Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML
Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML
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DOI：
10.1056/nejmoa1902688
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发表时间：
2019-10-31
影响因子：
158.5
通讯作者：
Levis, M. J.
中科院分区：
文献类型：
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作者：
Perl, A. E.;Martinelli, G.;Levis, M. J.
具有复发或难治性的急性髓样白血病（AML）的背景患者在FMS样酪氨酸激酶3基因（FLT3）中不经常反应挽救化学疗法。吉尔特替尼是一种口服，有效的选择性FLT3抑制剂，具有单代药活性，在复发或难治性的FLT3-突变的AML.Methodsin A期试验中，我们随机分配了具有复发或难治性的FLT3-MUTT3-MUTT3-MUTTAING AML，以接收2：1吉尔特替尼（每天120毫克）或打捞化疗。这两个主要终点是总生存期和完全或部分血液学恢复完全缓解的患者百分比。次要终点包括无事件生存期（免受治疗失败的自由[即复发或缺乏缓解]或死亡）以及完全缓解的患者的百分比。371名合格患者，247例被随机分配给吉尔特里替尼组和124到打捞化疗组。 Gilteritinib组的中位总生存期明显长于化学疗法组（9.3个月比5.6个月；死亡危害比率为0.64； 95％置信区间[CI]，0.49至0.83； p 0.49; p；
BACKGROUNDPatients with relapsed or refractory acute myeloid leukemia (AML) with mutations in the FMS-like tyrosine kinase 3 gene (FLT3) infrequently have a response to salvage chemotherapy. Gilteritinib is an oral, potent, selective FLT3 inhibitor with single-agent activity in relapsed or refractory FLT3-mutated AML.METHODSIn a phase 3 trial, we randomly assigned adults with relapsed or refractory FLT3-mutated AML in a 2:1 ratio to receive either gilteritinib (at a dose of 120 mg per day) or salvage chemotherapy. The two primary end points were overall survival and the percentage of patients who had complete remission with full or partial hematologic recovery. Secondary end points included event-free survival (freedom from treatment failure [i.e., relapse or lack of remission] or death) and the percentage of patients who had complete remission.RESULTSOf 371 eligible patients, 247 were randomly assigned to the gilteritinib group and 124 to the salvage chemotherapy group. The median overall survival in the gilteritinib group was significantly longer than that in the chemotherapy group (9.3 months vs. 5.6 months; hazard ratio for death, 0.64; 95% confidence interval [CI], 0.49 to 0.83; P