神经节苷脂联合脐血干细胞移植治疗脊髓小脑性共济失调的近期疗效

Short-term effects of combined ganglioside with umbilical cord blood stem cell transplantation for spinocerebellar ataxia

目的：探讨神经节苷脂联合脐血干细胞移植治疗脊髓小脑性共济失调的近期疗效。方法：回顾性分析我院2010年1月～2012年1月间收治的10例脊髓小脑性共济失调患者的临床病历资料，对患者进行神经节苷脂联合脐血干细胞移植治疗，使用量表评定移植前和移植结束后患者的状态。结果：患者经联合治疗后，世界合作共济失调量表（ International coopera-tive ataxia rating scale，ICARS）各项评分均有下降，治疗后动态功能、姿势和步态、言语障碍的评分明显低于治疗前（P＜0．05）；日常生活能力量表（ Activity of daily living scale ，ADL）的两量表评分均有下降，治疗后躯体生活自理量表总分、工具性日常生活能力量表总分明显低于治疗前（P＜0．05）。结论：神经节苷脂联合脐血干细胞移植治疗脊髓小脑性共济失调的近期疗效显著，有效改善患者的症状，提高日常生活自理能力和生活质量。

Objective：To observe the short-term effects of combined ganglioside with transplantation of umbilical cord blood stem cells for treatment of spi-nocerebellar ataxia.Methods：The clinical data were retrospectively analyzed in 10 patients with spinocerebellar ataxia undergone treatment with combined ganglioside with umbilical cord blood stem cell transplantation in our hospital in January of 2010 to 2012.International Cooperative Ataxia Rating Scale （ ICARS） was used to assess the conditions of patients before and after transplantation .Results：After joint therapy,ICARS scoring was generally fallen and the improvement was significant compared to pretherapy regarding the dynamic function,posture and gait as well as speech disorder（P〈0.05）.The sco-ring on Activity of Daily Living Scale（ADL） was also declined,including lower scores on scales of physical self-maintenance and instrumental activities of daily living（P〈0.05）.Conclusion： Combined ganglioside with transplantation of umbilical cord blood stem cells may produce short-term effect on pa-tients with spinocerebellar ataxia,and these effects include significant improvement of the patients′symptoms,activities of daily living and quality of life.