摘要
目的:回顾性分析自体外周血造血干细胞移植治疗重症肌无力的效果。方法:2005-08/2006-11安徽医科大学附属省立医院收治的2例重症肌无力患者,男女各1例,按美国重症肌无力协会新重症肌无力临床分型标准分别为IIIb型、IIIa型,初诊时分别合并有胸腺瘤及未分化结缔组织病,经服用拟胆碱药和多种免疫抑制剂均不能控制症状。移植前采用环磷酰胺+粒细胞集落刺激因子进行干细胞动员,在白细胞超过5×109L-1时用血细胞分离机采集造血干细胞,加入冷冻保护液,置-196℃液氮中冻存备用。预处理方案为胸腺放疗+BuCy+ATG。移植当天,42℃水浴箱中快速复温冻存的造血干细胞,2例患者输入活的有核细胞数分别为1.32×108/kg和3.06×108/kg,输入活CD34+细胞数为2.57×106/kg和11.22×106/kg。结果:2例患者均获得造血重建,都未出现肌无力危象,移植后1个月,在停用胆碱能受体激动剂后肌力正常。1例已随访30个月,不需服用药物,肌力仍正常;另1例移植后4个月,不服用药物肌力正常,移植后5个月死于间质性肺炎引起的呼吸衰竭。结论:对于重症、药物治疗不佳的重症肌无力患者,自体外周血造血干细胞移植近期疗效显著,是治疗的重要选择,需严密观察并及时处理并发症。
BACKGROUND： To retrospectively analyze effect of autologous peripheral blood hematopoietic stem cell transplantation for myasthenia gravis. METHODS： Two cases of patients （one male and one female） with myasthenia gravis were enrolled in this study, who were treated at the Anhui Provincial Hospital Affiliated to Anhui Medical University from August 2005 to November 2006. They were types Ⅲb and Ⅲa according to the Quantitative Myasthenia Gravis score of Myasthenia Gravis Foundation of America. During preliminary diagnosis, they were combined with thymoma and undifferentiated connective tissue disease, which could no be controlled by taking cholinomimetic and multiple immunodepressant. Prior to transplantation, stem cells were mobilized using cyclophosphamide＋granulocyte colony-stimulating factor. Hematopoietic stem cells were collected by using blood cell separator when white blood cells exceeded 5×10^9 L^-1, and frozen at -196 ℃ liquid nitrogen for reserve. Conditioning regimens were Busulfan, cyclopnosphamide, antithymocyte globulin （ATG） plus thymus irradiation. Hematopoietic stern cells （1.32×10^8/kg and 3.06×10^8/kg） we=e respectively infused into patients immediately after quickly thawed at 42 ℃ hot water bath, containing 2.57× 10^6/kg and 11.22×10^6/kg living CD34^＋ cells. RESULTS： The hematopoiesis of both patients were successfully reconstituted and muscle strength was completely recovered out of cholinergic receptor agonists after one month after transplantation. One patient was disease-free with a follow-up of 30 months till now. Muscle strength in the other patient was also persistently normal at 4 months after transplantation until his death of respiratory failure due to interstitial pneumonitis at 5 months after transplantation. CONCLUSION： Autologous peripheral blood stem cell transplantation have an obvious efficiency on the treatment of myasthenia gravis patients. It is also safe if complications related to transplantation were observed carefully and treated imm
出处
《中国组织工程研究与临床康复》
CAS
CSCD
北大核心
2008年第43期8509-8511,共3页
Journal of Clinical Rehabilitative Tissue Engineering Research
