Jia J S, Zhu H H, Gong L Z, Zhao T, Wang J, Jiang Q, Huang X J, Jiang H
Peking University People's Hospital, Peking University Institute of Hematology, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing 100044, China.
Zhonghua Xue Ye Xue Za Zhi. 2019 May 14;40(5):398-403. doi: 10.3760/cma.j.issn.0253-2727.2019.05.010.
To investigate the efficacy and prognostic factors of induction therapy in FLT3-ITD(+) acute myeloid leukemia (AML) in the real world data. 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. 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 (<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 ((2)=4.47, <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 ( values were 0.641 and 0.517, respectively) . 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.
在真实世界数据中研究FLT3-ITD(+)急性髓系白血病（AML）诱导治疗的疗效及预后因素。2013年1月至2016年12月，114例初诊FLT3-ITD(+)AML患者纳入本研究。114例患者中，男性75例，女性39例。中位年龄42岁（范围14至72岁）。采用化疗方案进行诱导治疗并对所有病例进行随访。通过MICM评估治疗反应，率的比较采用卡方检验分析，生存情况采用Kaplan-Meier分析进行估计，Cox比例风险模型用于识别独立预后因素。诊断时，52例FLT3-ITD(+)AML患者具有预后良好基因（46例NPM1、5例RUNX1-RUNX1T1、1例CEBPA双突变），62例为其他类型的FLT3-ITD(+)AML。所有患者均完成至少1个周期的诱导治疗并评估临床疗效，1个周期的完全缓解（CR）率为50.0%（57/114），2个周期的总CR率为72.5%（74/104）。具有预后良好基因的FLT3-ITD(+)AML患者1个周期的CR率为67.3%（35/52），2个周期为83.3%（40/48）；其他类型FLT3-ITD(+)AML患者1个周期的CR率为35.5%（22/62），2个周期为64.8%（35/54）。具有和不具有预后良好基因的FLT3-ITD(+)AML患者的CR率存在显著差异（<0.05）。这表明具有预后良好基因的FLT3-ITD(+)AML患者治疗效果相对较好。在1个周期化疗未缓解的其他类型FLT3-ITD(+)AML患者中，9例给予索拉非尼联合化疗，6例（66.7%）达到CR；23例给予传统化疗，7例（30.4%）达到CR。索拉非尼联合化疗组与传统化疗组之间存在显著差异（(2)=4.47，<0.05），这表明索拉非尼联合化疗可显著提高FLT3-ITD(+)AML患者的CR率。比较总生存（OS）和无病生存（DFS），索拉非尼联合化疗组与传统化疗组之间无显著差异（值分别为0.641和0.517）。FLT3-ITD(+)AML患者总体预后较差，索拉非尼联合化疗可改善无预后良好基因的FLT3-ITD(+)AML的分层治疗疗效。
