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

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

Battaglia, Manuela (författare): San Raffaele Scientific Institute,San Raffaele Hospital

Ahmed, Simi (författare): Juvenile Diabetes Research Foundation

Anderson, Mark S. (författare): University of California, San Francisco

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Atkinson, Mark A. (författare): University of Florida

Becker, Dorothy (författare): Children's Hospital of Pittsburgh of UPMC

Bingley, Polly J. (författare): University of Bristol

Bosi, Emanuele (författare): Vita-Salute San Raffaele University,San Raffaele Hospital

Brusko, Todd M. (författare): University of Florida

DiMeglio, Linda A. (författare): Indiana University

Evans-Molina, Carmella (författare): Indiana University

Gitelman, Stephen E. (författare): University of California, San Francisco

Greenbaum, Carla J. (författare): Benaroya Research Institute

Gottlieb, Peter A. (författare): University of Colorado

Herold, Kevan C. (författare): Yale University

Hessner, Martin J. (författare): Medical College of Wisconsin

Knip, Mikael (författare): University of Helsinki,Helsinki University Children's Hospital

Jacobsen, Laura (författare): University of Florida

Krischer, Jeffrey P. (författare): University of South Florida

Long, S. Alice (författare): Benaroya Research Institute

Lundgren, Markus (författare): Lund University,Lunds universitet,Pediatrisk endokrinologi,Forskargrupper vid Lunds universitet,Paediatric Endocrinology,Lund University Research Groups,Skåne University Hospital

McKinney, Eoin F. (författare): Addenbrooke's Hospital,University of Cambridge

Morgan, Noel G. (författare): University of Exeter,Royal Devon & Exeter Hospital

Oram, Richard A. (författare): Royal Devon & Exeter Hospital,University of Exeter

Pastinen, Tomi (författare): Children's Mercy Hospitals and Clinics, Kansas City

Peters, Michael C. (författare): University of California, San Francisco

Petrelli, Alessandra (författare): San Raffaele Scientific Institute,San Raffaele Hospital

Qian, Xiaoning (författare): Texas A and M University

Redondo, Maria J. (författare): Baylor College of Medicine,Texas Children’s Hospital

Roep, Bart O. (författare): Leiden University Medical Centre,City of Hope National Medical Center

Schatz, Desmond (författare): University of Florida

Skibinski, David (författare): Benaroya Research Institute

Peakman, Mark (författare): King's College London,King's Health Partners

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(creator_code:org_t)

2019-12-12
2020
Engelska 8 s.
Ingår i: Diabetes Care. - : American Diabetes Association. - 1935-5548 .- 0149-5992. ; 43:1, s. 5-12

Relaterad länk:: http://dx.doi.org/10...; visa fler...; https://care.diabete...; https://lup.lub.lu.s...; https://doi.org/10.2...; visa färre...

Tidskriftsartikel (refereegranskat)

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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.

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MEDICIN OCH HÄLSOVETENSKAP: MEDICIN OCH HÄLS ...; och Klinisk medicin; och Endokrinologi oc ...

Artiklar i publikationen: Diabetes Care

Av lärosätet: Lunds universitet

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

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