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2008-02南京医科大学附属无锡第二医院神经内科收治的男性患者1例,32岁,体质量38kg,四肢无力、肌萎缩18年,突发头晕、呕吐3d后入院,经基因分析和肌肉活检确诊为Emery-Dreifus型肌营养不良并发小脑梗死。经HLA配型在上海市脐血库中寻找到一个与受者非血源相关、HIA全相合、Rh血型相合、ABO血型主侧不合的脐血供体,于入院后第9天采用白消安+环磷酰胺+兔抗胸腺淋巴细胞球蛋白预处理后,通过外周静脉移植异基因脐血间充质干细胞悬液40mL,10滴/min,实际输入的有核细胞数为31.98×108。细胞移植5周后,患者头晕消失,吞咽困难、饮水呛咳症状明显好转,可连续饮水,双手协调动作较移植前好转,可操作电脑,体质量约增长4kg;移植后半年,血清肌酸磷酸激酶由移植前35.79μkat/L降至9.55μkat/L,血清肌酸磷酸激酶同工酶由移植前18.20μkat/L降至4.78μkat/L,肌红蛋白由移植前413.50μg/L降至213.20μg/L,功能独立性评分由71分提高至101分,未见免疫排斥反应及致瘤趋性。提示脐血间充质干细胞移植后能在短期内使血清肌酸磷酸激酶水平下降,改善运动功能,有助于Emery-Dreifuss型肌营养不良的治疗,近期安全性可靠。
2008-02 One male patient, aged 32, with a body mass of 38 kg, weakness in limbs and muscle atrophy for 18 years, admitted to Department of Neurology, Second Affiliated Hospital of Nanjing Medical University, admitted to Department of Neurology. The patients were admitted to hospital after 3 days of dizziness and vomiting. After gene analysis and muscle biopsy Diagnosis of Emery-Dreifus muscular dystrophy complicated by cerebellar infarction. HLA matching in the cord blood bank in Shanghai to find a non-associated with the recipient of blood, HIA full match, Rh blood type coincidence, ABO blood type main side of the cord blood donor, on the 9th day after admission with busulfan + Cyclophosphamide + rabbit anti-thymus lymphocyte globulin after pretreatment by peripheral vein allogeneic cord blood MSCs suspension 40mL, 10 drops / min, the actual number of nucleated cells was 31.98 × 108. After 5 weeks of cell transplantation, the patient dizziness disappeared, swallowing difficulties, symptoms of drinking water cough significantly improved, continuous drinking water, hands coordination better than before transplantation, can operate the computer, body weight about 4kg; six months after transplantation, serum creatine phosphate L decreased from 35.79μkat / L to 9.55μkat / L before transplantation, serum creatine phosphokinase isoenzymes decreased from 18.20μat / L to 4.78μkat / L before transplantation and myoglobin decreased from 413.50μg / L before transplantation to 213.20 μg / L, functional independence score increased from 71 to 101 points, no immune rejection and tumorigenicity. Prompted that umbilical cord blood-derived mesenchymal stem cells transplantation in a short period of time to serum creatine phosphokinase levels, improve motor function, contribute to the treatment of Emery-Dreifuss muscular dystrophy, the recent safe and reliable.