of statistics within the pharmaceutical industry. It covers all aspects of pharmaceutical statistical applications from discovery, through pre-clinical development, clinical development, post-marketing surveillance, consumer health, production, epidemiology, and health economics. The Journal is both international and multidisciplinary. It includes high quality practical papers, case studies and review papers.
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