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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。
Objective: To retrospectively analyze the effect of autologous peripheral blood stem cell transplantation in the treatment of myasthenia gravis. METHODS: Two patients with myasthenia gravis treated in Anhui Provincial Hospital Affiliated to Anhui Medical University from August 2005 to November 2006 were enrolled in this study. According to the American Myasthenia Gravis Association’s new myasthenia gravis clinical classification criteria, type IIIb, IIIa Type, newly diagnosed with thymoma and undifferentiated connective tissue disease, by taking cholinergic drugs and a variety of immunosuppressive agents can not control the symptoms. Before transplantation, cyclophosphamide + granulocyte colony-stimulating factor was used to mobilize stem cells. When leukocytes exceeded 5 × 109L-1, hematopoietic stem cells were collected by hemacytometer, added with cryoprotectant and stored at -196 ℃ in liquid nitrogen until use. Pretreatment regimen for thymus radiotherapy + BuCy + ATG. On the day of transplantation, cryopreserved hematopoietic stem cells were rapidly thawed in water bath at 42 ℃. The numbers of viable nucleated cells in two patients were 1.32 × 108 / kg and 3.06 × 108 / kg respectively, and the number of live CD34 + cells was 2.57 × 106 / kg and 11.22 × 106 / kg. Results: All of the 2 patients received hematopoietic reconstitution. None of them presented with myasthenic crisis. One month after transplantation, their muscle strength was normal after stopping the cholinergic receptor agonist. One patient had been followed up for 30 months and had no muscular strength, and the other one was 4 months after transplantation. Her muscle strength was normal after taking the drug and died of respiratory failure caused by interstitial pneumonia 5 months after transplantation. Conclusion: The recent curative effect of autologous peripheral blood stem cell transplantation is significant for the treatment of critically ill patients with myasthenia gravis, which is an important choice of treatment. Close observation and timely treatment of complications are needed.