Objective: To study the clinical features and prognosis of core binding factor acute myeloid leukemia (CBF-AML) in children.
Methods: A retrospective analysis was performed from the chart review data of children who were newly diagnosed with CBF-AML in the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, from August 2009 to November 2015. According to the type of fusion gene, the children were divided into CBFB-MYH11 and AML1-ETO groups. Clinical features and prognosis were analyzed and compared between the two groups.
Results: A total of 91 children with CBF-AML were enrolled in this study, among whom there were 74 (81%) in the AML1-ETO group and 17 (19%) in the CBFB-MYH11 group. Additional chromosomal abnormalities were observed in 38 children (42%), and deletion of sex chromosome was the most common abnormality and was observed in 28 children (31%). After the first course of induction treatment, the complete remission rate was 97% (88/91), the recurrence rate was 29% (26/91), the 5-year event-free survival (EFS) rate was 65%±6%, and the 5-year overall survival (OS) rate was 75%±5%. There were no significant differences between the AML1-ETO and CBFB-MYH11 groups in 5-year EFS rate (62%±7% vs 77%±11%, P>0.05) or 5-year OS rate (72%±6% vs 88%±9%, P>0.05).
Conclusions: AML1-ETO is the main type of fusion gene in children with CBF-AML, and deletion of sex chromosome is the most common type of additional chromosomal abnormalities. Children with CBF-AML often have a good prognosis, and the children with AML1-ETO have a similar prognosis to those with CBFB-MYH11.
目的: 分析儿童核心结合因子相关急性髓系白血病(CBF-AML)的临床特征及疗效。
方法: 回顾性分析2009年8月至2015年11月于中国医学科学院血液病医院确诊的初诊CBF-AML患儿的临床资料，根据融合基因类型，分为CBFB-MYH11组和AML1-ETO组。总结并比较其临床特征及预后。
结果: 共纳入91例初诊CBF-AML患儿，其中AML1-ETO组74例(81%)，CBFB-MYH11组17例(19%)。38例(42%)患儿伴有附加染色体异常，其中性染色体缺失28例(31%)最为常见。第1个疗程诱导治疗完全缓解率为97%(88/91)，复发率为29%(26/91)，5年EFS率为65%±6%，5年OS率为75%±5%。AML1-ETO、CBFB-MYH11组5年EFS率分别为62%±7%、77%±11%(P > 0.05)。AML1-ETO、CBFB-MYH11组5年OS率分别为72%±6%、88%±9%(P > 0.05)。
结论: 儿童CBF-AML以AML1-ETO为主，性染色体缺失为最常见的附加染色体异常，预后较好，AML1-ETO与CBFB-MYH11患儿预后相当。