Summary The prognostic factors to stratify acute myeloid leukaemia (AML) with double‐mutated CCAAT/enhancer‐binding protein alpha (CEBPAdm) into different risk groups remains to be determined. In this retrospective study, we evaluated 171 consecutive patients with newly diagnosed AML with CEBPAdm by a Cox proportional hazards regression model. In univariate analyses, colony stimulating factor 3 receptor (CSF3R) and Wilms tumour 1 (WT1) mutations were associated with poor relapse‐free survival (RFS). The induction regimens including homoharringtonine (omacetaxine mepesuccinate) or intermediate‐dose cytarabine was associated with favourable RFS and overall survival (OS). The induction regimen including both homoharringtonine and intermediate‐dose cytarabine was associated with the most favourable RFS (3‐year RFS 84.7%) and OS (3‐year OS 92.8%) compared to the conventional cytarabine and daunorubicin regimen (3‐year RFS 27.7%, hazard ratio [HR] 0.126, 95% confidence interval [CI] 0.051–0.313, Wald p < 0.001; and 3‐year OS 56.4%, HR 0.179, 95% CI 0.055–0.586, Wald p = 0.005). In multivariate analyses, the induction regimen including intermediate‐dose cytarabine (HR 0.364, 95% CI 0.205–0.646, Wald p < 0.001) and CSF3R mutations (HR 2.667, 95% CI 1.276–5.572, Wald p = 0.009) were independently associated with RFS. Taken together, we found that induction regimen and CSF3R mutations were independent prognostic factors for AML with CEBPAdm.
IntroductionAcute myeloid leukemia (AML) patients with CEBPA double mutation (CEBPAdm) were associated with distinct immunophenotypes and prognosis. Recently, both International Consensus Classification (ICC) and World Health Organization (WHO) classifications incorporated BZIP single mutations (CEBPAsmBZIP) into the favorable risk group. However, the immunophenotypes of CEBPAsmBZIP mutations have not been characterized, especially when compared with the immunophenotypes of CEBPAdm.MethodsRetrospectively, we investigated and compared the immunophenotypes of AML with CEBPA mutations. Randomforest model and XGBoost algorithm were used to set up a scoring system based on the immunophenotypes of those patients.ResultsIn a total of 967 AML patients: 218 were CEBPAdm (198 consisted of mutations in the BZIP region [CEBPAdmBZIP], 20 were double mutations outside BZIP region [CEBPAdm‐woBZIP]), 117 were CEBPAsm (54 CEBPAsmBZIP and 63 were single mutations outside BZIP region [CEBPAsm‐woBZIP]) and the others were wildtype CEBPA (CEBPAwt). Patients with CEBPAdmBZIP, CEBPAdm‐woBZIP and CEBPAsmBZIP shared the distinct immunophenotype of CD7+ CD34+ MPO+ HLA‐DR+ CD19−, in contrast to patients with CEBPAsm‐woBZIP and CEBPAwt who showed reduced expression of CD7, HLA‐DR, MPO, CD34 and a higher expression of CD19. Based on these immunophenotypes, we developed a scoring system to preemptively identify AML with CEBPAsmBZIP and CEBPAdm and validated it internally and externally.ConclusionsAML with CEBPAdmBZIP, CEBPAdm‐woBZIP, and CEBPAsmBZIP shared similar immunophenotypic profiles, whereas profoundly differed from the CEBPAsm‐woBZIP and CEBPAwt AML.
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