Azacitidine and Venetoclax Treatment in Acute Myeloid Leukemia: Real-Life Experience

Objective: This study evaluated the real-world efficacy, safety, and survival outcomes of azacitidine-venetoclax (AZA-VEN) therapy in newly diagnosed acute myeloid leukemia (AML) patients ineligible for intensive chemotherapy.
Materials and Methods: A retrospective analysis was conducted on 36 AML patients treated at two centers between 2022–2025. Data included demographics, treatment cycles, dose adjustments, response rates (complete remission, partial remission, refractory disease), hematologic recovery, side effects, and overall survival.
Results: The median age was 66 years (range: 27–98), with 61.1% achieving CR. Median OS (Overall Survival) was 22 months (95% CI: 13.1–30.9), significantly longer in patients receiving ≥3 cycles (23.7 vs. 6.4 months; p=0.031) and those with early neutrophil recovery (>1000/μL by day 7: 29.4 vs. 13.7 months; p=0.009). Grade 3 toxicity (33.3% of patients) correlated with poorer survival (6.4 vs. 22 months for Grade 2; p=0.006). No invasive fungal infections occurred.
Conclusion: AZA–VEN is effective and safe in chemotherapy-ineligible AML, providing survival outcomes that are comparable to, and in some aspects even consistent with, those observed in pivotal clinical trials. Our findings indicate that extended therapy, especially in patients who were able to continue for three or more cycles, was associated with a meaningful improvement in overall survival, thereby emphasizing the importance of maintaining treatment continuity whenever possible. The complete absence of invasive fungal infections in our cohort, in contrast to the variable rates reported in previous studies, suggests that our prophylaxis strategy may have contributed positively and could be of clinical relevance. In addition, the female survival advantage observed in our study, while noteworthy, should be interpreted with caution, considered as hypothesis-generating, and further validated in larger, well-designed studies.