Autologous umbilical cord blood transfusion in very young children with type 1 diabetes.
Autologous umbilical cord blood transfusion in very young children with type 1 diabetes.
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DOI：
10.2337/dc09-0967
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发表时间：
2009-11
期刊：
影响因子：
16.2
通讯作者：
Schatz DA
中科院分区：
文献类型：
--
作者：
Haller MJ;Wasserfall CH;McGrail KM;Cintron M;Brusko TM;Wingard JR;Kelly SS;Shuster JJ;Atkinson MA;Schatz DA
关于脐带血疗法调节自身免疫性疾病的治疗潜力的兴趣继续增长。我们进行了一项开放标签I期研究，使用自体脐带血输注改善1型糖尿病。15例诊断为1型糖尿病并储存自体脐带血的患者接受了单次静脉输注自体细胞，并完成了1年的输注后随访。强化胰岛素方案用于优化血糖控制。在输注前和输注后确定的时间段进行代谢和免疫学评估。输注时的中位（四分位距[IQR]）年龄为5.25（3.1-7.3）岁，诊断后至输注的中位时间为17.7（10.9-26.5）周。未观察到输注相关不良事件。输注后1年的代谢指标为C肽峰值中位数0.50 ng/ml（IQR 0.26-1.30），P = 0.002; A1 C 7.0%（IQR 6.5-7.7），P = 0.97;胰岛素剂量0.67单位· kg-1 ·天-1（IQR 0.55-0.77），P = 0.009。输注后1年，未观察到自身抗体滴度、调节性T细胞数量、CD 4/CD 8比值或其他T细胞表型的变化。1型糖尿病患儿自体脐带血输注是安全的，但尚未证明其在保护C肽方面的有效性。需要更大规模的随机研究以及该队列的2年输注后随访，以确定是否可以使用基于自体脐带血的方法来减缓1型糖尿病儿童内源性胰岛素产生的下降。
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. 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. 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. 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.