Boyalı Osman, Kabatas Serdar, Civelek Erdinç, Ozdemir Omer, Bahar-Ozdemir Yeliz, Kaplan Necati, Savrunlu Eyüp Can, Karaöz Erdal
Department of Neurosurgery, University of Health Sciences Turkey, Gaziosmanpaşa Training and Research Hospital, Istanbul 34360, Turkey.
Center for Stem Cell & Gene Therapy Research and Practice, University of Health Sciences Turkey, Istanbul 34360, Turkey.
World J Clin Cases. 2024 Mar 26;12(9):1585-1596. doi: 10.12998/wjcc.v12.i9.1585.
Cerebral palsy (CP) describes a group of disorders affecting movement, balance, and posture. Disturbances in motor functions constitute the main body of CP symptoms. These symptoms surface in early childhood and patients are affected for the rest of their lives. Currently, treatment involves various pharmacotherapies for different types of CP, including antiepileptics for epilepsy and Botox A for focal spasticity. However, none of these methods can provide full symptom relief. This has prompted researchers to look for new treatment modalities, one of which is mesenchymal stem cell therapy (MSCT). Despite being a promising tool and offering a wide array of possibilities, mesenchymal stem cells (MSCs) still need to be investigated for their efficacy and safety.
To analyze the efficacy and safety of MSCT in CP patients.
Our sample consists of four CP patients who cannot stand or walk without external support. All of these cases received allogeneic MSCT six times as 1 × 10/kg intrathecally, intravenously, and intramuscularly using umbilical cord-derived MSCs (UC-MSC). We monitored and assessed the patients pre- and post-treatment using the Wee Functional Independence Measure (WeeFIM), Gross Motor Function Classification System (GMFCS), and Manual Ability Classification Scale (MACS) instruments. We utilized the Modified Ashworth Scale (MAS) to measure spasticity.
We found significant improvements in MAS scores after the intervention on both sides. Two months: Right = 4000, = 0.046, left = 4000, = 0.046; four months: Right = 4000, = 0.046, left = 4000, = 0.046; 12 months: Right = 4000, = 0.046, left = 4000, = 0.046. However, there was no significant difference in motor functions based on WeeFIM results ( > 0.05). GMFCS and MACS scores differed significantly at 12 months after the intervention ( = 0.046, = 0.046). Finally, there was no significant change in cognitive functions ( > 0.05).
In light of our findings, we believe that UC-MSC therapy has a positive effect on spasticity, and it partially improves motor functions.
脑性瘫痪(CP)描述的是一组影响运动、平衡和姿势的病症。运动功能障碍构成了CP症状的主体。这些症状在儿童早期出现,患者会终生受到影响。目前,针对不同类型的CP有多种药物治疗方法,包括用于癫痫的抗癫痫药物和用于局灶性痉挛的A型肉毒毒素。然而,这些方法都无法完全缓解症状。这促使研究人员寻找新的治疗方式,其中之一是间充质干细胞疗法(MSCT)。尽管间充质干细胞(MSCs)是一种有前景的工具并提供了广泛的可能性,但仍需对其疗效和安全性进行研究。
分析MSCT对CP患者的疗效和安全性。
我们的样本包括四名在没有外部支撑的情况下无法站立或行走的CP患者。所有这些病例均接受了六次同种异体MSCT,使用脐带间充质干细胞(UC-MSC),通过鞘内、静脉内和肌肉内注射,剂量为1×10/kg。我们使用小儿功能独立性测量(WeeFIM)、粗大运动功能分类系统(GMFCS)和手动能力分类量表(MACS)工具在治疗前后对患者进行监测和评估。我们使用改良Ashworth量表(MAS)来测量痉挛程度。
我们发现干预后两侧的MAS评分均有显著改善。两个月时:右侧=4000,=0.046,左侧=4000,=0.046;四个月时:右侧=4000,=0.046,左侧=4000,=0.046;12个月时:右侧=4000,=0.046,左侧=4000,=0.046。然而,根据WeeFIM结果,运动功能没有显著差异(>0.05)。干预12个月后GMFCS和MACS评分有显著差异(=0.046,=0.046)。最后,认知功能没有显著变化(>0.05)。
根据我们的研究结果,我们认为UC-MSC疗法对痉挛有积极作用,并能部分改善运动功能。