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

Clinical characteristics and prognosis of core binding factor positive acute myeloid leukemia in children

目的 探讨核因子（即CBF,包含AML1/ETO和CBF/MYH11）阳性儿童急性髓细胞白血病（AML）临床特点及影响其预后的相关因素.方法 回顾性分析苏州大学附属儿童医院2007年6月至2012年6月初诊并至少进行1个疗程以上化疗的非急性早幼粒细胞白血病（APL）和Down综合征的85例AML病例,分为低危、中危、高危3组,了解3组患儿生存预后,分别分析性别、FAB分型、初诊白细胞数、CBF是否阳性、染色体、诱导缓解疗程数、中枢神经系统白血病（CNSL）、复发、是否行造血干细胞移植（HSCT）等因素对预后影响,Kaplan-Meier生存曲线分析其中40例CBF阳性AML患儿的5年总生存（OS）率和无事件生存（EFS）率,并以CBF阴性者为对照.结果 低、中、高危3组AML 5年OS率分别为（66.5±11.6）％、（54.5±14.4）％、（42.7±7.2）％,差异有统计学意义（x2=6.085,P=0.048）;5年EFS率分别为（62.2±14.0）％、（54.5±14.4）％、（41.9±7.3）％,差异无统计学意义（x2=1.060,P=2.589）.CBF阳性分布为低危组22例,中危组3例,高危组15例;40例CBF阳性者存活23例,45例CBF阴性者存活24例,2组5年OS率分别为（51.8±8.8）％、（50.6±7.6）％,差异无统计学意义.复发病例共10例,其中CBF阳性患儿6例（死亡3例）,阴性患儿4例（死亡2例）.HSCT患儿共20例,CBF阳性者5例（死亡1例）,阴性者15例（死亡3例）.COX回归模型多因素分析显示,性别、诱导缓解化疗疗程数和HSCT对各组AML预后均有统计学意义,而FAB分型、初诊白细胞计数及CNSL则对预后无明显统计学意义（P均＞0.05）.结论单纯化疗对核因子阳性或阴性AML患儿的5年EFS率差异无统计学意义,而HSCT可明显改善CBF阴性及整体AML患儿的5年OS率.

Objective To explore the clinical characteristics and prognosis of core binding factor（CBF） positive acute myeloid leukemia （AML） in children.Methods Eighty-five children with AML who were not acute promyelocytic leukemia （APL） and Downs syndrome were enrolled in this study and received more than one course of chemotherapy from Jun.2007 to Jun.2012 in Children＇s Hospital of Soochow University.They were divided into lowrisk group,medium-risk group,high-risk group according to national unity stratification criteria to confirm their survival condition,analyze the effection of prognosis of sex,FAB type,initial white blood cell number,CBF,chromosome,complete remission course,central nervous system leukemia （CNSL）,relapse,hematopoietic stem cell transplantation （HSCT）.Forty cases with CBF positive AML were evaluated with Kaplan-Meier survival curves of 5-year overall survival（OS） rate and event-free survival（EFS） rate,CBF negative AML as control group.Results Five-year OS rates of low-risk,intermediate-risk and high-risk group were （66.5 ± 11.6） %,（54.5 ± 14.4） % and（42.7 ± 7.2） % respectively,there was significant difference（x2 =6.085,P =0.048） ;and the 5-year EFS rates were （62.2 ± 14.0）%,（54.5 ± 14.4） % and （41.9 ± 7.3） % respectively,there was no significant difference （x2 =1.060,P =2.589）.Cases of distribution of CBF positive AML were 22/23 cases in low-risk group,3/1 1 cases in medium-risk group and 15/51 cases in high-risk group.Twenty-three cases survived in CBF positive AML group,24 cases survived in control group,and their 5-year OS rates were（51.8 ± 8.8） %,（50.6 ± 7.6） %,respectively.Ten cases relapsed,including 6 cases with CBF positive（3 cases death） and 4 control cases（2 cases death）.Twenty cases received HSCT,including 5 cases with CBF positive （1 case death） and 15 control cases （3 cases death）.COX regression multivariate analysis showed that gender,numbers of course of induction chemotherapy to remission and HSCT were individually statistic significance for pediatric AML＇s prognosis while the FAB classification,initial leukocyte count and central nervous system leukemia had no effect on prognosis.Conclusions There is no significantly statistic difference between CBF positive AML and its control in chemotherapy group.The survival rate in control group and all cases in pediatric AML could be obviously improvedby HSCT.