Dataset Information

Entropy Learning for Dynamic Treatment Regimes.

ABSTRACT: Estimating optimal individualized treatment rules (ITRs) in single or multi-stage clinical trials is one key solution to personalized medicine and has received more and more attention in statistical community. Recent development suggests that using machine learning approaches can significantly improve the estimation over model-based methods. However, proper inference for the estimated ITRs has not been well established in machine learning based approaches. In this paper, we propose a entropy learning approach to estimate the optimal individualized treatment rules (ITRs). We obtain the asymptotic distributions for the estimated rules so further provide valid inference. The proposed approach is demonstrated to perform well in finite sample through extensive simulation studies. Finally, we analyze data from a multi-stage clinical trial for depression patients. Our results offer novel findings that are otherwise not revealed with existing approaches.

SUBMITTER: Jiang B

PROVIDER: S-EPMC6750237 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Entropy Learning for Dynamic Treatment Regimes.

Jiang Binyan B Song Rui R Li Jialiang J Zeng Donglin D

Statistica Sinica 20190101 4

Estimating optimal individualized treatment rules (ITRs) in single or multi-stage clinical trials is one key solution to personalized medicine and has received more and more attention in statistical community. Recent development suggests that using machine learning approaches can significantly improve the estimation over model-based methods. However, proper inference for the estimated ITRs has not been well established in machine learning based approaches. In this paper, we propose a entropy lea ...[more]

PMID: 31534307

Similar Datasets

Project description:Precision medicine is currently a topic of great interest in clinical and intervention science. A key component of precision medicine is that it is evidence-based, i.e., data-driven, and consequently there has been tremendous interest in estimation of precision medicine strategies using observational or randomized study data. One way to formalize precision medicine is through a treatment regime, which is a sequence of decision rules, one per stage of clinical intervention, that map up-to-date patient information to a recommended treatment. An optimal treatment regime is defined as maximizing the mean of some cumulative clinical outcome if applied to a population of interest. It is well-known that even under simple generative models an optimal treatment regime can be a highly nonlinear function of patient information. Consequently, a focal point of recent methodological research has been the development of flexible models for estimating optimal treatment regimes. However, in many settings, estimation of an optimal treatment regime is an exploratory analysis intended to generate new hypotheses for subsequent research and not to directly dictate treatment to new patients. In such settings, an estimated treatment regime that is interpretable in a domain context may be of greater value than an unintelligible treatment regime built using 'black-box' estimation methods. We propose an estimator of an optimal treatment regime composed of a sequence of decision rules, each expressible as a list of "if-then" statements that can be presented as either a paragraph or as a simple flowchart that is immediately interpretable to domain experts. The discreteness of these lists precludes smooth, i.e., gradient-based, methods of estimation and leads to non-standard asymptotics. Nevertheless, we provide a computationally efficient estimation algorithm, prove consistency of the proposed estimator, and derive rates of convergence. We illustrate the proposed methods using a series of simulation examples and application to data from a sequential clinical trial on bipolar disorder.

Dataset Information

Entropy Learning for Dynamic Treatment Regimes.

Publications

Entropy Learning for Dynamic Treatment Regimes.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets