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, 100 mg) and should be swallowed whole without chewing, crushing, or breaking them. The dose is gradually increased over several weeks (called a ramp-up phase) 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 called BCL-2. This protein is found in high amounts in certain blood cancer cells and helps them survive longer than they should. By inhibiting BCL-2, venetoclax helps restore the natural process of cell death (apoptosis), allowing cancer cells to die off more effectively.
Venetoclax is especially useful in cancers where BCL-2 is overactive, such as chronic lymphocytic leukemia (CLL) and acute myeloid leukemia (AML). By promoting cancer cell death, venetoclax can help reduce the number of cancer cells in the body and improve outcomes for patients.
Common side effects
- Neutropenia (low white blood cell count)
- Thrombocytopenia (low platelet count)
- Anemia (low red blood cell count)
- Diarrhea
- Nausea
- Constipation
- Vomiting
- Fatigue
- Upper respiratory tract infection
- Cough
- Musculoskeletal pain
- Edema (swelling)
- Fever
- Infections (including pneumonia and sepsis)
Serious side effects can include tumor lysis syndrome (a rapid breakdown of cancer cells), severe infections, and low blood counts.
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 conditions that make them unable to receive intensive chemotherapy. In AML, it is used in combination with azacitidine, decitabine, or low-dose cytarabine.
Venetoclax is not routinely recommended for multiple myeloma outside of clinical trials, as studies have shown increased risk of death when used with certain other drugs in this setting.
Who should not take it
Venetoclax should not be taken by patients who are using strong CYP3A inhibitors (certain antifungals, antibiotics, or antivirals) at the start or during the ramp-up phase for CLL/SLL, as this can increase the risk of dangerous side effects.
It should also be avoided in people who are allergic to venetoclax or any of its ingredients. Caution is needed in pregnant women, as it may harm the unborn baby, and breastfeeding is not recommended during treatment and for one week after the last dose. Live vaccines should not be given during treatment. Venetoclax is not approved for use in children.
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
It may also be used with supportive medications to prevent side effects, such as anti-hyperuricemics (to prevent tumor lysis syndrome) and antibiotics (to prevent infections).
Commonly tested with
Venetoclax has been tested in combination with:
- Obinutuzumab and rituximab (for CLL/SLL)
- Azacitidine, decitabine, and low-dose cytarabine (for AML)
- Bortezomib and dexamethasone (for multiple myeloma, but this combination is not recommended outside of clinical trials due to increased risk of death)
It is also studied with other targeted therapies and chemotherapy agents in ongoing clinical trials.