Autologous umbilical cord blood transfusion in young children with type 1 diabetes fails to preserve C-peptide
OBJECTIVE: We conducted an open-label, phase I study using autologous umbilical cord blood (UCB) infusion to ameliorate type 1 diabetes (T1D). Having previously reported on the first 15 patients reaching 1 year of follow-up, herein we report on the complete cohort after 2 years of follow-up.
RESEARCH DESIGN AND METHODS: A total of 24 T1D patients (median age 5.1 years) received a single intravenous infusion of autologous UCB cells and underwent metabolic and immunologic assessments.
RESULTS: No infusion-related adverse events were observed. β-Cell function declined after UCB infusion. Area under the curve C-peptide was 24.3% of baseline 1 year postinfusion (P < 0.001) and 2% of baseline 2 years after infusion (P < 0.001). Flow cytometry revealed increased regulatory T cells (Tregs) (P = 0.04) and naive Tregs (P = 0.001) 6 and 9 months after infusion, respectively.
CONCLUSIONS: Autologous UCB infusion in children with T1D is safe and induces changes in Treg frequency but fails to preserve C-peptide.
Haller, M. J., Wasserfall, C. H., Hulme, M. A., Cintron, M., Brusko, T. M., McGrail, K. M., ... Schatz, D. A. (2011). Autologous umbilical cord blood transfusion in young children with type 1 diabetes fails to preserve C-peptide. Diabetes Care, 34(12), 2567-2569. DOI: 10.2337/dc11-1406