Heterogeneity in recent-onset type 1 diabetes - a clinical trial perspective DIABETES-METABOLISM RESEARCH AND REVIEWS Bollyky, J. B., Xu, P., Butte, A. J., Wilson, D. M., Beam, C. A., Greenbaum, C. J. 2015; 31 (6): 588-594


Type 1 diabetes (T1D) TrialNet is a National Institutes of Health-sponsored clinical trial network aimed at altering the disease course of T1D. The purpose of this study is to evaluate age-dependent heterogeneity in clinical, metabolic and immunologic characteristics of individuals with recent-onset T1D, to identify cohorts of interest and to aid in planning of future studies.Eight hundred eighty-three individuals with recent-onset T1D involved in five TrialNet studies were categorized by age as follows: =18 years, 12-17 years, 8-12 years and <8 years. Data were compared with healthy age-matched subjects in the National Health and Nutrition Examination Survey.Only 2.0% of the individuals overall were excluded from trial participation because of insufficient C-peptide values (<0.2?pmol/mL). A disproportionate number of these subjects were <8?years old. Leukopenia was present in 21.2% of individuals and lymphopenia in 11.6%; these frequencies were markedly higher than age-matched healthy National Health and Nutrition Examination Survey population. Of the cohort, 24.5% were overweight or obese. Neither high-risk human leukocyte antigen type DR3 nor DR4 was present in 31% of adults and 21% of children.The ability of recent-onset T1D patients to meet key entry criteria for TrialNet studies, including C-peptide >0.2?pmol/mL, varies by age. Lower C-peptide level requirements for younger participants and other aspects of heterogeneity of recent-onset T1D patients, such as white blood cell count abnormalities and body mass index should be considered in the design of future clinical studies. Copyright © 2015 John Wiley & Sons, Ltd.

View details for DOI 10.1002/dmrr.2643

View details for PubMedID 25689602