RT Journal Article
SR Electronic
T1 Autologous Umbilical Cord Blood Transfusion in Very Young Children With Type 1 Diabetes
JF Diabetes Care
JO Diabetes Care
FD American Diabetes Association
SP 2041
OP 2046
DO 10.2337/dc09-0967
VO 32
IS 11
A1 Haller, Michael J.
A1 Wasserfall, Clive H.
A1 McGrail, Kieran M.
A1 Cintron, Miriam
A1 Brusko, Todd M.
A1 Wingard, John R.
A1 Kelly, Susan S.
A1 Shuster, Jonathan J.
A1 Atkinson, Mark A.
A1 Schatz, Desmond A.
YR 2009
UL http://care.diabetesjournals.org/content/32/11/2041.abstract
AB 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. © 2009 by the American Diabetes Association.