Second Hematopoietic Stem Cell Transplantation for Post-Transplantation Relapsed Acute Leukemia in Children: A Retrospective EBMT-PDWP Study

Second Hematopoietic Stem Cell Transplantation for Post-Transplantation Relapsed Acute Leukemia in Children: A Retrospective EBMT-PDWP Study
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DOI:
10.1016/j.bbmt.2018.03.002
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发表时间:
2018-08-01
影响因子:
4.3
通讯作者:
Bader, Peter
Bader, Peter
中科院分区:
医学2区
文献类型:
--
作者:
Yaniv, Isaac;Krauss, Aviva C.;Bader, Peter

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从欧洲血液和骨髓移植学会登记处收集了来自120个中心的373名复发性白血病儿童(214名急性淋巴细胞白血病[ALL]和159名急性髓细胞白血病[AML])的结局数据,这些儿童在2004年至2013年期间接受了第二次异基因造血干细胞移植(HSCT)。2年总生存率(OS)为38%,5年为29%,2年无白血病生存率(LFS)为30%,5年为25%。ALL组第二次HSCT后的中位随访时间为36.4个月,AML组为50.2个月。在ALL组中,2年OS为43%,5年为33%,2年LFS为34%,5年为31%。在AML组中,2年OS为32%,5年为24%,2年LFS为24%,5年为17%。ALL组的2年非复发死亡率(NRM)为22%,AML组为18%。OS和LFS的有利预后因素(P <0.05)包括首次HSCT后移植和慢性移植物抗宿主病之间的时间间隔>12个月(两组),第二次HSCT前完全缓解(仅ALL组),年龄>12岁(仅AML组)。随着时间的推移,ALL组的结果更加一致,2年和5年复发率、NRM和LFS之间没有显著差异。复发性急性白血病儿童在第二次HSCT后长期生存的可能性很大。鉴于目前正在开发的许多新型靶向和免疫调节疗法,与更适合新方法的患者相比,确定可能从第二次HSCT中获益的特定患者亚群非常重要。(C)2018年美国血液和骨髓移植协会。
Outcome data were collected from the European Society for Blood and Marrow Transplantation registry on 373 children from 120 centers with relapsed leukemia (214 with acute lymphoblastic leukemia [ALL] and 159 with acute myelogenous leukemia [AML]) who underwent second allogeneic hematopoietic stem cell transplantation (HSCT) between 2004 and 2013. Overall survival (OS) was 38% at 2 years and 29% at 5 years, and leukemia-free survival (LFS) was 30% at 2 years and 25% at 5 years. Median follow-up after second HSCT was 36.4 months in the ALL group and 50.2 months in the AML group. In the ALL group, OS was 43% at 2 years and 33% at 5 years, and LFS was 34% at 2 years and 31% at 5 years. In the AML group, OS was 32% at 2 years and 24% at 5 years, and LFS was 24% at 2 years and 17% at 5 years. The 2-year non-relapse mortality (NRM) rate was 22% in the ALL group and 18% in the AML group. Favorable prognostic factors (P < .05) for OS and LFS included >12 months between transplantations and chronic graft-versus-host disease after the first HSCT (in both groups), complete response before the second HSCT (ALL group only), and age >12 years (AML group only). Findings were more consistent over time in the ALL group, with no significant differences between 2-year and 5-year rates of relapse, NRM, and LFS. Children with relapsed acute leukemias have a substantial likelihood of long-term survival following second HSCT. Given the many novel targeted and immunomodulation therapies currently under development, it is important to identify specific patient subpopulations that may benefit from a second HSCT compared with those better suited to new approaches. (C) 2018 American Society for Blood and Marrow Transplantation.