初诊儿童急性髓系白血病合并中枢神经系统白血病的临床治疗及预后分析  

Analysis of clinical treatment prognosis of children with newly diagnosed acute myeloid leukemia combined with central nervous system leukemia

作　　者：李怡蓉 李惠萍[1] 肖玉华 陈小敏 丁蓉芳[1] 温建芸[1] 易甜甜 赵娜娜 卢艳玲 余鑫 冯晓勤[1] LI Yirong;LI Huiping;XIAO Yuhua;CHEN Xiaomin;DING Rongfang;WEN Jianyun;YI Tiantian;ZHAO Nana;LU Yanling;YU Xin;FENG Xiaoqin(Department of Pediatrics,Nanfang Hospital,Southern Medical University,Guangzhou 510515,China;Department of Pediatric Internal Medicine,Women and Children's Hospital Affiliated to Xiamen University(Xiamen Maternal and Child Health Hospital),Xiamen 361001,China)

机构地区：[1]南方医科大学南方医院儿科,广州510515 [2]厦门大学附属妇女儿童医院(厦门市妇幼保健院)儿内科,厦门361001

出　　处：《中国小儿血液与肿瘤杂志》2024年第3期154-158,共5页Journal of China Pediatric Blood and Cancer

摘　　要：目的 分析华南儿童急性髓系白血病(C-HUNAN-AML-15)方案应用于中枢神经系统白血病(CNSL)的临床特征及治疗预后,为AML CNSL的治疗提供参考依据。方法 回顾性分析2015年1月—2020年12月收治的CNSL患儿的ELN风险分层以及AML相关融合/突变基因发生率、比较化疗组与移植组生存的差异。结果 研究纳入584例AML,其中CNSL 18例,发病比例为3%。ELN风险分层低危组占50%,中危组占39%,高危组占11%。AML最常见的合并基因变异是AML1/ETO 39%(7/18)、WT1突变22%(4/18)、c-KIT突变22%(4/18)、MLL/AF9 17%(3/18)。18例中采用单纯化疗11例,造血干细胞移植7例,中位随访时间为16.5(1~62)个月。5年总体OS和EFS分别为(74.2±11.2)%、(75.2±10.9)%。其中化疗组的预计5年OS和EFS分别为(90.9±8.7)%、(90.9±8.7)%,均高于移植组的5年OS和EFS分别为(50.5±20.4)%、(51.4±20.4)%,但两组间OS和EFS尚未见统计学差异(P=0.114)。化疗组中FLAG-IDA组预计5年OS和EFS分别为(83.1±11.0%)、(81.8±11.6)%,较DAE组的5年OS和EFS(53.3±24.8)%、(53.3±24.8)%升高,但两组间OS和EFS亦尚未见统计学差异(P=0.263)。结论 华南地区儿童急性髓系白血病协作组中枢神经系统白血病(CNSL)在低中危组病例数高于高危组,通过大剂量阿糖胞苷联合鞘注治疗对低危合并CNSL的患儿预后较好。Objective This study aimed to analyze the clinical characteristics and treatment outcomes of central nervous system leukemia(CNSL) in pediatric acute myeloid leukemia(AML) patients which received the C-HUNAN-AML-15 protocol in order to provide reference for the treatment of AML CNSL.Methods A retrospective analysis was conducted on pediatric patients diagnosed with CNSL from January 2015 to December 2020.The study evaluated the ELN risk stratification,occurrence rate of AML fusion/mutation genes,and compared the survival outcomes between the chemotherapy group and the transplantation group.Results A total of 584 cases of AML were included in this study,among which 18 cases were in initial diagnosis of CNSL,accounting for 3% of the total.Among the CNSL cases.The ELN risk stratification showed that 50% were classified as low risk,39% as intermediate risk,and 11% as high risk.The most common gene mutations in AML were AML1/ETO(39%),WT1(22%),c-KIT(22%),and MLL/AF9(17%).Among the 18 cases,11 children received chemotherapy alone and 7 children underwent hematopoietic stem cell transplantation.The median follow-up time was 16.5 months(1~62).The 5-year overall survival(OS) and event-free survival(EFS) rates were(74.2±11.2)% and(75.2±10.9)%,respectively.The 5-year OS and EFS rates in the chemotherapy group were(90.9±8.7)% and(90.9±8.7)%,which were higher than those in the transplantation group,(50.5±20.4)% and(51.4±20.4)%,respectively.However,there was no statistically significance in OS and EFS between the two groups(P=0.114).In the chemotherapy group,the 5-year OS and EFS rates for the FLAG-IDA regimen were(83.1±11.0)% and(81.8±11.6)%,respectively,which were higher compared to the DAE regimen,(53.3±24.8)% and(53.3±24.8)%.However,there was still no statistically significance in OS and EFS between the two regimens(P=0.263).Conclusions In the South China Pediatric AML Collaborative Group,children with AML CNSL in low and intermediate-risk group was more than that of high-risk group,the prognosis of low risk child

关 键 词：中枢神经系统白血病 儿童 急性髓系白血病 预后 

分 类 号：R733.7[医药卫生—肿瘤]