A phase 3 study of intensive chemotherapy with or without dasatinib in core-binding factor acute myeloid leukemia.

We report results from the randomized, open-label, phase 3 trial of intensive chemotherapy with or without the multikinase inhibitor dasatinib in adult patients with CBF-AML.

我们报告了一项随机、开放标签、第三阶段临床试验的结果，该试验评估了在成年CBF-AML患者中，使用或不使用多激酶抑制剂达沙替尼的强化化疗方案。

Patients received "3+7" induction therapy, followed by 4 cycles of high-dose cytarabine; in the investigational arm, patients received dasatinib 100 mg daily on days 8 to 21 in induction, and on days 6 to 28 in consolidation cycles, followed by 12-month single-agent dasatinib 100 mg daily.

患者接受了'3+7'诱导治疗，随后接受了4个周期的高剂量阿糖胞苷治疗；在研究组中，患者在诱导治疗的第8至21天以及巩固治疗周期的第6至28天每天口服100毫克的达沙替尼，之后继续接受为期12个月的每日100毫克单药达沙替尼治疗。

Primary end point was event-free survival (EFS).

主要终点是无事件生存（EFS）。

Secondary end points included overall survival, relapse-free survival, and cumulative incidenceof relapse.

次要终点包括总生存、无复发生存和复发生存的累积发生率。

A total of 202 patients were randomly assigned to the standard arm (n = 102) and to the dasatinib arm (n = 100).

共有202名患者被随机分配到标准治疗组（n = 102）和达沙替尼组（n = 100）。

Median age was 49 years (range, 18-77); 94 patients had t(8;21), 108 had inv(16)/t(16;16); and 58 (28.7%) patients had a KIT comutation.

中位年龄为49岁（范围，18-77岁）；94名患者存在t(8;21)易位，108名患者存在inv(16)/t(16;16)易位；58名（28.7%）患者存在KIT共突变。

There was no statistically significant difference in EFS (hazard ratio, 0.92; 95% confidence interval, 0.63-1.33; P = .66) or secondary end points between treatment arms.

在治疗组之间，无事件生存（EFS）（风险比，0.92；95%置信区间，0.63-1.33；P = .66）或次要终点之间没有统计学显著性差异。

There was also no significant difference in EFS in subgroup analyses according to age, CBF-AML type, and KIT mutation status.

在根据年龄、CBF-AML类型和KIT突变状态进行的亚组分析中，无进展生存期（EFS）也没有显著差异。

The incidenceof serious adverse events was higher in the investigational arm (64%) than in the standard arm (36%).

研究组中严重不良事件的发生率（64%）高于标准组（36%）。

In patients with CBF-AML, the addition of dasatinib to intensive chemotherapy failed to improve survival outcomes.

在携带CBF-AML的患者中，添加达沙替尼到强化化疗未能改善生存结果。

The addition of dasatinib was associated with an increase in toxicity.

添加达沙替尼与毒性增加相关。

This trial was registered at www.ClinicalTrials.gov as NCT02013648.

该试验已在www.ClinicalTrials.gov注册，注册号为NCT02013648。