Zhang Jingyang, Brown Elizabeth R
Fred Hutchinson Cancer Research Center, 1100 Fairview Ave N, M2-C200, Seattle, Washington 98109, U.S.A.
Department of Biostatistics, University of Washington, Seattle, Washington 98109, U.S.A.
Biometrics. 2014 Sep;70(3):745-53. doi: 10.1111/biom.12183. Epub 2014 May 20.
Estimating the effectiveness of a new intervention is usually the primary objective for HIV prevention trials. The Cox proportional hazard model is mainly used to estimate effectiveness by assuming that participants share the same risk under the covariates and the risk is always non-zero. In fact, the risk is only non-zero when an exposure event occurs, and participants can have a varying risk to transmit due to varying patterns of exposure events. Therefore, we propose a novel estimate of effectiveness adjusted for the heterogeneity in the magnitude of exposure among the study population, using a latent Poisson process model for the exposure path of each participant. Moreover, our model considers the scenario in which a proportion of participants never experience an exposure event and adopts a zero-inflated distribution for the rate of the exposure process. We employ a Bayesian estimation approach to estimate the exposure-adjusted effectiveness eliciting the priors from the historical information. Simulation studies are carried out to validate the approach and explore the properties of the estimates. An application example is presented from an HIV prevention trial.
评估新干预措施的有效性通常是艾滋病预防试验的主要目标。Cox比例风险模型主要用于通过假设参与者在协变量下具有相同风险且风险始终非零来估计有效性。事实上,只有当暴露事件发生时风险才非零,并且由于暴露事件模式不同,参与者传播风险可能各异。因此,我们提出一种新的有效性估计方法,通过使用潜在泊松过程模型来考虑研究人群中暴露程度的异质性,以调整各参与者的暴露路径。此外,我们的模型考虑了一部分参与者从未经历暴露事件的情况,并对暴露过程发生率采用零膨胀分布。我们采用贝叶斯估计方法,从历史信息中获取先验信息来估计经暴露调整后的有效性。进行模拟研究以验证该方法并探索估计值的性质。还给出了一个艾滋病预防试验的应用实例。