外周血干细胞移植治疗自身免疫性疾病对照研究及疗效随访  

Controlled study and follow-up of therapeutic effect of peripheral blood stem cell transplantation on autoimmune disease

作　　者：王金铠[1] 孟建波[1] 张金巧[1] 刑莉民[1] 常英军[1] 宋晓宁[1] 孙丽霞 王俊祥[1] 张敏[1] 吕鸿雁[1] 张运清[1] 李同健[1] 吴薇薇[1] 杨清云[1] 

机构地区：[1]河北医科大学第三医院血液科,050051

出　　处：《河北医药》2003年第11期806-808,共3页Hebei Medical Journal

摘　　要：目的　研究、评价大剂量环磷酰胺 (CTX)免疫清除治疗后用及不用造血干细胞救助治疗自身免疫性疾病的安全性及疗效。方法　 ( 1)治疗组自身免疫性疾病 (AD) 11例 ,其中严重性系统性红斑狼疮 9例 ,多发性硬化与晚期类风湿关节炎各 1例 ,采用大剂量CTX免疫清除治疗 (CTX 5 0mg·kg- 1 ·d- 1 × 3d) +造血干细胞 (HSC)救助 ;( 2 )对照组 9例严重性系统性红斑狼疮用血浆置换 +同步化CTX化疗 (CTX 12mg·kg- 1 ·d- 1 × 3d) ,不用造血干细胞救助。结果　 ( 1)治疗组 11例AD所有病人都成功进行了自体外周血干细胞移植 (APBSCT)的全过程 ,造血功能及免疫功能迅速恢复 ,未出现严重的毒性反应及并发症。 9例SLE病人完全缓解 ,相关抗体全部阴转 ,长期随访 (最长 2 4个月 )无 1例复发 ,仅 1例多发性硬化在治疗后 9个月复发 ;( 2 )对照组 9例SLE病人 ,有 3例病人分别在治疗后的第 1、3、8个月疾病复发 ,其中的 2例又进行了APBSCT。结论　 ( 1)血浆置换 +同步化免疫抑制剂CTX化疗 ,不用造血干细胞救助 ,近期疗效较好 ,复发率高 ( 3 3 % ) ;( 2 )大剂量CTX免疫清除治疗后用造血干细胞救助治疗 ,造血功能恢复较快 ,是安全有效的 ,而且持续完全缓解时间长 ,但还需进一步扩大病例 ,长期随访。Objective To investigate the clinical efficacy and safety of immunoablative high-dose cyclophosphamide, with or without stem-cell rescue in treating autoimmune disease(AD).Methods (1)11 patients with autoimmune disease in treatment group,including 9 cases of severe systemic lupus erythematosus (SLE),1 case of multiple sclerosis and 1 case of severe rheumatic arthritis,were treated by immunoablative high-dose cyclophosphamide (CTX 50 mg·kg -1·d -1×3 d) with stem-cell rescue;(2)9 patients with severe SLE in control group received plasmapheresis following synchronization of CTX (CTX 12 mg·kg -1·d -1×3 d) without stem-cell rescue.Results (1)In treatment group,autologous peripheral blood stem cell transplantation in 11 AD patients was successful,with rapid recoverence of marrow and immunological function,without serious toxic reaction.9 patients with SLE achieved complete remission with the related antibody turning to negative,and most of the patients didn't relapse after 3～24 months of follow-up;only one case of multiple sclerosis relapsed after APBSCT.(2)In control group 3 patients relapsed on 1st,3rd and 8th month after treatment,2 of which received APBSCT again.Conclusion (1)Plasmapheresis following synchronization of CTX without stem-cell rescue is effective in short time,but relapsing rate is high (33%);(2)Immunoablative high-dose cyclophosphamide with stem-cell rescue is safe and effective in treating AD,with long remission time ,however,which needs more cases and long-term follow-up to be confirmed.

关 键 词：外周血干细胞移植 治疗 自身免疫性疾病 对照研究 疗效随访 

分 类 号：R593.2[医药卫生—内科学]