摘要：
目的:探讨儿童急性非淋巴细胞白血病(非M3)采用NOPHO-AML 2004化疗方案的疗效及安全性.方法:2013年1月至2017年6月诊断的1-13岁急性非淋巴细胞白血病(非M3)33例.按FAB分型:M0 1例,M14例,M212例,M4 5例,M5 8例,M6 1例,M7 2例;高危14例,标危19例;均采用NOPHO-AML 2004方案化疗.应用SPSS 22.0软件统计,采用Kaplan-Meier生存分析法和Cox回归模型进行分析.结果:33例患儿诱导第1疗程(AIET)获完全缓解者27例,未缓解者5例,缓解率81.8％.在5例未缓解的患儿中,4例经诱导第2疗程(AM)后获完全缓解,1例未缓解,诱导总缓解率为96.9％.9例(占27.3％)骨髓复发,中位复发时间为完全持续缓解后30个月.单因素分析结果表明,年龄和输注红细胞次数是影响早期治疗反应的显著因素;Cox回归多因素分析显示,年龄＞7岁、MRD阳性、输RBC频率＞4次和早期治疗反应差为患儿复发的独立危险因素.患儿3年无事件生存率为59.9％,3年总体生存率为69.2％.8例高危儿童接受强化疗后行异基因造血干细胞移植,预后优于单纯化疗的患儿.接受化疗的患儿均出现不同程度的感染及骨髓抑制,或药物相关的消化道反应和过敏反应,经积极对症治疗后均可耐受.结论:NOPHO-AML 2004化疗方案诱导缓解率高,患者耐受性好,早期治疗反应是影响预后的重要因素.年龄和多次输注浓缩红细胞会显著影响早期治疗反应,且为患儿骨髓复发的重要因素.对于临床高危型急性非淋巴细胞白血病患儿,在NOPHO-AML 2004化疗方案的基础上联合造血干细胞移植可改善预后.%Objective:To investigate the efficacy and safety of NOPHO-AML 2004 chemotherapy regimen for treatment of children with acute myelocytic leukemia (non-M3).Methods:Thirty-three patients aged 1-13 with acute myelocytic leukemia (non-M3) were diagnosed from January 2013 to June 2017.FAB typing showed that 1 case in M0,4 cases in M1,12 cases in M2,5 cases in M4,8 cases in M5,1 case in M6,and 2 cases in M7;Risk stratification showed that:19 cases in standard risk,and 14 cases in high risk.All patients were treated with NOPHO-AML 2004 chemotherapy regimen.SPSS 22.0 software was used,the Kaplan-Meier survival analysis method and Cox regression model were used for statistical analysis.Results:In the first course of treatment (AIET),among 33 child patients there were 27 cases with complete remission,and 5 cases with non-remission,thus the remission rate was 81.8％.Out of the 5 child patients without remission,4 cases reached to the complete remission after the second course (AM),and 1 case did not remission,thus the total remission rate was 96.9％.9 cases (27.3％) underwent bone marrow recurrence and the median recurrence time was 30 months after complete continuous remission.Univariate analysis showed that age and erythrocyte transfusion frequency were significant factors to affect the early treatment response;the multiple Cox regression analysis showed that:age ＞ 7,MRD positive,erythrocyte transfusion ＞ 4 times and poor response to early treatment were independent risk factors for recurrence;Allogeneic hematopoietic stem cell transplantation(HSCT) in 8 high-risk children received enhanced chemotherapy had better efficacy as compared with the chemotherapy alone.The 3-year event-free survival rate was 59.9％,and 3-year overall survival rate was 69.2％.33 children patients experienced varying degrees of infection and myelosuppression,or drug-related gastrointestinal reactions and allergic reactions,patients were tolerable to these side reactions after active symptomatic treatment.Conclusion:NOPHO-AML 2004 chemotherapy regimen has high response rate and good tolerance,early treatment response is an important factor influencing prognosis.Age and repeated red blood cell infusions are the important factors influencing the prognosis,which promote bone marrow recurrence in AML children.For the children suffered from clinical high-risk AML,the NOPHO-AML 2004 chemotherapy regimen combined with HSCT can improve the prognosis of patients.
