Statistical Methods in Medical Research最新文献

筛选
英文 中文
Bayesian mixture models for phylogenetic source attribution from consensus sequences and time since infection estimates.
IF 1.6 3区 医学
Statistical Methods in Medical Research Pub Date : 2025-02-12 DOI: 10.1177/09622802241309750
Alexandra Blenkinsop, Lysandros Sofocleous, Francesco Di Lauro, Evangelia Georgia Kostaki, Ard van Sighem, Daniela Bezemer, Thijs van de Laar, Peter Reiss, Godelieve de Bree, Nikos Pantazis, Oliver Ratmann
{"title":"Bayesian mixture models for phylogenetic source attribution from consensus sequences and time since infection estimates.","authors":"Alexandra Blenkinsop, Lysandros Sofocleous, Francesco Di Lauro, Evangelia Georgia Kostaki, Ard van Sighem, Daniela Bezemer, Thijs van de Laar, Peter Reiss, Godelieve de Bree, Nikos Pantazis, Oliver Ratmann","doi":"10.1177/09622802241309750","DOIUrl":"https://doi.org/10.1177/09622802241309750","url":null,"abstract":"<p><p>In stopping the spread of infectious diseases, pathogen genomic data can be used to reconstruct transmission events and characterize population-level sources of infection. Most approaches for identifying transmission pairs do not account for the time passing since the divergence of pathogen variants in individuals, which is problematic in viruses with high within-host evolutionary rates. This prompted us to consider possible transmission pairs in terms of phylogenetic data and additional estimates of time since infection derived from clinical biomarkers. We develop Bayesian mixture models with an evolutionary clock as a signal component and additional mixed effects or covariate random functions describing the mixing weights to classify potential pairs into likely and unlikely transmission pairs. We demonstrate that although sources cannot be identified at the individual level with certainty, even with the additional data on time elapsed, inferences into the population-level sources of transmission are possible, and more accurate than using only phylogenetic data without time since infection estimates. We apply the proposed approach to estimate age-specific sources of HIV infection in Amsterdam tranamission networks among men who have sex with men between 2010 and 2021. This study demonstrates that infection time estimates provide informative data to characterize transmission sources, and shows how phylogenetic source attribution can then be done with multi-dimensional mixture models.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"9622802241309750"},"PeriodicalIF":1.6,"publicationDate":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143400099","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Hierarchical Bayesian bivariate spatial modeling of small area proportions with application to health survey data. 应用于健康调查数据的小面积比例的层次贝叶斯双变量空间建模。
IF 1.6 3区 医学
Statistical Methods in Medical Research Pub Date : 2025-02-11 DOI: 10.1177/09622802251316968
Hanjun Yu, Xinyi Xu, Lichao Yu
{"title":"Hierarchical Bayesian bivariate spatial modeling of small area proportions with application to health survey data.","authors":"Hanjun Yu, Xinyi Xu, Lichao Yu","doi":"10.1177/09622802251316968","DOIUrl":"https://doi.org/10.1177/09622802251316968","url":null,"abstract":"<p><p>In this article, we propose bivariate small area estimation methods for proportions based on the logit-normal mixed models with latent spatial dependence. We incorporate multivariate conditional autoregressive structures for the random effects under the hierarchical Bayesian modeling framework, and extend the methods to accommodate non-sampled regions. Posterior inference is obtained via adaptive Markov chain Monte Carlo algorithms. Extensive simulation studies are carried out to demonstrate the effectiveness of the proposed bivariate spatial models. The results suggest that the proposed methods are more efficient than the univariate and non-spatial methods in estimation and prediction, particularly when bivariate spatial dependence exists. Practical guidelines for model selection based on the simulation results are provided. We further illustrate the application of our methods by estimating the province-level heart disease rates and dyslipidemia rates among the middle-aged and elderly population in China's 31 mainland provinces in 2020.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"9622802251316968"},"PeriodicalIF":1.6,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143392034","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Statistical considerations for evaluating treatment effect under various non-proportional hazard scenarios.
IF 1.6 3区 医学
Statistical Methods in Medical Research Pub Date : 2025-02-11 DOI: 10.1177/09622802241313297
Xinyu Zhang, Erich J Greene, Ondrej Blaha, Wei Wei
{"title":"Statistical considerations for evaluating treatment effect under various non-proportional hazard scenarios.","authors":"Xinyu Zhang, Erich J Greene, Ondrej Blaha, Wei Wei","doi":"10.1177/09622802241313297","DOIUrl":"https://doi.org/10.1177/09622802241313297","url":null,"abstract":"<p><p>We conducted a systematic comparison of statistical methods used for the analysis of time-to-event outcomes under various proportional and non-proportional hazard (NPH) scenarios. Our study used data from recently published oncology trials to compare the Log-rank test, still by far the most widely used option, against some available alternatives, including the MaxCombo test, the Restricted Mean Survival Time difference test, the Generalized Gamma model and the Generalized F model. Power, type I error rate, and time-dependent bias with respect to the survival probability and median survival time were used to evaluate and compare the performance of these methods. In addition to the real data, we simulated three hypothetical scenarios with crossing hazards chosen so that the early and late effects \"cancel out\" and used them to evaluate the ability of the aforementioned methods to detect time-specific and overall treatment effects. We implemented novel metrics for assessing the time-dependent bias in treatment effect estimates to provide a more comprehensive evaluation in NPH scenarios. Recommendations under each NPH scenario are provided by examining the type I error rate, power, and time-dependent bias associated with each statistical approach.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"9622802241313297"},"PeriodicalIF":1.6,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143392085","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Causal survival embeddings: Non-parametric counterfactual inference under right-censoring.
IF 1.6 3区 医学
Statistical Methods in Medical Research Pub Date : 2025-02-11 DOI: 10.1177/09622802241311455
Carlos García Meixide, Marcos Matabuena
{"title":"Causal survival embeddings: Non-parametric counterfactual inference under right-censoring.","authors":"Carlos García Meixide, Marcos Matabuena","doi":"10.1177/09622802241311455","DOIUrl":"https://doi.org/10.1177/09622802241311455","url":null,"abstract":"<p><p>Counterfactual inference at the distributional level presents new challenges with censored targets, especially in modern healthcare problems. To mitigate selection bias in this context, we exploit the intrinsic structure of reproducing kernel Hilbert spaces (RKHS) harnessing the notion of kernel mean embedding. This enables the development of a non-parametric estimator of counterfactual survival functions. We provide rigorous theoretical guarantees regarding consistency and convergence rates of our new estimator under general hypotheses related to smoothness of the underlying RKHS. We illustrate the practical viability of our methodology through extensive simulations and a relevant case study: The SPRINT trial. Our estimatort presents a distinct perspective compared to existing methods within the literature, which often rely on semi-parametric approaches and confront limitations in causal interpretations of model parameters.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"9622802241311455"},"PeriodicalIF":1.6,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143391932","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Modeling the ratio of correlated biomarkers using copula regression.
IF 1.6 3区 医学
Statistical Methods in Medical Research Pub Date : 2025-02-11 DOI: 10.1177/09622802241313293
Moritz Berger, Nadja Klein, Michael Wagner, Matthias Schmid
{"title":"Modeling the ratio of correlated biomarkers using copula regression.","authors":"Moritz Berger, Nadja Klein, Michael Wagner, Matthias Schmid","doi":"10.1177/09622802241313293","DOIUrl":"https://doi.org/10.1177/09622802241313293","url":null,"abstract":"<p><p>Modeling the ratio of two dependent components as a function of covariates is a frequently pursued objective in observational research. Despite the high relevance of this topic in medical studies, where biomarker ratios are often used as surrogate endpoints for specific diseases, existing models are commonly based on oversimplified assumptions, assuming e.g. independence or strictly positive associations between the components. In this paper, we overcome such limitations and propose a regression model where the marginal distributions of the two components are linked by a copula. A key feature of our model is that it allows for both positive and negative associations between the components, with one of the model parameters being directly interpretable in terms of Kendall's rank correlation coefficient. We study our method theoretically, evaluate finite sample properties in a simulation study and demonstrate its efficacy in an application to diagnosis of Alzheimer's disease via ratios of amyloid-beta and total tau protein biomarkers.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"9622802241313293"},"PeriodicalIF":1.6,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143392048","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Distribution-free control charts for mixed-type data based on rank of interpoint distances.
IF 1.6 3区 医学
Statistical Methods in Medical Research Pub Date : 2025-02-10 DOI: 10.1177/09622802251316964
Guojun Liu, Jyun-You Chiang, Yajie Bai, Zhengcheng Mou
{"title":"Distribution-free control charts for mixed-type data based on rank of interpoint distances.","authors":"Guojun Liu, Jyun-You Chiang, Yajie Bai, Zhengcheng Mou","doi":"10.1177/09622802251316964","DOIUrl":"https://doi.org/10.1177/09622802251316964","url":null,"abstract":"<p><p>Multivariate control charts have found wide application in healthcare, yet they primarily cater to continuous or categorical variables. However, the emergence of mixed-type data has sparked interest in adapting traditional control charts to handle such complexity. Unfortunately, existing methods often struggle to effectively manage this complexity, particularly in scenarios with limited historical in-control data. In response, this article introduces three distribution-free control charts specifically designed for monitoring mixed-type processes. The proposed approach revolves around computing distances between observations and a specified point, thereby reducing the data to a single dimension. Subsequently, the ranks of these one-dimensional distances are leveraged to develop monitoring statistics. Furthermore, to facilitate dimensionality reduction, a novel distance measure tailored for mixed-type data is introduced. Extensive validation of our proposed method is conducted through comprehensive simulation experiments. Moreover, we demonstrate the practical applicability of the proposed method using an example related to heart disease.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"9622802251316964"},"PeriodicalIF":1.6,"publicationDate":"2025-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143391936","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Modelling extensions for multi-location studies in environmental epidemiology.
IF 1.6 3区 医学
Statistical Methods in Medical Research Pub Date : 2025-02-05 DOI: 10.1177/09622802241313284
Pierre Masselot, Antonio Gasparrini
{"title":"Modelling extensions for multi-location studies in environmental epidemiology.","authors":"Pierre Masselot, Antonio Gasparrini","doi":"10.1177/09622802241313284","DOIUrl":"10.1177/09622802241313284","url":null,"abstract":"<p><p>Multi-location studies are increasingly used in environmental epidemiology. Their application is supported by designs and statistical techniques developed in the last decades, which however have known limitations. In this contribution, we propose an improved modelling framework that addresses these issues. Specifically, this flexible framework allows the direct modelling of demographic differences across locations, defining geographical variations linked to multiple vulnerability factors, capturing spatial heterogeneity and predicting risks to new locations, and improving the assessment of uncertainty. We illustrate these new developments in an analysis of temperature-mortality associations in Italian cities, providing fully reproducible R code and data.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"9622802241313284"},"PeriodicalIF":1.6,"publicationDate":"2025-02-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143189642","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Estimating target population treatment effects in meta-analysis with individual participant-level data. 在个体参与者水平数据的meta分析中估计目标人群的治疗效果。
IF 1.6 3区 医学
Statistical Methods in Medical Research Pub Date : 2025-02-01 Epub Date: 2025-01-19 DOI: 10.1177/09622802241307642
Hwanhee Hong, Lu Liu, Elizabeth A Stuart
{"title":"Estimating target population treatment effects in meta-analysis with individual participant-level data.","authors":"Hwanhee Hong, Lu Liu, Elizabeth A Stuart","doi":"10.1177/09622802241307642","DOIUrl":"10.1177/09622802241307642","url":null,"abstract":"<p><p>Meta-analysis of randomized controlled trials is commonly used to evaluate treatments and inform policy decisions because it provides comprehensive summaries of all available evidence. However, meta-analyses are limited to draw population inference of treatment effects because they usually do not define target populations of interest specifically, and results of the individual randomized controlled trials in those meta-analyses may not generalize to the target populations. To leverage evidence from multiple randomized controlled trials in the generalizability context, we bridge the ideas from meta-analysis and causal inference. We integrate meta-analysis with causal inference approaches estimating target population average treatment effect. We evaluate the performance of the methods via simulation studies and apply the methods to generalize meta-analysis results from randomized controlled trials of treatments on schizophrenia to adults with schizophrenia who present to usual care settings in the United States. Our simulation results show that all methods perform comparably and well across different settings. The data analysis results show that the treatment effect in the target population is meaningful, although the effect size is smaller than the sample average treatment effect. We recommend applying multiple methods and comparing the results to ensure robustness, rather than relying on a single method.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"355-368"},"PeriodicalIF":1.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143012040","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Generalized Bayesian kernel machine regression. 广义贝叶斯核机器回归
IF 1.6 3区 医学
Statistical Methods in Medical Research Pub Date : 2025-02-01 Epub Date: 2024-12-12 DOI: 10.1177/09622802241280784
Xichen Mou, Hongmei Zhang, S Hasan Arshad
{"title":"Generalized Bayesian kernel machine regression.","authors":"Xichen Mou, Hongmei Zhang, S Hasan Arshad","doi":"10.1177/09622802241280784","DOIUrl":"10.1177/09622802241280784","url":null,"abstract":"<p><p>Kernel machine regression is a nonparametric regression method widely applied in biomedical and environmental health research. It employs a kernel function to measure the similarities between sample pairs, effectively identifying significant exposures and assessing their nonlinear impacts on outcomes. This article introduces an enhanced framework, the generalized Bayesian kernel machine regression. In comparison to traditional kernel machine regression, generalized Bayesian kernel machine regression provides substantial flexibility to accommodate a broader array of outcome variables, ranging from continuous to binary and count data. Simulations show generalized Bayesian kernel machine regression can successfully identify the nonlinear relationships between independent variables and outcomes of various types. In the real data analysis, we applied generalized Bayesian kernel machine regression to uncover cytosine phosphate guanine sites linked to health-related conditions such as asthma and smoking. The results identify crucial cytosine phosphate guanine sites and provide insights into their complex, nonlinear relationships with outcome variables.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"243-257"},"PeriodicalIF":1.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142819218","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Graphical methods to illustrate the nature of the relation between a continuous variable and the outcome when using restricted cubic splines with a Cox proportional hazards model. 在使用限制性三次样条和 Cox 比例危险模型时,用图形方法说明连续变量和结果之间关系的性质。
IF 1.6 3区 医学
Statistical Methods in Medical Research Pub Date : 2025-02-01 Epub Date: 2024-10-21 DOI: 10.1177/09622802241287707
Peter C Austin
{"title":"Graphical methods to illustrate the nature of the relation between a continuous variable and the outcome when using restricted cubic splines with a Cox proportional hazards model.","authors":"Peter C Austin","doi":"10.1177/09622802241287707","DOIUrl":"10.1177/09622802241287707","url":null,"abstract":"<p><p>Restricted cubic splines (RCS) allow analysts to model nonlinear relations between continuous covariates and the outcome in a regression model. When using RCS with the Cox proportional hazards model, there is no longer a single hazard ratio for the continuous variable. Instead, the hazard ratio depends on the values of the covariate for the two individuals being compared. Thus, using age as an example, when one assumes a linear relation between age and the log-hazard of the outcome there is a single hazard ratio comparing any two individuals whose age differs by 1 year. However, when allowing for a nonlinear relation between age and the log-hazard of the outcome, the hazard ratio comparing the hazard of the outcome between a 31- and a 30-year-old may differ from the hazard ratio comparing the hazard of the outcome between an 81- and an 80-year-old. We describe four methods to describe graphically the relation between a continuous variable and the outcome when using RCS with a Cox model. These graphical methods are based on plots of relative hazard ratios, cumulative incidence, hazards, and cumulative hazards against the continuous variable. Using a case study of patients presenting to hospital with heart failure and a series of mathematical derivations, we illustrate that the four methods will produce qualitatively similar conclusions about the nature of the relation between a continuous variable and the outcome. Use of these methods will allow for an intuitive communication of the nature of the relation between the variable and the outcome.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"277-285"},"PeriodicalIF":1.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11874503/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142475114","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
相关产品
×
本文献相关产品
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信