Design, analysis, and interpretation of treatment response heterogeneity in personalized nutrition and obesity treatment research

Author:

Zoh Roger S.1ORCID,Esteves Bridget H.2ORCID,Yu Xiaoxin1,Fairchild Amanda J.3ORCID,Vazquez Ana I.4,Chapple Andrew G.5ORCID,Brown Andrew W.6ORCID,George Brandon7,Gordon Derek8,Landsittel Douglas1,Gadbury Gary L.9,Pavela Greg10,de los Campos Gustavo11,Mestre Luis M.12ORCID,Allison David B.1

Affiliation:

1. Department of Epidemiology and Biostatistics Indiana University School of Public Health‐Bloomington Bloomington Indiana USA

2. Glanbia Performance Nutrition Downers Grove Illinois USA

3. Department of Psychology University of South Carolina Columbia South Carolina USA

4. Department of Epidemiology and Biostatistics Michigan State University Lansing Michigan USA

5. Biostatistics Program, School of Public Health LSU Health Sciences Center New Orleans Louisiana USA

6. Department of Applied Health Science Indiana University School of Public Health‐Bloomington Bloomington Indiana USA

7. College of Population Health Thomas Jefferson University Philadelphia Pennsylvania USA

8. Department of Genetics Rutgers Robert Wood Johnson Medical School New Brunswick New Jersey USA

9. Department of Statistics Kansas State University Manhattan Kansa USA

10. Department of Health Behavior University of Alabama at Birmingham Birmingham Alabama USA

11. Departments of Epidemiology & Biostatistics and Statistics & Probability, IQ ‐ Institute for Quantitative Health Science and Engineering Michigan State University Lansing Michigan USA

12. Department of Psychiatry Yale School of Medicine New Haven Connecticut USA

Abstract

SummaryIt is increasingly assumed that there is no one‐size‐fits‐all approach to dietary recommendations for the management and treatment of chronic diseases such as obesity. This phenomenon that not all individuals respond uniformly to a given treatment has become an area of research interest given the rise of personalized and precision medicine. To conduct, interpret, and disseminate this research rigorously and with scientific accuracy, however, requires an understanding of treatment response heterogeneity. Here, we define treatment response heterogeneity as it relates to clinical trials, provide statistical guidance for measuring treatment response heterogeneity, and highlight study designs that can quantify treatment response heterogeneity in nutrition and obesity research. Our goal is to educate nutrition and obesity researchers in how to correctly identify and consider treatment response heterogeneity when analyzing data and interpreting results, leading to rigorous and accurate advancements in the field of personalized medicine.

Funder

National Institutes of Health

National Institute on Aging

National Cancer Institute

Publisher

Wiley

Subject

Public Health, Environmental and Occupational Health,Endocrinology, Diabetes and Metabolism

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