Real-world outcomes of BCL2 inhibitor-based combination therapy in myelodysplastic syndrome and acute myeloid leukemia – Experience from a university hospital in northeastern Brazil

Abstract

Acute myeloid leukemia (AML) and high-risk myelodysplastic neoplasms (MDS) predominantly affect elderly patients who are often ineligible for intensive chemotherapy. The emergence of BCL2 inhibitors, particularly venetoclax, has changed the therapeutic landscape in these populations when combined with hypomethylating agents or low-dose cytarabine. This study aims to describe the clinical and epidemiological characteristics, treatment patterns, and outcomes of patients with AML and high-risk MDS treated with venetoclax-based regimens at a university hospital in northeastern Brazil. The methodology consists of a retrospective, descriptive study including patients aged ≥ 18 years diagnosed from January 2020 to October 2025. Twenty-six patients were included (19 AML, seven MDS), with a median age of 65 years (range: 37-81). Among AML cases, 42.1% were de novo and 57.9% were secondary, mostly post-MDS. Overall, 46.1% achieved complete remission, and 3.8% achieved partial remission. The median time to best response was one cycle. Three patients (11.5%) underwent allogeneic hematopoietic stem cell transplantation. Hematologic toxicity was universal, with febrile neutropenia in 78.3%. Venetoclax-based regimens demonstrated response rates comparable to international real-world data in AML and high-risk MDS, even with shorter venetoclax duration. Frailty remains the main barrier to transplantation, highlighting the importance of expanding access to targeted therapies within the public health system.