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An exhaustive ADDIS principle for online FWER control 在线 FWER 控制的详尽 ADDIS 原理
IF 1.7 3区 生物学
Biometrical Journal Pub Date : 2024-04-18 DOI: 10.1002/bimj.202300237
Lasse Fischer, Marta Bofill Roig, Werner Brannath
{"title":"An exhaustive ADDIS principle for online FWER control","authors":"Lasse Fischer,&nbsp;Marta Bofill Roig,&nbsp;Werner Brannath","doi":"10.1002/bimj.202300237","DOIUrl":"https://doi.org/10.1002/bimj.202300237","url":null,"abstract":"<p>In this paper, we consider online multiple testing with familywise error rate (FWER) control, where the probability of committing at least one type I error will remain under control while testing a possibly infinite sequence of hypotheses over time. Currently, adaptive-discard (ADDIS) procedures seem to be the most promising online procedures with FWER control in terms of power. Now, our main contribution is a uniform improvement of the ADDIS principle and thus of all ADDIS procedures. This means, the methods we propose reject as least as much hypotheses as ADDIS procedures and in some cases even more, while maintaining FWER control. In addition, we show that there is no other FWER controlling procedure that enlarges the event of rejecting any hypothesis. Finally, we apply the new principle to derive uniform improvements of the ADDIS-Spending and ADDIS-Graph.</p>","PeriodicalId":55360,"journal":{"name":"Biometrical Journal","volume":"66 3","pages":""},"PeriodicalIF":1.7,"publicationDate":"2024-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/bimj.202300237","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140619756","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","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 novel nonparametric time-dependent precision–recall curve estimator for right-censored survival data 用于右删失生存数据的新型非参数时间相关精度-召回曲线估计器
IF 1.7 3区 生物学
Biometrical Journal Pub Date : 2024-04-18 DOI: 10.1002/bimj.202300135
Kassu Mehari Beyene, Ding-Geng Chen, Yehenew Getachew Kifle
{"title":"A novel nonparametric time-dependent precision–recall curve estimator for right-censored survival data","authors":"Kassu Mehari Beyene,&nbsp;Ding-Geng Chen,&nbsp;Yehenew Getachew Kifle","doi":"10.1002/bimj.202300135","DOIUrl":"https://doi.org/10.1002/bimj.202300135","url":null,"abstract":"<p>In order to assess prognostic risk for individuals in precision health research, risk prediction models are increasingly used, in which statistical models are used to estimate the risk of future outcomes based on clinical and nonclinical characteristics. The predictive accuracy of a risk score must be assessed before it can be used in routine clinical decision making, where the receiver operator characteristic curves, precision–recall curves, and their corresponding area under the curves are commonly used metrics to evaluate the discriminatory ability of a continuous risk score. Among these the precision–recall curves have been shown to be more informative when dealing with unbalanced biomarker distribution between classes, which is common in rare event, even though except one, all existing methods are proposed for classic uncensored data. This paper is therefore to propose a novel nonparametric estimation approach for the time-dependent precision–recall curve and its associated area under the curve for right-censored data. A simulation is conducted to show the better finite sample property of the proposed estimator over the existing method and a real-world data from primary biliary cirrhosis trial is used to demonstrate the practical applicability of the proposed estimator.</p>","PeriodicalId":55360,"journal":{"name":"Biometrical Journal","volume":"66 3","pages":""},"PeriodicalIF":1.7,"publicationDate":"2024-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/bimj.202300135","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140619792","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Sharing information across patient subgroups to draw conclusions from sparse treatment networks 跨患者亚群共享信息,从稀疏的治疗网络中得出结论
IF 1.7 3区 生物学
Biometrical Journal Pub Date : 2024-04-18 DOI: 10.1002/bimj.202200316
Theodoros Evrenoglou, Silvia Metelli, Johannes-Schneider Thomas, Spyridon Siafis, Rebecca M. Turner, Stefan Leucht, Anna Chaimani
{"title":"Sharing information across patient subgroups to draw conclusions from sparse treatment networks","authors":"Theodoros Evrenoglou,&nbsp;Silvia Metelli,&nbsp;Johannes-Schneider Thomas,&nbsp;Spyridon Siafis,&nbsp;Rebecca M. Turner,&nbsp;Stefan Leucht,&nbsp;Anna Chaimani","doi":"10.1002/bimj.202200316","DOIUrl":"https://doi.org/10.1002/bimj.202200316","url":null,"abstract":"<p>Network meta-analysis (NMA) usually provides estimates of the relative effects with the highest possible precision. However, sparse networks with few available studies and limited direct evidence can arise, threatening the robustness and reliability of NMA estimates. In these cases, the limited amount of available information can hamper the formal evaluation of the underlying NMA assumptions of transitivity and consistency. In addition, NMA estimates from sparse networks are expected to be imprecise and possibly biased as they rely on large-sample approximations that are invalid in the absence of sufficient data. We propose a Bayesian framework that allows sharing of information between two networks that pertain to different population subgroups. Specifically, we use the results from a subgroup with a lot of direct evidence (a dense network) to construct informative priors for the relative effects in the target subgroup (a sparse network). This is a two-stage approach where at the first stage, we extrapolate the results of the dense network to those expected from the sparse network. This takes place by using a modified hierarchical NMA model where we add a location parameter that shifts the distribution of the relative effects to make them applicable to the target population. At the second stage, these extrapolated results are used as prior information for the sparse network. We illustrate our approach through a motivating example of psychiatric patients. Our approach results in more precise and robust estimates of the relative effects and can adequately inform clinical practice in presence of sparse networks.</p>","PeriodicalId":55360,"journal":{"name":"Biometrical Journal","volume":"66 3","pages":""},"PeriodicalIF":1.7,"publicationDate":"2024-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/bimj.202200316","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140619829","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Sample size planning for rank-based multiple contrast tests 基于等级的多重对比测试的样本量规划
IF 1.7 3区 生物学
Biometrical Journal Pub Date : 2024-04-18 DOI: 10.1002/bimj.202300240
Anna Pöhlmann, Edgar Brunner, Frank Konietschke
{"title":"Sample size planning for rank-based multiple contrast tests","authors":"Anna Pöhlmann,&nbsp;Edgar Brunner,&nbsp;Frank Konietschke","doi":"10.1002/bimj.202300240","DOIUrl":"https://doi.org/10.1002/bimj.202300240","url":null,"abstract":"<p>Rank methods are well-established tools for comparing two or multiple (independent) groups. Statistical planning methods for the computing the required sample size(s) to detect a specific alternative with predefined power are lacking. In the present paper, we develop numerical algorithms for sample size planning of pseudo-rank-based multiple contrast tests. We discuss the treatment effects and different ways to approximate variance parameters within the estimation scheme. We further compare pairwise with global rank methods in detail. Extensive simulation studies show that the sample size estimators are accurate. A real data example illustrates the application of the methods.</p>","PeriodicalId":55360,"journal":{"name":"Biometrical Journal","volume":"66 3","pages":""},"PeriodicalIF":1.7,"publicationDate":"2024-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/bimj.202300240","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140619793","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Mapping QTL controlling count traits with excess zeros and ones using a zero-and-one-inflated generalized Poisson regression model 利用零一膨胀广义泊松回归模型绘制控制具有过多零和一的计数性状的 QTL 图谱
IF 1.7 3区 生物学
Biometrical Journal Pub Date : 2024-04-14 DOI: 10.1002/bimj.202200342
Jinling Chi, Jimin Ye, Ying Zhou
{"title":"Mapping QTL controlling count traits with excess zeros and ones using a zero-and-one-inflated generalized Poisson regression model","authors":"Jinling Chi,&nbsp;Jimin Ye,&nbsp;Ying Zhou","doi":"10.1002/bimj.202200342","DOIUrl":"https://doi.org/10.1002/bimj.202200342","url":null,"abstract":"<p>The research on the quantitative trait locus (QTL) mapping of count data has aroused the wide attention of researchers. There are frequent problems in applied research that limit the application of the conventional Poisson model in the analysis of count phenotypes, which include the overdispersion and excess zeros and ones. In this article, a novel model, that is, the zero-and-one-inflated generalized Poisson (ZOIGP) model, is proposed to deal with these problems. Based on the proposed model, a score test is performed for the inflation parameter, in which the ZOIGP model with a constant proportion of excess zeros and ones is compared with a standard generalized Poisson model. To illustrate the practicability of the ZOIGP model, we extend it to the QTL interval mapping application that underpins count phenotype with excess zeros and excess ones. The genetic effects are estimated utilizing the expectation–maximization algorithm embedded with the Newton–Raphson algorithm, and the genome-wide scan and likelihood ratio test is performed to map and test the potential QTLs. The statistical properties exhibited by the proposed method are investigated through simulation. Finally, a real data analysis example is used to illustrate the utility of the proposed method for QTL mapping.</p>","PeriodicalId":55360,"journal":{"name":"Biometrical Journal","volume":"66 3","pages":""},"PeriodicalIF":1.7,"publicationDate":"2024-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140552879","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Bootstrap tests for simultaneous monotone ordering of effects in a two-way ANOVA 双向方差分析中效应同时单调排序的 Bootstrap 检验
IF 1.7 3区 生物学
Biometrical Journal Pub Date : 2024-04-05 DOI: 10.1002/bimj.202300238
Raju Dey, Anjana Mondal, Somesh Kumar
{"title":"Bootstrap tests for simultaneous monotone ordering of effects in a two-way ANOVA","authors":"Raju Dey,&nbsp;Anjana Mondal,&nbsp;Somesh Kumar","doi":"10.1002/bimj.202300238","DOIUrl":"https://doi.org/10.1002/bimj.202300238","url":null,"abstract":"<p>In a two-way additive analysis of variance (ANOVA) model, we consider the problem of testing for homogeneity of both row and column effects against their simultaneous ordering. The error variances are assumed to be heterogeneous with unbalanced samples in each cell. Two simultaneous test procedures are developed—the first one using the likelihood ratio test (LRT) statistics of two independent hypotheses and another based on the consecutive pairwise differences of estimators of effects. The parametric bootstrap (PB) approach is used to find critical points of both the tests and the asymptotic accuracy of the bootstrap is established. An extensive simulation study shows that the proposed tests achieve the nominal size and have very good power performance. The robustness of the tests is also analyzed under deviation from normality. An “R” package is developed and shared on “GitHub” for ease of implementation of users. The proposed tests are illustrated using a real data set on the mortality due to alcoholic liver disease and it is shown that age and gender have a significant impact on the increasing incidence of mortality.</p>","PeriodicalId":55360,"journal":{"name":"Biometrical Journal","volume":"66 3","pages":""},"PeriodicalIF":1.7,"publicationDate":"2024-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140351577","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Beta spending function based on conditional power in group sequential design 基于分组顺序设计中条件功率的贝塔支出函数
IF 1.7 3区 生物学
Biometrical Journal Pub Date : 2024-04-05 DOI: 10.1002/bimj.202300094
Senmiao Ni, Zihang Zhong, Zhiwei Jiang, Yang Zhao, Jingwei Wu, Hao Yu, Jianling Bai
{"title":"Beta spending function based on conditional power in group sequential design","authors":"Senmiao Ni,&nbsp;Zihang Zhong,&nbsp;Zhiwei Jiang,&nbsp;Yang Zhao,&nbsp;Jingwei Wu,&nbsp;Hao Yu,&nbsp;Jianling Bai","doi":"10.1002/bimj.202300094","DOIUrl":"https://doi.org/10.1002/bimj.202300094","url":null,"abstract":"<p>Conditional power (CP) serves as a widely utilized approach for futility monitoring in group sequential designs. However, adopting the CP methods may lead to inadequate control of the type II error rate at the desired level. In this study, we introduce a flexible beta spending function tailored to regulate the type II error rate while employing CP based on a predetermined standardized effect size for futility monitoring (a so-called CP-beta spending function). This function delineates the expenditure of type II error rate across the entirety of the trial. Unlike other existing beta spending functions, the CP-beta spending function seamlessly incorporates beta spending concept into the CP framework, facilitating precise stagewise control of the type II error rate during futility monitoring. In addition, the stopping boundaries derived from the CP-beta spending function can be calculated via integration akin to other traditional beta spending function methods. Furthermore, the proposed CP-beta spending function accommodates various thresholds on the CP-scale at different stages of the trial, ensuring its adaptability across different information time scenarios. These attributes render the CP-beta spending function competitive among other forms of beta spending functions, making it applicable to any trials in group sequential designs with straightforward implementation. Both simulation study and example from an acute ischemic stroke trial demonstrate that the proposed method accurately captures expected power, even when the initially determined sample size does not consider futility stopping, and exhibits a good performance in maintaining overall type I error rates for evident futility.</p>","PeriodicalId":55360,"journal":{"name":"Biometrical Journal","volume":"66 3","pages":""},"PeriodicalIF":1.7,"publicationDate":"2024-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140351594","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A framework to select tuning parameters for nonparametric derivative estimation 为非参数导数估计选择调整参数的框架
IF 1.7 3区 生物学
Biometrical Journal Pub Date : 2024-04-05 DOI: 10.1002/bimj.202300039
Sisheng Liu, Xiaoli Kong
{"title":"A framework to select tuning parameters for nonparametric derivative estimation","authors":"Sisheng Liu,&nbsp;Xiaoli Kong","doi":"10.1002/bimj.202300039","DOIUrl":"https://doi.org/10.1002/bimj.202300039","url":null,"abstract":"<p>In this paper, we propose a general framework to select tuning parameters for the nonparametric derivative estimation. The new framework broadens the scope of the previously proposed generalized <span></span><math>\u0000 <semantics>\u0000 <msub>\u0000 <mi>C</mi>\u0000 <mi>p</mi>\u0000 </msub>\u0000 <annotation>$C_p$</annotation>\u0000 </semantics></math> criterion by replacing the empirical derivative with any other linear nonparametric smoother. We provide the theoretical support of the proposed derivative estimation in a random design and justify it through simulation studies. The practical application of the proposed framework is demonstrated in the study of the age effect on hippocampal gray matter volume in healthy adults from the IXI dataset and the study of the effect of age and body mass index on blood pressure from the Pima Indians dataset.</p>","PeriodicalId":55360,"journal":{"name":"Biometrical Journal","volume":"66 3","pages":""},"PeriodicalIF":1.7,"publicationDate":"2024-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140351595","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Issue Information: Biometrical Journal 3'24 期刊信息:生物计量学杂志 3'24
IF 1.7 3区 生物学
Biometrical Journal Pub Date : 2024-04-04 DOI: 10.1002/bimj.202470003
{"title":"Issue Information: Biometrical Journal 3'24","authors":"","doi":"10.1002/bimj.202470003","DOIUrl":"https://doi.org/10.1002/bimj.202470003","url":null,"abstract":"","PeriodicalId":55360,"journal":{"name":"Biometrical Journal","volume":"66 3","pages":""},"PeriodicalIF":1.7,"publicationDate":"2024-04-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/bimj.202470003","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140348595","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","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 model-based reduced major axis regression 基于贝叶斯模型的还原主轴回归
IF 1.7 3区 生物学
Biometrical Journal Pub Date : 2024-04-04 DOI: 10.1002/bimj.202300279
Zhihua Ma, Ming-Hui Chen
{"title":"A Bayesian model-based reduced major axis regression","authors":"Zhihua Ma,&nbsp;Ming-Hui Chen","doi":"10.1002/bimj.202300279","DOIUrl":"https://doi.org/10.1002/bimj.202300279","url":null,"abstract":"<p>Reduced major axis (RMA) regression, widely used in the fields of zoology, botany, ecology, biology, spectroscopy, and among others, outweighs the ordinary least square regression by relaxing the assumption that the covariates are without measurement errors. A Bayesian implementation of the RMA regression is presented in this paper, and the equivalence of the estimates of the parameters under the Bayesian and the frequentist frameworks is proved. This model-based Bayesian RMA method is advantageous since the posterior estimates, the standard deviations, as well as the credible intervals of the estimates can be obtained through Markov chain Monte Carlo methods directly. In addition, it is straightforward to extend to the multivariate RMA case. The performance of the Bayesian RMA approach is evaluated in the simulation study, and, finally, the proposed method is applied to analyze a dataset in the plantation.</p>","PeriodicalId":55360,"journal":{"name":"Biometrical Journal","volume":"66 3","pages":""},"PeriodicalIF":1.7,"publicationDate":"2024-04-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140348596","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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