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venetoclax (Venclexta)
Targeted Therapy
Administration: oral

How it is administered

Venetoclax is taken by mouth as a tablet. The tablets come in strengths of 10 mg, 50 mg, and 100 mg. They should be swallowed whole with a meal and water, at the same time each day. Do not chew, crush, or break the tablets.

The dosing schedule for venetoclax usually starts with a low dose that is gradually increased over several weeks (known as a ramp-up phase) to reduce the risk of tumor lysis syndrome. Your doctor will provide a specific schedule based on your condition and any other medications you may be taking.

How it works

Venetoclax is a targeted therapy that works by blocking a protein called BCL-2. BCL-2 is found in high amounts in some blood cancers, including certain lymphomas and leukemias, and helps cancer cells survive longer than they should.

By inhibiting BCL-2, venetoclax helps restore the natural process of cell death (apoptosis) in cancer cells. This leads to the destruction of cancer cells, helping to control the disease. Venetoclax is especially useful in cancers where BCL-2 is overexpressed, such as chronic lymphocytic leukemia (CLL) and some forms of acute myeloid leukemia (AML). While not specifically approved for mantle cell lymphoma, venetoclax’s mechanism may be relevant for blood cancers with similar characteristics.

Common side effects

Common side effects of venetoclax may include:

  • Low white blood cell counts (neutropenia), increasing the risk of infection
  • Low red blood cell counts (anemia)
  • Low platelet counts (thrombocytopenia)
  • Diarrhea
  • Nausea
  • Upper respiratory tract infection
  • Cough
  • Musculoskeletal pain
  • Fatigue
  • Swelling (edema)

Serious side effects can include tumor lysis syndrome, severe infections, and low blood counts. Always report any unusual symptoms to your healthcare provider right away.

Who Should take it

Venetoclax is FDA-approved for adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), either alone or in combination with other medications. It is also approved in combination with certain other drugs for adults with newly diagnosed acute myeloid leukemia (AML) who are 75 years or older, or who have health problems that prevent them from receiving intensive chemotherapy.

For mantle cell lymphoma, venetoclax may be considered in certain situations, especially in clinical trials or when other treatments have not worked, but it is not currently an FDA-approved indication for this specific disease. Always discuss with your doctor whether venetoclax is appropriate for your situation.

Who should not take it

Venetoclax should not be taken by patients who are using strong CYP3A inhibitors at the start or during the dose ramp-up phase, as this can increase the risk of serious side effects like tumor lysis syndrome. Examples of strong CYP3A inhibitors include certain antifungal and antibiotic medications.

Patients who are pregnant or may become pregnant should avoid venetoclax, as it can harm an unborn baby. It is also not recommended for people who are breastfeeding. People with severe liver problems may require dose adjustments. Always inform your healthcare provider about all medications and supplements you are taking before starting venetoclax.

Commonly used with

Venetoclax is commonly used in combination with other medications for blood cancers. For CLL/SLL, it is often combined with obinutuzumab or rituximab. For AML, it is used with azacitidine, decitabine, or low-dose cytarabine.

The choice of combination depends on the type of blood cancer, previous treatments, and patient health. Your doctor will determine the best combination for your specific situation.

Commonly tested with

Venetoclax has been tested in clinical trials with a variety of other medications, including:

  • Obinutuzumab
  • Rituximab
  • Azacitidine
  • Decitabine
  • Low-dose cytarabine

These combinations are designed to improve treatment effectiveness for blood cancers such as CLL, SLL, and AML. In some research settings, venetoclax may also be studied with other targeted therapies or chemotherapy agents.