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儿童移植后复发急性白血病的二次造血干细胞移植:回顾性 EBMT-PDWP 研究。

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

机构信息

Rina Zaizov Hematology-Oncology Division, Schneider Children's Medical Center of Israel, Petach Tikva, Israel; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

Rina Zaizov Hematology-Oncology Division, Schneider Children's Medical Center of Israel, Petach Tikva, Israel.

出版信息

Biol Blood Marrow Transplant. 2018 Aug;24(8):1629-1642. doi: 10.1016/j.bbmt.2018.03.002. Epub 2018 Mar 13.

DOI:10.1016/j.bbmt.2018.03.002
PMID:29548831
Abstract

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 nonrelapse 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.

摘要

结果数据来自欧洲血液和骨髓移植学会的注册数据,涉及 120 个中心的 373 名复发白血病患儿(214 例急性淋巴细胞白血病[ALL]和 159 例急性髓细胞性白血病[AML]),他们在 2004 年至 2013 年间接受了第二次异基因造血干细胞移植(HSCT)。2 年和 5 年的总生存率(OS)分别为 38%和 29%,无白血病生存率(LFS)分别为 30%和 25%。第二次 HSCT 后中位随访时间在 ALL 组为 36.4 个月,AML 组为 50.2 个月。在 ALL 组中,2 年 OS 为 43%,5 年 OS 为 33%,2 年 LFS 为 34%,5 年 LFS 为 31%。在 AML 组中,2 年 OS 为 32%,5 年 OS 为 24%,2 年 LFS 为 24%,5 年 LFS 为 17%。ALL 组 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 < .05)包括两次移植之间的间隔时间>12 个月和第一次 HSCT 后慢性移植物抗宿主病(两组)、第二次 HSCT 前完全缓解(仅 ALL 组)和年龄>12 岁(仅 AML 组)。ALL 组的结果随时间更为一致,2 年和 5 年的复发率、NRM 和 LFS 之间无显著差异。接受第二次 HSCT 后,复发急性白血病患儿有很大的长期生存机会。鉴于目前正在开发许多新的靶向和免疫调节治疗方法,重要的是要确定与新方法相比可能从第二次 HSCT 中受益的特定患者亚群。

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