Between 15 December 2003 and 21 November 2008, 24 children with T1D (10 males, 14 females) underwent a single autologous UCB transfusion. No adverse events were observed in association with autologous UCB infusion. All aliquots of UCB had negative Gram stains, and none grew pathogenic organisms when cultured for virus, bacteria, or fungus.
Baseline and longitudinal postinfusion characteristics are provided in . Median age at infusion was 5.1 years (3.4–6.9). Median time from diagnosis to UCB infusion was 0.25 years (0.19–0.52). The median infused total nucleated cell count was 1.88 × 107
cells/kg. Median viability was 97% (95–99%). Overall, the total nucleated cell count recovered was commonly 1–2 log fold less than that typically observed in samples obtained from public banks (5
Baseline and postinfusion characteristics of autologous UCB recipients
Median AUC C-peptide at the time of autologous UCB infusion was 0.95 ng/mL (0.5–1.4). Median AUC C-peptide declined at all subsequent study visits compared with baseline (P
< 0.01 at all time points) (). At post-UCB infusion visits at 3, 6, and 9 months and 1 and 2 years, median AUC C-peptide was 0.66 ng/mL (0.2–1), 0.64 ng/mL (0.2–1.1), 0.46 ng/mL (0.03–1.1), 0.22 ng/mL (0.02–0.63), and 0.02 ng/mL (0–0.52), respectively (Supplementary Fig. 1
). AUC C-peptide was 24.3% of baseline at 1 year after infusion and 2% of baseline 2 years after infusion. Slope analysis of C-peptide demonstrated AUC C-peptide change of −2.4% per month (−3.09 to −1.43) (P
Baseline and 1- and 2-year median total peripheral white blood counts were 5.6 × 109, 5.5 × 109, and 5.5 × 109 cells/L, respectively (not significant vs. baseline). Total T-cell numbers, CD4-to-CD8 ratio, CD62L+ regulatory T cells (Tregs), CD62L+ T effectors, CD45RA+ T conventional cells, and CD45RO+ Tregs were not different at all study visits compared with baseline (data not shown). However, an increase in total Treg (CD4+CD25+FOXP3+) was observed at the 6-month visit (median 4.4%, P = 0.04), as was an increase in CD45RA+ Treg at the 9-month visit (median 43.5%, P = 0.001) ().