摘要
目的Duchenne型肌营养不良(Duchenne muscular dystrophy,DMD)是一种X链锁隐性遗传致死性肌病,抗肌萎缩蛋白(dystrophy)缺失如何引起肌肉变性、坏死具体机制不清,迄今尚无有效的治疗方法。该文试图探索应用自体分选干细胞治疗DMD的疗效。方法从2008年9月至今该科收治15例DMD患者,男14例,女1例;年龄6～23岁;病程2～18年。13例DMD患者有不同程度"鸭步"行走,全身骨骼肌进行性无力,萎缩,腓肠肌纤维化致假性肥大,2例关节畸形挛缩。全部患者肌电图示肌源性损害,多项肌酶升高,尤以磷酸肌酸激酶(CK)显著,可高达10600u/L,3例基因检测异常。自体干细胞采集15例中因4例体重不足20Kg者而直接采集骨髓;11例经G—CSF动员后COBE机采集外周血,CD34+干细胞经CliniMACS仪器分选,获得(1.0～5.6)×107的CD34+细胞,流式细胞仪检测阳性率80%。用分选CD34+细胞对患者做肌肉局部点状注射和细胞静脉回输;2个月后可再次治疗。结果治疗后随诊1～5个月,13例患者行走有明显改善,腓肠肌纤维化程度减轻,局部变软,全身力量增加,关节肌肉训练度提高。2例挛缩肢体有松弛,疼痛有好转,在帮助下可以肢体活动。增高的多项肌酶下降,CK可下降1000～5000u/L不等。肌电图将在半年后复查。结论应用自体分选干细胞为临床治疗DMD寻找到一条新途径,安全可行,其疗效可能与分选干细胞更利于提高调节自身免疫能力,使受损肌细胞再生修复有关。
【Objective】 Duchenne muscular dystrophy （DMD） is an X-linked genetic myopathy characterized by progressive skeletal-muscle degeneration,leading to muscle weakness in childhood,severe paresis in adolescence,and death in the 20s. Until now on there is no effective cures for this disease,we try to evaluate the action and effect of autologous purified blood stem cell transplantation for treatment of DMD.【Methods】 From September 2008 to now,we accepted 15 cases of DMD,14 of them are male,1 is female,ages are 6-23 years,and disease courses are 2-18 years. Thirteen cases were with different ＇wadding gait＇ walking,and their gastrocnemius muscle myofibrosis arrived to rigid level,2 cases＇ joints became deformed and contracture. All of DMD cases＇ electromyogram showed muscle origin impairment,many items of muscle biocatalysts were abnormal,especially creatine kinase （CK） level was high to 10600 u/L,and 3 cases＇ gene control function was disorder. Four cases＇ autologous stem cells were collected directly from bone marrow because of their body weight below 20 kg,other 11 cases＇ were collected from peripheral blood after G-CSF mobilizing by using COBE machine. CD34^＋ stem cells were purified by cliniMACS machine,then arrived the level to （1-5.6）×10^7,and the positive rate arrived 80 percent tested by flow cytometry. The purified CD34^＋ stem cells were used by local intramuscular injection or by vein transfusing back to the patients. After two months the treatment was repeated. 【Results】 Followed up for 1-5 months after treatment,13 cases＇ walking improved much better,gastrocnemius muscle myofibrosis level was lessen,local region turned soften,the body strength was buildup,and the articular muscle training level was increased. Two cases＇ contracture limbs were relaxed,pains were relived and the patients could do limb exercises under help. Multitude raised muscle biocatalysts were decreased,CK decreased 1000 u/L or 5000 u/L different level. ECG would test after 6 months. 【Conclusions】 Purified stem cell transplantation may be one of safe and practical new ways used to treat DMD,its curative effect may related to that the purified stem cell might be much better for regulating autoimmunity and repairing the damaged muscles.
出处
《中国现代医学杂志》
CAS
CSCD
北大核心
2010年第1期66-68,共3页
China Journal of Modern Medicine
