Autologous Umbilical Cord Blood Transfusion in Young Children With Type 1 Diabetes Fails to Preserve C-Peptide

  1. Desmond A. Schatz, MD1
  1. 1Department of Pediatrics, University of Florida, Gainesville, Florida
  2. 2Department of Pathology, University of Florida, Gainesville, Florida
  3. 3Department of Medicine, University of Florida, Gainesville, Florida
  4. 4Department of Epidemiology and Health Policy Research and the General Clinical Research Center, University of Florida, Gainesville, Florida
  1. Corresponding author: Michael J. Haller, 32610.hallemj{at}peds.ufl.edu.

Abstract

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.

  • Received July 26, 2011.
  • Accepted September 20, 2011.

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  1. Diabetes Care
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