Department of Pediatrics, Kochi Medical School, Kochi University, Kochi, Japan; Regenerative Medicine Research Center for Cerebral Palsy, Kochi Medical School Hospital, Kochi, Japan.
Department of Transfusion Medicine and Cell Therapy, Kochi Medical School Hospital, Kochi, Japan.
Brain Dev. 2022 Nov;44(10):681-689. doi: 10.1016/j.braindev.2022.08.004. Epub 2022 Aug 30.
Cerebral palsy (CP) is the most prevalent motor disorder of childhood. It typically results from in utero or perinatal brain injury. Recently, it has been reported that autologous cord blood (ACB) infusion for children with CP improved gross motor function and brain connectivity, but unfortunately, it has never been tried in Japan. We conducted a pilot study of the infusing of ACB, which was delivered from private bank, in the children with CP to assess the safety and feasibility to the procedure as well as any effect in improving neurological function.
After demonstrating the induction of tissue regeneration in animal model studies conducted a single-arm pilot study of intravenous ACB infusion in 6 young Japanese children with CP (ages 1-6 years). Primary outcomes were safety assessed by vital signs, clinical symptoms, and blood and urinary examinations at baseline and 1 weeks, 1, 2 and 3 years after treatment. In addition, motor function evaluations, neurodevelopmental examinations, magnetic resonance imaging, and electroencephalography (EEG) were performed at the same time.
Infusion was generally well-tolerated, although one patient experienced microhematuria 1 year after treatment and another one patient experienced febrile convulsion once 9 months after treatment. These events were transient, no relapse was seen during observation study. All patients improved a median of 6.8 points on the 1-year Gross Motor Functional Measure-66 (GMFM-66) scores, greater than predicted by age and severity. Furthermore, the 2-year and 3-year GMFM-66 scores were also greater than expected (median 6.2 points and 5.5 points, respectively). Overall scales and language-social scales of the developmental quotient (DQ) improved in 3 of 6 patients, who had greater changes in their GMFM-66 scores than the other cases after treatment. There were no significant correlations among the GMFM-66 scores, DQ, and infusion cell counts.
ACB infusion was safe and feasible for clinical use in patients with CP. However, much more clinical study with larger numbers of patients and in-depth studies of treatment mechanism of CP are needed.
脑瘫（CP）是儿童最常见的运动障碍。它通常是由于胎儿期或围产期的脑损伤引起的。最近，有报道称，自体脐带血（ACB）输注可改善脑瘫患儿的粗大运动功能和脑连接，但不幸的是，这种方法从未在日本尝试过。我们进行了一项关于将来自私人银行的 ACB 输注到脑瘫患儿中的试点研究，以评估该程序的安全性和可行性，以及对改善神经功能的任何影响。
在动物模型研究中证明了组织再生的诱导作用后，我们对 6 名患有 CP 的日本年轻儿童（年龄 1-6 岁）进行了单臂试点研究，进行了静脉内 ACB 输注。主要结果通过生命体征、临床症状以及治疗前后 1 周、1、2 和 3 年的血液和尿液检查来评估安全性。此外，还同时进行了运动功能评估、神经发育检查、磁共振成像和脑电图（EEG）。
输注总体上耐受性良好，尽管有 1 名患者在治疗后 1 年出现镜下血尿，另 1 名患者在治疗后 9 个月出现热性惊厥。这些事件是短暂的，在观察研究期间没有复发。所有患者的 1 年粗大运动功能测量-66（GMFM-66）评分中位数均提高了 6.8 分，高于年龄和严重程度的预期。此外，2 年和 3 年的 GMFM-66 评分也高于预期（中位数分别为 6.2 分和 5.5 分）。在治疗后，6 名患者中有 3 名的发育商（DQ）的整体和语言社会量表有所提高，他们的 GMFM-66 评分变化大于其他病例。GMFM-66 评分、DQ 和输注细胞计数之间没有显著相关性。
ACB 输注在 CP 患者的临床应用中是安全可行的。然而，还需要更多的临床研究，包括更多的患者和深入研究 CP 的治疗机制。
