目的 探讨单倍型造血干细胞移植(haplo-HSCT)治疗儿童获得性重型再生障碍性贫血(SAA)的疗效.方法 同顾性分析2011年12月1日至2017年12月1日接受haplo-HSCT的59例SAA患者.结果 ①全部59例SAA患儿中男37例、女22例,中位年龄4.5(1.2~ 14.8)岁,中位体重43(12~80)kg;SAA-Ⅰ型47例,SAA-Ⅱ型12例,26例为极重型再生障碍性贫血(VSAA);首次移植56例,二次移植3例.②预处理选用白消安+环磷酰胺+抗胸腺细胞球蛋白(ATG)方案或白消安+氟达拉滨+环磷酰胺+ATG方案;采用环孢素A(CsA)+霉酚酸酯+甲氨蝶呤方案预防急性GVHD;回输单个核细胞中位数为15.60(7.74~ 21.04)×108/kg,CD34+细胞中位数为4.86(3.74 ~ 7.14)× 106/kg.③全部59例患儿均获得中性粒细胞和血小板植入.中性粒细胞植入中位时间为13(10~19)d,血小板植入中位时间为19(9~ 62)d.④Ⅰ~Ⅱ、Ⅲ/Ⅳ度急性GVHD发生率分别为45.76%(27/59)、13.56% (8/59),慢性GVHD发生率为8.47% (5/59).巨细胞病毒血症发生率为59.32%(35/59)、EB病毒血症发生率为28.81%(17/59).⑤中位随访时间30(8~ 80)个月,57例无病存活,2例死亡(均死于GVHD),5年总生存率、无失败生存率均为(96.4±2.5)%.结论 haplo-HSCT是儿童SAA疗效较好的治疗方法.
Objective To investigate the efficacy of haplotype hematopoietic stem cell transplantation in the treatment of acquired severe aplastic anemia (SAA) in children.Methods The clinical characteristics of 59 pediatric patients with SAA,including 26 cases VSAA,37males and 22 females,47 cases type Ⅰ and 12 cases type Ⅱ,undrerwent haplo-HSCT in our hospital between December 1st,2011 and December 1st,2017 were retrospectively analyzed.Among 59 patients,56 patients with a median age of 4.5 (1.2-14.8)years and median weight of 43 (12-80)kg underwent their first HSCT and 3 patients underwent their second HSCT.All patients received the following conditioning regimen:busulfan,cyclophosphamide,and rabbit ATG or Bu (-,CTX),fludarabineand rabbit ATG.The prophylaxis of acute graft versus host disease (aGVHD) was cyclosporine (CsA),MMF and methotrexate.All patients received bone marrow transfusion on day 01 and peripheral stem cell transfusion on day 02 from haploid donor.The median dose of donor mononuclear cell counts was 15.60 (7.74-21.04) × 108/kg of recipient weight and CD34+ cell counts was 4.86(3.74-7.14)× 106/kg of recipient weight.Results Neutrophils and platelets of all 59 children were implanted.The median implantation time of granulocytes and platelets were 13 (10-19) d,19 (9-62) d,respectively.The incidence of grade Ⅰ-Ⅱ aGVHD was 45.76% (27cases) and grade Ⅲ/Ⅳ 13.56% (8 cases),The incidence of chronic GVHD was 8.47% (5 cases),The incidences of CMV and EBV viremia were 59.32% (35 cases) and 28.81% (17 cases),respectively.The median follow-up was 30 (8-80) months,57 patients survived with disease free,2 patients died of GVHD.Both of the estimated 5-year OS and DFS rates were (96.4±2.5)%.Conclusion Haplo-HSCT could improve the outcomes of SAA children.