Department of Pediatrics, University of Florida, Gainesville, Florida, USA.
Diabetes Care. 2011 Dec;34(12):2567-9. doi: 10.2337/dc11-1406. Epub 2011 Oct 19.
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.
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.
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.
Autologous UCB infusion in children with T1D is safe and induces changes in Treg frequency but fails to preserve C-peptide.
我们进行了一项开放性、I 期研究，使用自体脐带血（UCB）输注来改善 1 型糖尿病（T1D）。先前我们报告了前 15 名达到 1 年随访的患者，在此我们报告了 2 年随访后的完整队列。
共 24 名 T1D 患者（中位年龄 5.1 岁）接受单次静脉输注自体 UCB 细胞，并进行代谢和免疫评估。
未观察到输注相关不良事件。UCB 输注后β细胞功能下降。输注后 1 年 C 肽曲线下面积为基线的 24.3%（P<0.001），输注后 2 年为基线的 2%（P<0.001）。流式细胞术显示，输注后 6 个月和 9 个月分别分别增加了调节性 T 细胞（Tregs）（P=0.04）和幼稚 Tregs（P=0.001）。
在 T1D 儿童中自体 UCB 输注是安全的，并诱导 Treg 频率发生变化，但未能保留 C 肽。
