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Important Note

The following information is intended to supplement, not substitute for, the expertise and judgment of your physician, pharmacist or other healthcare professional. It should not be construed to indicate that use of the drug is safe, appropriate, or effective for you. Consult your healthcare professional before using this drug.




WARNING: Busulfan is FDA-approved only for treating chronic myelogenous leukemia (CML). This medication can lower the body's ability to fight an infection or cancer, as well as prevent normal blood clotting. Notify your doctor immediately if you develop any signs of an infection such as fever, chills, persistent sore throat; easy bruising/bleeding, or unusual tiredness/fatigue. Blood tests (e.g., CBC, platelets) will be performed to monitor your progress. Your busulfan dose may need to be decreased or stopped. If your leukemia symptoms reoccur or worsen, notify your doctor immediately. Busulfan itself may infrequently cause cancer.


Busulfan is used to treat leukemia (CML) when stem cell transplant is not an option. Busulfan works by stopping cancer cells from multiplying, but also affects some normal cells, such as bone marrow cells. Busulfan is not a cure, but can cause cancer remission.



This medication may also be used to treat other disorders of the blood or bone marrow (e.g., myeloproliferative disorder, thrombocytosis, myelofibrosis); or to prepare for stem cell transplants in certain CML patients.

How To Use

This medication is taken by mouth, usually once daily or as directed. Use this exactly as directed by your doctor. To minimize side effects, you may be given other medications (e.g., anti-nausea, anti-seizure agents) to take in addition to this drug. Follow all directions for the proper use of other medications; consult your doctor or pharmacist. Unless your doctor instructs you otherwise, drink plenty of fluids while using busulfan or any other anti-cancer medication. Follow all instructions for proper use, handling, and safe disposal of this medication. If you have questions regarding busulfan, ask your doctor or pharmacist. When used without stem cell transplantation, busulfan may cause cancer remission within 2 months of starting treatment.

Side Effects

Nausea, diarrhea or constipation, headache, skin darkening, or flushing may occur. If any of these effects persist or worsen, contact your doctor or pharmacist promptly. Tell your doctor immediately if any of these serious side effects occur: unusual tiredness, fast or irregular heartbeat, fever, persistent sore throat, easy bruising/bleeding, mental/mood changes, increased thirst, blurred vision, muscle cramps or weakness, chest pain, seizures, sudden weight gain or loss, stomach pain or swelling, change in amount or color of urine, mouth sores. Tell your doctor immediately if any of these unlikely but serious side effects occur: dizziness, fainting, vomiting. Tell your doctor immediately if any of these highly unlikely but very serious side effects occur: dark urine, yellowing eyes/skin, coughing up blood, vision problems. An allergic reaction to this drug is unlikely, but seek immediate medical attention if it occurs. Symptoms of an allergic reaction include: rash, itching, swelling, dizziness, trouble breathing. Rare, possibly fatal, lung problems may occur months to years after using busulfan. Symptoms include trouble breathing or persistent cough or congestion. Report any symptoms promptly. If your leukemia symptoms reoccur or worsen, notify your doctor immediately. Busulfan itself may infrequently cause cancer. If you notice other effects not listed above, contact your doctor or pharmacist.


Tell your doctor your medical history, especially: seizures, head injury, blood disorders (e.g., low blood counts, or thalassemia), diabetes, lung disease, any serious infections, any allergies. Because this medication can lower your body's ability to fight an infection, do not touch your eyes or the inside of your nose without first washing your hands. Do not have immunizations/vaccinations without the consent of your doctor, and avoid contact with people who have recently received oral polio vaccine. Use caution with sharp objects like safety razors or nail cutters, and avoid activities that might cause injury. Caution is advised when using this drug in children because they may clear the drug from their system faster, and therefore may require special dosing and monitoring. This drug may also cause fertility problems. This medication is not recommended for use during pregnancy. Busulfan may cause fetal harm. Avoid becoming pregnant while using this drug. Consult your doctor for more details. It is not known whether this drug passes into breast milk. Because of the potential risk to the infant, breast-feeding while using this drug is not recommended. Consult your doctor before breast-feeding.

Drug Interactions

Tell your doctor or pharmacist of all prescription and nonprescription drugs you may use, especially of: azole antifungals (e.g., itraconazole,), thioguanine, acetaminophen, drugs that can cause seizures (e.g., bupropion), drugs that can increase seizure risk (e.g., phenothiazines such as chlorpromazine), other drugs which affect the bone marrow/immune system (e.g., other anti-cancer drugs, or cyclosporine). Check your prescription and nonprescription labels carefully for acetaminophen-containing products. These should be used cautiously 3 days before and during therapy with busulfan. Acetaminophen may increase busulfan blood levels and side effects. If acetaminophen is used, do not exceed recommended daily doses. Do not start or stop any medicine without doctor or pharmacist approval.


If overdose is suspected, contact your local poison control center or emergency room immediately. Symptoms of overdose may include severe fatigue, persistent sore throat, fever; or easy bruising or bleeding.


Do not share this medication with others; keep busulfan away from children. Laboratory tests (e.g., blood, liver) will be performed to monitor your progress.

Missed Dose

If you miss a dose, immediately contact your doctor or pharmacist to establish a new dosing schedule. Do not double the dose to catch up.


Store at room temperature and keep away from moisture and sunlight. Do not store in the bathroom.

Medical Alert

Your condition can cause complications in a medical emergency. For enrollment information call MedicAlert at 1-800-854-1166 (USA), or 1-800-668-1507 (Canada).