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Introducing the Endotype Concept to Address the Challenge of Disease Heterogeneity in Type 1 Diabetes.

Abstract：

:The clinical diagnosis of new-onset type 1 diabetes has, for many years, been considered relatively straightforward. Recently, however, there is increasing awareness that within this single clinical phenotype exists considerable heterogeneity: disease onset spans the complete age range; genetic susceptibility is complex; rates of progression differ markedly, as does insulin secretory capacity; and complication rates, glycemic control, and therapeutic intervention efficacy vary widely. Mechanistic and immunopathological studies typically show considerable patchiness across subjects, undermining conclusions regarding disease pathways. Without better understanding, type 1 diabetes heterogeneity represents a major barrier both to deciphering pathogenesis and to the translational effort of designing, conducting, and interpreting clinical trials of disease-modifying agents. This realization comes during a period of unprecedented change in clinical medicine, with increasing emphasis on greater individualization and precision. For complex disorders such as type 1 diabetes, the option of maintaining the "single disease" approach appears untenable, as does the notion of individualizing each single patient's care, obliging us to conceptualize type 1 diabetes less in terms of phenotypes (observable characteristics) and more in terms of disease endotypes (underlying biological mechanisms). Here, we provide our view on an approach to dissect heterogeneity in type 1 diabetes. Using lessons from other diseases and the data gathered to date, we aim to delineate a roadmap through which the field can incorporate the endotype concept into laboratory and clinical practice. We predict that such an effort will accelerate the implementation of precision medicine and has the potential for impact on our approach to translational research, trial design, and clinical management.

journal_name

Diabetes Care

journal_title

Diabetes care

authors

Battaglia M,Ahmed S,Anderson MS,Atkinson MA,Becker D,Bingley PJ,Bosi E,Brusko TM,DiMeglio LA,Evans-Molina C,Gitelman SE,Greenbaum CJ,Gottlieb PA,Herold KC,Hessner MJ,Knip M,Jacobsen L,Krischer JP,Long SA,Lundgren M

doi

10.2337/dc19-0880

subject

Has Abstract

pub_date

2020-01-01 00:00:00

pages

5-12

issue

eissn

0149-5992

issn

1935-5548

pii

dc19-0880

journal_volume

pub_type

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Introducing the Endotype Concept to Address the Challenge of Disease Heterogeneity in Type 1 Diabetes.