评估时间不规则且信息丰富的试验的半参数敏感性分析。

IF 1.4 4区 数学 Q3 BIOLOGY Biometrics Pub Date : 2024-10-03 DOI:10.1093/biomtc/ujae154
Bonnie B Smith, Yujing Gao, Shu Yang, Ravi Varadhan, Andrea J Apter, Daniel O Scharfstein
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引用次数: 0

摘要

许多试验的目的是在随机化后的预定时间或前后收集结果。如果参与者实际接受评估的时间存在差异,这可能对了解治疗效果构成挑战,因为并非所有参与者都在感兴趣的时间接受结果评估。此外,观察到的结果值可能不能代表所有参与者在给定时间的结果。已经开发了一些方法来解释某些类型的这种不规则和信息性的评估时间;然而,由于这些方法依赖于不可检验的假设,因此需要进行敏感性分析。我们开发了一种敏感性分析方法,该方法以可解释评估(EA)假设为基准,在该假设下,每次的评估和结果仅通过在该时间之前收集的数据相关联。我们的方法使用指数倾斜假设,由敏感性分析参数控制,假设偏离EA假设。我们的推理策略基于一种新的基于影响函数的增广逆强度加权估计器。我们的方法允许对观测数据进行灵活的半参数建模,这与灵敏度参数的规格分离。我们将我们的方法应用于一项低收入哮喘患者的随机试验,并详细说明了我们的估计程序的实施。
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Semi-parametric sensitivity analysis for trials with irregular and informative assessment times.

Many trials are designed to collect outcomes at or around pre-specified times after randomization. If there is variability in the times when participants are actually assessed, this can pose a challenge to learning the effect of treatment, since not all participants have outcome assessments at the times of interest. Furthermore, observed outcome values may not be representative of all participants' outcomes at a given time. Methods have been developed that account for some types of such irregular and informative assessment times; however, since these methods rely on untestable assumptions, sensitivity analyses are needed. We develop a sensitivity analysis methodology that is benchmarked at the explainable assessment (EA) assumption, under which assessment and outcomes at each time are related only through data collected prior to that time. Our method uses an exponential tilting assumption, governed by a sensitivity analysis parameter, that posits deviations from the EA assumption. Our inferential strategy is based on a new influence function-based, augmented inverse intensity-weighted estimator. Our approach allows for flexible semiparametric modeling of the observed data, which is separated from specification of the sensitivity parameter. We apply our method to a randomized trial of low-income individuals with uncontrolled asthma, and we illustrate implementation of our estimation procedure in detail.

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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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