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

摘要:

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。

Abstract:

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 (P<0.05).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)%; P<0.01］. 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.