How effective is venetoclax/venetoclax plus azacitidine in treating disease?

Venetoclax/Venetoclax is an oral BCL-2 inhibitor mainly used to treat chronic lymphocytic leukemia (CLL) and certain types of acute myelogenous leukemia (AML). Azacitidine is a nucleoside analog mainly used to treat acute myeloid leukemia and myelodysplastic syndrome (MDS). In recent years, clinical studies of the combination of venetoclax and azacitidine have shown promising therapeutic effects, especially in older patients and those with leukemias that are resistant to traditional chemotherapy.

Studies have shown that the combination treatment of venetoclax and azacitidine can significantly improve the overall response rate and progression-free survival of patients. The mechanism of this combination therapy is that venetoclax promotes cancer cell apoptosis by inhibiting the function of BCL-2 protein, while azacitidine enhances cell cycle arrest and apoptosis by interfering with DNA synthesis and repair. This synergistic effect makes it easier for cancer cells to be eliminated, thereby improving efficacy.

In clinical trials, the combined use of venetoclax and azacitidine not only improved patient survival but also reduced the incidence of adverse reactions. Patients who receive combination therapy generally tolerate treatment better than azacitidine alone. In elderly AML patients who are not suitable for intensive chemotherapy, the low-intensity venetoclax combined with decitabine or azacitidine regimen shows promising efficacy and tolerable safety, with an overall response rate (CR+CRi) as high as 73% in the 400 mg venetoclax + HMA cohort, low early mortality, and an overall survival rate (OS) of more than 17 months. In addition, combination therapies have also shown effectiveness in certain subpopulations (such as patients with high-risk genetic mutations), providing more treatment options.

Reference materials:https://pmc.ncbi.nlm.nih.gov/articles/PMC6318429/#B36