Biometrics最新文献_第7页

Unit information Dirichlet process prior. 单位信息 Dirichlet 过程先验

IF 1.4 4区数学

Biometrics Pub Date : 2024-07-01 DOI: 10.1093/biomtc/ujae091

Jiaqi Gu, Guosheng Yin

引用次数: 0

Sensitivity analysis for publication bias in meta-analysis of sparse data based on exact likelihood. 基于精确似然法的稀疏数据荟萃分析中出版偏差的敏感性分析。

IF 1.4 4区数学

Biometrics Pub Date : 2024-07-01 DOI: 10.1093/biomtc/ujae092

Taojun Hu, Yi Zhou, Satoshi Hattori

{"title":"Sensitivity analysis for publication bias in meta-analysis of sparse data based on exact likelihood.","authors":"Taojun Hu, Yi Zhou, Satoshi Hattori","doi":"10.1093/biomtc/ujae092","DOIUrl":"10.1093/biomtc/ujae092","url":null,"abstract":"Meta-analysis is a powerful tool to synthesize findings from multiple studies. The normal-normal random-effects model is widely used to account for between-study heterogeneity. However, meta-analyses of sparse data, which may arise when the event rate is low for binary or count outcomes, pose a challenge to the normal-normal random-effects model in the accuracy and stability in inference since the normal approximation in the within-study model may not be good. To reduce bias arising from data sparsity, the generalized linear mixed model can be used by replacing the approximate normal within-study model with an exact model. Publication bias is one of the most serious threats in meta-analysis. Several quantitative sensitivity analysis methods for evaluating the potential impacts of selective publication are available for the normal-normal random-effects model. We propose a sensitivity analysis method by extending the likelihood-based sensitivity analysis with the $t$-statistic selection function of Copas to several generalized linear mixed-effects models. Through applications of our proposed method to several real-world meta-analyses and simulation studies, the proposed method was proven to outperform the likelihood-based sensitivity analysis based on the normal-normal model. The proposed method would give useful guidance to address publication bias in the meta-analysis of sparse data.","PeriodicalId":8930,"journal":{"name":"Biometrics","volume":null,"pages":null},"PeriodicalIF":1.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142280084","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Discussion on "LEAP: the latent exchangeability prior for borrowing information from historical data" by Ethan M. Alt, Xiuya Chang, Xun Jiang, Qing Liu, May Mo, H. Amy Xia, and Joseph G. Ibrahim. 关于 Ethan M. Alt、Xiuya Chang、Xun Jiang、Qing Liu、May Mo、H. Amy Xia 和 Joseph G. Ibrahim 所著《LEAP：从历史数据中借用信息的潜在可交换性先验》的讨论。

IF 1.4 4区数学

Biometrics Pub Date : 2024-07-01 DOI: 10.1093/biomtc/ujae085

Darren Scott, Alex Lewin

引用次数: 0

Reduced-rank clustered coefficient regression for addressing multicollinearity in heterogeneous coefficient estimation. 用于解决异质系数估算中多重共线性问题的降序聚类系数回归。

IF 1.4 4区数学

Biometrics Pub Date : 2024-07-01 DOI: 10.1093/biomtc/ujae076

Yan Zhong, Kejun He, Gefei Li

引用次数: 0

Visibility graph-based covariance functions for scalable spatial analysis in non-convex partially Euclidean domains. 基于可见性图的协方差函数，用于非凸部分欧几里得域的可扩展空间分析。

IF 1.4 4区数学

Biometrics Pub Date : 2024-07-01 DOI: 10.1093/biomtc/ujae089

Brian Gilbert, Abhirup Datta

{"title":"Visibility graph-based covariance functions for scalable spatial analysis in non-convex partially Euclidean domains.","authors":"Brian Gilbert, Abhirup Datta","doi":"10.1093/biomtc/ujae089","DOIUrl":"https://doi.org/10.1093/biomtc/ujae089","url":null,"abstract":"We present a new method for constructing valid covariance functions of Gaussian processes for spatial analysis in irregular, non-convex domains such as bodies of water. Standard covariance functions based on geodesic distances are not guaranteed to be positive definite on such domains, while existing non-Euclidean approaches fail to respect the partially Euclidean nature of these domains where the geodesic distance agrees with the Euclidean distances for some pairs of points. Using a visibility graph on the domain, we propose a class of covariance functions that preserve Euclidean-based covariances between points that are connected in the domain while incorporating the non-convex geometry of the domain via conditional independence relationships. We show that the proposed method preserves the partially Euclidean nature of the intrinsic geometry on the domain while maintaining validity (positive definiteness) and marginal stationarity of the covariance function over the entire parameter space, properties which are not always fulfilled by existing approaches to construct covariance functions on non-convex domains. We provide useful approximations to improve computational efficiency, resulting in a scalable algorithm. We compare the performance of our method with those of competing state-of-the-art methods using simulation studies on synthetic non-convex domains. The method is applied to data regarding acidity levels in the Chesapeake Bay, showing its potential for ecological monitoring in real-world spatial applications on irregular domains.","PeriodicalId":8930,"journal":{"name":"Biometrics","volume":null,"pages":null},"PeriodicalIF":1.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142153112","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Integrating external summary information in the presence of prior probability shift: an application to assessing essential hypertension. 在先验概率偏移的情况下整合外部摘要信息：在评估本质性高血压中的应用。

IF 1.4 4区数学

Biometrics Pub Date : 2024-07-01 DOI: 10.1093/biomtc/ujae090

Chixiang Chen, Peisong Han, Shuo Chen, Michelle Shardell, Jing Qin

{"title":"Integrating external summary information in the presence of prior probability shift: an application to assessing essential hypertension.","authors":"Chixiang Chen, Peisong Han, Shuo Chen, Michelle Shardell, Jing Qin","doi":"10.1093/biomtc/ujae090","DOIUrl":"10.1093/biomtc/ujae090","url":null,"abstract":"Recent years have witnessed a rise in the popularity of information integration without sharing of raw data. By leveraging and incorporating summary information from external sources, internal studies can achieve enhanced estimation efficiency and prediction accuracy. However, a noteworthy challenge in utilizing summary-level information is accommodating the inherent heterogeneity across diverse data sources. In this study, we delve into the issue of prior probability shift between two cohorts, wherein the difference of two data distributions depends on the outcome. We introduce a novel semi-parametric constrained optimization-based approach to integrate information within this framework, which has not been extensively explored in existing literature. Our proposed method tackles the prior probability shift by introducing the outcome-dependent selection function and effectively addresses the estimation uncertainty associated with summary information from the external source. Our approach facilitates valid inference even in the absence of a known variance-covariance estimate from the external source. Through extensive simulation studies, we observe the superiority of our method over existing ones, showcasing minimal estimation bias and reduced variance for both binary and continuous outcomes. We further demonstrate the utility of our method through its application in investigating risk factors related to essential hypertension, where the reduced estimation variability is observed after integrating summary information from an external data.","PeriodicalId":8930,"journal":{"name":"Biometrics","volume":null,"pages":null},"PeriodicalIF":1.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11381951/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142153110","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

A Bayesian latent-subgroup platform design for dose optimization. 用于剂量优化的贝叶斯潜在子组平台设计。

IF 1.9 4区数学

Biometrics Pub Date : 2024-07-01 DOI: 10.1093/biomtc/ujae093

Rongji Mu,Xiaojiang Zhan,Rui Sammi Tang,Ying Yuan

{"title":"A Bayesian latent-subgroup platform design for dose optimization.","authors":"Rongji Mu,Xiaojiang Zhan,Rui Sammi Tang,Ying Yuan","doi":"10.1093/biomtc/ujae093","DOIUrl":"https://doi.org/10.1093/biomtc/ujae093","url":null,"abstract":"The US Food and Drug Administration launched Project Optimus to reform the dose optimization and dose selection paradigm in oncology drug development, calling for the paradigm shift from finding the maximum tolerated dose to the identification of optimal biological dose (OBD). Motivated by a real-world drug development program, we propose a master-protocol-based platform trial design to simultaneously identify OBDs of a new drug, combined with standards of care or other novel agents, in multiple indications. We propose a Bayesian latent subgroup model to accommodate the treatment heterogeneity across indications, and employ Bayesian hierarchical models to borrow information within subgroups. At each interim analysis, we update the subgroup membership and dose-toxicity and -efficacy estimates, as well as the estimate of the utility for risk-benefit tradeoff, based on the observed data across treatment arms to inform the arm-specific decision of dose escalation and de-escalation and identify the OBD for each arm of a combination partner and an indication. The simulation study shows that the proposed design has desirable operating characteristics, providing a highly flexible and efficient way for dose optimization. The design has great potential to shorten the drug development timeline, save costs by reducing overlapping infrastructure, and speed up regulatory approval.","PeriodicalId":8930,"journal":{"name":"Biometrics","volume":null,"pages":null},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142200450","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Designing cancer screening trials for reduction in late-stage cancer incidence. 设计癌症筛查试验，降低晚期癌症发病率。

IF 1.4 4区数学

Biometrics Pub Date : 2024-07-01 DOI: 10.1093/biomtc/ujae097

Kehao Zhu, Ying-Qi Zhao, Yingye Zheng

{"title":"Designing cancer screening trials for reduction in late-stage cancer incidence.","authors":"Kehao Zhu, Ying-Qi Zhao, Yingye Zheng","doi":"10.1093/biomtc/ujae097","DOIUrl":"https://doi.org/10.1093/biomtc/ujae097","url":null,"abstract":"Before implementing a biomarker test for early cancer detection into routine clinical care, the test must demonstrate clinical utility, that is, the test results should lead to clinical actions that positively affect patient-relevant outcomes. Unlike therapeutical trials for patients diagnosed with cancer, designing a randomized controlled trial (RCT) to demonstrate the clinical utility of an early detection biomarker with mortality and related endpoints poses unique challenges. The hurdles stem from the prolonged natural progression of the disease and the lack of information regarding the time-varying screening effect on the target asymptomatic population. To facilitate the study design of screening trials, we propose using a generic multistate disease history model and derive model-based effect sizes. The model links key performance metrics of the test, such as sensitivity, to primary endpoints like the incidence of late-stage cancer. It also incorporates the practical implementation of the biomarker-testing program in real-world scenarios. Based on the chronological time scale aligned with RCT, our method allows the assessment of study powers based on key features of the new program, including the test sensitivity, the length of follow-up, and the number and frequency of repeated tests. The calculation tool from the proposed method will enable practitioners to perform realistic and quick evaluations when strategizing screening trials for specific diseases. We use numerical examples based on the National Lung Screening Trial to demonstrate the method.","PeriodicalId":8930,"journal":{"name":"Biometrics","volume":null,"pages":null},"PeriodicalIF":1.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11413908/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142280080","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Estimating the size of a closed population by modeling latent and observed heterogeneity. 通过对潜在和观察到的异质性建模来估算封闭种群的规模。

IF 1.9 4区数学

Biometrics Pub Date : 2024-03-27 DOI: 10.1093/biomtc/ujae017

Francesco Bartolucci, Antonio Forcina

{"title":"Estimating the size of a closed population by modeling latent and observed heterogeneity.","authors":"Francesco Bartolucci, Antonio Forcina","doi":"10.1093/biomtc/ujae017","DOIUrl":"10.1093/biomtc/ujae017","url":null,"abstract":"The paper extends the empirical likelihood (EL) approach of Liu et al. to a new and very flexible family of latent class models for capture-recapture data also allowing for serial dependence on previous capture history, conditionally on latent type and covariates. The EL approach allows to estimate the overall population size directly rather than by adding estimates conditional to covariate configurations. A Fisher-scoring algorithm for maximum likelihood estimation is proposed and a more efficient alternative to the traditional EL approach for estimating the non-parametric component is introduced; this allows us to show that the mapping between the non-parametric distribution of the covariates and the probabilities of being never captured is one-to-one and strictly increasing. Asymptotic results are outlined, and a procedure for constructing profile likelihood confidence intervals for the population size is presented. Two examples based on real data are used to illustrate the proposed approach and a simulation study indicates that, when estimating the overall undercount, the method proposed here is substantially more efficient than the one based on conditional maximum likelihood estimation, especially when the sample size is not sufficiently large.","PeriodicalId":8930,"journal":{"name":"Biometrics","volume":null,"pages":null},"PeriodicalIF":1.9,"publicationDate":"2024-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140304679","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Robustness of response-adaptive randomization. 反应自适应随机化的稳健性。

IF 1.9 4区数学

Biometrics Pub Date : 2024-03-27 DOI: 10.1093/biomtc/ujae049

Xiaoqing Ye, Feifang Hu, Wei Ma

{"title":"Robustness of response-adaptive randomization.","authors":"Xiaoqing Ye, Feifang Hu, Wei Ma","doi":"10.1093/biomtc/ujae049","DOIUrl":"https://doi.org/10.1093/biomtc/ujae049","url":null,"abstract":"Doubly adaptive biased coin design (DBCD), a response-adaptive randomization scheme, aims to skew subject assignment probabilities based on accrued responses for ethical considerations. Recent years have seen substantial advances in understanding DBCD's theoretical properties, assuming correct model specification for the responses. However, concerns have been raised about the impact of model misspecification on its design and analysis. In this paper, we assess the robustness to both design model misspecification and analysis model misspecification under DBCD. On one hand, we confirm that the consistency and asymptotic normality of the allocation proportions can be preserved, even when the responses follow a distribution other than the one imposed by the design model during the implementation of DBCD. On the other hand, we extensively investigate three commonly used linear regression models for estimating and inferring the treatment effect, namely difference-in-means, analysis of covariance (ANCOVA) I, and ANCOVA II. By allowing these regression models to be arbitrarily misspecified, thereby not reflecting the true data generating process, we derive the consistency and asymptotic normality of the treatment effect estimators evaluated from the three models. The asymptotic properties show that the ANCOVA II model, which takes covariate-by-treatment interaction terms into account, yields the most efficient estimator. These results can provide theoretical support for using DBCD in scenarios involving model misspecification, thereby promoting the widespread application of this randomization procedure.","PeriodicalId":8930,"journal":{"name":"Biometrics","volume":null,"pages":null},"PeriodicalIF":1.9,"publicationDate":"2024-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141178772","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0