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.


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.
Department of Pediatrics, University of Florida, Gainesville, Florida, USA. hallemj@peds.ufl.edu
Michael J
MJ
Haller
Clive H
CH
Wasserfall
Maigan A
MA
Hulme
Miriam
M
Cintron
Todd M
TM
Brusko
Kieran M
KM
McGrail
Theresa M
TM
Sumrall
John R
JR
Wingard
Douglas W
DW
Theriaque
Jonathan J
JJ
Shuster
Mark A
MA
Atkinson
Desmond A
DA
Schatz
D001804:Blood Transfusion, Autologous; D002096:C-Peptide / Q000097:blood; D002648:Child; D002675:Child, Preschool; D003922:Diabetes Mellitus, Type 1 / Q000276:immunology / Q000628:therapy*; D005260:Female; D005312:Fetal Blood / Q000637:transplantation*; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D008297:Male; D010865:Pilot Projects; D050378:T-Lymphocytes, Regulatory / Q000276:immunology
D002096:C-Peptide