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异基因造血干细胞移植治疗儿童 MLL 重排急性髓系白血病的疗效。

Outcomes of allogeneic haematopoietic stem cell transplantation for paediatric patients with MLL-rearranged acute myeloid leukaemia.

机构信息

Department of Hematology, Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.

Department of Pediatrics, Peking University People's Hospital, Peking University, Beijing, China.

出版信息

BMC Cancer. 2022 Aug 16;22(1):896. doi: 10.1186/s12885-022-09978-3.

Abstract

BACKGROUND

The presence of mixed-lineage leukaemia rearrangement (MLL-r) in paediatric patients with acute myeloid leukaemia (AML) is a poor prognostic predictor. Whether allogeneic haematopoietic stem cell transplantation (allo-HSCT) is beneficial in such cases remains unclear.

METHODS

We evaluated the outcomes and prognostic factors of allo-HSCT in 44 paediatric patients with MLL-r AML in the first complete remission (CR1) between 2014 and 2019 at our institution.

RESULTS

For all the 44 patients, the 3-year overall survival (OS), event-free survival (EFS), and cumulative incidence of relapse (CIR) were 74.5%, 64.1%, and 29.1%, respectively. Among them, 37 (84.1%) patients received haploidentical (haplo)-HSCT, and the 3-year OS, EFS, and CIR were 73.0%, 65.6%, and 26.4%, respectively. The 100-day cumulative incidence of grade II-IV acute graft-versus-host disease (aGVHD) post-transplantation was 27.3%, and that of grade III-IV aGVHD was 15.9%. The overall 3-year cumulative incidence of chronic graft-versus-host disease (cGVHD) post-transplantation was 40.8%, and that of extensive cGVHD was 16.7%. Minimal residual disease (MRD)-positive (MRD +) status pre-HSCT was significantly associated with lower survival and higher risk of relapse. The 3-year OS, EFS, and CIR differed significantly between patients with MRD + pre-HSCT (n = 15; 48.5%, 34.3% and 59%) and those with MRD-pre-HSCT (n = 29; 89.7%, 81.4% and 11.7%). Pre-HSCT MRD + status was an independent risk factor in multivariate analysis.

CONCLUSIONS

Allo-HSCT (especially haplo-HSCT) can be a viable strategy in these patients, and pre-HSCT MRD status significantly affected the outcomes.

摘要

背景

儿童急性髓系白血病(AML)患者存在混合谱系白血病重排(MLL-r)是预后不良的预测因素。在这种情况下,异体造血干细胞移植(allo-HSCT)是否有益尚不清楚。

方法

我们评估了本机构 2014 年至 2019 年间 44 例在首次完全缓解(CR1)期存在 MLL-rAML 的儿科患者接受 allo-HSCT 的结果和预后因素。

结果

所有 44 例患者的 3 年总生存率(OS)、无事件生存率(EFS)和累积复发率(CIR)分别为 74.5%、64.1%和 29.1%。其中 37 例(84.1%)患者接受了单倍体(haplo)-HSCT,其 3 年 OS、EFS 和 CIR 分别为 73.0%、65.6%和 26.4%。移植后 100 天 II-IV 级急性移植物抗宿主病(aGVHD)的累积发生率为 27.3%,III-IV 级 aGVHD 的累积发生率为 15.9%。移植后慢性移植物抗宿主病(cGVHD)的总 3 年累积发生率为 40.8%,广泛型 cGVHD 的累积发生率为 16.7%。HSCT 前微小残留病(MRD)阳性(MRD+)状态与生存率降低和复发风险增加显著相关。HSCT 前 MRD+(n=15;48.5%、34.3%和 59%)和 MRD-(n=29;89.7%、81.4%和 11.7%)患者的 3 年 OS、EFS 和 CIR 差异有统计学意义。多因素分析显示,HSCT 前 MRD+状态是独立的危险因素。

结论

allo-HSCT(尤其是 haplo-HSCT)是这些患者可行的治疗策略,HSCT 前 MRD 状态显著影响预后。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/abeb/9382754/bfa7a83c0955/12885_2022_9978_Fig1_HTML.jpg

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