Two-part test of vaccine effect

Two-part test of vaccine effect
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DOI:
10.1002/sim.6412
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发表时间:
2015-05-20
影响因子:
2
通讯作者:
Proschan, Michael
Proschan, Michael
中科院分区:
医学3区
文献类型:
--
作者:
Hu, Zonghui;Proschan, Michael

文献摘要

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疫苗的好处通常是双重的:(i)预防疾病,或者,如果不能预防疾病,(ii)减轻疾病的严重程度。为了评估疫苗的效果,我们提出了两个适应性测试。加权两部分检验是两个统计量的组合,一个关于疾病发生率,一个关于疾病严重程度。对先验效应量越大的统计量赋予更大的权重,并确定权重以最大化检验功效。随机化检验适用于感染总数相对较少的情况。它使用疾病严重程度的信息来增强权力,同时将疾病发病率作为主要利益。所提出的测试的性质进行了探讨渐近和数值研究。虽然是由疫苗研究推动的,但拟议的测试适用于任何涉及二元和连续结果的试验,以评估治疗效果。2015年出版。本条目属于美国政府作品,在美国属于公有领域。
Vaccine benefit is usually two-folded: (i) prevent a disease or, failing that, (ii) diminish the severity of a disease. To assess vaccine effect, we propose two adaptive tests. The weighted two-part test is a combination of two statistics, one on disease incidence and one on disease severity. More weight is given to the statistic with the larger a priori effect size, and the weights are determined to maximize testing power. The randomized test applies to the scenario where the total number of infections is relatively small. It uses information on disease severity to bolster power while preserving disease incidence as the primary interest. Properties of the proposed tests are explored asymptotically and by numerical studies. Although motivated by vaccine studies, the proposed tests apply to any trials that involve both binary and continuous outcomes for evaluating treatment effect. Published 2015. This article is a US Government work and is in the public domain in the USA.