自体外周血干细胞移植治疗系统性硬化病四例远期疗效观察  

Long-term efficacy of autologous hematopoietic stem cell transplantation in systemic sclerosis patients

作　　者：霍晓聪[1] 蓝梅[1] 汤杨明[1] 黄新翔[1] 黄玉葵[1] 雷静[1] 李永敢 朱霞[1] 李学军[1] 林金盈[1] Huo Xiaocong;Lan Mei;Tang Yangming;Huang Xinxiang;Huang Yukui;Lei Jing;Li Yonggan;Zhu Xia;Li Xuejun;Lin Jinying(Department of Rheumatology,the People's Hospital of Guangxi Zhuang Autonomous Region,Nanning 530021,China)

机构地区：[1]广西壮族自治区人民医院风湿免疫科,南宁530021

出　　处：《中华风湿病学杂志》2019年第10期673-678,共6页Chinese Journal of Rheumatology

基　　金：广西卫生厅自筹经费课题项目(Z2011455,Z2008048);广西医疗卫生适宜技术开发与推广应用项目(S201647)。

摘　　要：目的探讨自体外周血干细胞移植(AHSCT)治疗SSc的远期疗效。方法2007年5月至2009年6月对4例SSc患者进行AHSCT治疗。干细胞动员方案为环磷酰胺+粒细胞集落刺激因子(G-CSF),预处理方案为环磷酰胺50 mg·kg-1·d-1,4 d。移植后观察患者改良Rodnan皮肤评分(mRSS)、高分辨CT率(HRCT)评分、肺功能等指标的变化。结果移植后6个月内,4例患者mRSS改善,HRCT评分、肺功能好转或稳定。移植6个月至1年内,1例持续好转,2例病情出现反复,经免疫抑制治疗后再次稳定。随访8.7(4.1~9.8)年,3例稳定,1例死亡。移植相关并发症有感染、肝功能损害,无移植相关死亡。结论以环磷酰胺为预处理方案的AHSCT治疗SSc安全有效。对病程短、进展快、水肿期的患者移植后疗效显著。但长期疗效差,容易反复,需要巩固维持治疗。Objective To observe the long-term efficacy and safety of autologous hematopoietic stem cell transplantation(AHSCT)for systemic sclerosis(SSc)patients.Methods Between May 2007 and June 2009,4 patients with SSc were enrolled in the study.Peripheral blood stem cells were mobilized with cyclopho-sphamide(CTX)followed by granulocyte colony stimulating factor(G-CSF).Conditioning was performed with i.v.cyclophosphamide 50 mg·kg-1·d-1 for 4 days.The results of the modified Rodnan skin score(mRSS),thoracic high-resolution computer tomography and pulmonary function were collected after transplantation.Results There was an improvement in mRSS,lung function and HRCT in the six months after AHSCT.Within six month to one year after transplantation,one patient had sustained and two patients recurred.After active treatments two patients were improved again.During the follow-up of 8.7(4.1-9.8)years,three patients were stable and one patient died.Infection and hepatic function injury were the major complications.There was not transplant-related mortality.Conclusion AHSCT with CTX as a pre-conditioning regimen is safe and effective for SSc.The efficacy for patients with short course,rapid progress and edema is significant.However,long-term efficacy is poor,and long-term maintenance treatment is needed.

关 键 词：硬皮病 系统性 外周血干细胞移植 

分 类 号：R73[医药卫生—肿瘤]