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A novel longitudinal rank-sum test for multiple primary endpoints in clinical trials: Applications to neurodegenerative disorders

临床试验多主要终点的纵向秩和检验:神经退行性疾病应用

Neurodegenerative disorders such as Alzheimer's disease (AD) present a significant global health challenge, characterized by cognitive decline, functional impairment, and other debilitating effects. Current AD clinical trials often assess multiple longitudinal primary endpoints to comprehensively evaluate treatment efficacy. Traditional methods, however, may fail to capture global treatment effects, require larger sample sizes due to multiplicity adjustments, and may not…

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Random forests for binary geospatial data

用于二元地理空间数据的随机森林

The manuscript develops new method and theory for non-linear regression for binary dependent data using random forests. Existing implementations of random forests for binary data cannot explicitly account for data correlation common in geospatial and time-series settings. For continuous outcomes, recent work has extended random forests (RF) to RF-GLS that incorporate spatial covariance using the generalized least squares (GLS) loss. However, adoption of this idea for binary…

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Discrete-time Competing-Risks Regression with or without Penalization

离散时间竞争风险回归:含惩罚与不含惩罚

Many studies employ the analysis of time-to-event data that incorporates competing risks and right censoring. Most methods and software packages are geared towards analyzing data that comes from a continuous failure time distribution. However, failure-time data may sometimes be discrete either because time is inherently discrete or due to imprecise measurement. This paper introduces a new estimation procedure for discrete-time survival analysis with competing events. The…

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Causal survival analysis under competing risks using longitudinal modified treatment policies

利用纵向修正治疗策略开展竞争风险下的因果生存分析

Longitudinal modified treatment policies (LMTP) have been recently developed as a novel method to define and estimate causal parameters that depend on the natural value of treatment. LMTPs represent an important advancement in causal inference for longitudinal studies as they allow the non-parametric definition and estimation of the joint effect of multiple categorical, numerical, or continuous exposures measured at several time points. We extend the LMTP methodology to…

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Dealing with multiple intercurrent events using hypothetical and treatment policy strategies simultaneously

同时采用假想策略与治疗策略处理多个伴发事件

To precisely define the treatment effect of interest in a clinical trial, the ICH E9 estimand addendum describes that relevant so-called intercurrent events should be identified and strategies specified to deal with them. Handling intercurrent events with different strategies leads to different estimands. In this paper, we focus on estimands that involve addressing one intercurrent event with the treatment policy strategy and another with the hypothetical strategy. We define…

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Horowitz-Manski-Lee Bounds with Multilayered Sample Selection

多层样本选择下的 Horowitz–Manski–Lee 界限

This paper investigates the causal effect of job training on wage rates in the presence of firm heterogeneity. When training affects the sorting of workers to firms, sample selection is no longer binary but is ``multilayered". This paper extends the canonical Heckman (1979) sample selection model -- which assumes selection is binary -- to a setting where it is multilayered. In this setting Lee bounds set identifies a total effect that combines a weighted-average of the…

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Ordinal Patterns Based Change Points Detection

基于序模式的变点检测

The ordinal patterns of a fixed number of consecutive values in a time series is the spatial ordering of these values. Counting how often a specific ordinal pattern occurs in a time series provides important insights into the properties of the time series. In this work, we prove the asymptotic normality of the relative frequency of ordinal patterns for time series with linear increments. Moreover, we apply ordinal patterns to detect changes in the distribution of a time…

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Sufficient dimension reduction for regression with spatially correlated errors: application to prediction

空间相关误差回归的充分降维:预测应用

In this paper, we address the problem of predicting a response variable in the context of both, spatially correlated and high-dimensional data. To reduce the dimensionality of the predictor variables, we apply the sufficient dimension reduction (SDR) paradigm, which reduces the predictor space while retaining relevant information about the response. To achieve this, we impose two different spatial models on the inverse regression: the separable spatial covariance model…

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RieszBoost: Gradient Boosting for Riesz Regression

RieszBoost:用于 Riesz 回归的梯度提升

Answering causal questions often involves estimating linear functionals of conditional expectations, such as the average treatment effect or the effect of a longitudinal modified treatment policy. By the Riesz representation theorem, these functionals can be expressed as the expected product of the conditional expectation of the outcome and the Riesz representer, a key component in doubly robust estimation methods. Traditionally, the Riesz representer is estimated indirectly…

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Data-adaptive exposure thresholds for the Horvitz-Thompson estimator of the Average Treatment Effect in experiments with network interference

网络干扰实验中平均处理效应 Horvitz–Thompson 估计的自适应暴露阈值

Randomized controlled trials often suffer from interference, a violation of the Stable Unit Treatment Values Assumption (SUTVA) in which a unit's treatment assignment affects the outcomes of its neighbors. This interference causes bias in naive estimators of the average treatment effect (ATE). A popular method to achieve unbiasedness is to pair the Horvitz-Thompson estimator of the ATE with a known exposure mapping: a function that identifies which units in a given…

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Practically Effective Adjustment Variable Selection in Causal Inference

因果推断中具有实际效果的调整变量选择

In the estimation of causal effects, one common method for removing the influence of confounders is to adjust the variables that satisfy the back-door criterion. However, it is not always possible to uniquely determine sets of such variables. Moreover, real-world data is almost always limited, which means it may be insufficient for statistical estimation. Therefore, we propose criteria for selecting variables from a list of candidate adjustment variables along with an…

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Randomization Inference: Theory and Applications

随机化推断:理论与应用

We review approaches to statistical inference based on randomization. Permutation tests are treated as an important special case. Under a certain group invariance property, referred to as the ``randomization hypothesis,'' randomization tests achieve exact control of the Type I error rate in finite samples. Although this unequivocal precision is very appealing, the range of problems that satisfy the randomization hypothesis is somewhat limited. We show that randomization…

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Synthetic Random Environmental Time Series Generation with Similarity Control, Preserving Original Signal's Statistical Characteristics

相似度可控且保留原信号统计特征的随机环境时间序列生成

Synthetic datasets are widely used in many applications, such as missing data imputation, examining non-stationary scenarios, in simulations, training data-driven models, and analyzing system robustness. Typically, synthetic data are based on historical data obtained from the observed system. The data needs to represent a specific behavior of the system, yet be new and diverse enough so that the system is challenged with a broad range of inputs. This paper presents a method,…

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Multi-Study Causal Forest (MCF): A flexible framework for data borrowing in the presence of varying treatment effect heterogeneity

多研究因果森林:处理效应异质性变化下的数据借用框架

Tailoring treatment assignment to specific individuals can improve the health outcomes, but a single study may offer inadequate information for this purpose. The ability to leverage information from an auxiliary data source deemed to be `most similar' to a primary data source has been shown to improve estimates of treatment effects. In this paper, we introduce a framework, the Multi-Study Causal Forest (MCF), to borrow individual patient-level data from an auxiliary data…

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Heteroscedastic Double Bayesian Elastic Net

异方差双重贝叶斯弹性网

In many practical applications, regression models are employed to uncover relationships between predictors and a response variable, yet the common assumption of constant error variance is frequently violated. This issue is further compounded in high-dimensional settings where the number of predictors exceeds the sample size, necessitating regularization for effective estimation and variable selection. To address this problem, we propose the Heteroscedastic Double Bayesian…

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Estimating Network Models using Neural Networks

利用神经网络估计网络模型

Exponential random graph models (ERGMs) are very flexible for modeling network formation but pose difficult estimation challenges due to their intractable normalizing constant. Existing methods, such as MCMC-MLE, rely on sequential simulation at every optimization step. We propose a neural network approach that trains on a single, large set of parameter-simulation pairs to learn the mapping from parameters to average network statistics. Once trained, this map can be…

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Inference for multiple treatment effects using confounder importance learning

利用混杂变量重要性学习推断多个处理效应

We address modelling and computational issues for multiple treatment effect inference under many potential confounders. Our main contribution is providing a trade-off between preventing the omission of relevant confounders, while not running into an over-selection of instruments that significantly inflates variance. We propose a novel empirical Bayes framework for Bayesian model averaging that learns from data the prior inclusion probabilities of key covariates. Our…

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Regressions under Adverse Conditions

不利条件下的回归

We introduce a new regression method that relates the mean of an outcome variable to covariates, under the "adverse condition" that a distress variable falls in its tail. This allows to tailor classical mean regressions to adverse scenarios, which receive increasing interest in economics and finance, among many others. In the terminology of the systemic risk literature, our method can be interpreted as a regression for the Marginal Expected Shortfall. We propose a two-step…

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Higher-Order Causal Message Passing for Experimentation with Complex Interference

复杂干扰实验中的高阶因果消息传递

Accurate estimation of treatment effects is essential for decision-making across various scientific fields. This task, however, becomes challenging in areas like social sciences and online marketplaces, where treating one experimental unit can influence outcomes for others through direct or indirect interactions. Such interference can lead to biased treatment effect estimates, particularly when the structure of these interactions is unknown. We address this challenge by…

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Statistical inference for high-dimensional spectral density matrix

高维谱密度矩阵的统计推断

The spectral density matrix is a fundamental object of interest in time series analysis, and it encodes both contemporary and dynamic linear relationships between component processes of the multivariate system. In this paper we develop novel inference procedures for the spectral density matrix in the high-dimensional setting. Specifically, we introduce a new global testing procedure to test the nullity of the cross-spectral density for a given set of frequencies and across…

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Parameter Estimation of State Space Models Using Particle Importance Sampling

利用粒子重要性采样估计状态空间模型参数

State-space models have been used in many applications, including econometrics, engineering, medical research, etc. The maximum likelihood estimation (MLE) of the static parameter of general state-space models is not straightforward because the likelihood function is intractable. It is popular to use the sequential Monte Carlo(SMC) method to perform gradient ascent optimisation in either offline or online fashion. One problem with existing online SMC methods for MLE is that…

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Generalized Neyman Allocation for Locally Minimax Optimal Best-Arm Identification

局部极小极大最优最佳臂识别的广义 Neyman 分配

This study investigates an asymptotically locally minimax optimal algorithm for fixed-budget best-arm identification (BAI). We propose the Generalized Neyman Allocation (GNA) algorithm and demonstrate that its worst-case upper bound on the probability of misidentifying the best arm aligns with the worst-case lower bound under the small-gap regime, where the gap between the expected outcomes of the best and suboptimal arms is small. Our lower and upper bounds are tight,…

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Online Generalized Method of Moments for Time Series

时间序列的在线广义矩估计

Online learning has gained popularity in recent years due to the urgent need to analyse large-scale streaming data, which can be collected in perpetuity and serially dependent. This motivates us to develop the online generalized method of moments (OGMM), an explicitly updated estimation and inference framework in the time series setting. The OGMM inherits many properties of offline GMM, such as its broad applicability to many problems in econometrics and statistics, natural…

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Functional Clustering for Longitudinal Associations between Social Determinants of Health and Stroke Mortality in the US

美国健康社会决定因素与卒中死亡率纵向关联的功能聚类

Understanding the longitudinally changing associations between Social Determinants of Health (SDOH) and stroke mortality is essential for effective stroke management. Previous studies have uncovered significant regional disparities in the relationships between SDOH and stroke mortality. However, existing studies have not utilized longitudinal associations to develop data-driven methods for regional division in stroke control. To fill this gap, we propose a novel clustering…

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