脐血干细胞移植治疗中间型脊髓性肌萎缩症1例并文献复习

Umbilical Cord Blood Stem Cells Tansplantation for Intermediate Spinal Muscular Atrophy: Report and Review of Literature of One Case

目的:探讨脐血干细胞移植治疗中间型脊髓性肌萎缩症的临床治疗可行性及效果。方法:已确诊的中间型脊髓性肌萎缩症患儿,采用脐血干细胞移植治疗,4次为一疗程,移植途径采用静脉输注(1次)加蛛网膜下腔注射(3次)的方法,治疗前和治疗后半年均需完善神经系统体检、实验室检查、FIM评分、肌电图等。结果:移植治疗后患儿神经系统症状明显改善,FIM评分提高,实验室检查肌酶下降,肌电图提示重收缩每10.0ms所检肌运动单位较前增加。随访10月患儿未出现副反应。结论:应用脐血干细胞移植治疗中间型脊髓性肌萎缩症是有效安全的,可以改善患儿神经功能。

Objective： To observe the effect of the treatment to intermediate form of spinal muscular atrophy by human umbilical cord blood stem cells. Methods： All the patients of intermediate form of spinal muscular atrophy were accepted the transplantion with human umbilical cord blood stem cells The course include for four times, one by iv another three times by lumbar puncture. All the patients were accepted the neurological physical examination ,biochemical test, evaluation of the FIM scal and the examination of EMG. Result： After transplant the neurological function was improved, the scal of FIM increased, the muscle enzymes the muscle motor potential in- creased .no one presented the side effect after ten month follow up. Conclusion： The transplantation with human umbilical cord blood stem cells was safety and feasibility for the patient of intermediate form of spinal muscular atrophy.