Clinical features and prognosis of core binding factor acute myeloid leukemia in children
LIU Chao, CHEN Xiao-Yan, YI Mei-Hui, WU Wen-Qi, RUAN Min, ZHU Xiao-Fan
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology&Blood Diseases Hospital, Chinese Academy of Medical Sciences&Peking Union Medical College, Tianjin 300020, China
Abstract 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.
LIU Chao,CHEN Xiao-Yan,YI Mei-Hui et al. Clinical features and prognosis of core binding factor acute myeloid leukemia in children[J]. CJCP, 2020, 22(7): 739-743.
LIU Chao,CHEN Xiao-Yan,YI Mei-Hui et al. Clinical features and prognosis of core binding factor acute myeloid leukemia in children[J]. CJCP, 2020, 22(7): 739-743.
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.
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.
