How it is administered

Venetoclax is taken by mouth as a tablet, once daily, with a meal and water. Tablets come in different strengths (10 mg, 50 mg, 100 mg), and should be swallowed whole without chewing, crushing, or breaking. The dose is gradually increased over several days (ramp-up phase) to reduce the risk of side effects, especially tumor lysis syndrome. For Acute Myeloid Leukemia (AML), venetoclax is used in combination with other medications such as azacitidine, decitabine, or low-dose cytarabine.

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 many blood cancers, including AML, BCL-2 is overactive, allowing cancer cells to live longer than they should.

Venetoclax works by binding to the BCL-2 protein, blocking its function. This action releases signals inside the cancer cell that trigger apoptosis, leading to the death of cancer cells. By restoring the cell's ability to self-destruct, venetoclax helps reduce the number of cancer cells in the body. It is especially useful in patients whose cancer cells rely heavily on BCL-2 for survival.

Who Should take it

Venetoclax is indicated for adults with newly diagnosed Acute Myeloid Leukemia (AML) who are 75 years or older, or for those who have health conditions that prevent them from receiving intensive induction chemotherapy. It is always used in combination with other medications such as azacitidine, decitabine, or low-dose cytarabine.

Venetoclax is also used for certain other blood cancers, but for AML, it is specifically for patients who may not tolerate standard, more aggressive treatments due to age or other medical problems.

Who should not take it

Venetoclax should not be taken by patients who are using strong CYP3A inhibitors (such as certain antifungals or antibiotics) at the start and during the ramp-up phase, as this can increase the risk of severe side effects like tumor lysis syndrome.

It should also be avoided in patients who are allergic to venetoclax or any of its ingredients. Women who are pregnant or breastfeeding should not take venetoclax, as it may harm the fetus or nursing baby. Live vaccines should not be given before, during, or after treatment until the immune system recovers.

Commonly used with

For AML, venetoclax is always used in combination with one of the following:

• Azacitidine
• Decitabine
• Low-dose cytarabine

These combinations are chosen based on the patient's health and the doctor's recommendation. Venetoclax may also be used with other medications for different blood cancers.

Commonly tested with

Venetoclax has been tested in combination with azacitidine, decitabine, and low-dose cytarabine for AML. It has also been studied with other agents like rituximab and obinutuzumab in other blood cancers.

In clinical trials for AML, the focus has been on using venetoclax with hypomethylating agents (azacitidine or decitabine) or low-dose cytarabine, especially in older adults or those unable to tolerate intensive chemotherapy.