Lo Raymond Y, Luo Yuncin, Chen Shu-Cin, Wang Jen-Hung, Ko Chen-Yu, Chen Ying-Jie, Su Yu-Chin, Lee Tong-Young, Wang Jonas C, Lin Shinn-Zong
Department of Neurology, Taitung St. Mary's Hospital, Taitung, Taiwan.
Department of Neurology, Hualien Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation, Hualien, Taiwan.
Tzu Chi Med J. 2025 May 9;37(3):321-327. doi: 10.4103/tcmj.tcmj_249_24. eCollection 2025 Jul-Sep.
Transplantation of human umbilical cord blood cells (hUCB) may enhance neuroprotection, and thus, the intravenous (IV) infusion of hUCB in patients with acute ischemic stroke (AIS) is being tested for its safety and efficacy.
We conducted a 12-month, open-label, and single-center, phase I trial of hUCB treatment in AIS patients at the age of 45-80 years, with magnetic resonance imaging evidence of acute infarction in the internal carotid artery supplied territory and the National Institute of Health Stroke Scale (NIHSS) score between 6 and 18. Eligible participants received a single-dose IV infusion of hUCB followed by the two doses of mannitol infusion within 9 days after the onset of stroke symptoms. The primary endpoint was the incidence of adverse events (AEs) and the secondary endpoints were the changes in NIHSS, Barthel index (BI), and Berg Balance Scale (BBS) scores.
Six patients (Male: Female = 3: 3) were enrolled with a mean age at 65.8 years. A total of 40 AEs occurred in six participants during this study, which included nine serious adverse events. Only transient erythema multiforme and hematuria were probably and possibly related to hUCB infusion, respectively. The mean NIHSS score was 11.5 at baseline and it significantly improved at 1, 3, 6, 9, and 12 months after treatment (mean change from baseline: -4.0, -5.3, -6.8, -7.0, and -7.3). The mean BI score was 22.5 at baseline and it significantly increased at 3 and 6 months after treatment (mean change from baseline: 26.7 and 42.5, respectively). The BBS score increased numerically but did not reach statistical significance. The changes in cytokine levels and spleen size were unremarkable.
The IV hUCB was safe and well tolerated in AIS patients, and the preliminary efficacy results demonstrated its therapeutic potential, supporting the conduct of a randomized, placebo controlled, phase II clinical trial in future.
人脐带血细胞（hUCB）移植可能增强神经保护作用，因此，正在对急性缺血性卒中（AIS）患者静脉输注hUCB的安全性和有效性进行测试。
我们开展了一项为期12个月、开放标签、单中心的I期试验，纳入年龄在45至80岁之间的AIS患者，这些患者经磁共振成像证实颈内动脉供血区域存在急性梗死，且美国国立卫生研究院卒中量表（NIHSS）评分在6至18分之间。符合条件的参与者接受单次静脉输注hUCB，随后在卒中症状发作后9天内输注两剂甘露醇。主要终点是不良事件（AE）的发生率，次要终点是NIHSS、Barthel指数（BI）和Berg平衡量表（BBS）评分的变化。
共纳入6例患者（男∶女 = 3∶3），平均年龄65.8岁。在本研究期间，6名参与者共发生40起AE，其中包括9起严重不良事件。仅多形性红斑和血尿可能分别与hUCB输注有关。治疗前NIHSS平均评分为11.5分，治疗后1、3、6、9和12个月时显著改善（相对于基线的平均变化：-4.0、-5.3、-6.8、-7.0和-7.3）。治疗前BI平均评分为22.5分，治疗后3个月和6个月时显著升高（相对于基线的平均变化：分别为26.7和42.5）。BBS评分有数值上的增加，但未达到统计学意义。细胞因子水平和脾脏大小的变化不明显。
静脉输注hUCB在AIS患者中安全且耐受性良好，初步疗效结果显示了其治疗潜力，支持未来开展随机、安慰剂对照的II期临床试验。
