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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 study and evaluate the safety and efficacy of high-dose cyclophosphamide (CTX) immunosuppressive therapy for the treatment of autoimmune diseases with and without hematopoietic stem cell rescue. Methods (1) Eleven patients with autoimmune diseases (AD) were treated in the treatment group, including 9 severe systemic lupus erythematosus, 1 multiple sclerosis and advanced rheumatoid arthritis, and high dose CTX immunosuppression (CTX 50mg (2) In the control group, 9 patients with severe systemic lupus erythematosus were treated with CTX chemotherapy (CTX 12mg · kg-1 · d-1) 1 × 3d), without hematopoietic stem cell rescue. Results (1) All the 11 patients with AD in the treatment group were successfully performed the whole process of autologous peripheral blood stem cell transplantation (APBSCT), the hematopoietic function and immune function recovered rapidly, and no serious toxic reaction and complications occurred. Nine patients with SLE were completely relieved and all the antibodies were negative. All patients had long-term follow-up (up to 24 months) with no recurrence and only 1 case with multiple sclerosis relapsed 9 months after treatment. (2) In the control group, 9 cases of SLE Patients, 3 patients relapsed after the first 1,3,8 months of disease recurrence, two of which were APBSCT. Conclusions (1) CTX chemotherapy with plasma exchange and synchronized immunosuppressive agents, without the help of hematopoietic stem cells, has good short-term efficacy and high recurrence rate (33%). (2) After treatment with high-dose CTX immunosuppressive therapy with hematopoietic stem cell rescue, Quick recovery of hematopoietic function, is safe and effective, and lasting a long time to complete remission, but still need to further expand the case, long-term follow-up.