经CD34^+纯化的自体外周血干细胞移植治疗小儿难治性自身免疫性疾病的初步观察

Study of the Treatment with Autologous Peripheral Blood CD34^+ Cell Transplantation in Juvenile Severe Autoimmune Disease

目的探讨经CD34+纯化的自体外周血干细胞移植治疗小儿难治性自身免疫性疾病的疗效。方法3例难治性自身免疫性疾病患儿接受自体外周血CD34+细胞移植。首先采用环磷酰胺+非格司亭方案动员外周血干细胞,通过CliniMACS细胞分选仪分选CD34+细胞后冻存。然后选用卡莫司汀+依托泊苷+阿糖胞苷+美法仑+抗淋巴细胞球蛋白,或环磷酰胺+美法仑+抗淋巴细胞球蛋白两种方案预处理;当天回输CD34+细胞;观察造血重建、免疫恢复,以及症状、体征变化情况。结果动员获得了足够单个核细胞,纯化后CD34+细胞纯度高。粒细胞植入时间分别为+9d、+13d、+11d,血小板植入时间分别为+14d、+18d和+13d。3例原发病缓解。随访16～18个月,皮肌炎皮损消失,四肢肌力恢复正常,类风湿关节炎28个关节疾病活动评分由7.3分降至2.4分,狼疮活动指数评分由16分降至4分。结论自体外周血CD34+细胞移植是常规治疗无效的小儿难治性自身免疫性疾病的可选择治疗措施之一。

Objective To explore the efficacy of autologous peripheral blood CD34^＋ cell transplantation in the treatment of severe autoimmune disease. Methods Three cases of juvenile severe autoimmune disease received autologous peripheral blood CD34^＋ cell transplantation. Autologous peripheral blood cells were mobilized with the regimen of cycolphosphamide plus rhG-GSF, then the CD34^＋ cells were selected by CliniMACS. The preparative therapy consisted of CTX/Mel/ATG or BEAM/ATG. The patient received CD34^＋ cell transplantation, then hematopoiesis recovery immunity resume and the changes of symptom and sign were observed. Results After mobilization,the amount of monocyte cell was enough. Through selection, high purity of CD34^＋ cells was obtained. Granulocytes recovery was achieved on day 9,13 and 11 post autograft respectively. Platelets engraftment was achieved on day 14,18 and 13 post autograft respectively. The three patients showed improvement of the disease. At 16-18 months post autograft, the skin lesions disappeared and the muscle force of limb restored. The juvenile rheumatoid arthritis disease activity score 28（DAS28） decreased from 7.3 to 2.4 ,and the systemic lupus erythematosus disease activity index（DAI） score decreased from 16 to 4. Conclusion The autologous peripheral blood CD34＋ cell transplantation is an alternation choice for severe autoimmune disease not respond to conventional treatments.