本研究旨在比较在核心结合因子急性髓系白血病（CBF-AML）患者中，赫马哈林汀联合阿糖胞苷和阿格来替宁（HAA）方案与伊达鲁替宁联合阿糖胞苷（IA）方案作为首次诱导化疗的疗效。使用Cox回归模型和倾向性分数匹配（PSM）来确定与更好的缓解率和预后相关的方案。总共374例CBF-AML患者（243例RUNX1::RUXN1T1和131例CBFB::MYH11）被纳入本研究。患者首次诱导治疗接受HAA或IA方案（每组187例）。对于RUNX1::RUXN1T1携带者，多变量分析显示HAA方案与首次诱导后的较高完全缓解/缓解不归类（hazard ratio [HR] = 5.3 [95% CI 2.3, 12.2]; p < 0.001）和较好的无复发生存期（RFS）（HR = 0.5 [0.3, 0.8], p = 0.01）显著相关。PSM分析中，HAA方案在CR/CRi率上也较高（96% vs. 77%，p < 0.001），特别是对于携带野生型KIT（KITWT）（96% vs. 83%，p = 0.02）或非D816 KIT突变的患者（100% vs. 63%，p = 0.002），以及较好的RFS（p = 0.01），与IA方案相比。然而，对于CBFB::MYH11携带者，两种方案在缓解率和预后方面无差异。赫马哈林汀联合阿糖胞苷和阿格来替宁方案作为首次诱导化疗在RUNX1::RUNX1T1 AML患者中，特别是对于携带KITWT和非D816 KIT突变的患者，以及较好的RFS方面与IA方案相比表现出较高的CR/CRi率。两种方案在CBFB::MYH11携带者中的疗效无差异。© 2023. 作者，独家授权给Springer-Verlag GmbH Germany，属于Springer Nature。

To compare efficacy between homoharringtonine combined with cytarabine and aclarubicin (HAA) and idarubicin and cytarabine (IA) regimens as first induction chemotherapy in patients with core binding factor acute myeloid leukemia (CBF-AML). Cox regression model and propensity score matching (PSM) were used to identify the regimen associated with a better remission rate and outcomes. In total, 374 patients with CBF-AML (243 with RUNX1::RUXN1T1 and 131 with CBFB::MYH11) were included in this study. The patients received the HAA or IA regimen (187 each) as the first induction therapy. For patients with RUNX1::RUXN1T1, multivariate analyses showed that the HAA regimen was significantly associated with a higher CR/CRi rate after the first induction (hazard ratio [HR] = 5.3 [95% CI 2.3, 12.2]; p < 0.001) and more favorable relapse-free survival (RFS) (HR = 0.5 [0.3, 0.8], p = 0.01). In PSM analysis, the HAA regimen also had a higher CR/CRi rate (96% vs. 77%, p < 0.001), especially for those harboring wild-type KIT (KITWT) (96% vs. 83%, p = 0.02) or non-D816 KIT mutation (100% vs. 63%, p = 0.002), as well as more favorable RFS (p = 0.01), compared with the IA regimen. However, there was no difference in the remission rate or outcomes between the two regimens for patients with CBFB::MYH11. The HAA regimen as first induction chemotherapy resulted in a higher CR/CRi rate in AML patients with RUNX1::RUNX1T1, especially those harboring KITWT and non-D816 KIT mutation, and a more favorable RFS compared with the IA regimen. The efficacy between the two regimens did not differ in those with CBFB::MYH11.© 2023. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.