Autologous umbilical cord blood transfusion in very young children with type 1 diabetes.
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.


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.
Haller
Michael J
MJ
Department of Pediatrics, The University of Florida, Gainesville, Florida, USA. hallemj@peds.ufl.edu
Wasserfall
Clive H
CH
McGrail
Kieran M
KM
Cintron
Miriam
M
Brusko
Todd M
TM
Wingard
John R
JR
Kelly
Susan S
SS
Shuster
Jonathan J
JJ
Atkinson
Mark A
MA
Schatz
Desmond A
DA
D001786:Blood Glucose / Q000378:metabolism*; D001804:Blood Transfusion, Autologous / Q000379:methods; D002096:C-Peptide / Q000097:blood; D015496:CD4-Positive T-Lymphocytes / Q000276:immunology; D018414:CD8-Positive T-Lymphocytes / Q000276:immunology; D002648:Child; D002675:Child, Preschool; D003922:Diabetes Mellitus, Type 1 / Q000097:blood / Q000188:drug therapy / Q000276:immunology / Q000628:therapy*; D005312:Fetal Blood / Q000637:transplantation*; D005500:Follow-Up Studies; D006442:Glycated Hemoglobin / Q000378:metabolism; D006801:Humans; D007004:Hypoglycemic Agents / Q000627:therapeutic use; D007223:Infant; D007262:Infusions, Intravenous; D007328:Insulin / Q000627:therapeutic use; D007958:Leukocyte Count; D013997:Time Factors
D001786:Blood Glucose; D002096:C-Peptide; D006442:Glycated Hemoglobin; D007004:Hypoglycemic Agents; D007328:Insulin