Department of Neonatology, The First Affiliated Hospital of Xi'an Jiaotong University, No.277 West Yanta Road, Xi'an, 710061, Shaanxi, People's Republic of China.
Affiliated Taihe Hospital of Hubei University of Medicine, No. 32 Southern Renmin Road, Shiyan, 422000, Hubei, People's Republic of China.
Stem Cell Res Ther. 2020 Feb 3;11(1):43. doi: 10.1186/s13287-019-1545-x.
Cerebral palsy (CP) is a syndrome of childhood movement and posture disorders. Clinical evidence is still limited and sometimes inconclusive about the benefits of human umbilical cord mesenchymal stem cells (hUC-MSCs) for CP. We conducted a randomized trial to evaluate the safety and efficacy of hUC-MSC transplantation concomitant with rehabilitation in patients with CP.
Eligible patients were allocated into the hUC-MSC group and control group. In addition to rehabilitation, the patients in the hUC-MSC group received four transfusions of hUC-MSCs intravenously, while the control group received a placebo. Adverse events (AEs) were collected for safety evaluation in the 12-month follow-up phase. Primary endpoints were assessed as activities of daily living (ADL), comprehensive function assessment (CFA), and gross motor function measure (GMFM) scales. In addition, cerebral metabolic activity was detected by F-FDG-PET/CT to explore the possible mechanism of the therapeutic effects. Primary endpoint data were analyzed by ANOVA using SPSS version 20.0.
Forty patients were enrolled, and 1 patient withdrew informed consent. Therefore, 39 patients received treatments and completed the scheduled assessments. No significant difference was shown between the 2 groups in AE incidence. Additionally, significant improvements in ADL, CFA, and GMFM were observed in the hUC-MSC group compared with the control group. In addition, the standard uptake value of F-FDG was markedly increased in 3 out of 5 patients from the hUC-MSC group at 12 months after transplantation.
Our clinical data showed that hUC-MSC transplantation was safe and effective at improving the gross motor and comprehensive function of children with CP when combined with rehabilitation. Recovery of cerebral metabolic activity might play an essential role in the improvements in brain function in patients with CP. The therapeutic window, transfusion route, and dosage in our study were considerable for reference in clinical application.
Chictr.org.cn, ChiCTR1800016554. Registered 08 June 2018-retrospectively registered. The public title was "Randomized trial of umbilical cord-derived mesenchymal stem cells for cerebral palsy."
脑瘫（CP）是一种儿童运动和姿势障碍综合征。临床证据仍然有限，有时对人脐带间充质干细胞（hUC-MSCs）治疗 CP 的益处尚无定论。我们进行了一项随机试验，以评估 hUC-MSC 移植联合康复治疗 CP 患者的安全性和有效性。
符合条件的患者被分配到 hUC-MSC 组和对照组。除康复治疗外，hUC-MSC 组患者还接受了 4 次静脉内 hUC-MSC 输注，而对照组则接受了安慰剂。在 12 个月的随访期内收集不良事件（AE）以进行安全性评估。主要终点评估为日常生活活动（ADL）、综合功能评估（CFA）和粗大运动功能测量（GMFM）量表。此外，通过 F-FDG-PET/CT 检测脑代谢活性，以探索治疗效果的可能机制。使用 SPSS 版本 20.0 的 ANOVA 分析主要终点数据。
共纳入 40 例患者，1 例患者撤回知情同意。因此，39 例患者接受治疗并完成了预定评估。两组间 AE 发生率无显著差异。此外，与对照组相比，hUC-MSC 组的 ADL、CFA 和 GMFM 均有显著改善。此外，在移植后 12 个月，hUC-MSC 组的 5 例患者中有 3 例的 F-FDG 标准摄取值明显增加。
我们的临床数据显示，hUC-MSC 移植联合康复治疗可安全有效地改善 CP 儿童的粗大运动和综合功能。脑代谢活性的恢复可能在 CP 患者脑功能改善中发挥重要作用。我们研究中的治疗窗、输注途径和剂量可为临床应用提供参考。
Chictr.org.cn，ChiCTR1800016554。于 2018 年 6 月 8 日注册-回顾性注册。公众标题为“脐带衍生间充质干细胞治疗脑瘫的随机试验”。
