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Nansu Zong

Publications and source records attributed to Nansu Zong.

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Optimizing Clinical Trial Protocols Using EHR-Derived Heterogeneous Treatment Effects

Traditional randomized trials often obscure clinically meaningful heterogeneity in treatment response by focusing on average effects. Leveraging real-world data to emulate clinical trials and estimate heterogeneous treatment effects (HTEs) offers a promising path toward more precise and efficient trial design. In this study, we emulate the DAPA-HF trial using electronic health records from the Mayo Clinic Cloud (MCC) to investigate whether HTE-guided stratification can identify patient subgroups with distinct treatment responses to dapagliflozin versus placebo in patients with heart failure with reduced ejection fraction. All-cause mortality was evaluated using Cox proportional hazards models, with HTEs estimated using a Meta-S learner and subgroups defined using a decision tree-based thresholding approach. In the overall cohort of the emulation, no significant treatment difference was observed (HR, 1.681; 95% CI, 0.828-3.413; p = 0.1507). However, compared with the overall emulated cohort, in which dapagliflozin showed no statistically significant survival benefit, HTE-driven stratification identified subgroups with significant and directionally distinct treatment effects. The beneficial (low-HTE) subgroup showed a significant survival benefit from dapagliflozin (HR = 0.203, 95% CI, 0.087-0.476, p = 0.0002), whereas the harmful (high-HTE) subgroup showed a significant harmful association with markedly increased mortality risk (HR = 6.680, 95% CI, 2.759-16.171, p < 0.0001). These findings indicate that HTE-guided stratification can uncover clinically meaningful beneficial and harmful treatment-effect patterns that are masked in the full-cohort emulation.

stat.AP

Lightweight Retrieval-Augmented Generation and Large Language Model-Based Modeling for Scalable Patient-Trial Matching

Patient-trial matching requires reasoning over long, heterogeneous electronic health records (EHRs) and complex eligibility criteria, posing significant challenges for scalability, generalization, and computational efficiency. Existing approaches either rely on full-document processing with large language models (LLMs), which is computationally expensive, or use traditional machine learning methods that struggle to capture unstructured clinical narratives. In this work, we propose a lightweight framework that combines retrieval-augmented generation and large language model-based modeling for scalable patient-trial matching. The framework explicitly separates two key components: retrieval-augmented generation is used to identify clinically relevant segments from long EHRs, reducing input complexity, while large language models are used to encode these selected segments into informative representations. These representations are further refined through dimensionality reduction and modeled using lightweight predictors, enabling efficient and scalable downstream classification. We evaluate the proposed approach on multiple public benchmarks (n2c2, SIGIR, TREC 2021/2022) and a real-world multimodal dataset from Mayo Clinic (MCPMD). Results show that retrieval-based information selection significantly reduces computational burden while preserving clinically meaningful signals. We further demonstrate that frozen LLMs provide strong representations for structured clinical data, whereas fine-tuning is essential for modeling unstructured clinical narratives. Importantly, the proposed lightweight pipeline achieves performance comparable to end-to-end LLM approaches with substantially lower computational cost.

cs.CL

LLM-Match: An Open-Sourced Patient Matching Model Based on Large Language Models and Retrieval-Augmented Generation

Patient matching is the process of linking patients to appropriate clinical trials by accurately identifying and matching their medical records with trial eligibility criteria. We propose LLM-Match, a novel framework for patient matching leveraging fine-tuned open-source large language models. Our approach consists of four key components. First, a retrieval-augmented generation (RAG) module extracts relevant patient context from a vast pool of electronic health records (EHRs). Second, a prompt generation module constructs input prompts by integrating trial eligibility criteria (both inclusion and exclusion criteria), patient context, and system instructions. Third, a fine-tuning module with a classification head optimizes the model parameters using structured prompts and ground-truth labels. Fourth, an evaluation module assesses the fine-tuned model's performance on the testing datasets. We evaluated LLM-Match on four open datasets - n2c2, SIGIR, TREC 2021, and TREC 2022 - using open-source models, comparing it against TrialGPT, Zero-Shot, and GPT-4-based closed models. LLM-Match outperformed all baselines.

cs.CL

Launching Insights: A Pilot Study on Leveraging Real-World Observational Data from the Mayo Clinic Platform to Advance Clinical Research

Backgrounds: Artificial intelligence (AI) is transforming healthcare, yet translating AI models from theoretical frameworks to real-world clinical applications remains challenging. The Mayo Clinic Platform (MCP) was established to address these challenges by providing a scalable ecosystem that integrates real-world multiple modalities data from multiple institutions, advanced analytical tools, and secure computing environments to support clinical research and AI development. Methods: In this study, we conducted four research projects leveraging MCP's data infrastructure and analytical capabilities to demonstrate its potential in facilitating real-world evidence generation and AI-driven clinical insights. Utilizing MCP's tools and environment, we facilitated efficient cohort identification, data extraction, and subsequent statistical or AI-powered analyses. Results: The results underscore MCP's role in accelerating translational research by offering de-identified, standardized real-world data and facilitating AI model validation across diverse healthcare settings. Compared to Mayo's internal Electronic Health Record (EHR) data, MCP provides broader accessibility, enhanced data standardization, and multi-institutional integration, making it a valuable resource for both internal and external researchers. Conclusion: Looking ahead, MCP is well-positioned to transform clinical research through its scalable ecosystem, effectively bridging the divide between AI innovation and clinical deployment. Future investigations will build upon this foundation, further exploring MCP's capacity to advance precision medicine and enhance patient outcomes.

cs.CY

Detecting Reddit Users with Depression Using a Hybrid Neural Network SBERT-CNN

Depression is a widespread mental health issue, affecting an estimated 3.8% of the global population. It is also one of the main contributors to disability worldwide. Recently it is becoming popular for individuals to use social media platforms (e.g., Reddit) to express their difficulties and health issues (e.g., depression) and seek support from other users in online communities. It opens great opportunities to automatically identify social media users with depression by parsing millions of posts for potential interventions. Deep learning methods have begun to dominate in the field of machine learning and natural language processing (NLP) because of their ease of use, efficient processing, and state-of-the-art results on many NLP tasks. In this work, we propose a hybrid deep learning model which combines a pretrained sentence BERT (SBERT) and convolutional neural network (CNN) to detect individuals with depression with their Reddit posts. The sentence BERT is used to learn the meaningful representation of semantic information in each post. CNN enables the further transformation of those embeddings and the temporal identification of behavioral patterns of users. We trained and evaluated the model performance to identify Reddit users with depression by utilizing the Self-reported Mental Health Diagnoses (SMHD) data. The hybrid deep learning model achieved an accuracy of 0.86 and an F1 score of 0.86 and outperformed the state-of-the-art documented result (F1 score of 0.79) by other machine learning models in the literature. The results show the feasibility of the hybrid model to identify individuals with depression. Although the hybrid model is validated to detect depression with Reddit posts, it can be easily tuned and applied to other text classification tasks and different clinical applications.

cs.CL

Evaluation of GPT-3 for Anti-Cancer Drug Sensitivity Prediction

In this study, we investigated the potential of GPT-3 for the anti-cancer drug sensitivity prediction task using structured pharmacogenomics data across five tissue types and evaluated its performance with zero-shot prompting and fine-tuning paradigms. The drug's smile representation and cell line's genomic mutation features were predictive of the drug response. The results from this study have the potential to pave the way for designing more efficient treatment protocols in precision oncology.

cs.LG