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Estimation of treatment effect among treatment responders with a time-to-event endpoint 以时间为终点估算治疗应答者的治疗效果
IF 1 4区 数学 Q3 Mathematics Pub Date : 2024-01-18 DOI: 10.1111/sjos.12706
Andreas Nordland, Torben Martinussen
In a placebo-controlled clinical study one may calculate the average treatment effect to convey the effect of the active treatment on some outcome. However, if it is speculated that the treatment only has an effect if the patient responds to the treatment defined by a certain biomarker response, then it is arguably more relevant to estimate the treatment effect among such responders. We present such a causal parameter that is based on principal stratification and is identified under the exclusion of a treatment effect among the non-responders. We focus on time-;to-event outcomes allowing for right censoring, and construct a doubly robust and efficient estimator based on the associated efficient influence function. The properties of the estimator are showcased in a simulation study and the methodology is applied to the Leader trial investigating the effect of liraglutide on the occurrence of cardiovascular events.
在安慰剂对照临床研究中,我们可以计算平均治疗效果,以反映积极治疗对某些结果的影响。但是,如果推测只有当患者对治疗产生反应时,治疗才会产生效果,而这种反应是由某种生物标记物的反应所决定的,那么估计这种反应者的治疗效果可能更有意义。我们提出了这样一个基于主分层的因果参数,它是在排除非应答者治疗效果的情况下确定的。我们将重点放在允许右删减的时间-事件结果上,并根据相关的有效影响函数构建了一个双重稳健有效的估计器。我们在模拟研究中展示了该估计器的特性,并将该方法应用于调查利拉鲁肽对心血管事件发生影响的 Leader 试验。
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引用次数: 0
Nonparametric plug-in classifier for multiclass classification of S.D.E. paths 用于 S.D.E. 路径多类分类的非参数插件分类器
IF 1 4区 数学 Q3 Mathematics Pub Date : 2024-01-15 DOI: 10.1111/sjos.12702
Christophe Denis, Charlotte Dion-Blanc, Eddy Ella-Mintsa, Viet Chi Tran
We study the multiclass classification problem where the features come from a mixture of time-homogeneous diffusion.Specifically, the classes are discriminated by their drift functions while the diffusion coefficient is common to all classes and unknown.In this framework, we build a plug-in classifier which relies on nonparamateric estimators of the drift and diffusion functions.We first establish the consistency of our classification procedure under mild assumptions and then provide rates of convergence under different setof assumptions. Finally, a numerical study supports our theoretical findings.
我们研究的是多类分类问题,其中的特征来自于时间同质扩散的混合物。具体来说,类是通过它们的漂移函数来区分的,而扩散系数则是所有类共有的未知数。在这个框架下,我们建立了一个插件分类器,它依赖于漂移和扩散函数的非参数估计器。最后,一项数值研究支持了我们的理论发现。
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引用次数: 0
The effect of the working correlation on fitting models to longitudinal data 工作相关性对纵向数据模型拟合的影响
IF 1 4区 数学 Q3 Mathematics Pub Date : 2024-01-02 DOI: 10.1111/sjos.12704
Samuel Muller, Suojin Wang, A. H. Welsh
We present a detailed discussion of the theoretical properties of quadratic inference function estimators of the parameters in marginal linear regression models. We consider the effect of the choice of working correlation on fundamental questions including the existence of quadratic inference function estimators, their relationship with generalized estimating equations estimators, and the robustness and asymptotic relative efficiency of quadratic inference function and generalized estimating equations estimators. We show that the quadratic inference function estimators do not always exist and propose a way to handle this. We then show that they have unbounded influence functions and can be more or less asymptotically efficient than generalized estimating equations estimators. We also present empirical evidence to demonstrate these results. We conclude that the choice of working correlation can have surprisingly large effects.
我们详细讨论了边际线性回归模型参数二次推理函数估计器的理论特性。我们考虑了工作相关性的选择对基本问题的影响,包括二次推理函数估计器的存在性、它们与广义估计方程估计器的关系,以及二次推理函数估计器和广义估计方程估计器的稳健性和渐进相对效率。我们证明了二次推理函数估计子并不总是存在,并提出了一种处理方法。然后,我们证明它们的影响函数是无界的,其渐近效率可能高于或低于广义估计方程估计器。我们还提出了经验证据来证明这些结果。我们的结论是,工作相关性的选择可能会产生令人惊讶的巨大影响。
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引用次数: 0
Log-density gradient covariance and automatic metric tensors for Riemann manifold Monte Carlo methods† 黎曼流形蒙特卡罗方法的对数密度梯度协方差和自动度量张量†。
IF 1 4区 数学 Q3 Mathematics Pub Date : 2023-12-26 DOI: 10.1111/sjos.12705
Tore Selland Kleppe
A metric tensor for Riemann manifold Monte Carlo particularly suited for non-linear Bayesian hierarchical models is proposed. The metric tensor is built from symmetric positive semidefinite log-density gradient covariance (LGC) matrices, which are also proposed and further explored here. The LGCs generalize the Fisher information matrix by measuring the joint information content and dependence structure of both a random variable and the parameters of said variable. Consequently, positive definite Fisher/LGC-based metric tensors may be constructed not only from the observation likelihoods as is current practice, but also from arbitrarily complicated non-linear prior/latent variable structures, provided the LGC may be derived for each conditional distribution used to construct said structures. The proposed methodology is highly automatic and allows for exploitation of any sparsity associated with the model in question. When implemented in conjunction with a Riemann manifold variant of the recently proposed numerical generalized randomized Hamiltonian Monte Carlo processes, the proposed methodology is highly competitive, in particular for the more challenging target distributions associated with Bayesian hierarchical models.
本文提出了一种特别适用于非线性贝叶斯层次模型的黎曼流形蒙特卡罗度量张量。该度量张量由对称正半有限对数密度梯度协方差(LGC)矩阵构建而成。LGC 通过测量随机变量和所述变量参数的联合信息含量和依赖结构,对费雪信息矩阵进行了概括。因此,基于正定费雪/LGC 的度量张量不仅可以像目前的做法那样从观测似然构建,还可以从任意复杂的非线性先验/后验变量结构构建,前提是可以为用于构建上述结构的每个条件分布导出 LGC。所提出的方法具有很高的自动性,可以利用与相关模型有关的任何稀疏性。当与最近提出的数值广义随机哈密尔顿蒙特卡罗过程的黎曼流形变体结合使用时,所提出的方法具有很强的竞争力,特别是对于与贝叶斯层次模型相关的更具挑战性的目标分布。
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引用次数: 0
Characterization of valid auxiliary functions for representations of extreme value distributions and their max-domains of attraction 极值分布及其最大吸引域表示的有效辅助函数的特征
IF 1 4区 数学 Q3 Mathematics Pub Date : 2023-12-26 DOI: 10.1111/sjos.12701
Miriam Isabel Seifert
In this paper we study two important representations for extreme value distributions and their max-domains of attraction (MDA), namely von Mises representation (vMR) and variation representation (VR), which are convenient ways to gain limit results. Both VR and vMR are defined via so-called auxiliary functions ψ. Up to now, however, the set of valid auxiliary functions for vMR has neither been characterized completely nor separated from those for VR. We contribute to the current literature by introducing “universal” auxiliary functions which are valid for both VR and vMR representations for the entire MDA distribution families. Then we identify exactly the sets of valid auxiliary functions for both VR and vMR. Moreover, we propose a method for finding appropriate auxiliary functions with analytically simple structure and provide them for several important distributions.
本文研究了极值分布及其最大吸引域(MDA)的两个重要表示,即冯-米塞斯表示(vMR)和变异表示(VR),它们是获得极限结果的便捷方法。VR 和 vMR 都是通过所谓的辅助函数 ψ 来定义的。然而,到目前为止,vMR 的有效辅助函数集既没有被完全描述,也没有与 VR 的有效辅助函数集区分开来。我们通过引入对整个 MDA 分布族的 VR 和 vMR 表示都有效的 "通用 "辅助函数,为现有文献做出了贡献。然后,我们精确地确定了 VR 和 vMR 的有效辅助函数集。此外,我们还提出了一种寻找适当辅助函数的方法,该方法具有简单的分析结构,并提供了几种重要分布的辅助函数。
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引用次数: 0
Consistent covariances estimation for stratum imbalances under minimization method for covariate-adaptive randomization 协方差自适应随机化最小化方法下分层不平衡的一致协方差估计
IF 1 4区 数学 Q3 Mathematics Pub Date : 2023-12-26 DOI: 10.1111/sjos.12703
Zixuan Zhao, Yanglei Song, Wenyu Jiang, Dongsheng Tu
Pocock and Simon's minimization method is a popular approach for covariate-adaptive randomization in clinical trials. Valid statistical inference with data collected under the minimization method requires the knowledge of the limiting covariance matrix of within-stratum imbalances, whose existence is only recently established. In this work, we propose a bootstrap-based estimator for this limit and establish its consistency, in particular, by Le Cam's third lemma. As an application, we consider in simulation studies adjustments to existing robust tests for treatment effects with survival data by the proposed estimator. It shows that the adjusted tests achieve a size close to the nominal level, and unlike other designs, the robust tests without adjustment may have an asymptotic size inflation issue under the minimization method.
波科克和西蒙的最小化方法是临床试验中一种常用的协方差自适应随机方法。使用最小化方法收集的数据进行有效的统计推断,需要知道层内不平衡的极限协方差矩阵,而该矩阵的存在最近才被证实。在这项工作中,我们提出了一种基于自举法的极限估计方法,并特别通过 Le Cam 的第三个 Lemma 建立了其一致性。作为一项应用,我们在模拟研究中考虑用提出的估计器调整现有的生存数据治疗效果稳健检验。结果表明,调整后的检验规模接近名义水平,与其他设计不同的是,在最小化方法下,未经调整的稳健检验可能存在规模膨胀的渐近问题。
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引用次数: 0
Confidence Bands for Survival Curves from Outcome-Dependent Stratified Samples 依赖结果的分层抽样生存曲线的置信带
IF 1 4区 数学 Q3 Mathematics Pub Date : 2023-12-21 DOI: 10.1111/sjos.12700
Takumi Saegusa, Peter Nandori
We consider the construction of confidence bands for survival curves under the outcome-dependent stratified sampling. A main challenge of this design is that data are a biased dependent sample due to stratification and sampling without replacement. Most literature on regression approximates this design by Bernoulli sampling but variance is generally overestimated. Even with this approximation, the limiting distribution of the inverse probability weighted Kaplan-Meier estimator involves a general Gaussian process, and hence quantiles of its supremum is not analytically available. In this paper, we provide a rigorous asymptotic theory for the weighted Kaplan-Meier estimator accounting for dependence in the sample. We propose the novel hybrid method to both simulate and bootstrap parts of the limiting process to compute confidence bands with asymptotically correct coverage probability. Simulation study indicates that the proposed bands are appropriate for practical use. A Wilms tumor example is presented.
我们考虑了在依赖结果的分层抽样下构建生存曲线的置信区间。这种设计的主要挑战在于,由于分层抽样和无替换抽样,数据是有偏差的依赖样本。大多数关于回归的文献都用伯努利抽样来近似这种设计,但方差通常被高估。即使采用了这种近似方法,反概率加权卡普兰-梅耶估计器的极限分布也涉及一般高斯过程,因此无法对其上确值进行分析。在本文中,我们为加权卡普兰-梅耶估计器提供了严格的渐近理论,并考虑了样本中的依赖性。我们提出了一种新颖的混合方法,既能模拟极限过程,又能引导极限过程,从而计算出具有渐近正确覆盖概率的置信带。模拟研究表明,所提出的置信带适合实际应用。下面以 Wilms 肿瘤为例进行说明。
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引用次数: 0
G-optimal grid designs for kriging models 克里金模型的 G 优化网格设计
IF 1 4区 数学 Q3 Mathematics Pub Date : 2023-12-11 DOI: 10.1111/sjos.12699
Subhadra Dasgupta, Siuli Mukhopadhyay, Jonathan Keith
This work is focused on finding G -optimal designs theoretically for kriging models with two -dimensional inputs and separable exponential covariance structures. For design comparison, the notion of evenness of two-dimensional grid designs is developed. The mathematical relationship between the design and the supremum of the mean squared prediction error (SMSPE) function is studied and then optimal designs are explored for both prospective and retrospective design scenarios. In the case of prospective designs, the new design is developed before the experiment is conducted and the regularly spaced grid is shown to be the G -optimal design. Retrospective designs are constructed by adding or deleting points from an already existing design. Deterministic algorithms are developed to find the best possible retrospective designs (which minimizes the SMSPE). It is found that a more evenly spread design under the G -optimality criterion leads to the best possible retrospective design. For all the cases of finding the optimal prospective designs and the best possible retrospective designs, both frequentist and Bayesian frameworks have been considered. The proposed methodology for finding retrospective designs is illustrated with a spatio-temporal river water quality monitoring experiment.
这项工作的重点是为具有二维输入和可分离指数协方差结构的克里金模型从理论上找到 G 最佳设计。为了进行设计比较,提出了二维网格设计的均匀性概念。研究了设计与均方预测误差(SMSPE)函数上确界之间的数学关系,然后探讨了前瞻性设计和回顾性设计两种情况下的最优设计。在前瞻性设计的情况下,新设计是在实验进行之前开发的,而规则间隔的网格被证明是 G 最佳设计。回顾性设计是通过在已有设计中添加或删除点来构建的。我们开发了确定性算法来寻找最佳的回顾性设计(使 SMSPE 最小)。研究发现,在 G 最佳准则下,更均匀分布的设计会导致最佳的回顾性设计。在寻找最优前瞻性设计和最佳回顾性设计的所有情况下,都考虑了频繁主义和贝叶斯框架。我们用一个时空河流水质监测实验来说明所提出的寻找回溯设计的方法。
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引用次数: 0
Nonparametric conditional mean testing via an extreme-type statistic in high dimension 基于高维极值型统计量的非参数条件均值检验
IF 1 4区 数学 Q3 Mathematics Pub Date : 2023-12-02 DOI: 10.1111/sjos.12697
Yiming Liu, Guangming Pan, Guangren Yang, Wang Zhou
We propose a new test to investigate the conditional mean dependence between a response variable and the corresponding covariates in the high dimensional regimes. The test statistic is an extreme-type one built on the nonparametric method. The limiting null distribution of the proposed extreme type statistic under a mild mixing condition is established. Moreover, to make the test more powerful in general structures we propose a more general test statistic and develop its asymptotic properties. The power analysis of both methods is also considered. In real data analysis, we also propose a new way to conduct the feature screening based on our results. To evaluate the performance of our estimators and other methods, extensive simulations are conducted.
我们提出了一种新的检验方法来研究高维体系中响应变量与相应协变量之间的条件平均相关性。检验统计量是建立在非参数方法上的极值型统计量。建立了在轻度混合条件下所提出的极值型统计量的极限零分布。此外,为了使一般结构的检验更有效,我们提出了一个更一般的检验统计量,并发展了它的渐近性质。本文还考虑了两种方法的功率分析。在实际数据分析中,我们也提出了一种基于我们的结果进行特征筛选的新方法。为了评估我们的估计器和其他方法的性能,进行了大量的模拟。
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引用次数: 0
Modelling multivariate extreme value distributions via Markov trees* 用马尔可夫树建模多元极值分布*
IF 1 4区 数学 Q3 Mathematics Pub Date : 2023-11-30 DOI: 10.1111/sjos.12698
Shuang Hu, Zuoxiang Peng, Johan Segers
Multivariate extreme value distributions are a common choice for modelling multivariate extremes. In high dimensions, however, the construction of flexible and parsimonious models is challenging. We propose to combine bivariate max-stable distributions into a Markov random field with respect to a tree. Although in general not max-stable itself, this Markov tree is attracted by a multivariate max-stable distribution. The latter serves as a tree-based approximation to an unknown max-stable distribution with the given bivariate distributions as margins. Given data, we learn an appropriate tree structure by Prim's algorithm with estimated pairwise upper tail dependence coefficients as edge weights. The distributions of pairs of connected variables can be fitted in various ways. The resulting tree-structured max-stable distribution allows for inference on rare event probabilities, as illustrated on river discharge data from the upper Danube basin.
多变量极值分布是建模多变量极值的常用选择。然而,在高维中,构建灵活且简洁的模型是一项挑战。我们提出将二元最大稳定分布组合成一个关于树的马尔可夫随机场。虽然马尔可夫树本身通常不是极大稳定的,但它被多元极大稳定分布所吸引。后者以给定的二元分布作为边界,作为未知最大稳定分布的基于树的近似。给定数据,我们用估计的两两上尾相关系数作为边权,通过Prim算法学习到合适的树结构。连接变量对的分布可以用各种方法拟合。由此产生的树状结构最大稳定分布允许对罕见事件概率进行推断,如多瑙河上游流域的河流流量数据所示。
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引用次数: 4
期刊
Scandinavian Journal of Statistics
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