IST、脐血输注联合rhTPO治疗SAA的临床观察

Clinical Observation of IST, Cord Blood Infusion Combined with rhTPO in the Treat-ment of SAA

摘要: 目的：评价强化免疫抑制疗法(immunosuppressive therapy, IST)、脐血输注联合重组人血小板生成素(recombinant human thrombopoietin, rhTPO)治疗重型再生障碍性贫血(severe aplastic anemia, SAA)的疗效。方法：回顾性分析7例初治SAA患者的临床资料，其治疗方案为IST (兔抗人胸腺免疫球蛋白 + 环磷酰胺 + 环孢素) + 脐血输注 + rhTPO。结果：患者中位年龄为31(14~45)岁，治疗前中位病程为3 (1~132)个月，粒细胞恢复(>0.5 × 109/L)的中位时间为16 (10~24)天，血小板恢复(> 20 × 109/L)的中位时间为36 (11~80)天，治疗3月及6月时的血液学反应率分别为85.7%、71.4%。中位随访时间为46 (4~65)个月，患者3年总生存(overall survival, OS)率为71.4%，5年内疾病复发率为14.3%。结论：IST + 脐血输注 + rhTPO疗法可提高早期血液学反应率，迅速实现造血恢复，安全有效，在保持较高生存率的同时提高患者的生活品质。

Abstract: Objective: To evaluate the efficacy of intensive immunosuppressive therapy (IST), umbilical cord blood transfusion and recombinant human thrombopoietin (rhTPO) in the treatment of severe aplastic anemia (SAA). Methods: The clinical data of 7 newly diagnosed patients with SAA were ret-rospectively analyzed. The treatment regimen was IST (rabbit anti-human thymoglobulin + cyclo-phosphamide + cyclosporine) + cord blood transfusion + rhTPO. Results: The median age of the pa-tients was 31 (14~45) years. The median course of disease before treatment was 3 (1~132) months. The median time of granulocyte recovery (> 0.5 × 109/L) was 16 (10~24) days, the median time of platelet recovery (> 20 × 109/L) was 36 (11~80) days. The hematological response rates were 85.7% and 71.4% at 3 and 6 months, respectively. The median follow-up was 46 (4~65) months. The 3-year overall survival (OS) rate was 71.4%, and the 5-year recurrence rate was 14.3%. Conclu-sion: IST + cord blood transfusion + rhTPO therapy can increase the early hematological reaction rate, quickly achieve hematopoietic recovery, safe and effective, maintain a higher survival rate and improve the quality of life of patients.