Heterogeneity and endotypes in type 1 diabetes mellitus

There is a growing awareness that type 1 diabetes mellitus (T1DM) is a heterogeneous disease that can be characterized into distinct endotypes. This Review discusses the evidence for endotypes in T1DM and explores the implications for clinical practice. Despite major advances over the past decade, prevention and treatment of type 1 diabetes mellitus (T1DM) remain suboptimal, with large and unexplained variations in individual responses to interventions. The current classification schema for diabetes mellitus does not capture the complexity of this disease or guide clinical management effectively. One of the approaches to achieve the goal of applying precision medicine in diabetes mellitus is to identify endotypes (that is, well-defined subtypes) of the disease each of which has a distinct aetiopathogenesis that might be amenable to specific interventions. Here, we describe epidemiological, clinical, genetic, immunological, histological and metabolic differences within T1DM that, together, suggest heterogeneity in its aetiology and pathogenesis. We then present the emerging endotypes and their impact on T1DM prediction, prevention and treatment.

Automated summary

There is a recognition that T1DM is a heterogeneous disease with distinct endotypes, which has important implications for clinical practice. The current classification schema for diabetes mellitus does not capture the complexity of the disease or guide clinical management effectively. Identifying endotypes can help achieve precision medicine in diabetes mellitus and improve prediction, prevention, and treatment of T1DM.