摘要
目的探讨脐带间充质干细胞(UC-MSCs)移植治疗肌萎缩侧索硬化症(ALS)的效果及安全性。方法29例ALS患者接受4次UC-MSCs移植治疗(蛛网膜下腔注射,2×10^7~5×10^7个细胞/次)。比较治疗前后肌萎缩侧索硬化功能评分量表(ALSFRS-R)评分、改良医学研究委员会(MRC)评分,对安全性指标进行评价。治疗后6个月以呼吸衰竭、死亡等为终点事件进行随访。结果 ALSFRS-R评分:治疗前后总分比较差异无统计学意义(P〉0.05);治疗后第14天穿衣和卫生评分明显高于治疗前(P=0.036);治疗后第28天,言语、穿衣和卫生功能评分明显高于治疗前(P分别为0.032、0.015),且言语功能评分明显高于治疗后第14天(P=0.040)。MRC评分:治疗后第14天上肢肌群肌力评分明显高于治疗前(P=0.050),治疗第28天上肢肌群肌力评分进一步增加(P=0.036)。随访至治疗后6个月,1例患者于治疗后第3个月因呼吸衰竭死亡,其余患者无严重不良事件。结论 UC-MSCs移植治疗ALS短期效果好且较为安全。
Objective To evaluate the short-term clinical efficacy and safety of transplantation of umbilical cord mesenchymal stem cells( UC-MSCs) in treatment of amyotrophic lateral sclerosis( ALS). Methods Twenty-nine patients with ALS were treated with UC-MSCs transplantation for 4 times( lumbar puncture,2 × 10^7 to 5 × 10^7 cells / time). Revised Amyotrophic Lateral Sclerosis Functional Rating Scale( ALSFRS-R) and Medical Research Council( MRC) scale for assessment of muscle power were assessed before and after the transplant. Safety indexes were evaluated at the same time.Results No significant difference of ALSFRS-R scale was observed( P〉0. 05). There was a significant improvement in the dressing and health abilities in ALS patients on the 14 th day after treatment( P = 0. 036),and the scores of verbal,dressing and health abilities on the 28 th day after treatment were significantly higher than those before treatment( P =0. 032,0. 015). In addition,the speech function was further improved compared with that on the 14 th day after treatment( P = 0. 040). The strength of upper limb muscles was enhanced with the extension of treatment( P = 0. 050,0. 036,on the 14 th and 28 th day after treatment). Followed up for 6 months after treatment,only 1 patient died of respiratory failure at the 3th month,and the remaining patients had no serious adverse events. Conclusions UC-MSCs transplantation is safe and effective for ALS in the short-term.
出处
《山东医药》
CAS
北大核心
2016年第36期1-4,共4页
Shandong Medical Journal
基金
广东省自然科学基金资助项目(2015A030310436)
广东省中医药管理局科研资助项目(20151210)