Objective: To analyze the clinical outcome and the prognostic factor in pediatric patients with core binding factor-acute myeloid leukemia (CBF-AML). Methods: A total of 121 newly diagnosed pediatric CBF-AML patients enrolled from Aug. 2005 to Sep. 2017 were retrospectively reviewed. Cumulative incidence of relapse (CIR), event-free survival (EFS) and overall survival (OS) rates were estimated by Kaplan-Meier method and prognostic factors were evaluated by Cox regression with SPSS. Results: Of the 121 patients, 120 patients were assessed for bone marrow remission after induction chemotherapy. 100 cases (83.3%) achieved complete remission (CR) after the first course of chemotherapy. 119 cases (99.2%) achieved CR after the second course of chemotherapy. Of the 121 patients, 13 patients (10.7%) had recurrence with the median interval of recurrence as 13.8 months (3.7 to 58.8 months). 17 patients (14.0%) died. The CIR, EFS and OS at 3 years were 12.7%, 77.5% and 82.8%, respectively. The factors including age at diagnosis, sex, initial WBC count, presence of extramedullary leukemia, C-KIT expression, additional chromosomal abnormalities, and CR after the first course of chemotherapy were analyzed by multivariate regression analysis of Cox. Multivariate analysis identified that additional chromosomal abnormalities was the only independent risk factor affecting OS (HR=4.289, 95%CI 1.070-17.183, P=0.040). Conclusions: Pediatric CBF-AML was a unique setting of prognostic subtypes. Chemotherapy produced good responses. Additional chromosomal abnormalities was the only independent risk factor for OS in pediatric CBF-AML.
目的： 分析儿童核心结合因子相关性急性髓系白血病（CBF-AML）的疗效及预后因素。 方法： 对2005年8月至2017年9月收治住院的初诊CBF-AML患儿共121例进行回顾性研究。采用Kaplan-Meier曲线评估患儿的累积复发率（CIR）、无事件生存（EFS）率和总生存（OS）率，Cox回归模型评估预后因素。 结果： 121例患儿中，120例有化疗后骨髓缓解情况评估结果，1个疗程后完全缓解（CR）100例（83.3%），2个疗程后CR 119例（99.2%）。121例患儿中复发13例（10.7%），中位复发时间13.8（3.7~58.8）个月，死亡17例（14.0%）。全部患儿3年CIR、EFS、OS率分别为12.7%、77.5%、82.8%。对发病时年龄、性别、初诊时WBC、是否有髓外白血病、是否伴C-KIT突变、是否有附加染色体异常、第1个疗程是否达CR等进行Cox多因素回归分析，只有伴附加染色体异常是影响患儿OS的独立危险因素（HR=4.289，95%CI 1.070~17.183，P=0.040）。 结论： 儿童CBF-AML是一组独特的预后亚型，化疗疗效较好，伴附加染色体异常是影响患儿OS的独立危险因素。.
Keywords: Childhood; Core binding factor; Leukemia, myeloid, acute; Prognosis DOI: 10.3760/cma.j.issn.0253-2727.2019.01.010.