How it is administered

Venetoclax is taken by mouth as a tablet once daily, with a meal and water. The tablets should be swallowed whole and not chewed, crushed, or broken. Venetoclax is available in 10 mg, 50 mg, and 100 mg tablets. The dosing schedule often involves a gradual increase (ramp-up) to the target dose to reduce the risk of side effects, especially at the start of treatment.

How it works

Venetoclax is a targeted therapy known as a BCL-2 inhibitor. BCL-2 is a protein that helps cancer cells survive by preventing them from undergoing a natural process called apoptosis (programmed cell death). In certain blood cancers, such as acute myeloid leukemia (AML) and chronic lymphocytic leukemia (CLL), BCL-2 is overexpressed, making the cancer cells more resistant to dying.

Venetoclax works by binding directly to the BCL-2 protein, blocking its function. This allows the cancer cells to undergo apoptosis, leading to their death. By restoring the body's ability to remove these abnormal cells, venetoclax helps reduce the number of cancer cells in the blood and bone marrow. This mechanism is particularly important in blood cancers where BCL-2 plays a significant role in disease progression.

Who Should take it

Venetoclax is approved for adults with certain blood cancers:

• Chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
• In combination with azacitidine, decitabine, or low-dose cytarabine for newly diagnosed acute myeloid leukemia (AML) in adults 75 years or older, or those who have health conditions that prevent them from receiving intensive chemotherapy.

For patients with myelodysplastic syndromes (MDS), venetoclax is not currently FDA-approved specifically for this use, but it may be considered in clinical trials or special circumstances. Your doctor will determine if venetoclax is appropriate for your specific diagnosis and health status.

Who should not take it

Venetoclax should not be used in patients who are taking strong CYP3A inhibitors at the start and during the ramp-up phase for CLL/SLL, as this combination can significantly increase the risk of serious side effects, such as tumor lysis syndrome (TLS).

It should also be avoided in patients with known hypersensitivity to venetoclax or any of its ingredients. Pregnant women should not take venetoclax due to the risk of harm to the unborn baby. The safety and effectiveness of venetoclax have not been established in children. Always inform your healthcare provider about all medications and supplements you are taking before starting venetoclax.

Commonly used with

Venetoclax is often used in combination with other medications to treat blood cancers. For AML, it is commonly combined with azacitidine, decitabine, or low-dose cytarabine. For CLL/SLL, it may be used with obinutuzumab or rituximab. These combinations are designed to improve treatment effectiveness.

Your doctor will choose the combination based on your specific type of cancer, overall health, and previous treatments.

Commonly tested with

Venetoclax has been tested in clinical trials with several other medications, including azacitidine, decitabine, low-dose cytarabine, obinutuzumab, and rituximab. These studies have helped determine the best combinations and dosing schedules for different types of blood cancers.

If you are participating in a clinical trial, venetoclax may be tested with other investigational drugs or supportive therapies depending on the study design.