脐带间充质干细胞移植治疗遗传性痉挛性截瘫一例

Umbilical Cord Mesenchymal Stem Cell Transplantation in Treatment of a Patient with Hereditary Spastic Paraplegia

目的观察脐带间充质干细胞移植治疗遗传性痉挛性截瘫（hereditary spastic paraplegia,HSP）的效果和安全性。方法将细胞总数为（2~6）×107个脐带间充质干细胞通过静脉输注和鞘内注射途径移植到我院收治的HSP自愿接受干细胞移植1例的体内。观察患者临床症状及各项指标的变化并随访。结果本例脐带间充质干细胞移植后双下肢肌张力明显降低,Ashwoth痉挛评定分级由3级降至1级,无需借助拐杖或他人帮助可独立行走且步态平稳,各项生化指标正常,疾病严重程度分级由5级降至2级,未出现明显不良反应。随访15个月症状持续缓解无复发。结论脐带间充质干细胞移植治疗HSP近期效果明显,可改善患者临床症状,延缓病情进展,是一种值得借鉴的治疗方法。

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 A patient with HSP received UC-MSCs transplantation with a total cellular score of（2～6）×107 by intravenous infusion and intrathecal injections.The patient was followed up for 15 months after transplantation,and the changes of clinical symptoms and various indexes were observed.Results After transplantation,the clinical symptoms of the patient improved significantly： muscle tonus of both lower extremities were reduced obviously,Ashwoth degree was reduced from degree 3 to degree 1,with stable walking capability without help of crutches and other person.Various biochemical indicators were normal,severity of disease decreased from grade 5 to grade 2 without severe complications and obvious side effects.The patient＇s condition was relieved continuously and no recurrence was found after a 15-month follow-up.Conclusion UC-MSCs transplantation in treatment of HSP is effective in a quick term,and is a worthy therapy,which can improve clinical manifestations and delay progression of the disease.