Abstract

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1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(χ2=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。

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