目的:对达沙替尼作为第二代酪氨酸激酶抑制剂靶向抑制存在C-KIT突变及KIT蛋白高表达的核心结合因子阳性的急性髓系白血病(AML)患儿的临床有效性和安全性进行评估。方法:选取我中心7例核心结合因子阳性的AML患儿,完善其发病时白细胞总数、骨髓形态、流式免疫分型、染色体核型分析、融合基因定量及突变基因筛查,给予达沙替尼单药治疗或者联合化疗治疗,根据血药物浓度,调整达沙替尼用量,以15天为1个周期评估患儿骨髓形态、流式免疫分型、融合基因定量拷贝数的变化情况。结果:4例患儿经达沙替尼治疗后融合基因降低至低拷贝数,其中2例C-KIT突变的患儿接受治疗后融合基因均降至低拷贝数。1例巩固治疗5个疗程后融合基因高拷贝数的患儿单用达沙替尼,15天融合基因降至低拷贝数。结论:达沙替尼可以增加化疗对核心结合因子阳性的AML的疗效,尤其是存在C-KIT突变及KIT蛋白过表达的患儿。 OBJECTIVE: To evaluate the clinical efficacy of dasatinib as a second-generation tyrosine kinase inhibitor in the treatment of children with core-associated acute myeloid leukemia (AML) with C-KIT mutations and high expression of KIT and Safety assessment. Methods: Seven children with AML positive core binding factor in our center were selected to improve the total number of leucocytes, bone marrow morphology, flow immunophenotyping, chromosome karyotype analysis, quantitative fusion gene and mutation gene screening, Monotherapy or chemotherapy combined with chemotherapy. According to the concentration of blood drug, the dosage of dasatinib was adjusted. The changes of bone marrow morphology, flow immunophenotyping and quantitative copy number of fusion gene in 15 days were evaluated. RESULTS: In 4 children, the fusion gene was reduced to low copy number after treatment with dasatinib. Two of the two children with C-KIT mutation had their fusion gene reduced to low copy number after treatment. One case of consolidation therapy for 5 courses of fusion gene high copy number of children with dasatinib alone, 15 days fusion gene down to low copy number. Conclusion: Dasatinib can increase the efficacy of chemotherapy on coreline-positive AML, especially in children with C-KIT mutations and KIT protein overexpression.