Journal of the Royal Statistical Society Series C-Applied Statistics最新文献

The aim of dynamic prediction is to provide individualized risk predictions over time, which are updated as new data become available. In pursuit of constructing a dynamic prediction model for a progressive eye disorder, age-related macular degeneration (AMD), we propose a time-dependent Cox survival neural network (tdCoxSNN) to predict its progression using longitudinal fundus images. tdCoxSNN builds upon the time-dependent Cox model by utilizing a neural network to capture the nonlinear effect of time-dependent covariates on the survival outcome. Moreover, by concurrently integrating a convolutional neural network with the survival network, tdCoxSNN can directly take longitudinal images as input. We evaluate and compare our proposed method with joint modelling and landmarking approaches through extensive simulations. We applied the proposed approach to two real datasets. One is a large AMD study, the Age-Related Eye Disease Study, in which more than 50,000 fundus images were captured over a period of 12 years for more than 4,000 participants. Another is a public dataset of the primary biliary cirrhosis disease, where multiple laboratory tests were longitudinally collected to predict the time-to-liver transplant. Our approach demonstrates commendable predictive performance in both simulation studies and the analysis of the two real datasets.

Survival is poor for patients with metastatic cancer, and it is vital to examine new biomarkers that can improve patient prognostication and identify those who would benefit from more aggressive therapy. In metastatic prostate cancer, 2 new assays have become available: one that quantifies the number of cancer cells circulating in the peripheral blood, and the other a marker of the aggressiveness of the disease. It is critical to determine the magnitude of the effect of these biomarkers on the discrimination of a model-based risk score. To do so, most analysts frequently consider the discrimination of 2 separate survival models: one that includes both the new and standard factors and a second that includes the standard factors alone. However, this analysis is ultimately incorrect for many of the scale-transformation models ubiquitous in survival, as the reduced model is misspecified if the full model is specified correctly. To circumvent this issue, we developed a projection-based approach to estimate the impact of the 2 prostate cancer biomarkers. The results indicate that the new biomarkers can influence model discrimination and justify their inclusion in the risk model; however, the hunt remains for an applicable model to risk-stratify patients with metastatic prostate cancer.

Network meta-analysis is a powerful tool to synthesize evidence from independent studies and compare multiple treatments simultaneously. A critical task of performing a network meta-analysis is to offer ranks of all available treatment options for a specific disease outcome. Frequently, the estimated treatment rankings are accompanied by a large amount of uncertainty, suffer from multiplicity issues, and rarely permit possible ties of treatments with similar performance. These issues make interpreting rankings problematic as they are often treated as absolute metrics. To address these shortcomings, we formulate a ranking strategy that adapts to scenarios with high-order uncertainty by producing more conservative results. This improves the interpretability while simultaneously accounting for multiple comparisons. To admit ties between treatment effects in cases where differences between treatment effects are negligible, we also develop a Bayesian non-parametric approach for network meta-analysis. The approach capitalizes on the induced clustering mechanism of Bayesian non-parametric methods, producing a positive probability that two treatment effects are equal. We demonstrate the utility of the procedure through numerical experiments and a network meta-analysis designed to study antidepressant treatments.

The motivation for this paper is to determine factors associated with time-to-fertility treatment (TTFT) among women currently attempting pregnancy in a cross-sectional sample. Challenges arise due to dependence between time-to-pregnancy (TTP) and TTFT. We propose appending a marginal accelerated failure time model to identify risk factors of TTFT with a model for TTP where fertility treatment is included as a time-varying treatment to account for their dependence. The latter requires extending backwards recurrence survival methods to incorporate time-varying covariates with time-varying coefficients. Since backwards recurrence survival methods are a function of mean survival, computational difficulties arise in formulating mean survival when fertility treatment is unobserved, i.e. when TTFT is censored. We address these challenges by developing computationally friendly forms for the double expectation of TTP and TTFT. The performance is validated via comprehensive simulation studies. We apply our approach to the National Survey of Family Growth and explore factors related to prolonged TTFT in the U.S.

We consider the problem of predicting an individual's identity from accelerometry data collected during walking. In a previous paper, we transformed the accelerometry time series into an image by constructing the joint distribution of the acceleration and lagged acceleration for a vector of lags. Predictors derived by partitioning this image into grid cells were used in logistic regression to predict individuals. Here, we (a) implement machine learning methods for prediction using the grid cell-derived predictors; (b) derive inferential methods to screen for the most predictive grid cells while adjusting for correlation and multiple comparisons; and (c) develop a novel multivariate functional regression model that avoids partitioning the predictor space. Prediction methods are compared on two open source acceleometry data sets collected from: (a) 32 individuals walking on a $1.06$ km path; and (b) six repetitions of walking on a 20 m path on two occasions at least 1 week apart for 153 study participants. In the 32-individual study, all methods achieve at least 95% rank-1 accuracy, while in the 153-individual study, accuracy varies from 41% to 98%, depending on the method and prediction task. Methods provide insights into why some individuals are easier to predict than others.

Wildland fire smoke exposures are an increasing threat to public health, highlighting the need for studying the effects of protective behaviours on reducing health outcomes. Emerging smartphone applications provide unprecedented opportunities to deliver health risk communication messages to a large number of individuals in real-time and subsequently study the effectiveness, but also pose methodological challenges. Smoke Sense, a citizen science project, provides an interactive smartphone app platform for participants to engage with information about air quality, and ways to record their own health symptoms and actions taken to reduce smoke exposure. We propose a doubly robust estimator of the structural nested mean model that accounts for spatially and time-varying effects via a local estimating equation approach with geographical kernel weighting. Moreover, our analytical framework also handles informative missingness by inverse probability weighting of estimating functions. We evaluate the method using extensive simulation studies and apply it to Smoke Sense data to increase the knowledge base about the relationship between health preventive measures and health-related outcomes. Our results show that the protective behaviours' effects vary over space and time and find that protective behaviours have more significant effects on reducing health symptoms in the Southwest than the Northwest region of the U.S.

Motivated by the questions of risk assessment in climatology (temperature change in North America) and medicine (impact of statin usage and coronavirus disease 2019 on hospitalized patients), we address the problem of estimating the set in the domain of a function whose image equals a predefined subset of the real line. Existing methods require strict assumptions. We generalize the estimation of such sets to dense and nondense domains with protection against inflated Type I error in exploratory data analysis. This is achieved by proving that confidence sets of multiple upper, lower, or interval sets can be simultaneously constructed with the desired confidence nonasymptotically through inverting simultaneous confidence intervals. Nonparametric bootstrap algorithm and code are provided.

Functional magnetic resonance imaging (fMRI) is a noninvasive and in-vivo imaging technique essential for measuring brain activity. Functional connectivity is used to study associations between brain regions, either while study subjects perform tasks or during periods of rest. In this paper, we propose a rigorous definition of task-evoked functional connectivity at the population level (ptFC). Importantly, our proposed ptFC is interpretable in the context of task-fMRI studies. An algorithm for estimating the ptFC is provided. We present the performance of the proposed algorithm compared to existing functional connectivity frameworks using simulations. Lastly, we apply the proposed algorithm to estimate the ptFC in a motor-task study from the Human Connectome Project.

The use of digital devices to collect data in mobile health studies introduces a novel application of time series methods, with the constraint of potential data missing at random or missing not at random (MNAR). In time-series analysis, testing for stationarity is an important preliminary step to inform appropriate subsequent analyses. The Dickey-Fuller test evaluates the null hypothesis of unit root non-stationarity, under no missing data. Beyond recommendations under data missing completely at random for complete case analysis or last observation carry forward imputation, researchers have not extended unit root non-stationarity testing to more complex missing data mechanisms. Multiple imputation with chained equations, Kalman smoothing imputation, and linear interpolation have also been used for time-series data, however such methods impose constraints on the autocorrelation structure and impact unit root testing. We propose maximum likelihood estimation and multiple imputation using state space model approaches to adapt the augmented Dickey-Fuller test to a context with missing data. We further develop sensitivity analyses to examine the impact of MNAR data. We evaluate the performance of existing and proposed methods across missing mechanisms in extensive simulations and in their application to a multi-year smartphone study of bipolar patients.

In many contexts, particularly when study subjects are adolescents, peer effects can invalidate typical statistical requirements in the data. For instance, it is plausible that a student's academic performance is influenced both by their own mother's educational level as well as that of their peers. Since the underlying social network is measured, the Add Health study provides a unique opportunity to examine the impact of maternal college education on adolescent school performance, both direct and indirect. However, causal inference on populations embedded in social networks poses technical challenges, since the typical no interference assumption no longer holds. While inverse probability-of-treatment weighted (IPW) estimators have been developed for this setting, they are often highly unstable. Motivated by the question of maternal education, we propose doubly robust (DR) estimators combining models for treatment and outcome that are consistent and asymptotically normal if either model is correctly specified. We present empirical results that illustrate the DR property and the efficiency gain of DR over IPW estimators even when the treatment model is misspecified. Contrary to previous studies, our robust analysis does not provide evidence of an indirect effect of maternal education on academic performance within adolescents' social circles in Add Health.