Effect of a single autologous cord blood infusion on beta-cell and immune function in children with new onset type 1 diabetes: A non-randomized, controlled trial

Background: The application of autologous cord blood in children with type 1 diabetes has been found to be safe, but not to preserve beta-cell function in a previous study, which, however, had not included a control group. Objective: To compare the changes of metabolic and immune function over time between cord blood infused children and natural controls. Subjects and methods: Seven children with newly diagnosed type 1 diabetes underwent a single autologous cord blood infusion and 10 children were enrolled as natural controls in a non-randomized, controlled, open label intervention trial. Primary analyses were performed 1 year following cord blood infusion. Cases and controls were compared regarding metabolic [area under the curve (AUC) and peak C-peptide, insulin use, and HbA1c] and immune outcome (islet autoantibody titer and T-cell response), adjusted for age, gender, diabetes duration, and baseline levels. Results: There were no significant adverse events related to the infusion. Metabolic and immune outcomes were not significantly different at 12months follow-up between infused children and controls (e.g., adjusted p=0.244 for AUC C-peptide, adjusted p=0.820 for insulin use, adjusted p=0.772 for peripheral regulatory T cells). Six-month change of AUC C-peptide correlated significantly with the number of infused CD34+ cells (r=0.931, p=0.002). Conclusions: An autologous cord blood infusion does not change the natural course of metabolic and immune parameters after disease onset. However, the content of CD34+ cells in the stored blood sample might offer potential for improvement of future cell therapies.