USE OF AZACITIDINE AND VENETOCLAX (AZA+VEN) IN HIGH-RISK MYELODYSPLASTIC SYNDROME AND ACUTE MYELOID LEUKEMIA FOLLOWING MYELODYSPLASTIC SYNDROME: EX-PERIENCE OF THE NATIONAL RESEARCH ONCOLOGY CENTER

A. Umargaliyeva, Vadim Kemaykin, R. Vildanov, Azat Baglanovich Karabekov, Zhandos K. Burkitbayev, K. Mukhambetiyar, Alexandr Kolesnev

2026

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DOI 10.52532/3135-3940-2026-3-724
Data confidence 70 medium
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Abstract

Relevance: Myelodysplastic syndrome (MDS) represents a heterogeneous group of clonal disorders characterized by cytopenia, bone marrow cell dysplasia, and a risk of transformation into acute myeloid leukemia (AML). The combination of azacitidine and venetoclax (AZA+VEN) improves disease control, while allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains a potentially effective treatment option for patients with high-risk MDS. Clinical experience with AZA+VEN in Kazakhstan remains limited.Objective: To evaluate the efficacy and safety of AZA+VEN in patients with high-risk MDS and AML transformed from MDS, based on the clinical experience of the National Research Oncology Center (Astana, Kazakhstan).Materials and Methods: This retrospective, descriptive, single-center study included 17 patients with high-risk MDS and AML transformed from MDS. AZA+VEN was administered as first-line therapy in 3 (17.6%) patients and as second-line therapy in 14 (82.4%). Before starting AZA+VEN, 9 patients receiving second-line therapy had received azacitidine, and 5 had received decitabine. We assessed treatment response after 2, 4, and 6 cycles based on bone marrow and peripheral blood parameters. Overall survival (OS) and progression-free survival (PFS) were estimated using the Kaplan-Meier method.Results: Bone marrow remission was achieved in 9 (52.9%) patients; 4 (23.5%) underwent allo-HSCT after achieving remission. Median OS was 8 months, and median PFS was 3 months. A total of 13 (76.5%) deaths were recorded: 5 due to disease progression or refractory disease, 5 due to infectious complications, and 3 due to other causes. Febrile neutropenia was observed in 7 (41.2%) patients. No permanent treatment discontinuations due to toxicity were recorded.Conclusion: In the study cohort, AZA+VEN therapy was associated with bone marrow remission in more than half of the patients. Allo-HSCT performed in a subset of patients after achieving remission is of clinical interest in terms of the potential use of AZA+VEN as a therapeutic step before transplantation. The small sample size, lack of a comparison group, and cohort heterogeneity require cautious interpretation of the findings and further investigation of this approach in larger studies.