PT  - JOURNAL ARTICLE
AU  - Haller, Michael J.
AU  - Wasserfall, Clive H.
AU  - McGrail, Kieran M.
AU  - Cintron, Miriam
AU  - Brusko, Todd M.
AU  - Wingard, John R.
AU  - Kelly, Susan S.
AU  - Shuster, Jonathan J.
AU  - Atkinson, Mark A.
AU  - Schatz, Desmond A.
TI  - Autologous Umbilical Cord Blood Transfusion in Very Young Children With Type 1 Diabetes
AID  - 10.2337/dc09-0967
DP  - 2009 Nov 01
TA  - Diabetes Care
PG  - 2041--2046
VI  - 32
IP  - 11
4099  - http://care.diabetesjournals.org/content/32/11/2041.short
4100  - http://care.diabetesjournals.org/content/32/11/2041.full
SO  - Diabetes Care2009 Nov 01; 32
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.