Autologous umbilical cord blood transfusion in young children with type 1 diabetes fails to preserve C-peptide

Diabetes Care. 2011 Dec;34(12):2567-9. doi: 10.2337/dc11-1406. Epub 2011 Oct 19.


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.

Trial registration: NCT00305344.

Publication types

  • Clinical Trial, Phase I
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Blood Transfusion, Autologous*
  • C-Peptide / blood
  • Child
  • Child, Preschool
  • Diabetes Mellitus, Type 1 / immunology
  • Diabetes Mellitus, Type 1 / therapy*
  • Female
  • Fetal Blood / transplantation*
  • Follow-Up Studies
  • Humans
  • Infant
  • Male
  • Pilot Projects
  • T-Lymphocytes, Regulatory / immunology


  • C-Peptide

Associated data