Venetoclax and decitabine vs intensive chemotherapy as induction for young patients with newly diagnosed AML

作者：Jing Lü, Shengli Xue, Ying Wang, Xuefeng He, Xiaohui Hu, Miao Miao, Yang Zhang, Zaixiang Tang, Jun-dan Xie, Xiaofei Yang, Mingzhu Xu, Yaoyao Shen, Feng Du, Qian Wu, Mengxing Xue, Yun Wang, Ailing Deng, Xueqing Dou, Yang Xu, Haiping Dai, Depei Wu, Suning Chen · 发表于：Blood · 年份：2025 · DOI：10.1182/blood.2024027217 · 被引用次数：47 · 研究领域：Acute Myeloid Leukemia Research、Histone Deacetylase Inhibitors Research、Multiple Myeloma Research and Treatments

ABSTRACT: Venetoclax (VEN) combined with hypomethylating agents is approved for frontline therapy in older/unfit patients with acute myeloid leukemia (AML). However, prospective data on this low-intensity therapy in treatment-naive younger patients with AML are lacking. This study investigated the efficacy and safety of VEN plus decitabine (VEN-DEC) as induction in untreated young fit patients with AML in a randomized trial. Patients aged 18 to 59 years eligible for intensive chemotherapy were randomized 1:1 to receive VEN-DEC or IA-12 (idarubicin and cytarabine). All patients achieved composite complete remission (CRc) underwent high-dose cytarabine consolidation. The primary end point was CRc rate after induction. Of 255 screened, 188 were enrolled and randomly assigned, with 94 in each group. In the intention-to-treat population, CRc was 89% (84/94) in the VEN-DEC group vs 79% (74/94) in the IA-12 group (noninferiority P = .0021), with measurable residual disease negativity rates of 80% (67/84) vs 76% (56/74), respectively. VEN-DEC showed superior CRc in patients aged ≥40 years (91% vs 75%) and those with adverse risk (91% vs 42%) or epigenetic mutations (91% vs 67%), but lower CRc in RUNX1::RUNX1T1 fusion cases (44% vs 88%) than IA-12. Patients in the VEN-DEC group experienced fewer grade ≥3 infections (32% vs 67%) and shorter severe thrombocytopenia duration (median, 13 vs 19 days; P < .001). At a median follow-up of 12.1 months, overall and progression-free survival wer...