真实世界中FLT3-ITD(+)急性髓系白血病诱导治疗的疗效及预后因素分析

OBJECTIVE: To investigate the efficacy and prognostic factors of induction therapy in FLT3-ITD(+) acute myeloid leukemia (AML) in the real world data. METHODS: From January 2013 to December 2016, 114 de novo patients with FLT3-ITD(+)AML were enrolled in this study. Out of 114 cases, 75 were male, and 39 were female. The median age was 42 years old (ranged from 14 to 72 years old). The chemotherapy regimens were used for induction therapy and all cases were followed up. The treatment response was evaluated by MICM and the comparison of the ratio were analyzed by chi-square test and the survival was estimated by Kaplan-Meier analysis and Cox proportional hazards model was used to identify independent prognostic factors. RESULTS: There were 52 FLT3-ITD(+)AML patients with favorable prognosis genes (46 cases with NPM1, 5 cases with RUNX1-RUNX1T1, 1 case with CEBPA double mutation) and 62 patients with other types of FLT3-ITD(+)AML at diagnosis. All patients completed at least one cycle of induction therapy and the clinical curative effect was evaluated, complete remission (CR) rate was 50.0% (57/114) in one cycle and total CR rate was 72.5% (74/104) in two cycles. The CR rate of the FLT3-ITD(+) AML patients with favorable prognosis genes was 67.3% (35/52) in one cycle and 83.3% (40/48) in two cycles; for the other types FLT3-ITD(+)AML patients, the CR rate was 35.5% (22/62) in one cycle and 64.8% (35/54) in two cycles. There was a significant difference in CR rate between the FLT3-ITD(+)AML patients with and without favorable prognosis genes (P<0.05). This indicates that the FLT3-ITD(+)AML patients with favorable prognosis gene had relatively good therapeutic effect. Among other types of FLT3-ITD(+)AML patients who did not achieve remission from one cycle of chemotherapy, 9 patients were given sorafenib plus chemotherapy and 6 cases (66.7%) achieved CR; 23 patients were given conventional chemotherapy and 7 cases (30.4%) achieved CR. There was a significant difference between sorafenib plus chemotherapy and conventional chemotherapy groups (χ(2)=4.47, P<0.05) and this indicates that sorafenib plus chemotherapy can significantly improve the CR rate of FLT3-ITD(+)AML patients. Comparing overall survival (OS) and disease free survival (DFS), there was no significant difference between sorafenib plus chemotherapy and conventional chemotherapy groups (P values were 0.641 and 0.517, respectively). CONCLUSION: The overall prognosis of FLT3-ITD(+)AML patients is poor, and the stratification therapeutic efficacy of FLT3-ITD(+)AML without favorable prognosis gene can be improved by sorafenib combined with chemotherapy.