Background:CCAAT/enhancer-binding protein alpha–basic leucine zipper in-frame (CEBPAbZIP-inf) mutations are associated with favorable outcomes in acute myeloid leukemia (AML). So far, there are limited data on integrating clinical and genomic features impacting the outcomes.Methods:Clinical and genomic data from consecutive patients withCEBPAbZIP-infwere reviewed. A Cox proportional hazards regression was used to identify the variables associated with event-free survival (EFS), relapse-free survival (RFS) and survival.Results:224CEBPAbZIP-infpatients were included in this study. In the 201 patients, except for the 19 receiving the transplant in the first complete remission with no events (the transplant cohort), multivariate analyses showed thatIKZF1mutations/deletions were significantly associated with poor EFS (p= 0.001) and RFS (p< 0.001);FLT3-ITD mutations, poor RFS (p= 0.048). In addition, increasing WBC count, lower hemoglobin concentration, non-intensive induction, and MRD positivity after first consolidation predicted poor outcomes. On the basis of the number of adverse prognostic covariates for RFS, the 201 patients were classified into low-, intermediate- or high-risk subgroups, and there were significant differences in the 3-year EFS, RFS and survival rates (allp< 0.001); however, except for survival in the low-risk group, these metrics were lower than those in the transplant cohort.Conclusions:We identified a potential high-risk population with adverse prognostic factors inCEBPAbZIP-infAML patients for which transplantation should be considered.
背景：CCAAT/增强子结合蛋白α-碱性亮氨酸拉链框内（CEBPAbZIP-inf）突变与急性髓系白血病（AML）的良好预后相关。迄今为止，关于整合影响预后的临床和基因组特征的数据有限。 方法：回顾性分析了连续收治的CEBPAbZIP-inf患者的临床和基因组数据。采用Cox比例风险回归模型分析与无事件生存期（EFS）、无复发生存期（RFS）和总生存期相关的变量。 结果：本研究共纳入224例CEBPAbZIP-inf患者。在201例患者中（排除了19例在首次完全缓解期接受移植且无事件发生的患者，即移植队列），多变量分析显示，IKZF1突变/缺失与较差的EFS（p=0.001）和RFS（p<0.001）显著相关；FLT3-ITD突变与较差的RFS相关（p=0.048）。此外，白细胞计数升高、血红蛋白浓度降低、非强化诱导治疗以及首次巩固治疗后微小残留病阳性均预示着不良预后。根据RFS不良预后协变量的数量，将201例患者分为低危、中危或高危亚组，其3年EFS、RFS和总生存率存在显著差异（均p<0.001）；然而，除低危组的总生存率外，这些指标均低于移植队列。 结论：我们在CEBPAbZIP-inf AML患者中识别出一个具有不良预后因素的高危人群，应考虑对该人群进行移植治疗。
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