Biometrics最新文献

This paper introduces a model for longitudinal functional data analysis that accounts for pointwise skewness. The proposed procedure decouples the marginal pointwise variation from the complex longitudinal and functional dependence using copula methodology. Pointwise variation is described through parametric distribution functions that capture varying skewness and change smoothly both in time and over the functional argument. Joint dependence is quantified through a Gaussian copula with a low-rank approximation-based covariance. The introduced class of models provides a unifying platform for both pointwise quantile estimation and prediction of complete trajectories at new times. We investigate the methods numerically in simulations and discuss their application to a diffusion tensor imaging study of multiple sclerosis patients. This approach is implemented in the R package sLFDA that is publicly available on GitHub.

Mouse-tracking data, which record computer mouse trajectories while participants perform an experimental task, provide valuable insights into subjects' underlying cognitive processes. Neuroscientists are interested in clustering the subjects' responses during computer mouse-tracking tasks to reveal patterns of individual decision-making behaviors and identify population subgroups with similar neurobehavioral responses. These data can be combined with neuroimaging data to provide additional information for personalized interventions. In this article, we develop a novel hierarchical shrinkage partition (HSP) prior for clustering summary statistics derived from the trajectories of mouse-tracking data. The HSP model defines a subjects' cluster as a set of subjects that gives rise to more similar (rather than identical) nested partitions of the conditions. The proposed model can incorporate prior information about the partitioning of either subjects or conditions to facilitate clustering, and it allows for deviations of the nested partitions within each subject group. These features distinguish the HSP model from other bi-clustering methods that typically create identical nested partitions of conditions within a subject group. Furthermore, it differs from existing nested clustering methods, which define clusters based on common parameters in the sampling model and identify subject groups by different distributions. We illustrate the unique features of the HSP model on a mouse tracking dataset from a pilot study and in simulation studies. Our results show the ability and effectiveness of the proposed exploratory framework in clustering and revealing possible different behavioral patterns across subject groups.

Model averaging is an important tool for treating uncertainty from model selection process and fusing information from different models, and has been widely used in various fields. However, the most existing model averaging criteria are proposed based on the methods of ordinary least squares or maximum likelihood, which possess high sensitivity to outliers or violation of certain model assumption. For the mean regression, no optimal robust methods are developed. To fill this gap, in our paper, we propose an outlier-robust model averaging approach by Mallows-type criterion. The idea is that we first construct a generalized M (GM) estimator for each candidate model, and then build robust weighting schemes by the asymptotic expansion of the final prediction error based on the GM-type loss function. So, we can still achieve a trustworthy result even if the dataset is contaminated by outliers in response and/or covariates. Asymptotic properties of the proposed robust model averaging estimators are established under some regularity conditions. The consistency of our weight estimators tending to the theoretically optimal weight vectors is also derived. We prove that our model averaging estimator is robust in terms of having bounded influence function. Further, we define the empirical prediction influence function to evaluate the quantitative robustness of the model averaging estimator. A simulation study and a real data analysis are conducted to demonstrate the finite sample performance of our estimators and compare them with other commonly used model selection and averaging methods.

With the advent of massive survival data with a cure fraction, large-scale regression for analyzing the effects of risk factors on a general population has become an emerging challenge. This article proposes a new probability-weighted method for estimation and inference for semiparametric cure regression models. We develop a flexible formulation of the mixture cure model consisting of the model-free incidence and the latency assumed by the semiparametric proportional hazards model. The susceptible probability assesses the concordance between the observations and the latency. With the susceptible probability as weight, we propose a weighted estimating equation method in a small-scale setting. Robust nonparametric estimation of the weight permits stable implementation of the estimation of regression parameters. A recursive probability-weighted estimation method based on data blocks with smaller sizes is further proposed, which achieves computational and memory efficiency in a large-scale or online setting. Asymptotic properties of the proposed estimators are established. We conduct simulation studies and a real data application to demonstrate the empirical performance of the proposed method.

One of the goals of precision psychiatry is to characterize mental disorders in an individualized manner, taking into account the underlying dynamic processes. Recent advances in mobile technologies have enabled the collection of ecological momentary assessments that capture multiple responses in real-time at high frequency. However, ecological momentary assessment data are often multi-dimensional, correlated, and hierarchical. Mixed-effect models are commonly used but may require restrictive assumptions about the fixed and random effects and the correlation structure. The recurrent temporal restricted Boltzmann machine (RTRBM) is a generative neural network that can be used to model temporal data, but most existing RTRBM approaches do not account for the potential heterogeneity of group dynamics within a population based on available covariates. In this paper, we propose a new temporal generative model, the HDRBM, to learn the heterogeneous group dynamics and demonstrate the effectiveness of this approach on simulated and real-world ecological momentary assessment datasets. We show that by incorporating covariates, HDRBM can improve accuracy and interpretability, explore the underlying drivers of the group dynamics of participants, and serve as a generative model for ecological momentary assessment studies.

Randomized trials seek efficient treatment effect estimation within target populations, yet scientific interest often also centers on subpopulations. Although there are typically too few subjects within each subpopulation to efficiently estimate these subpopulation treatment effects, one can gain precision by borrowing strength across subpopulations, as is the case in a basket trial. While dynamic borrowing has been proposed as an efficient approach to estimating subpopulation treatment effects on primary endpoints, additional efficiency could be gained by leveraging the information found in secondary endpoints. We propose a multisource exchangeability model (MEM) that incorporates secondary endpoints to more efficiently assess subpopulation exchangeability. Across simulation studies, our proposed model almost uniformly reduces the mean squared error when compared to the standard MEM that only considers data from the primary endpoint by gaining efficiency when subpopulations respond similarly to the treatment and reducing the magnitude of bias when the subpopulations are heterogeneous. We illustrate our model's feasibility using data from a recently completed trial of very low nicotine content cigarettes to estimate the effect on abstinence from smoking within three priority subpopulations. Our proposed model led to increases in the effective sample size two to four times greater than under the standard MEM.

Analyses of cluster randomized trials (CRTs) can be complicated by informative missing outcome data. Methods such as inverse probability weighted generalized estimating equations have been proposed to account for informative missingness by weighing the observed individual outcome data in each cluster. These existing methods have focused on settings where missingness occurs at the individual level and each cluster has partially or fully observed individual outcomes. In the presence of missing clusters, for example, all outcomes from a cluster are missing due to drop-out of the cluster, these approaches ignore this cluster-level missingness and can lead to biased inference if the cluster-level missingness is informative. Informative missingness at multiple levels can also occur in CRTs with a multi-level structure where study participants are nested in subclusters such as healthcare providers, and the subclusters are nested in clusters such as clinics. In this paper, we propose new estimators for estimating the marginal treatment effect in CRTs accounting for missing outcome data at multiple levels based on weighted generalized estimating equations. We show that the proposed multi-level multiply robust estimator is consistent and asymptotically normally distributed provided that one of the multiple propensity score models postulated at each clustering level is correctly specified. We evaluate the performance of the proposed method through extensive simulations and illustrate its use with a CRT evaluating a Malaria risk-reduction intervention in rural Madagascar.

We commend Alt et al.'s innovative approach for analysis with a hybrid control arm while offering insights into two key considerations: the necessity for extrapolation and the potential benefits of curating historical control data before analysis.

Recent breakthroughs in spatially resolved transcriptomics (SRT) technologies have enabled comprehensive molecular characterization at the spot or cellular level while preserving spatial information. Cells are the fundamental building blocks of tissues, organized into distinct yet connected components. Although many non-spatial and spatial clustering approaches have been used to partition the entire region into mutually exclusive spatial domains based on the SRT high-dimensional molecular profile, most require an ad hoc selection of less interpretable dimensional-reduction techniques. To overcome this challenge, we propose a zero-inflated negative binomial mixture model to cluster spots or cells based on their molecular profiles. To increase interpretability, we employ a feature selection mechanism to provide a low-dimensional summary of the SRT molecular profile in terms of discriminating genes that shed light on the clustering result. We further incorporate the SRT geospatial profile via a Markov random field prior. We demonstrate how this joint modeling strategy improves clustering accuracy, compared with alternative state-of-the-art approaches, through simulation studies and 3 real data applications.

The scope of this paper is a multivariate setting involving categorical variables. Following an external manipulation of one variable, the goal is to evaluate the causal effect on an outcome of interest. A typical scenario involves a system of variables representing lifestyle, physical and mental features, symptoms, and risk factors, with the outcome being the presence or absence of a disease. These variables are interconnected in complex ways, allowing the effect of an intervention to propagate through multiple paths. A distinctive feature of our approach is the estimation of causal effects while accounting for uncertainty in both the dependence structure, which we represent through a directed acyclic graph (DAG), and the DAG-model parameters. Specifically, we propose a Markov chain Monte Carlo algorithm that targets the joint posterior over DAGs and parameters, based on an efficient reversible-jump proposal scheme. We validate our method through extensive simulation studies and demonstrate that it outperforms current state-of-the-art procedures in terms of estimation accuracy. Finally, we apply our methodology to analyze a dataset on depression and anxiety in undergraduate students.