Venetoclax, azacitidine, and G-CSF targeting molecular residual disease in CBFB::MYH11 AML resistant to high-dose cytarabine

Acute myeloid leukemia (AML) with the CBFB::MYH11 rearrangement is generally considered a favorable-risk subtype highly responsive to intensive cytarabine-based chemotherapy [ 1 ]. However, up to 30% of patients ultimately relapse, driven largely by persistent measurable residual disease (MRD) [ 1 ]. Patients failing to achieve deep molecular responses during early consolidation therapy, typically defined as a >3-log reduction in transcript levels or an absolute threshold below 0.1%, face a significantly elevated risk of relapse [ 2 , 3 ]. While allogeneic hematopoietic stem cell transplantation (allo-HSCT) can effectively overcome this adverse prognosis [ 4 ], its widespread application is frequently precluded by patient comorbidities and donor availability. This underscores an urgent clinical need for effective non-transplant regimens capable of eradicating MRD.