How it Works

Busulfan is a bifunctional alkylating agent which damages the DNA of cancer cells and interferes with their ability to grow and divide. Having a selective immunosuppressive effect on the bone marrow. 

Busulfan has a selective effect on granulocytopoiesis, the process by which the body produces granulocytes, a type of white blood cell involved in the immune system's response to infection and inflammation. Being also effective for the reduction of masses made of granulocytes.

How it’s Administered

• In treating acute myeloid leukemia (AML), busulfan is usually given as an intravenous (IV) infusion directly into a vein. It is typically given in combination with other chemotherapy medications as a multi-drug therapy.
• Before starting treatment with Busulfan, your doctor will perform a physical examination and may order specific tests to assess your overall health and to determine the appropriate dosage. The dosage and duration of treatment will depend on the individual patient and the specific type of AML being treated.

Who Should Take Busulfan

• Busulfan is indicated for conditioning treatment before stem cell transplant in eligible patients considered for busulfan and cyclophosphamide combination induction therapy. 
• Palliative care patients in a chronic phase of Acute Myeloid Leukemia 
• Effective for a prolonged remission in patients with polycythemia vera,  a type of cancer that produces too many abnormal red blood cells, especially with thrombocytosis (an overproduction of platelets). 
• Can be very helpful in selected cases of essential thrombocytopenia (a low platelet count in the blood) and myelofibrosis, a type of cancer that affects the blood-forming cells in the bone marrow. 

Who Shouldn’t Take Busulfan

• People who are allergic to the medication or any of its components.
• Patients who presented resistance or refractory disease to the medication 
• Pregnant women and breastfeeding are counterindicated. Busulfan can harm a developing fetus and may pass into breast milk.
• Men must use contraceptives while taking the medication
• People planning to have children in the future, must be advised to preserve sperm/eggs before the treatment starts
• People who have certain medical conditions that may make them more sensitive to the side effects of busulfan. For example, people with liver or kidney problems, bone marrow disorders, or a history of blood clots may be at increased risk of side effects from busulfan.

The Most Common Side Effects of Taking Busulfan Include:

• Nausea and vomiting (usually mild with standard doses).
• Diarrhea (usually mild with standard doses).
• Poor appetite.
• Mouth sores.
• Loss of fertility. This effect is dose-dependent.
• Decreased blood cell counts: Your levels of platelets and white and red blood cells may temporarily drop. Which may cause fatigue, weakness, shortness of breath, increased risk of infections, bleeding, or bruising.

Commonly Used in Acute Myeloid Leukemia With

• Cyclophosphamide and then an autologous stem cell transplantation.
• Fludarabine and then an allogeneic stem cell transplantation.

Take a look at the available clinical trials for AML

Date last updated 12/22/22