摘要
目的探讨自体骨髓间充质干细胞移植治疗唐氏综合征患儿的有效性。方法海军总医院儿科自2012年11月—2014年11月收治的骨髓间充质干细胞移植的唐氏综合征患儿51例（年龄0.5-7岁）,骨髓动员后进行骨髓采集间充质分选,分别行右侧侧脑室穿刺1次及腰椎穿刺2次骨髓间充质干细胞移植。每次移植的细胞数为1×10^6/kg。每例患儿进行Gesell发育诊断量表评估,对术前6个月及术后6个月2个相同长度时间内发育速率进行自身对照观察。同时,对移植术后1、3、6个月患儿的发育情况进行追踪随访。结果 51例中1例失访,余50例完成观察。其中32例在术后1-6个月逐渐出现不同程度智力、语言、运动进步,术后6个月患儿细动作能、应物能、应人能进步月份值较术前6个月明显升高,差异有统计学意义（P〈0.05）。结论骨髓间充质干细胞移植安全可靠,能改善唐氏综合征患儿智力和运动发育水平,且主要表现在情绪、细动作能、应人能及应物能的改善。
Objective To observe the clinical efficacy of transplantation with autologous bone marrow mesenchymal stem cells（ BMMSCs） in the treatment of children with Down syndrome.Methods Fifty-one children with Down syndrome（ 0. 5 — 7 years old）,admitted to our hospital from November 2012 to November 2014,were chosen; Autologous bone marrow collected and sorted for BMMSCs by once right ventricle and twice lumbar puncture. Each time the number of cell transplantation is 1×10^6/ kg. Each child for assessment of Gesell mental and motor developments in the same time periods was performed 6 months before and 6 months after the autologous BMMSCs transplantation. Meanwhile,the improvement of each patient was followed up and analyzed by l,3 and 6months after the transplantation. Results One patient failed in follow-up; the other 50 were observed completely. Gradual mental and motor improvements appeared in these 32 patients 1 month after the transplantation,and lasted till the 6 months of transplantation. There are more improve ment in the fine motor,reactant and personal,social behavior in 6 months after transplantation（ P〈0. 05）. Conclusion Autologous BMMSCs transplantation can improve the intellectual development in a short time,mainly in the fine motor,reactant and personal,social behavior in children with Down syndrome. BMMSCs transplantation for Down syndrome is safe and effective in a short-term.
出处
《转化医学杂志》
2016年第5期288-291,共4页
Translational Medicine Journal
基金
科技部国际合作专项项目(2012DFA30880)
关键词
骨髓间充质干细胞
细胞移植
唐氏综合征
Bone marrow mesenchymal stem cell（BMMSCs）
Transplantation
Down syndrome
