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Matched Learning for Optimizing Individualized Treatment Strategies Using Electronic Health Records.

ABSTRACT: Current guidelines for treatment decision making largely rely on data from randomized controlled trials (RCTs) studying average treatment effects. They may be inadequate to make individualized treatment decisions in real-world settings. Large-scale electronic health records (EHR) provide opportunities to fulfill the goals of personalized medicine and learn individualized treatment rules (ITRs) depending on patient-specific characteristics from real-world patient data. In this work, we tackle challenges with EHRs and propose a machine learning approach based on matching (M-learning) to estimate optimal ITRs from EHRs. This new learning method performs matching instead of inverse probability weighting as commonly used in many existing methods for estimating ITRs to more accurately assess individuals' treatment responses to alternative treatments and alleviate confounding. Matching-based value functions are proposed to compare matched pairs under a unified framework, where various types of outcomes for measuring treatment response (including continuous, ordinal, and discrete outcomes) can easily be accommodated. We establish the Fisher consistency and convergence rate of M-learning. Through extensive simulation studies, we show that M-learning outperforms existing methods when propensity scores are misspecified or when unmeasured confounders are present in certain scenarios. Lastly, we apply M-learning to estimate optimal personalized second-line treatments for type 2 diabetes patients to achieve better glycemic control or reduce major complications using EHRs from New York Presbyterian Hospital.

SUBMITTER: Wu P

PROVIDER: S-EPMC7539620 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature

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Matched Learning for Optimizing Individualized Treatment Strategies Using Electronic Health Records.

Wu Peng P Zeng Donglin D Wang Yuanjia Y

Journal of the American Statistical Association 20190423 529

Current guidelines for treatment decision making largely rely on data from randomized controlled trials (RCTs) studying average treatment effects. They may be inadequate to make individualized treatment decisions in real-world settings. Large-scale electronic health records (EHR) provide opportunities to fulfill the goals of personalized medicine and learn individualized treatment rules (ITRs) depending on patient-specific characteristics from real-world patient data. In this work, we tackle cha ...[more]

PMID: 33041401

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Project description:BackgroundNowadays, patients with chronic diseases such as diabetes and hypertension have reached alarming numbers worldwide. These diseases increase the risk of developing acute complications and involve a substantial economic burden and demand for health resources. The widespread adoption of Electronic Health Records (EHRs) is opening great opportunities for supporting decision-making. Nevertheless, data extracted from EHRs are complex (heterogeneous, high-dimensional and usually noisy), hampering the knowledge extraction with conventional approaches.MethodsWe propose the use of the Denoising Autoencoder (DAE), a Machine Learning (ML) technique allowing to transform high-dimensional data into latent representations (LRs), thus addressing the main challenges with clinical data. We explore in this work how the combination of LRs with a visualization method can be used to map the patient data in a two-dimensional space, gaining knowledge about the distribution of patients with different chronic conditions. Furthermore, this representation can be also used to characterize the patient's health status evolution, which is of paramount importance in the clinical setting.ResultsTo obtain clinical LRs, we considered real-world data extracted from EHRs linked to the University Hospital of Fuenlabrada in Spain. Experimental results showed the great potential of DAEs to identify patients with clinical patterns linked to hypertension, diabetes and multimorbidity. The procedure allowed us to find patients with the same main chronic disease but different clinical characteristics. Thus, we identified two kinds of diabetic patients with differences in their drug therapy (insulin and non-insulin dependant), and also a group of women affected by hypertension and gestational diabetes. We also present a proof of concept for mapping the health status evolution of synthetic patients when considering the most significant diagnoses and drugs associated with chronic patients.ConclusionOur results highlighted the value of ML techniques to extract clinical knowledge, supporting the identification of patients with certain chronic conditions. Furthermore, the patient's health status progression on the two-dimensional space might be used as a tool for clinicians aiming to characterize health conditions and identify their more relevant clinical codes.

Project description:BackgroundThe main goal of this study is to explore the use of features representing patient-level electronic health record (EHR) data, generated by the unsupervised deep learning algorithm autoencoder, in predictive modeling. Since autoencoder features are unsupervised, this paper focuses on their general lower-dimensional representation of EHR information in a wide variety of predictive tasks.MethodsWe compare the model with autoencoder features to traditional models: logistic model with least absolute shrinkage and selection operator (LASSO) and Random Forest algorithm. In addition, we include a predictive model using a small subset of response-specific variables (Simple Reg) and a model combining these variables with features from autoencoder (Enhanced Reg). We performed the study first on simulated data that mimics real world EHR data and then on actual EHR data from eight Advocate hospitals.ResultsOn simulated data with incorrect categories and missing data, the precision for autoencoder is 24.16% when fixing recall at 0.7, which is higher than Random Forest (23.61%) and lower than LASSO (25.32%). The precision is 20.92% in Simple Reg and improves to 24.89% in Enhanced Reg. When using real EHR data to predict the 30-day readmission rate, the precision of autoencoder is 19.04%, which again is higher than Random Forest (18.48%) and lower than LASSO (19.70%). The precisions for Simple Reg and Enhanced Reg are 18.70 and 19.69% respectively. That is, Enhanced Reg can have competitive prediction performance compared to LASSO. In addition, results show that Enhanced Reg usually relies on fewer features under the setting of simulations of this paper.ConclusionsWe conclude that autoencoder can create useful features representing the entire space of EHR data and which are applicable to a wide array of predictive tasks. Together with important response-specific predictors, we can derive efficient and robust predictive models with less labor in data extraction and model training.

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Matched Learning for Optimizing Individualized Treatment Strategies Using Electronic Health Records.

Publications

Matched Learning for Optimizing Individualized Treatment Strategies Using Electronic Health Records.

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