【摘要】 目的比较一线使用单倍体造血干细胞移植(HID-HSCT)或强化免疫抑制治疗(IST)治疗中性粒细胞为0的暴发型再生障碍性贫血(FAA)的疗效差异。方法收集2016年1月至2021年1月于中国贫血东部协作组各医院60例一线接受HID-HSCT或IST的中性粒细胞绝对值(ANC)为0的FAA的临床资料,回顾性分析比较2种治疗方法的疗效及预后。结果分别有25例、35例FAA患者接受HID-HSCT或IST治疗。HID-HSCT组和IST组相较:中位起效时间、6个月总反应率(ORR)、完全缓解(CR)均占显著优势:12.0(11.0,15.0)d比87.0(65.0,95.0)d,P<0.001;84.0%比40.0%,P=0.001;80.0%比17.1%,P=0.001。中位随访18.5(4.3,30.8)个月,HID-HSCT组无事件生存率(EFS)和总生存率(OS)均优于IST组(78.3%比20.0%,P=0.000;80.0%比57.1%,P=0.049)。结论 HID-HSCT治疗FAA造血重建快,长期生存好,值得探索做为FAA一线治疗方案。更多还原

【Abstract】 Objective To compare the efficacies of haplo-identical donor hematopoietic stem cell transplantation(HIDHSCT)and intensive immunosuppressive therapy( IST)in treating adult patients with severe aplastic anemia(SAA)with an absolute neutrophil count(ANC)of zero. Methods A retrospective analysis was conducted on clinical data from 60 cases of SAA with an ANC of zero treated with haplo-identical donor HSCT or IIST between January 2016 and January 2021 to compare the efficacy and prognosis of the two regimens. Results Of the 60 patients,25 received first-line allogeneic HID-HSCT and 35 received IST. The median onset time of the HID-HSCT group was 12.0 days(11.0,15.0)shorter than87.0 days(65.0,95.0)in the IST group(P<0.001). At six months,the overall response rate(ORR)and complete response(CR)were significantly higher in the HID-HSCT group than in the IST group( 84.0% vs. 40.0%,P=0.001;80.0% vs.17.1%,P=0.001). Over an median follow-up of 18.5 months(4.3,30.8),at which point the better event free survival (EFS),and overall survival(OS)were also showedbetter in HID-HSCT group: 78.3% vs. 20.0%,P=0.000;80.0% vs. 57.1%, P=0.049. Conclusion HID-HSCT for FAA has a fast hematopoieticreconstruction and good long-term survival. It isworth exploring as the frontline treatment for FAA更多还原