非清髓性自体外周血造血干细胞移植治疗难治性自身免疫病五年随访

Nonmyeloablative autologons peripheral blood stem cell transplantation for refractory autoimmune disease： 5-year follow-up

目的 探讨非清髓性自体外周血造血干细胞移植（NAST）治疗难治性自身免疫病（AD）的远期疗效。方法总结5例接受NAST的AD患者移植后的随访情况。非清髓性预处理方案：移植前2d阿糖胞苷（200mg·kg-1·d-1）及环磷酰胺（40mg·kg-1·d-1），均为静脉滴注。评价患者移植前后的相关症状体征的改变及远期并发症，流式细胞仪检测免疫功能的变化。结果5例患者均成功植入。白细胞总数恢复正常的平均时间12d，血小板〉100×10^9／L的平均时间为10d，血红蛋白〉120g／L的平均时间为22d。随访中，NAST后5例患者临床症状和体症明显缓解，淋巴细胞亚群检测显示：CD4圾CD4＋／CD8＋均恢复正常。1例男性患者移植4年后妻子正常受孕并产下一健康女婴。4例女性均恢复正常工作与生活。结论与经典的清髓性HSCT治疗AD比较，NAST造血重建快，远期疗效确切。AD患者NAST治疗后生活质量好于清髓性HSCT。

Objective To investigate the long-term efficacy of nonmyeloablative autologous peripheral blood stem cell transplantation （NAST） to cure refractory autoimmune disease （AD）. Method Long-term follow up of four cases of AD patients with NAST were summarized. The pretreatment regimen was intravenous injection of cytarabin （200 mg·kg-1· d-1） and cyclophosphamide （40 mg·kg-1·d-1）. The therapeutic effect was evaluated by the change of symptoms and signs and long term complications. Changes of immune function were detected by flow-cytometry. Results Five cases of patients had been successfully engrafted. The average time for peripheral leucocytes count to reach 4.0×10^9/L was 12 days. It needed 10 days for platelets to return to 100×10^9/L and 22 days for hemoglobin to 120 g/L. Apparent remission of symptoms and signs was observed after transplantation. Lymphocyte subtypes analysis pre- and post- NAST showed that count of CD4＋ and the ratio of CD4 ＋/CD8 ＋ was returned to normal. One patient gave birth to a healthy baby four years after transplantation. Three female patients returned to normal life. Conclusions Compared with classical myeloablative stem cell transplantation, NAST has a rapid hematopoietic recovery and good long-term therapeutic effect in AD. The quality of life in AD patients treated with NAST is higher than those treated with myeloablative hematopoietic stem cell transplantation.