造血干细胞移植治疗重型再生障碍性贫血的疗效：43例报告
[Efficacies of hematopoietic stem cell transplantation for severe aplastic anemia: a report of 43 patients].
作者信息
Wang Xiuli, Jin Song, Liu Limin, Chen Suning, Zhang Xiang, Jin Zhengming, Miao Miao, Tang Xiaowen, Fu Zhengzheng, Han Yue, Ma Xiao, Chen Feng, Qiu Huiying, Sun Aining, Wu Depei
机构信息
Jiangsu Institute of Hematology, First Affiliated Hospital of Soochow University, Key Laboratory of Thrombosis and Hemostasis under Ministry of Health, Collaborative Innovation Center of Hematology, Suzhou 215006, China.
Jiangsu Institute of Hematology, First Affiliated Hospital of Soochow University, Key Laboratory of Thrombosis and Hemostasis under Ministry of Health, Collaborative Innovation Center of Hematology, Suzhou 215006, China. Email:
出版信息
Zhonghua Yi Xue Za Zhi. 2014 Nov 4;94(40):3140-4.
OBJECTIVE
To evaluate the efficacies of hematopoietic stem cell transplantation (HSCT) for severe aplastic anemia (SAA).
METHODS
A total of 43 SAA patients (SAA-I, n = 29; SAA-II, n = 14) underwent HSCT from 2002 January to 2013 December. There were 22 males and 21 females with a median age of 31 (12-49) years. And 35 patients received HLA-matched sibling HSCT (Sib-HSCT) while another 8 had unrelated donor HSCT (UD-HSCT). The hematopoietic stem cells were collected from bone marrow (n = 10), peripheral blood (n = 23) and bone marrow & peripheral blood (n = 10). Conditioning regimens were mostly composed of Fludarabine, antihuman thymocyte gloBulin and cyclophosphamide. Cyclosporine and methotrexate (including mycophenolate mofetil for UD) were offered for preventing graft-versus-host disease (GVHD). The median counts of mononuclear cell and CD34(+) stem cell were 8.1 (2.4-13.5) ×10(8)/kg and 3.7 (2.3-14.7) ×10(6)/kg respectively.
RESULTS
Hematopoiesis reconstitution was achieved in 42 patients. The median periods for neutrophils to 0.5×10(9)/L and platelets to 20×10(9)/L were +10 (+8-+25) days and +14 (+8-+80) days respectively. The median of survival time was not reached. Overall survival (OS) and failure-free survival (FFS) at 5 years had no differences between UD-HSCT and Sib-HSCT groups (OS: 87.6% vs 84.5%, P = 0.87; FFS: 86.2% vs 79.5%, P = 0.64). The same results also were seen between age ≤ 20 group and > 20 group (OS: 92.0% vs 82.5%, P = 0.39; FFS: 91.0% vs 77.3%, P = 0.38). The median follow-up time was 17.0 (0.4-140.0) months. And 8/43 patients died from thrombotic microangiopathy (TMA) (n = 1), TMA associated with capillary leak syndrome (n = 1), pulmonary infection (n = 3), acute GVHD of grade IV (n = 1) and unknown causes (n = 2).
CONCLUSIONS
Sib-HSCT is preferred for SAA patients under 40 years. Searching of HLA-matched unrelated donor should be performed soon after diagnosis if HLA-matched sibling is unavailable.
目的
评估造血干细胞移植（HSCT）治疗重型再生障碍性贫血（SAA）的疗效。
方法
2002年1月至2013年12月期间，共有43例SAA患者（SAA-I型29例，SAA-II型14例）接受了HSCT。其中男性22例，女性21例，中位年龄31（12 - 49）岁。35例患者接受了人类白细胞抗原（HLA）匹配的同胞造血干细胞移植（Sib-HSCT），另外8例接受了非血缘供者造血干细胞移植（UD-HSCT）。造血干细胞采集来源为骨髓（10例）、外周血（23例）以及骨髓加外周血（10例）。预处理方案主要由氟达拉滨、抗人胸腺细胞球蛋白和环磷酰胺组成。使用环孢素和甲氨蝶呤（UD-HSCT患者使用霉酚酸酯）预防移植物抗宿主病（GVHD）。单个核细胞和CD34(+)干细胞的中位计数分别为8.1（2.4 - 13.5）×10(8)/kg和3.7（2.3 - 14.7）×10(6)/kg。
结果
42例患者实现造血重建。中性粒细胞升至0.5×10(9)/L和血小板升至20×10(9)/L的中位时间分别为 +10（+8 - +25）天和 +14（+8 - +80）天。生存时间中位数未达到。UD-HSCT组和Sib-HSCT组5年总生存率（OS）和无失败生存率（FFS）无差异（OS：87.6% 对 84.5%，P = 0.87；FFS：86.2% 对 79.5%，P = 0.64）。年龄≤20岁组和＞20岁组之间也有相同结果（OS：92.0% 对 82.5%，P = 0.39；FFS：91.0% 对 77.3%，P = 0.38）。中位随访时间为17.0（0.4 - 140.0）个月。43例患者中有8例死于血栓性微血管病（TMA）（1例）、与毛细血管渗漏综合征相关的TMA（1例）、肺部感染（3例）、IV级急性GVHD（1例）以及不明原因（2例）。
结论
40岁以下的SAA患者首选Sib-HSCT。若无法找到HLA匹配的同胞供者，确诊后应尽快寻找HLA匹配的非血缘供者。
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