Effect of extramedullary disease on allogeneic hematopoietic cell transplantation for pediatric acute myeloid leukemia: a nationwide retrospective study
Effect of extramedullary disease on allogeneic hematopoietic cell transplantation for pediatric acute myeloid leukemia: a nationwide retrospective study
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DOI：
10.1038/s41409-021-01250-9
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发表时间：
2021-03-10
影响因子：
4.8
通讯作者：
Atsuta, Yoshiko
中科院分区：
文献类型：
--
作者：
Sakaguchi, Hirotoshi;Miyamura, Takako;Atsuta, Yoshiko
患有急性髓细胞白血病（AML）的儿童通常会发生髓外疾病（EMD），包括中枢神经系统（CNS）病变和髓样肉瘤（MS）。在这项回顾性分析中，我们的目的是确定EMD对2006年至2016年期间678例儿童原发性AML患者（中位年龄，7岁;范围，0.3-15岁）的异基因造血细胞移植（HCT）结局的影响。我们比较了诊断时有EMD（EMD组，n = 158; CNS病变，n = 47; CNS病变+ MS，n = 9; MS，n = 102）和无EMD（非EMD组，n = 520）患者的结局。生存者的中位随访时间为4.5年，EMD组和非EMD组的4年总生存率（OS）分别为60.6%和56.4%（P = 0.60）。除骨病变外，EMD部位的OS无显著差异，骨病变与非缓解状态下HCT后OS较差相关。多因素分析显示EMD不影响HCT的结局。总之，研究结果表明，EMD不应被视为急性髓细胞白血病儿童HCT的不良预后因素。
Children with acute myeloid leukemia (AML) commonly develop extramedullary disease (EMD), which comprises central nervous system (CNS) lesions and myeloid sarcoma (MS). In this retrospective analysis, we aimed to determine the effect of EMD on the outcomes of allogeneic hematopoietic cell transplantation (HCT) in 678 pediatric patients with de novo AML (median age, 7 years; range, 0.3-15 years) between 2006 and 2016. We compared the outcomes between patients with (EMD group, n = 158; CNS lesion, n = 47, CNS lesion + MS, n = 9, and MS, n = 102) and without EMD at diagnosis (non-EMD group, n = 520). Survivors were followed for a median of 4.5 years, and the 4-year overall survival (OS) rates were 60.6% and 56.4% in the EMD and non-EMD groups, respectively (P = 0.60). No significant differences in OS were observed with respect to the EMD site, except bone lesions, which were associated with poor OS after HCT in a non-remission status. A multivariate analysis revealed that EMD did not affect the outcomes of HCT. In conclusion, the study findings suggest that EMD should not be considered a poor prognostic factor in HCT for children with AML.