Benjamin A. Goldstein

Minghui Sun, Matthew M. Engelhard, Benjamin A. Goldstein

Risk assessments for a pediatric population are often conducted across multiple stages. For example, clinicians may evaluate risks prenatally, at birth, and during Well-Child visits. Although predictions made at later stages typically achieve higher precision, it is clinically desirable to make reliable risk assessments as early as possible. Therefore, this study focuses on improving prediction performance in early-stage risk assessments. Our solution, \textbf{Borrowing From the Future (BFF)}, is a contrastive multi-modal framework that treats each time window as a distinct modality. In BFF, a model is trained on all available data throughout the time while performing a risk assessment using up-to-date information. This contrastive framework allows the model to ``borrow'' informative signals from later stages (e.g., Well-Child visits) to implicitly supervise the learning at earlier stages (e.g., prenatal/birth stages). We validate BFF on two real-world pediatric outcome prediction tasks, demonstrating consistent improvements in early risk assessments. The code is available at https://github.com/scotsun/bff.

Minghui Sun, Benjamin A. Goldstein, Matthew M. Engelhard

Learning representations unaffected by superficial characteristics is important to ensure that shifts in these characteristics at test time do not compromise downstream prediction performance. For instance, in healthcare applications, we might like to learn features that contain information about pathology yet are unaffected by race, sex, and other sources of physiologic variability, thereby ensuring predictions are equitable and generalizable across all demographics. Here we propose Contrastive LEarning with Anti-contrastive Regularization (CLEAR), an intuitive and easy-to-implement framework that effectively separates essential (i.e., task-relevant) characteristics from superficial (i.e., task-irrelevant) characteristics during training, leading to better performance when superficial characteristics shift at test time. We begin by supposing that data representations can be semantically separated into task-relevant content features, which contain information relevant to downstream tasks, and task-irrelevant style features, which encompass superficial attributes that are irrelevant to these tasks, yet may degrade performance due to associations with content present in training data that do not generalize. We then prove that our anti-contrastive penalty, which we call Pair-Switching (PS), minimizes the Mutual Information between the style attributes and content labels. Finally, we instantiate CLEAR in the latent space of a Variational Auto-Encoder (VAE), then perform experiments to quantitatively and qualitatively evaluate the resulting CLEAR-VAE over several image datasets. Our results show that CLEAR-VAE allows us to: (a) swap and interpolate content and style between any pair of samples, and (b) improve downstream classification performance in the presence of previously unseen combinations of content and style. Our code will be made publicly available.

Zigui Wang, Minghui Sun, Jiang Shu et al.

Unstructured Electronic Health Record (EHR) data, such as clinical notes, contain clinical contextual observations that are not directly reflected in structured data fields. This additional information can substantially improve model learning. However, due to their unstructured nature, these data are often unavailable or impractical to use when deploying a model. We introduce a multimodal learning framework that leverages unstructured EHR data during training while producing a model that can be deployed using only structured EHR data. Using a cohort of 3,466 children evaluated for late talking, we generated note embeddings with BioClinicalBERT and encoded structured embeddings from demographics and medical codes. A note-based teacher model and a structured-only student model were jointly trained using contrastive learning and contrastive knowledge distillation loss, producing a strong classifier (AUROC = 0.985). Our proposed model reached an AUROC of 0.705, outperforming the structured-only baseline of 0.656. These results demonstrate that incorporating unstructured data during training enhances the model's capacity to identify task-relevant information within structured EHR data, enabling a deployable structured-only phenotype model.

Mengying Yan, Ziye Tian, Siqi Li et al.

Clinical decision support tools built on electronic health records often experience performance drift due to temporal population shifts, particularly when changes in the clinical environment initially affect only a subset of patients, resulting in a transition to mixed populations. Such case-mix changes commonly arise following system-level operational updates or the emergence of new diseases, such as COVID-19. We propose TRACER (Transfer Learning-based Real-time Adaptation for Clinical Evolving Risk), a framework that identifies encounter-level transition membership and adapts predictive models using transfer learning without full retraining. In simulation studies, TRACER outperformed static models trained on historical or contemporary data. In a real-world application predicting hospital admission following emergency department visits across the COVID-19 transition, TRACER improved both discrimination and calibration. TRACER provides a scalable approach for maintaining robust predictive performance under evolving and heterogeneous clinical conditions.

Pengxi Liu, Yi Shen, Matthew M. Engelhard et al.

Fairness metrics utilizing the area under the receiver operator characteristic curve (AUC) have gained increasing attention in high-stakes domains such as healthcare, finance, and criminal justice. In these domains, fairness is often evaluated over risk scores rather than binary outcomes, and a common challenge is that enforcing strict fairness can significantly degrade AUC performance. To address this challenge, we propose Fair Proportional Optimal Transport (FairPOT), a novel, model-agnostic post-processing framework that strategically aligns risk score distributions across different groups using optimal transport, but does so selectively by transforming a controllable proportion, i.e., the top-lambda quantile, of scores within the disadvantaged group. By varying lambda, our method allows for a tunable trade-off between reducing AUC disparities and maintaining overall AUC performance. Furthermore, we extend FairPOT to the partial AUC setting, enabling fairness interventions to concentrate on the highest-risk regions. Extensive experiments on synthetic, public, and clinical datasets show that FairPOT consistently outperforms existing post-processing techniques in both global and partial AUC scenarios, often achieving improved fairness with slight AUC degradation or even positive gains in utility. The computational efficiency and practical adaptability of FairPOT make it a promising solution for real-world deployment.

Zidi Xiu, Chenyang Tao, Michael Gao et al.

Combining the increasing availability and abundance of healthcare data and the current advances in machine learning methods have created renewed opportunities to improve clinical decision support systems. However, in healthcare risk prediction applications, the proportion of cases with the condition (label) of interest is often very low relative to the available sample size. Though very prevalent in healthcare, such imbalanced classification settings are also common and challenging in many other scenarios. So motivated, we propose a variational disentanglement approach to semi-parametrically learn from rare events in heavily imbalanced classification problems. Specifically, we leverage the imposed extreme-distribution behavior on a latent space to extract information from low-prevalence events, and develop a robust prediction arm that joins the merits of the generalized additive model and isotonic neural nets. Results on synthetic studies and diverse real-world datasets, including mortality prediction on a COVID-19 cohort, demonstrate that the proposed approach outperforms existing alternatives.

Zidi Xiu, Chenyang Tao, Benjamin A. Goldstein et al.

The abundance of modern health data provides many opportunities for the use of machine learning techniques to build better statistical models to improve clinical decision making. Predicting time-to-event distributions, also known as survival analysis, plays a key role in many clinical applications. We introduce a variational time-to-event prediction model, named Variational Survival Inference (VSI), which builds upon recent advances in distribution learning techniques and deep neural networks. VSI addresses the challenges of non-parametric distribution estimation by ($i$) relaxing the restrictive modeling assumptions made in classical models, and ($ii$) efficiently handling the censored observations, {\it i.e.}, events that occur outside the observation window, all within the variational framework. To validate the effectiveness of our approach, an extensive set of experiments on both synthetic and real-world datasets is carried out, showing improved performance relative to competing solutions.

Benjamin A. Goldstein, Matthew Phelan, Neha J. Pagidipati et al.

Participants enrolled into randomized controlled trials (RCTs) often do not reflect real-world populations. Previous research in how best to translate RCT results to target populations has focused on weighting RCT data to look like the target data. Simulation work, however, has suggested that an outcome model approach may be preferable. Here we describe such an approach using source data from the 2x2 factorial NAVIGATOR trial which evaluated the impact of valsartan and nateglinide on cardiovascular outcomes and new-onset diabetes in a pre-diabetic population. Our target data consisted of people with pre-diabetes serviced at our institution. We used Random Survival Forests to develop separate outcome models for each of the 4 treatments, estimating the 5-year risk difference for progression to diabetes and estimated the treatment effect in our local patient populations, as well as sub-populations, and the results compared to the traditional weighting approach. Our models suggested that the treatment effect for valsartan in our patient population was the same as in the trial, whereas for nateglinide treatment effect was stronger than observed in the original trial. Our effect estimates were more efficient than the weighting approach.