Autologous Umbilical Cord Blood Transfusion in Very Young Children With Type 1 Diabetes

  1. Michael J. Haller, MD1,
  2. Clive H. Wasserfall, MS2,
  3. Kieran M. McGrail, BS2,
  4. Miriam Cintron, BS1,
  5. Todd M. Brusko, PHD3,
  6. John R. Wingard, MD4,
  7. Susan S. Kelly, MD5,
  8. Jonathan J. Shuster, PHD6,
  9. Mark A. Atkinson, PHD2 and
  10. Desmond A. Schatz, MD1
  1. 1Department of Pediatrics, The University of Florida, Gainesville, Florida;
  2. 2Department of Pathology, The University of Florida, Gainesville, Florida;
  3. 3Diabetes Center, University of California at San Francisco, San Francisco, California;
  4. 4Department of Medicine, The University of Florida, Gainesville, Florida;
  5. 5Department of Pediatrics, The University of Texas, Houston, Texas;
  6. 6Department of Epidemiology and Health Policy Research and the General Clinical Research Center, The University of Florida, Gainesville, Florida.
  1. Corresponding author: Michael Haller, hallemj{at}peds.ufl.edu.

Abstract

OBJECTIVE Interest continues to grow regarding the therapeutic potential for umbilical cord blood therapies to modulate autoimmune disease. We conducted an open-label phase I study using autologous umbilical cord blood infusion to ameliorate type 1 diabetes.

RESEARCH DESIGN AND METHODS Fifteen patients diagnosed with type 1 diabetes and for whom autologous umbilical cord blood was stored underwent a single intravenous infusion of autologous cells and completed 1 year of postinfusion follow-up. Intensive insulin regimens were used to optimize glycemic control. Metabolic and immunologic assessments were performed before infusion and at established time periods thereafter.

RESULTS Median (interquartile range [IQR]) age at infusion was 5.25 (3.1–7.3) years, with a median postdiagnosis time to infusion of 17.7 (10.9–26.5) weeks. No infusion-related adverse events were observed. Metabolic indexes 1 year postinfusion were peak C-peptide median 0.50 ng/ml (IQR 0.26–1.30), P = 0.002; A1C 7.0% (IQR 6.5–7.7), P = 0.97; and insulin dose 0.67 units · kg−1 · day−1 (IQR 0.55–0.77), P = 0.009. One year postinfusion, no changes were observed in autoantibody titers, regulatory T-cell numbers, CD4-to-CD8 ratio, or other T-cell phenotypes.

CONCLUSIONS Autologous umbilical cord blood transfusion in children with type 1 diabetes is safe but has yet to demonstrate efficacy in preserving C-peptide. Larger randomized studies as well as 2-year postinfusion follow-up of this cohort are needed to determine whether autologous cord blood–based approaches can be used to slow the decline of endogenous insulin production in children with type 1 diabetes.

Footnotes

  • Clinical trial reg. no. NCT00305344, clinicaltrials.gov.

  • The costs of publication of this article were defrayed in part by the payment of page charges. This article must therefore be hereby marked “advertisement” in accordance with 18 U.S.C. Section 1734 solely to indicate this fact.

  • See accompanying editorial, p. 2138.

    • Received May 31, 2009.
    • Accepted August 21, 2009.
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