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venetoclax (Venclexta)
Targeted Therapy
Administration: oral

How it is administered

Venetoclax is taken by mouth as a tablet, once daily, with a meal and water. The tablets come in different strengths (10 mg, 50 mg, and 100 mg). It is important to swallow the tablets whole, without chewing, crushing, or breaking them. The dose is gradually increased over several weeks (called a ramp-up schedule) to reduce the risk of side effects, especially at the start of treatment.

How it works

Venetoclax is a targeted therapy that works by blocking a protein in cancer cells called BCL-2. BCL-2 helps cancer cells survive longer than they should. By inhibiting BCL-2, venetoclax allows the cancer cells to undergo a natural process of cell death called apoptosis. This is particularly effective in blood cancers where BCL-2 is often overactive, such as chronic lymphocytic leukemia (CLL) and acute myeloid leukemia (AML).

Venetoclax is not specifically approved for Peripheral T-Cell Lymphoma, but it is used in other blood cancers. Its action helps to restore the balance between cell survival and cell death, making it harder for cancer cells to grow and spread.

Common side effects

  • Neutropenia (low white blood cell count)
  • Thrombocytopenia (low platelet count)
  • Anemia (low red blood cell count)
  • Diarrhea
  • Nausea
  • Constipation
  • Vomiting
  • Fatigue
  • Fever
  • Infections (such as upper respiratory tract infections, pneumonia, sepsis)
  • Edema (swelling)
  • Rash
  • Cough
  • Muscle or joint pain

Serious side effects can include tumor lysis syndrome (a rapid breakdown of cancer cells), severe infections, and low blood counts. Your doctor will monitor your blood work regularly to check for these side effects.

Who Should take it

Venetoclax is approved for adults with certain types of blood cancers:

  • Chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), either alone or in combination with other medicines.
  • Newly diagnosed acute myeloid leukemia (AML) in adults 75 years or older, or in adults who have health problems that prevent them from receiving intensive chemotherapy. In AML, it is used in combination with azacitidine, decitabine, or low-dose cytarabine.

Venetoclax is not specifically approved for Peripheral T-Cell Lymphoma, but it may be used in clinical trials or off-label in some cases. Always discuss with your doctor if venetoclax is appropriate for your specific type of blood cancer.

Who should not take it

Venetoclax should not be used in patients who are taking strong CYP3A inhibitors (certain medications that affect how venetoclax is broken down in the body) at the start and during the ramp-up phase, especially for those with CLL/SLL, due to the risk of serious side effects like tumor lysis syndrome.

It should also be avoided in patients who have a known allergy to venetoclax or any of its ingredients. Women who are pregnant or breastfeeding should not take venetoclax, as it may harm the unborn baby or nursing infant. Live vaccines should not be given before, during, or after treatment until your immune system recovers. Always inform your healthcare provider about all medications and supplements you are taking.

Commonly used with

Venetoclax is often used in combination with other medications to treat blood cancers:

  • With obinutuzumab or rituximab for CLL/SLL
  • With azacitidine, decitabine, or low-dose cytarabine for AML

These combinations help improve the effectiveness of treatment. Your doctor will decide the best combination based on your specific diagnosis and health status.

Commonly tested with

Venetoclax has been tested in clinical trials with:

  • Obinutuzumab and rituximab (for CLL/SLL)
  • Azacitidine, decitabine, and low-dose cytarabine (for AML)
  • Other investigational agents in various blood cancers

It is important to note that venetoclax is not approved for use with bortezomib and dexamethasone in multiple myeloma outside of clinical trials, due to increased risk of death seen in studies.