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单侧自体骨髓间充质干细胞移植治疗帕金森病的开放标签研究。

Open-labeled study of unilateral autologous bone-marrow-derived mesenchymal stem cell transplantation in Parkinson's disease.

机构信息

Advanced Neuroscience Institute, BGS-Global Hospital, Bangalore, India.

出版信息

Transl Res. 2010 Feb;155(2):62-70. doi: 10.1016/j.trsl.2009.07.006. Epub 2009 Aug 6.

DOI:10.1016/j.trsl.2009.07.006
PMID:20129486
Abstract

Parkinson's disease (PD) is a progressive neurodegenerative disease for which stem cell research has created hope in the last few years. Seven PD patients aged 22 to 62 years with a mean duration of disease 14.7+/-7.56 years were enrolled to participate in the prospective, uncontrolled, pilot study of single-dose, unilateral transplantation of autologous bone-marrow-derived mesenchymal stem cells (BM-MSCs). The BM-MSCs were transplanted into the sublateral ventricular zone by stereotaxic surgery. Patients were followed up for a period that ranged from 10 to 36 months. The mean baseline "off" score was 65+/-22.06, and the mean baseline "on" score was 50.6+/-15.85. Three of 7 patients have shown a steady improvement in their "off"/"on" Unified Parkinson's Disease Rating Scale (UPDRS). The mean "off" score at their last follow-up was 43.3 with an improvement of 22.9% from the baseline. The mean "on" score at their last follow-up was 31.7, with an improvement of 38%. Hoehn and Yahr (H&Y) and Schwab and England (S&E) scores showed similar improvements from 2.7 and 2.5 in H&Y and 14% improvement in S&E scores, respectively. A subjective improvement was found in symptoms like facial expression, gait, and freezing episodes; 2 patients have significantly reduced the dosages of PD medicine. These results indicate that our protocol seems to be safe, and no serious adverse events occurred after stem-cell transplantation in PD patients. The number of patients recruited and the uncontrolled nature of the trial did not permit demonstration of effectiveness of the treatment involved. However, the results encourage future trials with more patients to demonstrate efficacy.

摘要

帕金森病(PD)是一种进行性神经退行性疾病,近年来干细胞研究为其带来了希望。本前瞻性、非对照、单剂量、单侧自体骨髓间充质干细胞(BM-MSCs)移植的初步研究共纳入 7 例年龄 22-62 岁、平均病程 14.7+/-7.56 年的 PD 患者。通过立体定向手术将 BM-MSCs 移植到侧脑室下区。患者随访时间为 10-36 个月。平均基线“关”期评分为 65+/-22.06,平均基线“开”期评分为 50.6+/-15.85。7 例患者中有 3 例“关”/“开”期统一帕金森病评定量表(UPDRS)评分持续改善。末次随访时,平均“关”期评分为 43.3,与基线相比改善 22.9%。末次随访时平均“开”期评分为 31.7,改善 38%。Hoehn 和 Yahr(H&Y)和 Schwab 和 England(S&E)评分分别从基线的 2.7 和 2.5 分别提高了 27%和 14%。症状如面部表情、步态和冻结发作方面有主观改善;2 例患者显著减少了 PD 药物的剂量。这些结果表明,我们的方案似乎是安全的,PD 患者干细胞移植后没有发生严重不良事件。纳入的患者数量和试验的非对照性质不允许证明所涉及治疗的有效性。然而,这些结果鼓励未来进行更多患者的试验以证明疗效。

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