脐带间充质干细胞移植治疗遗传性痉挛性截瘫2例

背景：遗传性痉挛性截瘫是一种具有临床及遗传异质性的神经系统遗传病,目前临床治疗无明显效果。目的：观察脐带间充质干细胞移植治疗遗传性痉挛性截瘫的效果及安全性。方法：将细胞总数为（2～6）×107个人胎儿脐带间充质干细胞通过静脉输注和腰穿鞘内注射途径移植到自愿接受干细胞移植的2例遗传性痉挛性截瘫患者体内。移植后定期随访观察患者临床症状及各项指标的变化并进行综合分析。结果与结论：2例患者经脐带间充质干细胞移植治疗后临床症状均明显好转,双下肢肌张力明显降低,不需借助拐杖或他人帮助可独立行走,并且步态平稳,移植后各项生化指标正常,未出现严重的并发症和明显的不良反应。随访1年余2例患者的症状持续缓解无复发。说明脐带间充质干细胞移植治疗遗传性痉挛性截瘫近期疗效明显,可以改善患者的临床症状,延缓病情的进展。

Application of umbilical cord mesenchymal stem cell transplantation in the treatment of two cases of hereditary spastic paraplegia

BACKGROUND：Hereditary spastic paraplegia is a kind of nervous system genetic disease which has clinical and genetic heterogeneity. Now,the treatment effectiveness of this disease is poor. OBJECTIVE：To observe the clinical effect and safety of umbilical cord mesenchymal stem cells （UC-MSCs） transplantation in the treatment of hereditary spastic paraplegia （HSP）. METHODS：Two patients with HSP received UC-MSCs transplantation which total cellular score of UC-MSCs was （2-6）×107 by intravenous infusion and lumbar puncture intrathecal injections. Both patients were followed up after transplantation. Clinical symptoms and various indexes were observed and literature review was performed. RESULTS AND CONCLUSION：After transplantation,the clinical symptoms of both patients improved obviously：muscle tonus of both lower extremities reduced obviously,independent ambulation independent of crutches and another person＇s aid,and walking was stable. Various biochemical indicators were normal and both patients had no severe complications or clear side effects after transplantation. Both patients＇ conditions were continuously catabatic and no recurrence was found after one year follow up. These indicated that UC-MSCs transplantation is effective and can ameliorate clinical manifestations and delay progression of the disease in the treatment of HSP.