Objective: To investigate the clinical significance and prognosis factors of acute myeloid leukemia (AML) with FLT3 mutation-internal tandem duplication (FLT3-ITD) and provide more evidence for prognosis evaluation in AML with FLT3-ITD. Methods: The clinical characteristics and outcomes of 98 AML with FLT3-ITD were analyzed. Results: In patients with FLT3-ITD positive AML, the median of WBC and peripheral blood blast at initial diagnosis were 58.2 (0.3-461.8) ×109/L and 42.2 (0-397.1) ×109/L, respectively. The complete remission (CR) rate after one course induction therapy was 60.6% in 71 patients with intermediate or adverse risk. The primary refractory rate was 26.8% in 71 patients with intermediate or adverse risk. The lower number of peripheral blood blast was a favorable factor to achieve CR after one course induction therapy (P=0.009). CR after one course (HR=0.395, 95% CI 0.183-0.85, P=0.001) and allo-transplantation in CR1 (HR=0.180, 95% CI 0.043-0.752, P=0.018) favored longer relapse-free survival (RFS). CR after one course (HR=0.251, 95% CI 0.121-0.523, P< 0.001) and lower number of peripheral blood blast (HR=0.219, 95% CI 0.088-0.545, P=0.003) favored longer overall survival (OS) in AML with FLT3-ITD. Conclusion: FLT3-ITD positive AML with lower number of peripheral blood blast has a relative favorable prognosis. CR after one course and allotransplantation in CR1 are important strategy to improve prognosis in AML with FLT3-ITD.
目的: 探讨FLT3-ITD突变阳性急性髓系白血病（AML）的临床特征和预后因素，为FLT3-ITD突变阳性AML患者的预后评估提供更多证据。
方法: 收集98例FLT3-ITD突变阳性AML初诊患者的临床资料，对其临床特征与预后的相关性进行统计学分析。
结果: 98例FLT3-ITD突变阳性AML患者初诊时中位WBC 58.2（0.3~461.8）×109/L，外周血原始细胞绝对计数中位数42.2(0～397.1）×109/L。71例细胞遗传学中高危组患者1个疗程完全缓解（CR）率为60.6%，原发耐药率为26.8%。单因素分析结果显示外周血原始细胞绝对计数低是影响患者1个疗程CR的良好预后因素（P=0.009）。多因素分析结果显示，1个疗程达CR（HR=0.395，95%CI 0.183~0.850，P=0.001）及第1次CR期行异基因造血干细胞移植（HR=0.180，95%CI 0.043~0.752，P=0.018）是影响患者无复发生存的良好预后因素，1个疗程达CR（HR=0.251，95%CI 0.121~0.523，P<0.001）及外周血原始细胞绝对计数低（HR=0.219, 95%CI 0.088~0.545，P=0.003）是影响患者总生存的良好预后因素。
结论: 外周血原始细胞计数低的FLT3-ITD突变阳性AML患者预后相对良好，1个疗程达CR和第1次CR期行异基因造血干细胞移植是改善FLT3-ITD突变阳性AML患者预后的重要治疗策略。