Title:

Efficacy and Safety of Venetoclax Plus Azacitidine in Acute Myeloid Leukemia Patients Ineligible for Intensive Chemotherapy

Authors:

A. Farheen, F. Nadeem, and Srijamya

Introduction:

Acute myeloid leukemia (AML) is characterized by clonal proliferation of immature myeloid cells and is associated with poor outcomes in elderly and medically unfit patients. Venetoclax, a selective B-cell lymphoma-2 (BCL-2) inhibitor, in combination with azacitidine, has emerged as an effective low-intensity therapeutic strategy, improving remission and survival outcomes in newly diagnosed AML patients unsuitable for intensive chemotherapy.

Aim:

To evaluate the efficacy and safety of venetoclax plus azacitidine in AML patients ineligible for intensive chemotherapy.

Methods:

A review of phase 1b–3 clinical trials, pooled subgroup analyses, and observational studies evaluating venetoclax plus azacitidine in AML patients ineligible for intensive chemotherapy was performed. Outcomes assessed included complete remission/complete remission with incomplete hematologic recovery (CR/CRi), overall survival (OS), measurable residual disease (MRD) negativity, and treatment-related adverse events.

Results:

Venetoclax plus azacitidine demonstrated significant clinical activity in newly diagnosed AML patients unfit for intensive chemotherapy. Median overall survival was 14.7–16.4 months, with a 24-month overall survival of 37.5%. Composite CR/CRi rates were 66–74%, while MRD negativity among responders reached up to 80%. Clinical benefit was maintained across elderly, frail, adverse-risk, and molecular subgroups, including IDH1/2 mutations. The most common grade ≥3 adverse events included neutropenia (20–89%), thrombocytopenia (23–82%), febrile neutropenia (39–65%), and infections.

Conclusion:

Venetoclax plus azacitidine provides durable remissions, meaningful survival benefit, and high response rates in AML patients unsuitable for intensive chemotherapy. Despite frequent hematologic toxicities, the regimen maintains a manageable and predictable safety profile.

Acknowledgements:

Medications team mentored and guided in writing the manuscript and submission of the abstract at the conference.

Contact:

ashfarheen.125@gmail.com