Safety and efficacy of venetoclax-azacitidine combined with low-dose Idarubicin and cytarabine regimens for the treatment of newly diagnosed acute myeloid leukemia patients compared to the standard chemotherapy: a propensity score-matched real-world

Despite advances in treatment, long -term survival in patients with acute myeloid leukemia (AML) remains unsatisfactory. Standard induction chemotherapy achieves complete remission (CR) rates of 60%-80% in newly diagnosed AML patients; however, relapse and chemoresistance persist as major challenges. Combined multi-target therapeutic regimens have the potential to enhance response rates and improve survival outcomes. In this propensity score matching (PSM), single-center retrospective study, we evaluated the efficacy and safety of a novel regimen-venetoclax-azacitidine combined with low-dose idarubicin and cytarabine (VAIA)།versus standard idarubicin-cytarabine (IA) therapy in newly diagnosed AML patients. Twenty-six patients treated with VAIA were compared with 52 patients treated with IA, with well-matched clinical and molecular baseline characteristics. The VAIA cohort demonstrated significantly higher CR and measurable residual disease (MRD)-negative CR rates compared to the IA cohort (84.3% vs. 61.5%, P = 0.037; and 80.8% vs. 53.8%, P = 0.026, respectively). Moreover, VAIA was associated with improved overall survival (1-year OS: 76.9% vs. 57.7%, P = 0.031) and leukemia-free survival (1-year LFS: 65.4% vs. 46.2%, P = 0.042). Subgroup analysis revealed that adverse-risk patients particularly benefited from VAIA (1-year OS: 69.2% vs. 32%, P = 0.045; 1-year LFS: 61.5% vs. 20%, P = 0.046). In both treatment groups, MRD-negative patients exhibited significantly longer survival than MRD-positive patients. The VAIA regimen also showed superior therapeutic effects in patients particularly in patients with adverse or complex cytogenetics and adverse risk, with an acceptable and manageable toxicity profile. These findings support further prospective evaluation of the VAIA regimen in AML.