The effect of venetoclax/venetoclax in the treatment of myelodysplasia

Venetoclax/Venetoclax, as an oral drug targeting the BCL-2 protein, has attracted widespread attention in recent years in the treatment of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). These two diseases are more common in older patients and are often accompanied by drug resistance problems, which limits the effectiveness of treatment. Venetoclax improves the prognosis of these patients to some extent by lowering the apoptosis threshold and increasing the sensitivity of cells to chemotherapy drugs.

In studies, venetoclax has shown modest efficacy as a monotherapy forAML, but its effects were more pronounced in combination therapy. Especially when combined with chemotherapy drugs such as low-dose cytarabine, considerable positive results have been achieved. This combination enables better responses in patients who are not candidates for intensive chemotherapy and has shown the potential to increase overall survival. Additionally, venetoclax-based combination therapies have shown promising preliminary results in high-risk MDS patients, especially when combined with epigenetic drugs such as azacitidine. Recent clinical studies have shown that this combination can effectively induce responses and improve patients' quality of life.

However, although combination therapy with venetoclax has shown clinical promise, drug resistance and associated toxicities remain major obstacles in its application. On the one hand, some patients may develop resistance to venetoclax due to factors such as genetic mutations and changes in the tumor microenvironment, which greatly reduces the effectiveness of treatment. Therefore, it is important to identify and monitor these possible resistance mechanisms to ensure that patients can benefit from treatment.

At the same time, the side effects of venetoclax also require attention, especially in elderly patients, as drug-related toxicity may lead to serious health risks. Therefore, in clinical practice, doctors need to carefully consider the use of venetoclax and adjust the treatment plan based on the patient's specific situation to minimize adverse reactions and improve efficacy.

Reference materials:https://pmc.ncbi.nlm.nih.gov/articles/PMC10241752/