伴t(8;21)/AML1-ETO阳性的儿童急性髓系白血病的临床特点及预后研究

Clinical features and prognosis of t(8;21)/AML1-ETO-positive childhood acute myeloid leukemia

目的分析伴t(8;21)/AML1-ETO阳性的儿童急性髓系白血病(AML)的临床特点、生物学特征及预后。方法对伴t(8;21)/AML1-ETO阳性的55例AML患儿的临床资料进行回顾性分析,采用Kaplan-Meier曲线评估患儿的无事件生存(EFS)率、无病生存(DFS)率和总生存(OS)率,COX回归模型评估预后因素。结果①55例伴t(8;21)/AML1-ETO阳性患儿中,4例放弃治疗,4例化疗1疗程后失访,47例患儿进行了双诱导方案化疗,1疗程、2疗程完全缓解率分别为71%和94%,复发10例(21%),47例患儿的5年EFS率、DFS率、OS率分别为(56.1±7.9)%、(59.8±8.1)%、(72.0±8.1)%。②多因素分析显示年龄是影响患儿预后的独立危险因素,年龄越大出现事故或死亡的风险性越大(P<0.05)。③缓解后继续巩固强化规范化疗的患儿(n=27)5年OS率明显高于不规范化疗的患儿(n=13)[(47.5±17.1)%vs(38.9±17.3)%,P<0.01]。结论伴t(8;21)/AML1-ETO阳性儿童AML是一类具有高度异质性的疾病,其治疗完全缓解率高,远期疗效好,年龄是决定远期疗效的重要因素之一,完全缓解后进行巩固强化规范化疗疗效较好。

Objective To study the clinical and biological characteristics and prognosis of t（8;21）/AML1-ETO-positive childhood acute myeloid leukemia（AML）.Methods The clinical data of 55 children who were diagnosed as t（8;21）/AML1-ETO-positive AML were retrospectively studied.Event-free survival（EFS）,disease-free survival（DFS）,and overall survival（OS） rates were estimated by the Kaplan-Meier method.Prognostic factors were evaluated by COX regression analysis software.Results Of the 55 patients,4 patients gave up treatment after the diagnosis was confirmed and 4 patients were lost to follow-up after the first chemotherapy course.The remaining 47 patients received a double-induction therapy.The total complete remission（CR） rate was 71% and 94% after the first and second chemotherapy course,respectively.The disease was relapsed in 10 patients（21%）.The 5-year EFS,DFS and OS rates were（56.1±7.9）%,（59.8±8.1）%,and（72.0±8.1）%,respectively.Multivariate analysis showed that age was an independent risk factor for the long-term prognosis.The older children had a greater risk of experiencing an accident or death（P0.05）.The 5-year OS rate in 27 patients with regular consolidation chemotherapy was significantly higher than 13 patients with irregular chemotherapy after CR [（47.5±17.1）% vs（38.9±17.3）%;P0.01].Conclusions Childhood t（8;21）/AML1-ETO-positive AML is a highly heterogeneous disease,with a high CR rate and a good long-term prognosis.Age is one of the important factors affecting the long-term therapeutic effect.Regular consolidation chemotherapy applied after CR usually is helpful.