1 Institute of Neurobiology and Department of Human Anatomy & Histoembriology, School of Basic Medical Sciences, Xi'an Jiaotong University, Xi'an, Shanxi, People Republic of China.
2 Taihe Hospital Affiliated to Hubei Medical College, Shiyan, Hubei, People Republic of China.
Cell Transplant. 2018 Feb;27(2):325-334. doi: 10.1177/0963689717729379.
Cerebral palsy (CP) is a common disability which results in permanent chronic motor disability appearing in early childhood. Recently human umbilical cord blood mesenchymal stem cell (hUCB-MSC) infusion has emerged as a promising therapeutic strategy for CP, and the treatment efficacy remains to be confirmed by clinical trials. All 54 patients received basic rehabilitation as a background treatment. The infusion group comprising 27 patients received 4 infusions of hUCB-MSCs (intravenous infusions at a fixed dose of 5 × 10) and basic rehabilitation treatment, whereas 27 patients in the control group received 0.9% normal saline and basic rehabilitation treatment. Several indices were tested from baseline up to 24 months posttreatment regarding efficacy and safety evaluations, including the gross motor function measurement 88 (GMFM-88) scores, the comprehensive function assessment (CFA), lab tests, electroencephalogram (EEG), routine magnetic resonance imaging (MRI), and adverse events. The changes in the total proportion of GMFM-88 and total scores of CFA in the hUCB-MSC infusion group were significantly higher than that in control group at 3, 6, 12, 24 months posttreatment. Less diffuse slow waves were noticed after hUCB-MSC infusion in patients with slowing of EEG background rhythms at baseline. Based on the routine MRI exams, improvements in cerebral structures were rare after treatment. Serious adverse events were not observed during the whole study period. The results of the study indicated that hUCB-MSC infusion with basic rehabilitation was safe and effective in improving gross motor and comprehensive functions in children with CP.
脑性瘫痪（CP）是一种常见的残疾，会导致儿童早期出现永久性慢性运动障碍。最近，人脐带血间充质干细胞（hUCB-MSC）输注已成为 CP 的一种有前途的治疗策略，但其治疗效果仍需临床试验证实。所有 54 名患者均接受基础康复治疗作为背景治疗。输注组 27 例患者接受 4 次 hUCB-MSCs（静脉输注固定剂量 5×10）和基础康复治疗，对照组 27 例患者接受 0.9%生理盐水和基础康复治疗。从基线到治疗后 24 个月，对疗效和安全性评估进行了多项指标测试，包括粗大运动功能测量 88（GMFM-88）评分、综合功能评估（CFA）、实验室检查、脑电图（EEG）、常规磁共振成像（MRI）和不良事件。治疗后 3、6、12、24 个月，hUCB-MSC 输注组 GMFM-88 总分和 CFA 总分的总比例变化明显高于对照组。在脑电图背景节律减慢的患者中，hUCB-MSC 输注后可见弥漫性慢波减少。根据常规 MRI 检查，治疗后大脑结构改善罕见。整个研究期间未观察到严重不良事件。研究结果表明，hUCB-MSC 输注联合基础康复治疗在改善 CP 儿童粗大运动和综合功能方面是安全有效的。
