Azacitidine is a chemotherapy drug which may be used to treat a group of disorders that affect the bone marrow and blood known as myelodysplastic syndromes (MDS). It's also used to treat chronic myelomonocytic leukaemia (CMML) and acute myeloid leukaemia (AML).
Azacitidine is used when treatment with a stem cell transplant is not suitable.
This information should ideally be read with our general information about chemotherapy and your type of leukaemia.
Azacitidine is licensed and can be prescribed in the UK. The National Institute for Health and Clinical Excellence (NICE) currently gives advice on which new drugs or treatments should be available on the NHS.
The Scottish Medicines Consortium (SMC) is an organisation similar to NICE for the NHS in Scotland. Azacitidine has been approved by both NICE and SMC for use within the NHS for people who are not able to have a stem cell transplant.
You can read our section on access to cancer treatment for more information on the availability of cancer treatments.
Azacitidine is used to treat some blood conditions that produce abnormal blood cells. It's thought to work by:
stopping abnormal cells from reproducing (growing)
helping to repair damage in abnormal cells.
What azacitidine looks like
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Azacitidine is a white powder that forms a cloudy solution when dissolved in water for injections.
Azacitidine is usually given as a course of several sessions (cycles) of treatment. You’ll usually be given azacitidine as an outpatient. The length of your treatment and the number of cycles you have will depend on the type of MDS or leukaemia you're being treated for. Your nurse or doctor will discuss your treatment plan with you.
Azacitidine is given by injection through a fine needle into the layer of fat under the skin (subcutaneously). It’s usually given every day for one week followed by a three-week break when no treatment is given. This is called a treatment cycle. Because azacitidine is normally given for seven days in a row, you may have to attend hospital at weekends to have the drug.
Before you begin your treatment your doctor will arrange for you to have blood tests. You'll usually be given anti-sickness drugs before and/or during your treatment.
Each person’s reaction to chemotherapy is different. Some people have very few side effects while others may experience more. The side effects described here won’t affect everyone who has azacitidine. Side effects are usually temporary and will gradually disappear once your chemotherapy is over.
We have outlined the most common side effects but haven’t included those that are rare and unlikely to affect you. If you notice any effects that aren’t listed here, discuss them with your doctor, chemotherapy nurse or pharmacist.
Risk of infection
Azacitidine can reduce the number of white blood cells, which help fight infection. White blood cells are produced by the bone marrow. If the number of your white blood cells is low you will be more prone to infections. A low white blood cell count is called neutropenia.
Your resistance to infection will reach its lowest point around 21 days after you start chemotherapy. The number of white blood cells in your blood will then increase gradually, with levels usually returning to normal before your next treatment is due.
Contact your doctor or the hospital straight away if:
your temperature goes above 38°C (100.4°F)
you suddenly feel unwell, even with a normal temperature.
You'll have a blood test before having more azacitidine, to check the number of white blood cells in your blood. Occasionally, it may be necessary to delay your treatment if the number of your blood cells (blood count) is still low.
Bruising or bleeding
Azacitidine can reduce the production of platelets, which help the blood to clot. Tell your doctor if you have any unexplained bruising or bleeding, such as nosebleeds, bleeding gums, blood spots or rashes on the skin. Also let your doctor know straight away if you have black or tarry stools.You may need to have a platelet transfusion if your platelet count is low.
Azacitidine can reduce the number of red blood cells, which carry oxygen around the body. A low red blood cell count is called anaemia. This may make you feel tired and breathless. Tell your doctor or nurse if you have these symptoms. You may need to have a blood transfusion if the number of red blood cells becomes too low.
Feeling sick (nausea) or being sick (vomiting)
Your doctor can prescribe very effective anti-sickness (anti-emetic) drugs to prevent or greatly reduce nausea or vomiting. If the sickness isn't controlled, or if it continues, tell your doctor; they can prescribe other anti-sickness drugs that may be more effective.
Some anti-sickness drugs can cause constipation. Let your doctor or nurse know if this is a problem.
Constipation or diarrhoea
Azacitidine can cause constipation or diarrhoea. Constipation can usually be helped by drinking plenty of fluids, eating more fibre and doing some exercise.
You may need to take medicine (laxatives) to help. Your doctor can prescribe these or you can buy them at a pharmacy.
Diarrhoea can usually be easily controlled with medicine, but tell your doctor if it’s severe or continues. It's important to drink plenty of fluids if you have diarrhoea.
Problems at the injection site
These can include bruising, redness and swelling, which can be uncomfortable or painful. Injections will be rotated between your upper arm, thigh and abdomen (tummy). Using a warm or cool pack may help ease pain and discomfort at the injection site. Your doctor can prescribe painkillers if needed.
Feeling tired is a common side effect of chemotherapy, especially towards the end of treatment and for some weeks after it’s over. It’s important to try to pace yourself and get as much rest as you need. Try to balance this with some gentle exercise, such as short walks, which will help. If tiredness is making you feel sleepy, don’t operate or drive machinery.
Loss of appetite
Some people lose their appetite while they’re having chemotherapy. This can be mild and may only last a few days. If it doesn’t improve you can ask to see a dietitian or specialist nurse at your hospital. They can give you advice on improving your appetite and keeping to a healthy weight. You might find our section on eating well useful.
Less common side effects
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Very rarely people experience an allergic reaction to azacitidine. This happens shortly after injection. Signs of a reaction are a skin rash and itching, a feeling of swelling in the tongue or throat, wheezing, or a cough and breathlessness. Let your nurse or doctor know straight away if you feel unwell as allergic reactions can be effectively treated.
Azacitidine can cause you to feel breathless for a short while. Contact your doctor if this happens.
Pain in joints or muscles
This may be caused by azacitidine. It's important to tell your doctor about any pain, so that they can prescribe painkillers to help.
It’s important to let your doctor know straight away if you feel unwell or have any severe side effects, even if they’re not mentioned above.
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Risk of developing a blood clot
Cancer can increase your risk of developing a blood clot (thrombosis), and having chemotherapy may increase this risk further. A blood clot may cause symptoms such as pain, redness and swelling in a leg, or breathlessness and chest pain. Blood clots can be very serious, so it's important to tell your doctor straight away if you have any of these symptoms. Most clots can usually be successfully treated with drugs that thin the blood.
Some medicines, including those you can buy in a shop or chemist, can be harmful to take when you're having chemotherapy. Tell your doctor about any medicines you're taking, including over-the-counter drugs, complementary therapies and herbal drugs.
It's not advisable to become pregnant or father a child while taking azacitidine as it may harm the developing baby. It’s important to use effective contraception while taking this drug and for at least a few months afterwards. You can discuss this with your doctor.
It’s not known whether chemotherapy drugs can be present in semen or vaginal fluids. To protect your partner, it’s safest to either avoid sex or use a barrier form of contraception for about 48 hours after chemotherapy.
Your ability to become pregnant or father a child is likely to be affected by having azacitidine. It’s important to discuss fertility with your doctor before starting treatment.
There's a potential risk that chemotherapy drugs may be present in breast milk. Women are advised not to breastfeed during chemotherapy and for a few months afterwards.
If you’re admitted to hospital for a reason not related to the cancer, it’s important to tell the doctors and nurses looking after you that you're having chemotherapy treatment. You should tell them the name of your cancer specialist so that they can ask for advice.
It’s a good idea to know who you should contact if you have any problems or troublesome side effects when you’re at home. Your chemotherapy nurse or doctor will give you details of who to contact for advice. This should include ‘out-of hours’ contact details if you need to call someone at evenings, overnight or at the weekend.
This section is based on our Azacitidine factsheet which has been compiled using information from a number of reliable sources, including:
British National Formulary. 62nd edition. 2011. British Medical Association and Royal Pharmaceutical Society of Great Britain.
Sweetman, et al. Martindale: The Complete Drug Reference. 37th edition. 2011. Pharmaceutical Press.
electronic Medicines Compendium (eMC). www.medicines.org.uk (accessed October 2011).
Demakos E P, Linebaugh J A. Advances in myelodysplastic syndrome: nursing implications of azacitidine. Clinical Journal of Oncology Nursing. 2005. 9, 4: 417.