PDF(1496 KB)
儿童核心结合因子相关急性髓系白血病临床特征及预后分析
刘超, 陈晓燕, 易美慧, 吴文齐, 阮敏, 竺晓凡
中国当代儿科杂志 ›› 2020, Vol. 22 ›› Issue (7) : 739-743.
PDF(1496 KB)
PDF(1496 KB)
儿童核心结合因子相关急性髓系白血病临床特征及预后分析
Clinical features and prognosis of core binding factor acute myeloid leukemia in children
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。
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.
急性髓系白血病 / 核心结合因子 / 临床特征 / 预后 / 儿童
Acute myeloid leukemia / Core binding factor / Clinical feature / Prognosis / Child
[1] Pui CH, Carroll WL, Meshinchi S, et al. Biology, risk stratification, and therapy of pediatric acute leukemias:an update[J]. J Clin Oncol, 2011, 29(5):551-565.
[2] Badr P, Elsayed GM, Eldin DN, et al. Detection of KIT mutations in core binding factor acute myeloid leukemia[J]. Leuk Res Rep, 2018, 10:20-25.
[3] Faber ZJ, Chen X, Gedman AL, et al. The genomic landscape of core-binding factor acute myeloid leukemias[J]. Nat Genet, 2016, 48(12):1551-1556.
[4] 陈晓燕, 刘超, 吴文齐, 等. 儿童非核心结合因子急性髓系白血病的疗效及预后因素分析[J]. 中国当代儿科杂志, 2020, 22(5):466-472.
[5] 沈悌, 赵永强. 血液病诊断及疗效标准[M]. 第4版. 北京:科学出版社, 2018:91-94.
[6] 车琳, 许云云, 庞丽, 等. 核因子阳性儿童急性髓细胞性白血病临床特征及预后因素分析[J]. 中华实用儿科临床杂志, 2014, 29(3):207-211.
[7] Chen X, Dou H, Wang X, et al. KIT mutations correlate with adverse survival in children with core-binding factor acute myeloid leukemia[J]. Leuk Lymphoma, 2018, 59(4):829-836.
[8] Zwaan CM, Kolb EA, Reinhardt D, et al. Collaborative efforts driving progress in pediatric acute myeloid leukemia[J]. J Clin Oncol, 2015, 33(27):2949-2962.
[9] Klein K, Kaspers G, Harrison CJ, et al. Clinical impact of additional cytogenetic aberrations, cKIT and RAS mutations, and treatment elements in pediatric t(8;21)-AML:results from an international retrospective study by the International Berlin-Frankfurt-Münster Study Group[J]. J Clin Oncol, 2015, 33(36):4247-4258.
[10] Kolb EA, Meshinchi S. Acute myeloid leukemia in children and adolescents:identification of new molecular targets brings promise of new therapies[J]. Hematology Am Soc Hematol Educ Program, 2015, 2015:507-513.
[11] 吴珺, 陆爱东, 张乐萍, 等. 儿童核心结合因子相关性急性髓系白血病疗效及预后因素分析[J]. 中华血液学杂志, 2019, 40(1):52-57.
[12] 阮敏, 戚本泉, 刘芳, 等. 儿童急性髓系白血病82例长期随访研究[J]. 中华儿科杂志, 2018, 56(10):730-734.
[13] Marcucci G, Mrózek K, Ruppert AS, et al. Prognostic factors and outcome of core binding factor acute myeloid leukemia patients with t(8;21) differ from those of patients with inv(16):a Cancer and Leukemia Group B study[J]. J Clin Oncol, 2005, 23(24):5705-5717.
[14] Rubnitz JE, Inaba H, Dahl G, et al. Minimal residual disease-directed therapy for childhood acute myeloid leukaemia:results of the AML02 multicentre trial[J]. Lancet Oncol, 2010, 11(6):543-552.
[15] Tsukimoto I, Tawa A, Horibe K, et al. Risk-stratified therapy and the intensive use of cytarabine improves the outcome in childhood acute myeloid leukemia:the AML99 trial from the Japanese Childhood AML Cooperative Study Group[J]. J Clin Oncol, 2009, 27(24):4007-4013.
[16] Ishikawa Y, Kawashima N, Atsuta Y, et al. Prospective evaluation of prognostic impact of KIT mutations on acute myeloid leukemia with RUNX1-RUNX1T1 and CBFB-MYH11[J]. Blood Adv, 2020, 4(1):66-75.
[17] Paschka P, Schlenk RF, Weber D, et al. Adding dasatinib to intensive treatment in core-binding factor acute myeloid leukemia-results of the AMLSG 11-08 trial[J]. Leukemia, 2018, 32(7):1621-1630.
[18] Duployez N, Marceau-Renaut A, Boissel N, et al. Comprehensive mutational profiling of core binding factor acute myeloid leukemia[J]. Blood, 2016, 127(20):2451-2459.
[19] Creutzig U, Zimmermann M, Bourquin JP, et al. Second induction with high-dose cytarabine and mitoxantrone:different impact on pediatric AML patients with t(8;21) and with inv(16)[J]. Blood, 2011, 118(20):5409-5415.
[20] 贾月萍, 左英熹, 陆爱东, 等. 儿童急性髓系白血病M2型性染色体缺失的预后意义[J]. 中国当代儿科杂志, 2015, 17(2):168-171.
[21] 姚新原, 赵利师, 安曦洲, 等. 附加染色体异常的t(8;21)阳性儿童急性髓细胞白血病临床分析[J]. 现代医药卫生, 2018, 34(17):2613-2615, 2618.
