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Optimal Treatment Regimes: A Review and Empirical Comparison 最佳治疗方案:综述与实证比较
IF 2 3区 数学 Q1 STATISTICS & PROBABILITY Pub Date : 2023-02-22 DOI: 10.1111/insr.12536
Z. Li, Jie Chen, Eric B. Laber, Fang Liu, Richard Baumgartner
A treatment regime is a sequence of decision rules, one per decision point, that maps accumulated patient information to a recommended intervention. An optimal treatment regime maximises expected cumulative utility if applied to select interventions in a population of interest. As a treatment regime seeks to improve the quality of healthcare by individualising treatment, it can be viewed as an approach to formalising precision medicine. Increased interest and investment in precision medicine has led to a surge of methodological research focusing on estimation and evaluation of optimal treatment regimes from observational and/or randomised studies. These methods are becoming commonplace in biomedical research, although guidance about how to choose among existing methods in practice has been somewhat limited. The purpose of this review is to describe some of the most commonly used methods for estimation of an optimal treatment regime, and to compare these estimators in a series of simulation experiments and applications to real data. The results of these simulations along with the theoretical/methodological properties of these estimators are used to form recommendations for applied researchers.
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引用次数: 3
A Statistical Review of Template Model Builder: A Flexible Tool for Spatial Modelling 模板模型生成器的统计回顾:一个灵活的空间建模工具
IF 2 3区 数学 Q1 STATISTICS & PROBABILITY Pub Date : 2022-12-18 DOI: 10.1111/insr.12534
Aaron Osgood-Zimmerman, Jon Wakefield

The integrated nested Laplace approximation (INLA) is a well-known and popular technique for spatial modelling with a user-friendly interface in the R-INLA package. Unfortunately, only a certain class of latent Gaussian models are amenable to fitting with INLA. In this paper, we review template model builder (TMB), an existing technique and software package which is well-suited to fitting complex spatio-temporal models. TMB is relatively unknown to the spatial statistics community, but it is a flexible random effects modelling tool which allows users to define customizable and complex mixed effects models through C++ templates. After contrasting the methodology behind TMB with INLA, we provide a large-scale simulation study assessing and comparing R-INLA and TMB for continuous spatial models, fitted via the stochastic partial differential equations (SPDE) approximation. The results show that the predictive fields from both methods are comparable in most situations even though TMB estimates for fixed or random effects may have slightly larger bias than R-INLA. We also present a smaller discrete spatial simulation study, in which both approaches perform well. We conclude with a joint analysis of breast cancer incidence and mortality data implemented in TMB which requires a model which cannot be fit with R-INLA.

集成嵌套拉普拉斯近似(INLA)是一种众所周知的、流行的空间建模技术,在R - INLA包中有一个用户友好的界面。不幸的是,只有一类潜在高斯模型适合用INLA拟合。本文综述了模板模型构建器(template model builder, TMB),这是一种适合于拟合复杂时空模型的现有技术和软件包。TMB对于空间统计社区来说相对陌生,但它是一个灵活的随机效果建模工具,允许用户通过c++模板定义可定制的复杂混合效果模型。在对比了TMB和INLA背后的方法之后,我们提供了一项大规模的模拟研究,通过随机偏微分方程(SPDE)近似拟合,评估和比较了R - INLA和TMB对连续空间模型的影响。结果表明,两种方法的预测场在大多数情况下是可比较的,尽管固定效应或随机效应的TMB估计可能比R - INLA偏差略大。我们还提出了一个较小的离散空间模拟研究,其中两种方法都表现良好。最后,我们对TMB患者的乳腺癌发病率和死亡率数据进行了联合分析,这需要一个无法用R - INLA拟合的模型。
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引用次数: 1
Improving Probabilistic Record Linkage Using Statistical Prediction Models 利用统计预测模型改进概率记录链接
IF 2 3区 数学 Q1 STATISTICS & PROBABILITY Pub Date : 2022-12-04 DOI: 10.1111/insr.12535
Angelo Moretti, N. Shlomo
Record linkage brings together information from records in two or more data sources that are believed to belong to the same statistical unit based on a common set of matching variables. Matching variables, however, can appear with errors and variations and the challenge is to link statistical units that are subject to error. We provide an overview of record linkage techniques and specifically investigate the classic Fellegi and Sunter probabilistic record linkage framework to assess whether the decision rule for classifying pairs into sets of matches and non‐matches can be improved by incorporating a statistical prediction model. We also study whether the enhanced linkage rule can provide better results in terms of preserving associations between variables in the linked data file that are not used in the matching procedure. A simulation study and an application based on real data are used to evaluate the methods.
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引用次数: 0
A joint normal-binary (probit) model 一个联合正态二值(probit)模型
IF 2 3区 数学 Q1 STATISTICS & PROBABILITY Pub Date : 2022-11-08 DOI: 10.1111/insr.12532
Margaux Delporte, Steffen Fieuws, Geert Molenberghs, Geert Verbeke, Simeon Situma Wanyama, Elpis Hatziagorou, Christiane De Boeck

In biomedical research, often hierarchical binary and continuous responses need to be jointly modelled. In joint generalised linear mixed models, this can be done with correlated random effects, which allows examining the association structure between the various responses and the evolution of this association over time. In addition, the effect of covariates on all outcomes can be assessed simultaneously. Still, investigating this association is often limited to examining the correlations between the responses on an underlying scale. In addition, the interpretation of this hierarchical model is conditional on the subject-specific random effects. This paper extends this approach and shows how manifest correlations can be computed, that is, the associations between the observed responses. Further, a marginal model is formulated, in which the interpretation is no longer conditional on the random effects. In addition, prediction intervals are derived of one subvector of responses conditional on the other. These methods are applied in a case study of the lung function and allergic bronchopulmonary aspergillosis in patients with cystic fibrosis.

在生物医学研究中,通常需要对分层二值响应和连续响应进行联合建模。在联合广义线性混合模型中,这可以通过相关随机效应来完成,这允许检查各种响应之间的关联结构以及这种关联随时间的演变。此外,协变量对所有结果的影响可以同时评估。尽管如此,调查这种联系往往仅限于检查潜在规模上的反应之间的相关性。此外,该分层模型的解释取决于特定主题的随机效应。本文扩展了这种方法,并展示了如何计算明显的相关性,即观察到的响应之间的关联。进一步,建立了一个边际模型,其中解释不再以随机效应为条件。此外,还推导了响应的一个子向量以另一个子向量为条件的预测区间。这些方法应用于肺功能和过敏性支气管肺曲菌病的囊性纤维化患者的个案研究。
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引用次数: 1
Likelihood-Based Inference for the Finite Population Mean with Post-Stratification Information Under Non-Ignorable Non-Response 不可忽略非响应下具有分层后信息的有限总体均值的似然推断
IF 2 3区 数学 Q1 STATISTICS & PROBABILITY Pub Date : 2022-10-25 DOI: 10.1111/insr.12527
Sahar Z. Zangeneh, Roderick J. Little

We describe models and likelihood-based estimation of the finite population mean for a survey subject to unit non-response, when post-stratification information is available from external sources. A feature of the models is that they do not require the assumption that the data are missing at random (MAR). As a result, the proposed models provide estimates under weaker assumptions than those required in the absence of post-stratification information, thus allowing more robust inferences. In particular, we describe models for estimation of the finite population mean of a survey outcome with categorical covariates and externally observed categorical post-stratifiers. We compare inferences from the proposed method with existing design-based estimators via simulations. We apply our methods to school-level data from California Department of Education to estimate the mean academic performance index (API) score in years 1999 and 2000. We end with a discussion.

当从外部来源获得分层后信息时,我们描述了受单位无响应调查的有限总体均值的模型和基于似然的估计。这些模型的一个特点是,它们不需要假设数据是随机丢失的。因此,所提出的模型在较弱的假设下提供估计,而不是在缺乏分层后信息的情况下提供估计,从而允许更可靠的推断。特别是,我们描述了用分类协变量和外部观察的分类后分层来估计调查结果的有限总体均值的模型。我们通过仿真比较了所提出的方法与现有的基于设计的估计方法的推断。我们将我们的方法应用于加州教育部的校级数据,以估计1999年和2000年的平均学业表现指数(API)分数。我们以讨论结束。
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引用次数: 1
Global seasonal and pandemic patterns in influenza: An application of longitudinal study designs 流感的全球季节性和大流行模式:纵向研究设计的应用
IF 2 3区 数学 Q1 STATISTICS & PROBABILITY Pub Date : 2022-10-23 DOI: 10.1111/insr.12529
Elena N. Naumova, Ryan B. Simpson, Bingjie Zhou, Meghan A. Hartwick

The confluence of growing analytic capacities and global surveillance systems for seasonal infections has created new opportunities to further develop statistical methodology and advance the understanding of the global disease dynamics. We developed a framework to characterise the seasonality of infectious diseases for publicly available global health surveillance data. Specifically, we aimed to estimate the seasonal characteristics and their uncertainty using mixed effects models with harmonic components and the δ-method and develop multi-panel visualisations to present complex interplay of seasonal peaks across geographic locations. We compiled a set of 2 422 weekly time series of 14 reported outcomes for 173 Member States from the World Health Organization's (WHO) international influenza virological surveillance system, FluNet, from 02 January 1995 through 20 June 2021. We produced an analecta of data visualisations to describe global travelling waves of influenza while addressing issues of data completeness and credibility. Our results offer directions for further improvements in data collection, reporting, analysis and development of statistical methodology and predictive approaches.

日益增长的分析能力和全球季节性感染监测系统的汇合为进一步发展统计方法和促进对全球疾病动态的了解创造了新的机会。我们开发了一个框架,为公开的全球卫生监测数据描述传染病的季节性特征。具体来说,我们的目标是使用谐波分量和δ方法的混合效应模型来估计季节特征及其不确定性,并开发多面板可视化来呈现不同地理位置的季节峰值的复杂相互作用。从1995年1月2日至2021年6月20日,我们编制了一套2422个每周时间序列,其中包括世界卫生组织(世卫组织)国际流感病毒学监测系统fluet的173个会员国的14项报告结果。我们制作了一份数据可视化的analecata,以描述全球流感传播波,同时解决数据完整性和可信度问题。我们的研究结果为进一步改进数据收集、报告、分析以及统计方法和预测方法的发展提供了方向。
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引用次数: 0
Synergy of Biostatistics and Epidemiology in Air Pollution Health Effects Studies 生物统计学和流行病学在空气污染健康影响研究中的协同作用
IF 2 3区 数学 Q1 STATISTICS & PROBABILITY Pub Date : 2022-10-21 DOI: 10.1111/insr.12525
Douglas W. Dockery

The extraordinary advances in quantifying the health effects of ambient air pollution over the last five decades have led to dramatic improvement in air quality in the United States. This work has been possible through innovative epidemiologic study designs coupled with advanced statistical analytic methods. This paper presents a historical perspective on the coordinated developments of epidemiologic designs and statistical methods for air pollution health effects studies at the Harvard School of Public Health.

在过去的五十年里,在量化环境空气污染对健康的影响方面取得了非凡的进步,这使得美国的空气质量得到了巨大的改善。通过创新的流行病学研究设计与先进的统计分析方法相结合,这项工作成为可能。本文介绍了哈佛大学公共卫生学院空气污染健康影响研究的流行病学设计和统计方法的协调发展的历史观点。
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引用次数: 1
Path algorithms for fused lasso signal approximator with application to COVID-19 spread in Korea 融合套索信号逼近器路径算法及其在国内COVID-19传播中的应用
IF 2 3区 数学 Q1 STATISTICS & PROBABILITY Pub Date : 2022-10-19 DOI: 10.1111/insr.12521
Won Son, Johan Lim, Donghyeon Yu

The fused lasso signal approximator (FLSA) is a smoothing procedure for noisy observations that uses fused lasso penalty on unobserved mean levels to find sparse signal blocks. Several path algorithms have been developed to obtain the whole solution path of the FLSA. However, it is known that the FLSA has model selection inconsistency when the underlying signals have a stair-case block, where three consecutive signal blocks are either strictly increasing or decreasing. Modified path algorithms for the FLSA have been proposed to guarantee model selection consistency regardless of the stair-case block. In this paper, we provide a comprehensive review of the path algorithms for the FLSA and prove the properties of the recently modified path algorithms' hitting times. Specifically, we reinterpret the modified path algorithm as the path algorithm for local FLSA problems and reveal the condition that the hitting time for the fusion of the modified path algorithm is not monotone in a tuning parameter. To recover the monotonicity of the solution path, we propose a pathwise adaptive FLSA having monotonicity with similar performance as the modified solution path algorithm. Finally, we apply the proposed method to the number of daily-confirmed cases of COVID-19 in Korea to identify the change points of its spread.

融合套索信号逼近器(FLSA)是一种用于噪声观测的平滑过程,它在未观测到的平均水平上使用融合套索惩罚来寻找稀疏信号块。已经开发了几种路径算法来获得FLSA的整个求解路径。然而,已知当基础信号具有阶梯块时,FLSA具有模型选择不一致性,其中三个连续信号块严格增加或减少。已经提出了FLSA的改进路径算法,以保证模型选择的一致性,而不考虑楼梯间块。在本文中,我们对FLSA的路径算法进行了全面的回顾,并证明了最近修改的路径算法的命中时间的性质。具体来说,我们将改进的路径算法重新解释为局部FLSA问题的路径算法,并揭示了改进的路径方法的融合命中时间在调谐参数上不是单调的条件。为了恢复解路径的单调性,我们提出了一种具有单调性的路径自适应FLSA,其性能与改进的解路径算法相似。最后,我们将所提出的方法应用于韩国每日确诊的新冠肺炎病例数,以确定其传播的变化点。
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引用次数: 1
Accounting for Non-ignorable Sampling and Non-response in Statistical Matching 统计匹配中不可忽略抽样和无响应的解释
IF 2 3区 数学 Q1 STATISTICS & PROBABILITY Pub Date : 2022-10-19 DOI: 10.1111/insr.12524
Daniela Marella, Danny Pfeffermann

Data for statistical analysis is often available from different samples, with each sample containing measurements on only some of the variables of interest. Statistical matching attempts to generate a fused database containing matched measurements on all the target variables. In this article, we consider the use of statistical matching when the samples are drawn by informative sampling designs and are subject to not missing at random non-response. The problem with ignoring the sampling process and non-response is that the distribution of the data observed for the responding units can be very different from the distribution holding for the population data, which may distort the inference process and result in a matched database that misrepresents the joint distribution in the population. Our proposed methodology employs the empirical likelihood approach and is shown to perform well in a simulation experiment and when applied to real sample data.

用于统计分析的数据通常来自不同的样本,每个样本只包含对感兴趣的一些变量的测量。统计匹配尝试生成包含所有目标变量的匹配测量的融合数据库。在这篇文章中,当样本是通过信息采样设计绘制的,并且在随机无响应时不会丢失时,我们考虑使用统计匹配。忽略采样过程和非响应的问题是,响应单元观测到的数据分布可能与总体数据的分布非常不同,这可能会扭曲推理过程,并导致匹配的数据库歪曲总体中的联合分布。我们提出的方法采用了经验似然法,并在模拟实验中和应用于真实样本数据时表现良好。
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引用次数: 1
Thinking Clearly with Data: A Guide to Quantitative Reasoning and AnalysisEthan BuenodeMesquita and AnthonyFowlerPrinceton University Press, 2021, 400 pages, $95.00/£74.00, hardback ISBN: 978‐0‐691‐21436‐8 用数据清晰思考:定量推理和分析指南伊桑·布埃诺·德梅斯基塔和安东尼·福斯特普林斯顿大学出版社,2021年,400页,95.00美元/ 74.00英镑,精装本ISBN: 978‐0‐691‐21436‐8
IF 2 3区 数学 Q1 STATISTICS & PROBABILITY Pub Date : 2022-10-19 DOI: 10.1111/insr.12530
G. Dekkers
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引用次数: 3
期刊
International Statistical Review
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