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目的观察脐带间充质干细胞(UC-MSC)治疗遗传性痉挛性截瘫(HSP)的临床疗效及安全性。方法 2010年9月及2011年4月,分别给予一HSP家系父子2人行UC-MSC鞘内注射治疗,两个疗程,每次1×106cells/Kg,每周1次,4次为1个疗程。采用改良的Ashworth肌张力分级标准(MAS)、国际合作共济失调评分量表(ICARS)及日常生活量表(ADL),对患者治疗前后神经功能、日常生活能力进行评定。结果第一疗程结束1个月与治疗前比较,2人MAS分级、ICARS及ADL评分均降低,两人行走站立稳定性及言语流利程度较治疗前改善;第二疗程结束后1个月与该疗程治疗前比较,2人ICARS及ADL评分降低,儿子肌张力进一步降低,父亲双上肢共济失调减轻。2人治疗后均未见明显不良反应发生。末次治疗结束后随访20个月,父、子俩分别于第二疗程治疗结束7个月及8个月后,症状继续加重。结论 UC-MSC鞘内注射治疗是安全的,可在一定时间内一定程度上减轻患者临床症状,提高患者生活质量,延缓疾病进展,但疗效不能持久。
Objective To observe the clinical efficacy and safety of umbilical cord mesenchymal stem cells (UC-MSCs) in the treatment of hereditary spastic paraplegia (HSP). Methods In September 2010 and April 2011, two pedigrees including 2 father and son of HSP family were treated with intrathecal injection of UC-MSC respectively. The two courses of treatment were 1 × 106cells / Kg each time, once a week and 4 times as a course of treatment . The modified Ashworth Muscle Tension Grading Standard (MAS), International Cooperative Ataxia Scale (ICARS) and Daily Life Scale (ADL) were used to assess the neurological function and daily living ability of patients before and after treatment. Results The scores of MAS classification, ICARS and ADL in 2 patients decreased at 1 month after the end of the first course of treatment compared with that before treatment. The standing stability and verbal fluency of the two men were better than those before treatment. The second month after the end of the second course of treatment was Before treatment, ICARS and ADL score of 2 patients decreased, son’s muscle tension decreased further, and his father’s upper extremity ataxia reduced. No significant adverse reactions occurred after 2 treatments. After the last treatment was followed up for 20 months, the father and son continued to worsen the symptoms after 7 months and 8 months after the end of the second course of treatment. Conclusion Intrathecal injection of UC-MSC is safe and can relieve clinical symptoms to some extent, improve quality of life of patients and delay disease progression, but the effect can not be sustained.