Zhang Yu, Feng Zinan, Du Jing, Liu Hui, Yu Sijian, Liang Xinquan, Zhao Weihua, Zhang Qing, Zhang Xiong, Nie Danian, Sun Zhiqiang, Du Xin, Xu Xiaojun, Yu Guopan, Shi Pengcheng, Liu Qianwei, Shao Ruoyang, Qu Hong, Xiong Wenjie, Wang Shunqing, Jiang Yirong, Zhang Hongyu, Guo Ziwen, Dai Min, Jiang Xuejie, Xu Dan, Huang Fen, Fan Zhiping, Xu Na, Liu Can, Wu Meiqing, Lin Ren, Jin Hua, Sun Jing, Liu Qifa, Xuan Li
Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China.
Clinical Medical Research Center of Hematology Diseases of Guangdong Province, Guangzhou, China.
Leukemia. 2025 May 28. doi: 10.1038/s41375-025-02655-x.
Whether adding anthracycline to intermediate- or high-dose cytarabine as consolidation is beneficial remains unclear in acute myeloid leukemia (AML). Eligible AML patients in first complete remission were randomly assigned (1:1) to receive either high-dose cytarabine with idarubicin (IA3 + 3) (idarubicin 10 mg/m, d1-3 and cytarabine 2 g/m, every 12 h, d1-3) or high-dose cytarabine (HDAC) (cytarabine 3 g/m, every 12 h, d1-3) regimens as first consolidation. The primary endpoint was the rate of negative measurable residual disease (MRD) after first consolidation. Between November 2018 and December 2021, 407 patients were assigned to IA3 + 3 (n = 204) or HDAC (n = 203) groups. MRD after first consolidation for IA3 + 3 and HDAC groups was 65.2% (95%CI: 58.6-71.8%) and 53.2% (46.3-60.1%) (P = 0.009). The 3-year cumulative incidence of relapse was 22.6% (95%CI :16.8-29.0%) and 34.0% (27.1-41.1%) (P = 0.014), DFS was 68.4% (61.5-75.3%) and 52.9% (45.4-60.5%) (P = 0.003), OS was 75.5% (69.0-82.1%) and 69.6% (62.4-76.7%) (P = 0.18) and treatment-related mortality was 8.8% (5.2-13.6%) and 13.0% (8.5-18.5%) (P = 0.23) in two groups, respectively. Eighty-seven (43%) and 114 (56%) patients underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT), respectively (P = 0.006). IA3 + 3 regimen results in deeper remissions and reduces relapse compared to HDAC. This deeper remission improves DFS and translates into treatment advantage, with fewer patients undergoing allo-HSCT. (ClinicalTrials.gov, NCT03620955).
在急性髓系白血病（AML）中，作为巩固治疗，在中剂量或高剂量阿糖胞苷中添加蒽环类药物是否有益仍不清楚。首次完全缓解的符合条件的AML患者被随机分配（1:1）接受高剂量阿糖胞苷联合伊达比星（IA3 + 3）（伊达比星10mg/m²，第1 - 3天；阿糖胞苷2g/m²，每12小时一次，第1 - 3天）或高剂量阿糖胞苷（HDAC）（阿糖胞苷3g/m²，每12小时一次，第1 - 3天）方案作为首次巩固治疗。主要终点是首次巩固治疗后可测量残留病（MRD）阴性率。2018年11月至2021年12月期间，407例患者被分配到IA3 + 3组（n = 204）或HDAC组（n = 203）。IA3 + 3组和HDAC组首次巩固治疗后的MRD分别为65.2%（95%CI：58.6 - 71.8%）和53.2%（46.3 - 60.1%）（P = 0.009）。3年累积复发率分别为22.6%（95%CI：16.8 - 29.0%）和34.0%（27.1 - 41.1%）（P = 0.014），无病生存期（DFS）分别为68.4%（61.5 - 75.3%）和52.9%（45.4 - 60.5%）（P = 0.003），总生存期（OS）分别为75.5%（69.0 - 82.1%）和69.6%（62.4 - 76.7%）（P = 0.18），两组治疗相关死亡率分别为8.8%（5.2 - 13.6%）和13.0%（8.5 - 18.5%）（P = 0.23）。分别有87例（43%）和114例（56%）患者接受了异基因造血干细胞移植（allo - HSCT）（P = 0.006）。与HDAC相比，IA3 + 3方案能实现更深的缓解并降低复发率。这种更深的缓解改善了DFS并转化为治疗优势，接受allo - HSCT的患者更少。（ClinicalTrials.gov，NCT03620955）
