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  

Analysis of induction efficacy and prognostic factors in FLT3-ITD positive acute myeloid leukemia in the real world

作　　者：贾晋松[1] 主鸿鹄[1] 宫立众 赵婷[1] 王婧[1] 江倩[1] 黄晓军[1] 江浩[1] Jia Jinsong;Zhu Honghu;Gong Lizhong;Zhao Ting;Wang Jing;Jiang Qian;Huang Xiaojun;Jiang Hao(Peking University People’s Hospital,Peking University Institute of Hematology,Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation,Beijing 100044,China)

机构地区：[1]北京大学人民医院、北京大学血液病研究所、造血干细胞移植治疗血液病北京市重点实验室,100044

出　　处：《中华血液学杂志》2019年第5期398-403,共6页Chinese Journal of Hematology

摘　　要：目的探讨真实世界FLT3-ITD突变阳性急性髓系白血病(FLT3-ITD^+AML)诱导治疗的疗效及预后影响因素.方法收集2013年1月至2016年12月收治的初发FLT3-ITD^+AML患者114例,了解患者初诊时生物学特征,采用化疗方案进行诱导治疗,随访病例,并对其缓解率、预后等进行分析.结果114例FLT3-ITD^+AML患者初诊时分子学检测发现伴随预后良好基因突变52例(NPM1突变46例,RUNX1-RUNX1T1 5例,CEBPA双突变l例);其他类型FLT3-ITD^+AML 62例.所有患者均至少完成1个疗程的诱导治疗并进行了疗效评估,1个疗程完全缓解(CR)率为50.0%(57/114),2个疗程CR率为72.5%(74/104).52例伴随预后良好基因突变的FLT3-ITD^+AML患者1个疗程CR率为67.3%(35/52),2个疗程CR率为83.3%(40/48);62例其他类型FLT3-ITD^+AML患者1个疗程CR率为35.5%(22/62),2个疗程CR率为64.8%(35/54);两组CR率差异有统计学意义(P<0.05),显示FLT3-ITD伴随预后良好基因突变的AML患者治疗效果相对较好.对于其他类型FLT3-ITD^+AML 1个疗程未缓解的患者,随后采用索拉非尼联合化疗的9例患者中6例达到CR,缓解率为66.7%;采用常规化疗方案的23例患者中7例达到CR,缓解率为30.4%,两者之间差异有统计学意义(x^2=4.47,P<0.05),显示索拉非尼联合化疗方案可以显著提高FLT3-ITD^+AML不缓解患者的CR率.索拉非尼联合化疗与常规方案化疗患者总生存和无病生存比较,差异均无统计学意义(P值分别为0.641和0.517).结论FLT3-ITD^+AML患者整体预后不良,通过索拉非尼联合化疗的方案对不伴随预后良好基因突变的FLT3-ITD^+AML进行分层治疗可以提高疗效.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(x^2=4.47,P<0.05)and this indicates th

关 键 词：FLT3-ITD突变 白血病 髓样 急性 索拉非尼 治疗结果 

分 类 号：R733.71[医药卫生—肿瘤]