Busulfan

Busulfan is a chemotherapy drug used in high doses with other chemotherapy drugs before a stem cell transplant. It is sometimes used in low doses to treat blood cancers.

What is busulfan?

Busulfan is used to treat blood cancers. It is used in high doses together with other chemotherapy drugs before a stem cell transplant. It is also used in low doses to treat other types of blood cancers, such as essential thrombocythaemia and polycythaemia vera. It may sometimes be used to treat chronic myeloid leukaemia (CML)

It is best to read this information with our general information about chemotherapy and the type of cancer you have. 

Your doctor will talk to you about this treatment and its possible side effects before you agree (consent) to have treatment.

More information about this treatment

This information is correct at time of publishing. But sometimes the types of cancer this treatment is used for, or treatment side effects, may change between revision dates.

You can talk to your cancer team if you want more detailed information about this treatment. Or visit the electronic Medicines Compendium (eMC) website, which has patient information leaflets (PIL) for individual drugs.

How busulfan is given

Busulfan can be given on its own or in combination with other cancer drugs. If you are having:

  • high-dose busulfan, a chemotherapy nurse will give it to you as a drip during a stay in hospital
  • low-dose busulfan, you have it as tablets to take at home.

During a course of treatment, you usually see a:

  • blood cancer doctor (haematologist)
  • chemotherapy nurse or a specialist nurse
  • specialist pharmacist.

This is who we mean when we mention doctor, nurse or pharmacist in this information.

High-dose busulfan

Before or on the day of each treatment, a nurse or person trained to take blood (phlebotomist) will take a blood sample from you. This is to check that it is safe for you to have chemotherapy.

You will speak to a doctor, nurse or pharmacist before you have chemotherapy. They will talk to you about your blood results and ask you how you have been feeling. If your blood results are okay, the pharmacy team will prepare your chemotherapy.

Your nurse usually gives you anti-sickness (anti-emetic) and other drugs before the chemotherapy.

Busulfan is given as a drip (infusion) into a vein. It usually take 2 to 3 hours.

You may have busulfan through:

  • a cannula – a short, thin tube the nurse puts into a vein in your arm or hand
  • a central line – a fine tube that goes under the skin of your chest and into a vein close by
  • PICC line – a fine tube that is put into a vein in your arm and goes up into a vein in your chest.
  • an implantable port (portacath) – a disc that is put under the skin on your chest or arm and goes into a vein in your chest.

Low-dose busulfan

The tablets must be swallowed whole with a glass of water. They should not be chewed, broken or crushed. Take them at the same time every day.

If you forget to take the tablets, you should take the missed dose as soon as possible within the same day. If a full day has gone by, let your doctor or nurse know. Do not take a double dose unless your doctor tells you to.

Other things to remember about your tablets:

  • Wash your hands after taking your tablets.
  • Other people should avoid direct contact with the chemotherapy drugs. 
  • Keep them in the original package and at room temperature, away from moisture, heat and direct sunlight.
  • Keep them safe, somewhere children cannot see or reach them.
  • If you are sick just after taking the tablets, contact the hospital. Do not take another dose.
  • If your treatment is stopped, return any unused tablets to the hospital pharmacist.

Lactose

Busulfan tablets contain lactose. If you have a lactose intolerance, you should tell your doctor before taking busulfan tablets.

Your course of chemotherapy

High-dose busulfan is usually given for a few days as part of your conditioning treatment before a stem cell transplant.

If you are having low dose busulfan, your doctor or nurse will explain how often you have it. Your nurse or doctor will discuss your treatment plan with you. They may give you a copy of a treatment plan to take home with you.

Busulfan and paracetamol

You may be advised to avoid taking paracetamol for 72 hours before treatment and during treatment with busulfan as it can affect how busulfan works. Your doctor, nurse or pharmacist can tell you more about this.

About side effects

We explain the most common side effects of this treatment here. We also include some that are less common. 

You may get some of the side effects we mention, but you are unlikely to get all of them. And you may have some side effects, including rarer ones, that we have not listed here. 

Other cancer treatments may cause different side effects. If you are also having other cancer treatment, you may have other side effects.

Always tell your doctor, nurse or pharmacist about any side effects you have. They can give you: 

  • drugs to help control some side effects 
  • advice about managing side effects. 

It is important to take any drugs exactly as explained. This means they will be more likely to work for you.

Serious and life-threatening side effects

Some cancer treatments can cause serious side effects. Sometimes, these may be life-threatening. Your doctor, nurse or pharmacist can explain the risk of these side effects to you.

Contact the hospital

Your doctor, nurse or pharmacist will give you 24-hour contact numbers for the hospital. If you feel unwell or need advice, you can call at any time of the day or night. Save these numbers in your phone or keep them somewhere safe.

Side effects while treatment is being given

Some people may have side effects while they are being given the chemotherapy or shortly after they have it:

Allergic reaction

Some people have an allergic reaction while having this treatment. Before treatment, you will have medicines to help prevent or reduce any reaction. 

Signs of a reaction can include: 

  • feeling hot or flushed
  • shivering
  • itching
  • a skin rash
  • feeling dizzy or sick
  • a headache
  • feeling breathless or wheezy
  • swelling of your face or mouth
  • pain in your back, tummy or chest. 

Your nurse will check you for signs of a reaction during your treatment. If you feel unwell or have any of these signs, tell them straight away. If you do have a reaction, they can treat it quickly. 

Sometimes a reaction happens a few hours after treatment. If you develop any of these signs or feel unwell after you get home, contact the hospital straight away on the 24-hour number.

The drug leaks outside the vein

The drug may leak outside the vein. If this happens it can damage the tissue around the vein. This is called extravasation. Extravasation is not common but if it happens it is important that it is dealt with quickly. Tell your nurse straight away if you have any stinging, pain, redness or swelling around the vein. 

Common side effects

The side effects will depend on the dose of busulfan. If you are having low-dose busulfan, the side effects are likely to be mild.

Risk of infection

This treatment can reduce the number of white blood cells in your blood. These cells fight infection. If your white blood cell count is low, you may be more likely to get an infection. A low white blood cell count is called neutropenia.

An infection can be very serious when the number of white blood cells is low. It is important to get any infection treated as soon as possible. If you have any of the following symptoms, contact the hospital straight away on the 24-hour number:

  • a temperature above 37.5°C  
  • a temperature below 36°C 
  • you feel unwell, even with a normal temperature
  • you have symptoms of an infection.

Symptoms of an infection include:

  • feeling shivery and shaking
  • a sore throat
  • a cough 
  • breathlessness
  • diarrhoea
  • needing to pass urine (pee) often, or discomfort when you pass urine.

It is important to follow any specific advice your cancer treatment team gives you.

Your white blood cell count will usually return to normal before your next treatment. You will have a blood test before having more treatment. If your white blood cell count is low, your doctor may delay your treatment for a short time, until your cell count increases.

Bruising and bleeding

This treatment can reduce the number of platelets in your blood. Platelets are cells that help the blood to clot.

If the number of platelets is low, you may bruise or bleed easily. You may have:

  • nosebleeds
  • bleeding gums
  • heavy periods
  • blood in your urine (pee) or stools (poo)
  • tiny red, brown or purple spots that may look like a rash – these spots can be harder to see if you have black or brown skin. 

If you have any unexplained bruising or bleeding, contact the hospital straight away on the 24-hour number. You may need a drip to give you extra platelets. This is called a platelet transfusion.

Anaemia (low number of red blood cells)

This treatment can reduce the number of red blood cells in your blood. Red blood cells carry oxygen around the body. If the number of red blood cells is low, this is called anaemia. You may feel:

  • very low in energy
  • breathless 
  • dizzy and light-headed. 

If you have these symptoms, contact the hospital straight away on the 24-hour number. You may need treatment for anaemia. If you are very anaemic, you may need a drip to give you extra red blood cells. This is called a blood transfusion.

Feeling tired

Feeling tired is a common side effect of this treatment. It is often worse towards the end of treatment and for some weeks after it ends. Try to pace yourself and plan your day so you have time to rest. Gentle exercise, like short walks, can help you feel less tired. 

If you feel sleepy, do not drive or use machinery.

Feeling sick

The nurses will give you anti-sickness drugs regularly to help prevent or control sickness during your treatment. If you feel sick or are sick (vomit), tell your nurse or doctor. They may change the anti-sickness drug to one that works better for you. If you are having busulfan tablets, or low doses of busulfan, sickness is not usually a common side effect.

Loss of appetite

This treatment can affect your appetite. Don’t worry if you do not eat much for 1 or 2 days. But if your appetite does not come back after a few days, or if you are losing weight, tell your doctor, nurse or pharmacist. They can give you advice. They may give you food or drink supplements. Or they may suggest changes to your diet or eating habits to help.

Sore mouth and throat

This treatment may cause a sore mouth and throat. You may also get mouth ulcers. This can make you more likely to get an infection in your mouth or throat. Use a soft toothbrush to clean your teeth or dentures in the morning, at night and after meals.

If your mouth or throat is sore:

  • tell your nurse or doctor – they can give you a mouthwash or medicines to help
  • try to drink plenty of fluids
  • avoid alcohol, tobacco and foods that irritate your mouth and throat.

Skin changes

Chemotherapy may affect your skin. Your doctor or nurse can tell you what to expect. If your skin feels dry, try using an unperfumed moisturising cream every day. Busulfan can cause a rash, which may be itchy.

Your skin may darken. It will usually return to its normal colour after you finish treatment.

Always tell your doctor or nurse about any skin changes. They can give you advice and may give you creams or medicines to help. Any changes to your skin are usually temporary and improve when treatment finishes.

Hair loss

Hair loss is rare with low doses of busulfan.

If you are having high-dose treatment with busulfan, you will lose all the hair from your head. Your eyelashes, eyebrows and other body hair may also thin or fall out. It is almost always temporary. Your hair will usually grow back after chemotherapy ends.

It is important to cover your scalp when you are out in the sun until your hair grows back. Your nurse can give you advice about coping with hair loss.

Bladder irritation

High-dose busulfan can irritate your bladder and cause discomfort when you pass urine. Drink plenty of fluids – at least 2 litres (3½ pints) during the 24 hours following chemotherapy. It is also important to empty your bladder regularly and to try to pass urine (pee) as soon as you feel the need to go.

You may be given fluids through a drip (infusion) to help prevent bladder irritation. Contact the hospital straight away on the number you have been given if you:

  • feel any discomfort or stinging when you pass urine
  • notice any blood in your urine.

Constipation

This treatment can cause constipation. Constipation means that you are not able to pass stools (poo) as often as you normally do. It can become difficult or painful. Here are some tips that may help:

  • Drink at least 2 litres (3½ pints) of fluids each day.
  • Eat high-fibre foods, such as fruit, vegetables and wholemeal bread.
  • Do regular gentle exercise, like going for short walks.

If you have constipation, contact the hospital on the 24-hour number for advice. They can give you drugs called laxatives to help. 

If you have not been able to pass stools for over 2 days and are being sick, contact the 24-hour number straight away. 

Tummy pain

You may get pain in your tummy (abdomen), or have indigestion. Your doctor, nurse or pharmacist can give you advice or treatment to help. Contact the hospital straight away on the 24-hour number if your symptoms do not go away, or get worse.

Diarrhoea

This treatment may cause diarrhoea. Diarrhoea means passing more stools (poo) than is normal for you, or having watery or loose stools. You may also have stomach cramps. If you have a stoma, it may be more active than usual. 

If you are passing loose stools 3 or more times a day and this is not normal for you, contact the hospital as soon as possible on the 24-hour number. Follow the advice they give you about:

  • taking anti-diarrhoea medicines 
  • drinking enough fluids to keep you hydrated and to replace lost salts and minerals
  • any changes to your diet that might help. 

They might also ask you for a specimen of your stool to check for infection.

Effects on the kidneys

This treatment can affect how the kidneys work. This is usually mild and goes back to normal after treatment ends. You will have blood tests to check how well your kidneys are working. Contact the hospital on the 24-hour number if you:

  • have blood in your urine (pee) 
  • are passing less urine or peeing less often than usual.

Drinking fluids helps protect your kidneys. The advice is usually to try to drink at least 2 litres (3½ pints) of fluid each day. But follow any advice from your doctor, nurse or pharmacist about how much is right for you.

Effects on the liver

This treatment can affect how your liver works. You will have regular blood tests to check this.

Sometimes, high-dose busulfan can cause more serious liver problems. You may be given drugs to help prevent liver problems.

Contact the hospital straight away if you have any of these symptoms:

  • yellow skin or eyes
  • feeling very sleepy
  • dark urine (pee)
  • unexplained bleeding or bruising
  • pain in the right side of your tummy (abdomen)
  • loss of appetite.

Headaches

This treatment may cause headaches. If you have headaches, tell your doctor, nurse or pharmacist. They can give you advice about painkillers that may help. Tell them if the headache does not get better, or gets worse.

Eye problems

Busulfan may make your eyes dry. Your doctor can give you eye drops to help with this. Rarely, it may cause cataracts years later – this is clouding of the eye's lens. Let your doctor know if you notice any changes in your vision.

Less common side effects

Effects on the lungs

This treatment can cause changes to the lungs. Tell your doctor, nurse or pharmacist if you develop: 

  • a cough that does not go away
  • wheezing
  • breathlessness.

You should also tell them if any existing breathing problems get worse. You may have tests to check your lungs.

Effects on the nervous system

Busulfan can affect your mood. High-dose busulfan can also make it difficult to sleep. Talk to your doctor, nurse or pharmacist if you have sleep problems or if you feel low or anxious.

It can also make you feel dizzy, confused or restless, but this is less common. Tell your doctor or nurse straight away if you notice any of these symptoms.

Rarely, this treatment can cause:

  • you to see or hear things that are not there (hallucinations)
  • seizures (fits).

You may be given drugs to help prevent fits. Your doctor will explain more about this.

Effects on the heart

This treatment can affect how the heart works. You may have tests to check how well your heart is working. These may be done before, during and after treatment.

If the treatment is causing heart problems, your doctor may change the type of treatment you are having.

Contact the hospital straight away on the 24-hour number if you have any of these symptoms during or after treatment:

  • breathlessness
  • dizziness
  • changes to your heartbeat
  • swollen feet and ankles.

Other conditions can cause these symptoms, but it is important to get them checked by a doctor. 

Always call 999 if you have:

  • chest pain, pressure, heaviness, tightness or squeezing across the chest
  • difficulty breathing.

Second cancer

Rarely, busulfan can increase the risk of developing a second cancer years later. But the benefits of treatment usually far outweigh this risk. Your doctor can talk to you about this.

Other information

Blood clot risk

Cancer and some cancer treatments can increase the risk of a blood clot. Contact the hospital straight away on the 24-hour number if you have any of these symptoms during or after treatment:

  • throbbing pain or swelling in a leg or arm
  • reddening of the skin in the area – if you have black or brown skin, this can be harder to notice, but the skin might become darker
  • suddenly feeling breathless or coughing.

Always call 999 if you have:

  • chest pain
  • difficulty breathing.

A blood clot is serious, but it can be treated with drugs called anticoagulants. These thin the blood. Your doctor, nurse or pharmacist can give you more information about preventing and treating blood clots.

Other medicines

Some medicines can affect how this treatment works or be harmful while you are having it. Always tell your cancer doctor, nurse or pharmacist about any drugs you are taking or planning to take, such as: 

Vaccinations

Doctors usually recommend that people with cancer have vaccinations for flu and for coronavirus (covid). These help reduce your risk of serious illness from these infections. Most people can have these vaccines, including people with weak immune systems.

If your immune system is weak, you should not have live vaccinations. Live vaccines can make you unwell because they contain a very weak version of the illness they will protect you against. Live vaccines include Zostavax®, which is a shingles vaccine, and the yellow fever vaccine.

It is important to ask your doctor, nurse or pharmacist for advice about having vaccinations. They can explain what vaccines are right for you and when it is best to have them.

Contraception

Your doctor, nurse or pharmacist will advise you not to get pregnant or make someone pregnant while having this treatment and for some time afterwards. The drugs may harm a developing baby. It is important to use contraception to prevent pregnancy. Follow their advice about:

  • what types of contraception to use 
  • how long after treatment you should continue to use contraception. 

Breastfeeding

You are advised not to breastfeed while having this treatment, or for some time after treatment ends. This is because the drugs could be passed to the baby through breast milk. 

Your doctor, nurse or pharmacist can give you more information.

Fertility

Some cancer drugs can affect whether you can get pregnant or make someone pregnant. If you are worried about this, it is important to talk with your doctor before you start treatment.

Changes to periods

If you have a period, these may become irregular or stop while you are having this treatment. This may be temporary, but it can sometimes be permanent. Your menopause may start sooner than it would have done. Your doctor or nurse can give you more information.

Sex

It is possible that small amounts of chemotherapy may be passed on through vaginal fluids or semen. If you have sex in the first few days after treatment, your cancer team will usually advise using condoms or a dental dam to protect your partner.

Medical and dental treatment

If you need medical treatment for any reason other than cancer, always tell the healthcare professional that you are having cancer treatment. Give them the contact details for your cancer doctor or cancer team so they can ask for advice.

If you have appointments with a dentist, always tell them you are having cancer treatment. Talk to your cancer team before you have any dental treatment.

About our information

  • Reviewers

    This information has been written, revised and edited by Macmillan Cancer Support’s Cancer Information Development team. It has been reviewed by expert health professionals and people living with cancer.

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Date reviewed

Reviewed: 01 January 2024
|
Next review: 01 January 2026
Trusted Information Creator - Patient Information Forum
Trusted Information Creator - Patient Information Forum

Our cancer information meets the PIF TICK quality mark.

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