Autologous Umbilical Cord Blood Transfusion in Young Children With Type 1 Diabetes Fails to Preserve C-Peptide
- Michael J. Haller, MD1⇓,
- Clive H. Wasserfall, MS2,
- Maigan A. Hulme, BS2,
- Miriam Cintron, BS1,
- Todd M. Brusko, PHD2,
- Kieran M. McGrail, BS2,
- Theresa M. Sumrall, BS2,
- John R. Wingard, MD3,
- Douglas W. Theriaque, MS4,
- Jonathan J. Shuster, PHD4,
- Mark A. Atkinson, PHD2 and
- Desmond A. Schatz, MD1
- 1Department of Pediatrics, University of Florida, Gainesville, Florida
- 2Department of Pathology, University of Florida, Gainesville, Florida
- 3Department of Medicine, University of Florida, Gainesville, Florida
- 4Department of Epidemiology and Health Policy Research and the General Clinical Research Center, University of Florida, Gainesville, Florida
- Corresponding author: Michael J. Haller, .
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.
This article contains Supplementary Data online at http://care.diabetesjournals.org/lookup/suppl/doi:10.2337/dc11-1406/-/DC1.
- Received July 26, 2011.
- Accepted September 20, 2011.
- © 2011 by the American Diabetes Association.
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