Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial.
Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial.
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DOI：
10.1002/sctm.17-0102
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发表时间：
2017-12
影响因子：
6
通讯作者：
Kurtzberg J
中科院分区：
文献类型：
--
作者：
Sun JM;Song AW;Case LE;Mikati MA;Gustafson KE;Simmons R;Goldstein R;Petry J;McLaughlin C;Waters-Pick B;Chen LW;Wease S;Blackwell B;Worley G;Troy J;Kurtzberg J
脑性瘫痪(CP)是一种影响幼儿的疾病，会导致终身残疾。脐带血细胞通过旁分泌信号改善实验系统的运动功能。在证明了安全性后，我们对患有CP的儿童进行了自体脐血输注的II期试验，以测试自体脐血是否可以改善功能(ClinicalTrials.gov，NCT01147653；IND 14360)。在这项双盲、安慰剂对照的交叉研究中，一次静脉注射每公斤ACB总有核细胞1-5×107个，患有CP的1-6岁儿童被随机分配到基线接受ACB或安慰剂治疗，然后在一年后交替输注。在基线、治疗后1年和2年后进行运动功能和磁共振成像脑连通性研究。主要终点是基线输注1年后运动功能的变化。另外的分析在两年后进行。在基线时，63名儿童(中位年龄2.1岁)被随机分为治疗组(n = 32)和安慰剂组(n = 31)。虽然在安慰剂组和治疗组之间，1年后粗大运动功能测量-66(GMFM-66)评分的平均变化没有差异，但发现了剂量效应。在ACB治疗一年后的一项分析中，那些接受2×107/kg剂量≥的患者的GMFM-66得分显著高于年龄和严重程度预测的得分，以及皮博迪发育运动量表-2总运动商得分和标准化脑连接。这项研究的结果表明，适当剂量的ACB输注改善了脑瘫儿童的脑连接和粗大运动功能。干细胞转化医学2017；6：2071-2078
Cerebral palsy (CP) is a condition affecting young children that causes lifelong disabilities. Umbilical cord blood cells improve motor function in experimental systems via paracrine signaling. After demonstrating safety, we conducted a phase II trial of autologous cord blood (ACB) infusion in children with CP to test whether ACB could improve function (ClinicalTrials.gov, NCT01147653; IND 14360). In this double‐blind, placebo‐controlled, crossover study of a single intravenous infusion of 1–5 × 107 total nucleated cells per kilogram of ACB, children ages 1 to 6 years with CP were randomly assigned to receive ACB or placebo at baseline, followed by the alternate infusion 1 year later. Motor function and magnetic resonance imaging brain connectivity studies were performed at baseline, 1, and 2 years post‐treatment. The primary endpoint was change in motor function 1 year after baseline infusion. Additional analyses were performed at 2 years. Sixty‐three children (median age 2.1 years) were randomized to treatment (n = 32) or placebo (n = 31) at baseline. Although there was no difference in mean change in Gross Motor Function Measure‐66 (GMFM‐66) scores at 1 year between placebo and treated groups, a dosing effect was identified. In an analysis 1 year post‐ACB treatment, those who received doses ≥2 × 107/kg demonstrated significantly greater increases in GMFM‐66 scores above those predicted by age and severity, as well as in Peabody Developmental Motor Scales‐2 Gross Motor Quotient scores and normalized brain connectivity. Results of this study suggest that appropriately dosed ACB infusion improves brain connectivity and gross motor function in young children with CP. Stem Cells Translational Medicine 2017;6:2071–2078
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影响因子：
4.2
作者：
Englander, Zoe A.;Sun, Jessica;Case, Laura;Mikati, Mohamad A.;Kurtzberg, Joanne;Song, Allen W.
通讯作者：
Song, Allen W.
DOI：
10.1073/pnas.92.22.10119
发表时间：
1995-10-24
影响因子：
11.1
作者：
RUBINSTEIN, P;DOBRILA, L;STEVENS, CE
通讯作者：
STEVENS, CE
影响因子：
120.7
作者：
Rosenbaum, PL;Walter, SD;Galuppi, BE
通讯作者：
Galuppi, BE
影响因子：
2.1
作者：
Rubinstein, P;Stevens, CE
通讯作者：
Stevens, CE
影响因子：
5.7
作者：
Avants BB;Tustison NJ;Song G;Cook PA;Klein A;Gee JC
通讯作者：
Gee JC