homoharringtonine + azacytidine + venetoclax

The combination of homoharringtonine, azacytidine, and venetoclax (often referred to as the VAH regimen) is an investigational chemotherapy protocol for acute myeloid leukemia (AML), particularly in relapsed/refractory (R/R) or high-risk patients. - **Homoharringtonine** is a plant-derived cytotoxic alkaloid that inhibits protein synthesis by binding to the ribosomal A-site, leading to apoptosis in leukemic cells. - **Azacytidine** is a hypomethylating agent that incorporates into DNA and RNA, inhibiting DNA methyltransferase and resulting in hypomethylation of DNA; this reactivates tumor suppressor genes and induces cell differentiation or apoptosis. - **Venetoclax** is a selective B-cell lymphoma 2 (BCL-2) inhibitor that promotes apoptosis by blocking the anti-apoptotic function of BCL-2. This triple-drug regimen has demonstrated improved rates of complete remission, measurable residual disease negativity, event-free survival, and overall survival compared with dual regimens such as venetoclax plus azacytidine alone. The addition of homoharringtonine appears to mitigate negative prognostic impacts from certain genetic mutations common in AML (e.g., FLT3-ITD/TKD, N/KRAS). The safety profile remains tolerable with no significant increase in severe adverse events[1][6][4].

Other names

VAH regimenVenetoclax/Azacytidine/Homoharringtonine combination