Evidence map›Paper›PMID 30034060›Full record

ArticleJournal of the American Statistical Association2018

Learning Optimal Personalized Treatment Rules in Consideration of Benefit and Risk: with an Application to Treating Type 2 Diabetes Patients with Insulin Therapies.

Yuanjia Wang, Haoda Fu, Donglin Zeng

Open access · greenAbstract read
In one paragraph

Article in Journal of the American Statistical Association, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers.

0numbers the graph read from it
0cells of the map it votes in
32citing papers in PubMed
5.7field-weighted citation impact, top 3% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

32 citing papers in PubMed, 78 citations in OpenAlex.

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  9. Fusing Individualized Treatment Rules Using Secondary Outcomes.Proceedings of machine learning research · 2024
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

3 authors at 3 institutions in 1 country.

Yuanjia WangDepartment of Biostatistics, Mailman School of Public Health, Columbia University, New York, NY 10032.
Haoda FuEli Lilly and Company.
Donglin ZengDepartment of Biostatistics, University of North Carolina at Chapel Hill.
Columbia University · USEli Lilly (United States) · USUniversity of North Carolina at Chapel Hill · US

Funding

Statistical Methods for Integrating Mixed-type Biomarkers and Phenotypes in Neurodegenerative Disease ModelingR01NS073671 · NINDS · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI WANG, YUANJIA · 2011 to 2025
$3.9M
Semiparametric Regression Analysis of Censored DataR37GM047845 · NIGMS · UNIV OF NORTH CAROLINA CHAPEL HILL · PI LIN, DANYU · 2005 to 2014
$3.3M
Statistical and Machine Learning Methods to Improve Dynamic Treatment Regimens Estimation Using Real World Data.R01GM124104 · NIGMS · UNIV OF NORTH CAROLINA CHAPEL HILL · PI Yuanjia Wang, Donglin Zeng · 2018 to 2026
$3.1M
Semiparametric Regression Analysis of Censored DataR01GM047845 · NIGMS · UNIV OF NORTH CAROLINA CHAPEL HILL · PI LIN, DANYU · 1997 to 2018
$2.9M
Identifying Huntington's disease markers by modern statistical learning methods.U01NS082062 · NINDS · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI WANG, YUANJIA · 2014 to 2016
$1.1M
NIGMS NIH HHS R01 GM047845NIGMS NIH HHS R01 GM124104NIGMS NIH HHS R37 GM047845NINDS NIH HHS R01 NS073671NINDS NIH HHS U01 NS082062
6 · The paper itself

Abstract

Individualized medical decision making is often complex due to patient treatment response heterogeneity. Pharmacotherapy may exhibit distinct efficacy and safety profiles for different patient populations. An "optimal" treatment that maximizes clinical benefit for a patient may also lead to concern of safety due to a high risk of adverse events. Thus, to guide individualized clinical decision making and deliver optimal tailored treatments, maximizing clinical benefit should be considered in the context of controlling for potential risk. In this work, we propose two approaches to identify personalized optimal treatment strategy that maximizes clinical benefit under a constraint on the average risk. We derive the theoretical optimal treatment rule under the risk constraint and draw an analogy to the Neyman-Pearson lemma to prove the theorem. We present algorithms that can be easily implemented by any off-the-shelf quadratic programming package. We conduct extensive simulation studies to show satisfactory risk control when maximizing the clinical benefit. Lastly, we apply our method to a randomized trial of type 2 diabetes patients to guide optimal utilization of the first line insulin treatments based on individual patient characteristics while controlling for the rate of hypoglycemia events. We identify baseline glycated hemoglobin level, body mass index, and fasting blood glucose as three key factors among 18 biomarkers to differentiate treatment assignments, and demonstrate a successful control of the risk of hypoglycemia in both the training and testing data set.

Indexed as

Benefit Risk AnalysisHypoglycemiaMachine LearningNeyman-Pearson LemmaPersonalized Medicine

Identifiers

PMID30034060
PMCPMC6051551
OpenAlexW2599189192

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.