Time-to-event analysis often relies on prior parametric assumptions, or, if a non-parametric approach is chosen, Cox's model. This is inherently tied to the assumption of proportional hazards, with the analysis potentially invalidated if this assumption is not fulfilled. In addition, most interpretations focus on the hazard ratio, that is often misinterpreted as the relative risk. In this paper, we introduce an alternative to current methodology for assessing a treatment effect in a two-group situation, not relying on the proportional hazards assumption but assuming proportional risks. Precisely, we propose a new non-parametric model to directly estimate the relative risk of two groups to experience an event under the assumption that the risk ratio is constant over time. In addition to this relative measure, our model allows for calculating the number needed to treat as an absolute measure, providing the possibility of an easy and holistic interpretation of the data. We demonstrate the validity of the approach by means of a simulation study and present an application to data from a large randomized controlled trial investigating the effect of dapagliflozin on the risk of first hospitalization for heart failure.
相關內容
Motivated by recent findings that within-subject (WS) visit-to-visit variabilities of longitudinal biomarkers can be strong risk factors for health outcomes, this paper introduces and examines a new joint model of a longitudinal biomarker with heterogeneous WS variability and competing risks time-to-event outcome. Specifically, our joint model consists of a linear mixed-effects multiple location-scale submodel for the individual mean trajectory and WS variability of the longitudinal biomarker and a semiparametric cause-specific Cox proportional hazards submodel for the competing risks survival outcome. The submodels are linked together via shared random effects. We derive an expectation-maximization algorithm for semiparametric maximum likelihood estimation and a profile-likelihood method for standard error estimation. We implement efficient computational algorithms that scales to biobank-scale data with tens of thousands of subjects. Our simulation results demonstrate that, in the presence of heterogeneous WS variability, the proposed method has superior performance for estimation, inference, and prediction, over the classical joint model with homogeneous WS variability. An application of our method to a Multi-Ethnic Study of Atherosclerosis (MESA) data reveals that there is substantial heterogeneity in systolic blood pressure (SBP) WS variability across MESA individuals and that SBP WS variability is an important predictor for heart failure and death, (independent of, or in addition to) the individual SBP mean level. Furthermore, by accounting for both the mean trajectory and WS variability of SBP, our method leads to a more accurate dynamic prediction model for heart failure or death. A user-friendly R package \textbf{JMH} is developed and publicly available at \url{//github.com/shanpengli/JMH}.
Determining causal effects of interventions onto outcomes from real-world, observational (non-randomized) data, e.g., treatment repurposing using electronic health records, is challenging due to underlying bias. Causal deep learning has improved over traditional techniques for estimating individualized treatment effects (ITE). We present the Doubly Robust Variational Information-theoretic Deep Adversarial Learning (DR-VIDAL), a novel generative framework that combines two joint models of treatment and outcome, ensuring an unbiased ITE estimation even when one of the two is misspecified. DR-VIDAL integrates: (i) a variational autoencoder (VAE) to factorize confounders into latent variables according to causal assumptions; (ii) an information-theoretic generative adversarial network (Info-GAN) to generate counterfactuals; (iii) a doubly robust block incorporating treatment propensities for outcome predictions. On synthetic and real-world datasets (Infant Health and Development Program, Twin Birth Registry, and National Supported Work Program), DR-VIDAL achieves better performance than other non-generative and generative methods. In conclusion, DR-VIDAL uniquely fuses causal assumptions, VAE, Info-GAN, and doubly robustness into a comprehensive, performant framework. Code is available at: //github.com/Shantanu48114860/DR-VIDAL-AMIA-22 under MIT license.
When an exposure of interest is confounded by unmeasured factors, an instrumental variable (IV) can be used to identify and estimate certain causal contrasts. Identification of the marginal average treatment effect (ATE) from IVs relies on strong untestable structural assumptions. When one is unwilling to assert such structure, IVs can nonetheless be used to construct bounds on the ATE. Famously, Balke and Pearl (1997) proved tight bounds on the ATE for a binary outcome, in a randomized trial with noncompliance and no covariate information. We demonstrate how these bounds remain useful in observational settings with baseline confounders of the IV, as well as randomized trials with measured baseline covariates. The resulting bounds on the ATE are non-smooth functionals, and thus standard nonparametric efficiency theory is not immediately applicable. To remedy this, we propose (1) under a novel margin condition, influence function-based estimators of the bounds that can attain parametric convergence rates when the nuisance functions are modeled flexibly, and (2) estimators of smooth approximations of these bounds. We propose extensions to continuous outcomes, explore finite sample properties in simulations, and illustrate the proposed estimators in a randomized experiment studying the effects of vaccination encouragement on flu-related hospital visits.
Advanced science and technology provide a wealth of big data from different sources for extreme value analysis.Classic extreme value theory was extended to obtain an accelerated max-stable distribution family for modelling competing risk-based extreme data in Cao and Zhang (2021). In this paper, we establish probability models for power normalized maxima and minima from competing risks. The limit distributions consist of an extensional new accelerated max-stable and min-stable distribution family (termed as the accelerated p-max/p-min stable distribution), and its left-truncated version. The limit types of distributions are determined principally by the sample generating process and the interplay among the competing risks, which are illustrated by common examples. Further, the statistical inference concerning the maximum likelihood estimation and model diagnosis of this model was investigated. Numerical studies show first the efficient approximation of all limit scenarios as well as its comparable convergence rate in contrast with those under linear normalization, and then present the maximum likelihood estimation and diagnosis of accelerated p-max/p-min stable models for simulated data sets. Finally, two real datasets concerning annual maximum of ground level ozone and survival times of Stanford heart plant demonstrate the performance of our accelerated p-max and accelerated p-min stable models.
Experimental designs with hierarchically-structured errors are pervasive in many biomedical areas; it is important to take into account this hierarchical architecture in order to account for the dispersion and make reliable inferences from the data. This paper addresses the question of estimating a proportion or a ratio from positive or negative count data akin to those generated by droplet digital polymerase chain reaction experiments when the number of biological or technical replicates is limited. We present and discuss a Bayesian framework, for which we provide and implement a Gibbs sampler in R and compare it to a random effect model.
The population protocol model introduced by Angluin et al. in 2006 offers a theoretical framework for designing and analyzing distributed algorithms among limited-resource mobile agents. While the original population protocol model considers the concept of anonymity, the issue of privacy is not investigated thoroughly. However, there is a need for time- and space-efficient privacy-preserving techniques in the population protocol model if these algorithms are to be implemented in settings handling sensitive data, such as sensor networks, IoT devices, and drones. In this work, we introduce several formal definitions of privacy, ranging from assuring only plausible deniability of the population input vector to having a full information-theoretic guarantee that knowledge beyond an agent's input and output bear no influence on the probability of a particular input vector. We then apply these definitions to both existing and novel protocols. We show that the Remainder-computing protocol given by Delporte-Gallet et al. in 2007 (which is proven to satisfy output independent privacy under adversarial scheduling) is not information-theoretically private under probabilistic scheduling. In contrast, we provide a new algorithm and demonstrate that it correctly and information-theoretically privately computes Remainder under probabilistic scheduling.
SARSA, a classical on-policy control algorithm for reinforcement learning, is known to chatter when combined with linear function approximation: SARSA does not diverge but oscillates in a bounded region. However, little is known about how fast SARSA converges to that region and how large the region is. In this paper, we make progress towards this open problem by showing the convergence rate of projected SARSA to a bounded region. Importantly, the region is much smaller than the region that we project into, provided that the magnitude of the reward is not too large. Existing works regarding the convergence of linear SARSA to a fixed point all require the Lipschitz constant of SARSA's policy improvement operator to be sufficiently small; our analysis instead applies to arbitrary Lipschitz constants and thus characterizes the behavior of linear SARSA for a new regime.
This paper considers a downlink satellite communication system where a satellite cluster, i.e., a satellite swarm consisting of one leader and multiple follower satellites, serves a ground terminal. The satellites in the cluster form either a linear or circular formation moving in a group and cooperatively send their signals by maximum ratio transmission precoding. We first conduct a coordinate transformation to effectively capture the relative positions of satellites in the cluster. Next, we derive an exact expression for the orbital configuration-dependent outage probability under the Nakagami fading by using the distribution of the sum of independent Gamma random variables. In addition, we obtain a simpler approximated expression for the outage probability with the help of second-order moment-matching. We also analyze asymptotic behavior in the high signal-to-noise ratio regime and the diversity order of the outage performance. Finally, we verify the analytical results through Monte Carlo simulations. Our analytical results provide the performance of satellite cluster-based communication systems based on specific orbital configurations, which can be used to design reliable satellite clusters in terms of cluster size, formation, and orbits.
In many fields, including environmental epidemiology, researchers strive to understand the joint impact of a mixture of exposures. This involves analyzing a vector of exposures rather than a single exposure, with the most significant exposure sets being unknown. Examining every possible interaction or effect modification in a high-dimensional vector of candidates can be challenging or even impossible. To address this challenge, we propose a method for the automatic identification and estimation of exposure sets in a mixture with explanatory power, baseline covariates that modify the impact of an exposure and sets of exposures that have synergistic non-additive relationships. We define these parameters in a realistic nonparametric statistical model and use machine learning methods to identify variables sets and estimate nuisance parameters for our target parameters to avoid model misspecification. We establish a prespecified target parameter applied to variable sets when identified and use cross-validation to train efficient estimators employing targeted maximum likelihood estimation for our target parameter. Our approach applies a shift intervention targeting individual variable importance, interaction, and effect modification based on the data-adaptively determined sets of variables. Our methodology is implemented in the open-source SuperNOVA package in R. We demonstrate the utility of our method through simulations, showing that our estimator is efficient and asymptotically linear under conditions requiring fast convergence of certain regression functions. We apply our method to the National Institute of Environmental Health Science mixtures workshop data, revealing correct identification of antagonistic and agonistic interactions built into the data. Additionally, we investigate the association between exposure to persistent organic pollutants and longer leukocyte telomere length.
AI is undergoing a paradigm shift with the rise of models (e.g., BERT, DALL-E, GPT-3) that are trained on broad data at scale and are adaptable to a wide range of downstream tasks. We call these models foundation models to underscore their critically central yet incomplete character. This report provides a thorough account of the opportunities and risks of foundation models, ranging from their capabilities (e.g., language, vision, robotics, reasoning, human interaction) and technical principles(e.g., model architectures, training procedures, data, systems, security, evaluation, theory) to their applications (e.g., law, healthcare, education) and societal impact (e.g., inequity, misuse, economic and environmental impact, legal and ethical considerations). Though foundation models are based on standard deep learning and transfer learning, their scale results in new emergent capabilities,and their effectiveness across so many tasks incentivizes homogenization. Homogenization provides powerful leverage but demands caution, as the defects of the foundation model are inherited by all the adapted models downstream. Despite the impending widespread deployment of foundation models, we currently lack a clear understanding of how they work, when they fail, and what they are even capable of due to their emergent properties. To tackle these questions, we believe much of the critical research on foundation models will require deep interdisciplinary collaboration commensurate with their fundamentally sociotechnical nature.
注冊地址： 北京市海淀區羊坊店路18號2幢3層301-191