自体外周血干细胞移植治疗难治性免疫性血小板减少性紫癜及随访

Outcome of refractory autoimmune thrombocytopenia purpura treated with autologous peripheral blood stem cell transplantation

目的探讨自体外周血干细胞移植(APBSCT)治疗难治性免疫性血小板减少性紫癜(ITP)的疗效及移植后免疫重建。方法对一例难治ITP患者进行APBSCT并观察临床疗效和免疫重建的过程。结果经环磷酰胺(CY)+G-CSF动员,获得CD34+细胞4·7×106/kg。在TBI+CY预处理后,中性粒细胞、血小板分别在第11、12天植入,无严重并发症。移植后6个月开始淋巴细胞各亚群逐渐恢复,CD4+/CD8+比例仍倒置,记忆T细胞(CD4+CD45RO+)的恢复先于纯真T细胞(CD4+CD45RA+)。随访至31个月时血小板始终大于100×109/L,疾病处于完全缓解中。结论APBSCT可能是治疗难治性ITP的有效手段。

Objective To explore the efficacy and immune reconstitution of autologous peripheral blood stem cell transplantation （APBSCT） in the treatment of refractory autoimmune thrombocytopenia purpura （ITP）. Methods One patient with refractory ITP was treated with APBSCT, the effectiveness and the process of immune reconstitution were analyzed retrospectively. Results The case was mobilized by cyclophosphamide （CY） plus G-CSF and 4. 7 × 10^6/kg CD34^＋ cell was gained totally. Neutrophil and platelet were engrafted at 1 lth and 12th day without severe complications following the conditioning of TBI and CY. Although the each phenotype of the peripheral blood lymphocytes recovered gradually 6 months after the transplant, inverted CD4/CD8 ratio exited and memory T cell was improved much earlier than naive T cell. The platelete count was constantly over 100 × 10^9/L and the patient was in complete remission after a follow-up of 31 months. Conclusion APBSCT is a potential approach in the treatment of refractory ITP.