Individualized chemotherapy, which is at the forefront of acute myeloid leukemia (AML) treatment, has moderately improved outcomes over the past decade. Monitoring the peripheral blood blast burden during induction by flow cytometry has shown significant value in the evaluation of treatment responses. Our previous study reported the day 5 peripheral blast clearance rate (D5-PBCR) as an indicator of early treatment response, and D5-PBCR (+) patients showed poor outcomes. We performed the present phase 2 trial of early intervention in D5-PBCR (+) patients with homoharringtonine (HHT) introduced in the traditional induction regimen with anthracycline and cytarabine. The primary endpoint was complete remission (CR). This study enrolled 151 patients, 65 patients were D5-PBCR (+) and 55 patients completed induction with HHT addition. The overall CR rate after one course of induction was 84.4%, with 87.5% and 80.0% for the D5-PBCR (−) and D5-PBCR (+) groups, respectively. The incidence of grade 3/4 adverse events was comparable between the two groups. At the median follow-up of 53.1 months, median overall survival (OS) was not reached in the entire cohort, and median event-free survival (EFS) was 42.2 months. Neither the OS nor EFS showed significant differences between the D5-PBCR (−) and D5-PBCR (+) groups. Compared to historical data, significant improvements in both OS (p = .020) and EFS (p = .020) were observed in the D5-PBCR (+) group. In conclusion, optimization of induction chemotherapy with idarubicin and cytarabine according to D5-PBCR is feasible in patients with newly diagnosed AML. The addition of HHT demonstrated a good efficacy and safety profile.

中文翻译：

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根据外周原始细胞清除率优化伊达比星和阿糖胞苷与高灵素对新诊断急性髓细胞白血病患者的诱导方案：单臂 2 期试验 (RJ-AML 2014)

处于急性髓性白血病 (AML) 治疗前沿的个体化化疗在过去十年中适度改善了结果。通过流式细胞术监测诱导期间的外周血母细胞负荷在评估治疗反应中显示出重要价值。我们之前的研究报告了第 5 天的外周原始细胞清除率 (D5-PBCR) 作为早期治疗反应的指标，而 D5-PBCR (+) 患者的结果不佳。我们在传统的蒽环类和阿糖胞苷诱导方案中对 D5-PBCR (+) 患者进行了早期干预的 2 期试验。主要终点是完全缓解（CR）。该研究招募了 151 名患者，其中 65 名患者为 D5-PBCR（+），55 名患者完成了添加 HHT 的诱导。一个疗程后的总体 CR 率为 84.4%，D5-PBCR (-) 和 D5-PBCR (+) 组分别为 87.5% 和 80.0%。两组之间 3/4 级不良事件的发生率具有可比性。在中位随访 53.1 个月时，整个队列未达到中位总生存期 (OS)，中位无事件生存期 (EFS) 为 42.2 个月。OS 和 EFS 均未显示 D5-PBCR (-) 和 D5-PBCR (+) 组之间的显着差异。与历史数据相比，两个操作系统都有显着改进（中位无事件生存期 (EFS) 为 42.2 个月。OS 和 EFS 均未显示 D5-PBCR (-) 和 D5-PBCR (+) 组之间的显着差异。与历史数据相比，两个操作系统都有显着改进（中位无事件生存期 (EFS) 为 42.2 个月。OS 和 EFS 均未显示 D5-PBCR (-) 和 D5-PBCR (+) 组之间的显着差异。与历史数据相比，两个操作系统都有显着改进（ 在 D5-PBCR (+) 组中观察到p  = .020) 和 EFS ( p = .020)。总之，根据 D5-PBCR 优化伊达比星和阿糖胞苷的诱导化疗在新诊断的 AML 患者中是可行的。HHT 的添加表现出良好的疗效和安全性。