儿童急性单核细胞性白血病63例疗效分析

目的 评估儿童急性单核细胞性白血病(AML-M5)的疗效,探讨异基因造血干细胞移植(allo-HSCT)治疗儿童AML-M5的指征.方法 对AML-M5患儿进行回顾性分析,与同期非AML-M5的AML进行比较,采用Kaplan-Meier曲线评估患儿的无事件生存(EFS)率和总生存(OS)率,COX回归模型评估预后因素.结果 ①75例AML-M5患儿中12例放弃治疗;63例纳入分析,其中2例化疔第2天死亡,61例患儿2个疗程完全缓解(CR)率为73.8%(45例),5年EFS为(34.5±6.8)%,与同期117例非AML-M5、非急性早幼粒细胞性白血病(AML-M3)的AML患儿(51.0±4.9)%的5年EFS相比差异有统计学意义(P＜0.01).②多因素分析显示患儿年龄≥10岁、1疗程后骨髓原幼稚细胞比例≥15%、2疗程后未CR是独立危险因素.③20例1疗程后骨髓原幼稚细胞比例≥15%的AML-M5患儿选择allo-HSCT者(n=5)预后优于单纯化疗者(n=15)[(60.0±21.9)%vs.(7.3±7.1)%,P=0.024].结论 AML-M5患儿预后明显差于其他AML;化疗1疗程后骨髓原幼稚细胞比例≥15%患儿选择allo-HSCT能明显提高生存率;目前依据尚不足以提示1疗程后骨髓原幼稚细胞比例＜15%患儿应选择非血缘相关供体移植治疗.

Abstract：

Objective To evaluate the outcomes of childhood acute monocytic leukemia (AML-M5) and explore the indications of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for children with AML-M5. Method Seventy-five AML-M5 patients and 201 non-AML-M5 AML patients were enrolled in this retrospective analysis. Event-free survival (EFS) and overall survival (OS) rates were estimated by Kaplan-Meier method and prognostic factors were evaluated by COX regression with SPSS.Result ①Twelve patients gave up treatment after confirmed diagnosis. Two patients died on the second day after chemotherapy. Of the 61 patients, 73. 8％ (45/61) achieved complete remission (CR) after two courses of chemotherapy. The 5-year EFS rate was 34. 5％ ±6. 8％. But of the 117 non-AML-M5/M3 AML patients,the 5-year EFS rate was 51.0％ ±4. 9％. ② Multivariate analysis showed that age ≥ 10 y, the proportion of bone marrow blast cell counts≥ 15％ after the first induction therapy, not CR after two courses of chemotherapy were risk factors for the long-term prognosis. ③ Of the 20 patients whose bone marrow blast cell counts≥ 15％ after the first induction therapy, 5 patients who choose allo-HSCT had a better OS than the other 15 patients who choose chemotherapy only ( 60. 0％ ± 21.9％ vs. 7. 3％ ± 7. 1％, P = 0. 024 ).Conclusion Children with AML-M5 had a poorer prognosis than the other AML patients; patients whose bone marrow blast cell counts ≥ 15％ after the first induction therapy chose allo-HSCT had a better prognosis. At present, there is no enough evidence to support that patients whose bone marrow blast cell counts ＜ 15％ after the first induction therapy should choose unrelated donor for allo-HSCT.

Analysis of clinical outcomes of 63 children with acute monocytic leukemia

Objective To evaluate the outcomes of childhood acute monocytic leukemia (AML-M5) and explore the indications of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for children with AML-M5. Method Seventy-five AML-M5 patients and 201 non-AML-M5 AML patients were enrolled in this retrospective analysis. Event-free survival (EFS) and overall survival (OS) rates were estimated by Kaplan-Meier method and prognostic factors were evaluated by COX regression with SPSS.Result ①Twelve patients gave up treatment after confirmed diagnosis. Two patients died on the second day after chemotherapy. Of the 61 patients, 73. 8％ (45/61) achieved complete remission (CR) after two courses of chemotherapy. The 5-year EFS rate was 34. 5％ ±6. 8％. But of the 117 non-AML-M5/M3 AML patients,the 5-year EFS rate was 51.0％ ±4. 9％. ② Multivariate analysis showed that age ≥ 10 y, the proportion of bone marrow blast cell counts≥ 15％ after the first induction therapy, not CR after two courses of chemotherapy were risk factors for the long-term prognosis. ③ Of the 20 patients whose bone marrow blast cell counts≥ 15％ after the first induction therapy, 5 patients who choose allo-HSCT had a better OS than the other 15 patients who choose chemotherapy only ( 60. 0％ ± 21.9％ vs. 7. 3％ ± 7. 1％, P = 0. 024 ).Conclusion Children with AML-M5 had a poorer prognosis than the other AML patients; patients whose bone marrow blast cell counts ≥ 15％ after the first induction therapy chose allo-HSCT had a better prognosis. At present, there is no enough evidence to support that patients whose bone marrow blast cell counts ＜ 15％ after the first induction therapy should choose unrelated donor for allo-HSCT.