不同供体异基因造血干细胞移植一线治疗56例极重型再生障碍性贫血的疗效及安全性观察

付安蝶.不同供体异基因造血干细胞移植一线治疗56例极重型再生障碍性贫血的疗效及安全性观察[J].内科急危重症杂志,2022,28(5):378-382

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DOI：10.11768/nkjwzzzz20220506

中文关键词: 极重型再生障碍性贫血异基因造血干细胞移植同胞全相合供体替代供体

英文关键词:

基金项目:湖北省自然科学基金（No:2021CFB561)

作者	单位	E-mail
付安蝶	华中科技大学同济医学院附属同济医院	doctormeng@163.com

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中文摘要:

目的：评价不同供体异基因造血干细胞移植（allo-HSCT）一线治疗极重型再生障碍性贫血（VSAA）的疗效与安全性。方法：回顾性分析接受一线allo-HSCT治疗的VSAA患者临床资料，统计分析总生存(OS)率、移植物抗宿主病（GVHD）发生率、植入成功率及无病生存(EFS)率等指标。结果：共纳入56例患者，男33例，女23例，中位年龄为25（14，36）岁。同胞全相合造血干细胞移植（MSD-HSCT）25例，替代供体造血干细胞移植（AD-HSCT）31例。56例患者均获得造血重建，MSD组、AD组中性粒细胞植入时间分别为12（10，15）、13（11，15）（P=0.140），血小板植入时间分别为11（9，16）、12（10，15）（P=0.372），急性GVHD（aGVHD）发生率分别为16.00%、45.16%（P=0.027），Ⅰ~Ⅱ度aGVHD发生率分别为12.00%、38.71%（P=0.025），Ⅲ/Ⅳ度aGVHD发生率分别为4.00%、6.45%（P=0.685），慢性GVHD(cGVHD)发生率分别为44.00%、22.58%（P=0.149），巨细胞病毒（CMV）血症发生率分别为40.00%、61.29%（P=0.133），EB病毒(EBV)血症发生率分别为68.00%、67.74%(P=0.984)。2组患者的3年预期OS率分别为92.00%、87.09%（P=0.682），EFS率分别为84.00%、77.42%（P=0.737）。结论：MSD-HSCT、AD-HSCT一线治疗VSAA的总体疗效和安全性大致相当。对于无同胞全相合供体的VSAA患者，一线接受AD-HSCT是一个可行的选择。

英文摘要:

Objective: To evaluate the efficacy and safety of allogenic hematopoietic stem cell transplantation(allo-HSCT) with different donors in the first-line treatment for very severe aplastic anemia(VSAA). Methods: The clinical data were retrospectively reviewed on VSAA patients who underwent Allo-HSCT in the first-line treatment. The estimated overall survival(OS), incidence of graft-versus-host disease(GVHD), implantation success rate and event-free survival(EFS) were analyzed. Results: A total of 56 VSAA patients were enrolled, including 33 males and 23 females, with a median age of 25 (14, 36) years. Based upon donor sources, they were divided into matched sibling donors (MSD) group (25 cases)and alternative donors (AD) group (31 cases).Successful engraftment was observed in all patients. The median time of neutrophil engraftment in the MSD group and AD group was 12 (10, 15) days and 13(11, 15) days respectively (P=0.140). The median time of platelet engraftment in the MSD group and AD group was 11(9, 16) days and 12(10, 15) days respectively (P=0.372). The cumulative incidence of aGVHD in the MSD group and AD group was 16.00% and 45.16% respectively (P=0.027). The incidence of grade I-Ⅱ aGVHD in the MSD group and AD group was 12.00% and 38.71% respectively (P=0.025), and that of grade Ⅲ/Ⅳ aGVHD was 4.00% and 6.45% respectively (P=0.685). The cumulative incidence of cGVHD in the MSD group and AD group was 44.00% and 22.58% respectively (P=0.149). The incidence of CMV viremia in the the MSD group and AD group was 40% and 61.29% respectively (P=0.133) and that of EBV viremia was 68.00% and 67.74% respectively (P=0.984).There were no differences in the estimated 3-year OS(92.00% vs.87.09%, P=0.682) or EFS(84.00% vs. 77.42%, P=0.737)between MSD and AD groups. Conclusions: The efficacy and safety of MSD-HSCT and AD-HSCT were comparable for VSAA patients. In the absence of MSD, it's reasonable to consider AD-HSCT as the first line treatment.