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.
BMC Cancer. 2022 Aug 16;22(1):896. doi: 10.1186/s12885-022-09978-3.
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.
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.
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.
Allo-HSCT (especially haplo-HSCT) can be a viable strategy in these patients, and pre-HSCT MRD status significantly affected the outcomes.
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。
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。
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。
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。
