强化免疫抑制治疗和单倍体移植治疗年轻重型再生障碍性贫血的比较

目的比较强化免疫抑制治疗(IST)与单倍体供者异基因造血干细胞移植(HID-HSCT)治疗年轻重型再生障碍性贫血(SAA)患者的疗效和转归。方法回顾性分析55例年龄14~30岁的SAA患者,其中IST组治疗29例,HID-HSCT组治疗26例。结果IST组和HID-HSCT组完全缓解率分别为58.6%和84.6%(P<0.05),总反应率分别为86.2%和84.6%。两组5年总生存率(IST组为77.9%±11.7%,HID-HSCT组为82.1%±8.4%)和无事件生存率(IST为67.6%±11.7%,HID-HSCT为69.2%±9.1%)差异无统计学意义。IST组复发率为3.4%,克隆转化率为13.8%;HID-HSCT组复发率和克隆转化率均为0%,但2~4级aGVHD和cGVHD的发生率分别为19.2%和15.4%。IST组的住院费用明显低于HID-HSCT组(中位费用16.8万元vs 37.8万元,P<0.001)。结论IST与HID-HSCT相比总反应率和生存率差异无统计学意义。鉴于费用低,IST仍然是无同胞供者的年轻SAA患者的合适选择。

Comparison of intensive immunosuppressive therapy with haploidentical transplantation for young severe aplastic anemia

Objective A retrospective study was performed to compare intensive immunosuppressive therapy(IST)and haploidentical donor hematopoietic stem cell transplantation(HID-HSCT)for young patients with severe aplastic anaemia(SAA).Methods Fifty-five SAA patients aged 14-30 years were included.The patients were treated with anti-thymocyte immunoglobulin(ATG)combined with cyclosporine A(CsA)(29 cases,IST group)or HID-HSCT(26 cases,HID-HSCT group).Results The complete response(CR)rates in the IST and HID-HSCT groups were 58.6%vs 84.6%(P=0.034),and the overall response(OR)rates were 86.2%vs 84.6%.No significant difference was found in estimated 5-year overall survival(IST 77.9%±11.7%vs HID-HSCT 82.1%±8.4%)andevent-free survival(IST 67.6%±11.7%vs HID-HSCT 69.2%±9.1%).The relapse rate of the IST group was 3.4%,and the clonal transformation rate was 13.8%.In HID-HSCT group,the relapse and clonal transformation rates were both 0%,and the incidence rates of grades 2-4 aGVHD and cGVHD were 19.2%and 15.4%respectively.The hospitalization expenses were significantly less in the IST group than in that of HID-HSCT group(median 168000 yuan vs 378000 yuan,P<0.001).Conclusions IST shows similar OR,OS and EFS but much less expense comparing with that of HID-HSCT.IST remains an appropriate choice for young SAA patients without sibling donors.