临床试验应用中多指标综合评价治疗效果。

IF 3.1 Q2 PHARMACOLOGY & PHARMACY
Abdullah Al Masud, Samaradasa Weerahandi, Ching-Ray Yu
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引用次数: 0

摘要

在临床试验中,可以使用各种临床和实验室措施来评估一种新治疗方法相对于标准护理的益处。临床研究的数据通常通过将单个结果合并为一个主要结果来分析。该主要终点被称为复合终点或联合终点。我们建议采用Gehan(1965)的排序方法对复合终点进行分析,其中以成对方式将治疗组的每个受试者与对照组的每个受试者进行比较。我们的方法减少了计算时间和复杂性来构建一个学科水平的两两复合分数。当使用分层分数时,我们开发了一个用于分析复合终点的统计测试程序。在本文中,我们提出了两个测试(参数测试和非参数自举程序)来评估治疗效果。所提出的参数检验具有基于标准统计假设的渐近f分布。我们进行了广泛的模拟研究,以评估所提出的方法的操作特性,并将它们与现有方法进行比较。我们使用两项临床研究的公开数据来说明这些方法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Evaluating Treatment Efficacy by Combining Multiple Measures in Clinical Trial Applications.

A variety of clinical and laboratory measures can be used in clinical trials to assess the benefit of a new treatment over the standard of care. Data from clinical studies are often analyzed by combining individual outcomes into one primary outcome. That primary outcome is then referred to as a composite endpoint or a combined endpoint. We propose an analysis on the composite endpoint with Gehan's (1965) ranking approach where each subject in the treatment group is compared with each subject in the control group in a pair-wise manner. Our approach reduces computational time and complexity to construct a subject-level pairwise composite score. We develop a statistical testing procedure for the analysis of composite endpoints when using the hierarchical scores. In this article, we propose two tests (a parametric test and a non-parametric bootstrap procedure) for evaluating the effect of treatment. The proposed parametric test has an asymptotic F-distribution based on standard statistical assumptions. We conduct an extensive simulation study to assess the operating characteristics of the proposed methods and to compare them with an existing method. We illustrate the methods using publicly available data from two clinical studies.

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来源期刊
Pharmaceutical Medicine
Pharmaceutical Medicine PHARMACOLOGY & PHARMACY-
CiteScore
5.10
自引率
4.00%
发文量
36
期刊介绍: Pharmaceutical Medicine is a specialist discipline concerned with medical aspects of the discovery, development, evaluation, registration, regulation, monitoring, marketing, distribution and pricing of medicines, drug-device and drug-diagnostic combinations. The Journal disseminates information to support the community of professionals working in these highly inter-related functions. Key areas include translational medicine, clinical trial design, pharmacovigilance, clinical toxicology, drug regulation, clinical pharmacology, biostatistics and pharmacoeconomics. The Journal includes:Overviews of contentious or emerging issues.Comprehensive narrative reviews that provide an authoritative source of information on topical issues.Systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by PRISMA statement.Original research articles reporting the results of well-designed studies with a strong link to wider areas of clinical research.Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Pharmaceutical Medicine may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.
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