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首页> 外文期刊>Translational research: the journal of laboratory and clinical medicine >Open-labeled study of unilateral autologous bone-marrow-derived mesenchymal stem cell transplantation in Parkinson's disease.
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Open-labeled study of unilateral autologous bone-marrow-derived mesenchymal stem cell transplantation in Parkinson's disease.

机译:帕金森氏病单侧自体骨髓间充质干细胞移植的开放标签研究。

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摘要

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.
机译:帕金森氏病(PD)是一种进行性神经退行性疾病,最近几年干细胞研究为其带来了希望。纳入了7名22至62岁,平均病程14.7 +/- 7.56年的PD患者,参加了单剂量,单侧自体骨髓间充质干细胞单剂量移植的前瞻性,非对照,前瞻性研究( BM-MSC)。通过立体定向手术将BM-MSCs移植到下侧心室区。对患者进行了为期10到36个月的随访。基线“关闭”的平均得分是65 +/- 22.06,基线“打开”的平均得分是50.6 +/- 15.85。 7名患者中有3名在其“关” /“统一”帕金森病综合评分量表(UPDRS)上显示出稳定的改善。他们最后一次随访的平均“关闭”评分是43.3,比基线提高了22.9%。在他们的最后一次随访中,平均“上”分数是31.7,提高了38%。 Hoehn和Yahr(H&Y)以及Schwab和England(S&E)的分数分别显示出H&Y的2.7和2.5的相似改进以及S&E的14%的改进。在面部表情,步态和冰冻发作等症状上发现了主观的改善; 2例患者PD药物剂量明显减少。这些结果表明,我们的方案似乎是安全的,PD患者干细胞移植后未发生严重的不良事件。招募的患者数量以及试验的不受控制的性质均无法证明所涉及治疗的有效性。但是,结果鼓励将来有更多患者进行试验以证明疗效。

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