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Trade/other name(s): Vidaza, azacytidine, 5-azacytidine, AZA

Why would this drug be used?

Azacytidine is used to treat myelodysplastic syndromes, which are diseases of the bone marrow. It is used to treat several types of cancer, including chronic myelomonocytic leukemia.

How does this drug work?

Azacitidine is a chemotherapy drug known as an anti-metabolite. The drug prevents the body from making DNA and RNA that cells need to grow. This stops the growth of cancer cells and causes them to die. Azacitidine also appears to help restore normal growth and differentiation (cell specialization) in the cells of the bone marrow. This makes the bone marrow better able to make red blood cells, white blood cells, and platelets.

Before taking this medicine

Tell your doctor…

  • If you are allergic to anything, including medicines, dyes, additives, or foods.
  • If you have any medical conditions such as kidney disease, liver disease (including hepatitis), heart disease, congestive heart failure, diabetes, gout, or infections. These conditions may require that your medicine dose, regimen, or timing be changed.
  • If you have cancer in your liver. The drug can worsen liver function.
  • If you are pregnant, trying to get pregnant, or if there is any chance of pregnancy. This drug may cause birth defects if either the male or female is taking it at the time of conception or during pregnancy. Check with your doctor about what kinds of birth control can be used with this medicine.
  • If you are breast-feeding. It is not known whether this drug passes into breast milk. If it does, it could harm the baby.
  • If you think you might want to have children in the future. Some drugs can cause sterility. Talk with your doctor about the possible risk with this drug and the options that may preserve your ability to have children.
  • About any other prescription or over-the-counter medicines you are taking, including vitamins and herbs. In fact, keeping a written list of each of these medicines (including the doses of each and when you take them) with you in case of emergency may help prevent complications if you get sick.

Interactions with other drugs

Any drugs or supplements that interfere with blood clotting can raise the risk of bleeding during treatment with azacitidine. These include:

  • Vitamin E
  • Non-steroidal anti-inflammatory drugs (NSAIDs) such as aspirin, ibuprofen (Advil, Motrin), naproxen (Aleve, Naprosyn), and many others
  • Warfarin (Coumadin)
  • Ticlopidine (Ticlid)
  • Clopidogrel (Plavix)

Note that many cold, flu, fever, and headache remedies contain aspirin or ibuprofen. Ask your pharmacist if you aren't sure what's in the medicines you take.

Check with your doctor, nurse, or pharmacist about other medicines, vitamins, herbs, and supplements, and whether alcohol can cause problems with this medicine.

Interactions with foods

No serious interactions with food are known at this time. Check with your doctor, nurse, or pharmacist about whether foods may be a problem.

Tell all the doctors, dentists, nurses, and pharmacists you visit that you are taking this drug.

How is this drug taken or given?

Azacitidine is given as a shot (injection) under the skin every day for 7 days. This is repeated every 4 weeks for at least 4 cycles of treatment. The drug can also be infused into a vein (intravenously or IV) over a period of 20-60 minutes, or continuously for up to 5 days for some cancer treatments. The dose depends on how tall you are and what you weigh, how well your kidneys work, and blood counts. You or a family member can be taught to give the injection under the skin at home.

If you take it at home, don't let the liquid medicine splash onto the skin. If it does, wash right away with soap and water. If it gets into eyes, nose, mouth, or other mucous membranes, flush with plenty of water.

Store the medicine in its original container in the refrigerator, and keep the shot equipment in a safe place away from children, pets, and others. Store the used needles in a closed needle bucket and take them back to your doctor or nurse.


Your doctor will likely test your blood throughout your treatment, looking for possible effects of the drug on blood counts or on other body organs. Based on the test results, you may be given medicines to help treat any effects. Your doctor may also need to reduce or delay your next dose of this drug, or even stop it completely. Be sure to keep all your appointments for lab tests and doctor visits.

This drug can lower your white blood cell count, especially in the weeks after the drug is given. This can increase your chance of getting an infection. Be sure to let your doctor or nurse know right away if you have any signs of infection, such as fever (100.5° or higher), chills, pain when passing urine, a new cough, or bringing up sputum.

This drug may lower your platelet count in the weeks after it is given, which can increase your risk of bleeding. Speak with your doctor before taking any drugs or supplements that might affect your body's ability to stop bleeding, such as aspirin or aspirin-containing medicines, warfarin (Coumadin), or vitamin E. Tell your doctor right away if you have unusual bruising, or bleeding such as nosebleeds, bleeding gums when you brush your teeth, or black, tarry stools.

This drug may lower your red blood cell count. If this occurs, it is usually a few weeks after starting treatment. A low red blood cell count (known as anemia) can cause shortness of breath, or make you to feel weak or tired all the time. Your doctor may give you medicines to help prevent or treat this condition, or you may need to get blood transfusions.

Do not get any immunizations (vaccines), either during or after treatment with this drug, without your doctor's OK. Azacitidine may affect your immune system. This could make vaccinations ineffective, or could even lead to serious infections if you get a live virus vaccine during or soon after treatment. Try to avoid contact with people who have recently received a live virus vaccine, such as the oral polio vaccine or smallpox vaccine. Check with your doctor about this.

You may have nausea and vomiting on the day you receive this drug or in the first few days afterward. Your doctor may give you medicine before your treatment to help prevent nausea and vomiting. You will likely also get a prescription for an anti-nausea medicine that you can take at home. It is important to have these medicines on hand and to take them as prescribed by your doctor. Let your doctor know if these drugs do not control your vomiting.

This drug can cause the rapid killing of tumor cells, which in some cases has led to a serious imbalance of electrolytes in the blood, and even kidney damage within the first 3 days of treatment. This condition is known as tumor lysis syndrome. It is more likely if you have a large number of cancer cells in the body. If your doctor thinks you might be at risk, he or she will give you medicines and/or fluids to help prevent it.

Men and women should avoid conceiving a child while taking this drug and for some time after. Talk to your doctor about this.

Possible side effects

You will probably not have most of the following side effects, but if you have any talk to your doctor or nurse. They can help you understand the side effects and cope with them.


  • Low platelet count with increased risk of bruising or bleeding*
  • Low red blood cell count (anemia) with symptoms like tiredness (fatigue), shortness of breath, fast heartbeat, pale skin or lips*
  • Nausea*
  • Vomiting*
  • Diarrhea
  • Constipation
  • Tiredness
  • Irritation, itching, or redness at the injection site
  • Fever

Less common

  • Low white blood cell count with increased risk of infection*
  • Loss of appetite
  • Shortness of breath
  • Headache
  • Dizziness
  • Bruising or bleeding
  • Muscle aches
  • Chest or stomach pain
  • Skin rash
  • Nervousness, anxiety, or sadness
  • Weakness
  • Trouble sleeping
  • High blood pressure
  • Weight loss
  • Low potassium level in the blood (might cause weakness, tingling, muscle cramps, and other problems)
  • Pain or swelling in the arms or legs
  • Infections


  • Sores in mouth or on lips
  • Feeling sleepy or drowsy
  • Confusion or coma (very rare)
  • Rash with itching
  • Low blood pressure, which can make you feel dizzy or lightheaded
  • Kidney damage*
  • Liver damage
  • Skin damage, ulcer, or sore at the place the drug was injected
  • Severe allergic reaction, with symptoms like itching, hives, swelling in the mouth or throat, or shock
  • Death due to liver damage (for those who had cancer in the liver or prior liver damage) or kidney damage

*See "Precautions" section for more detailed information.

There are other side effects not listed above that can also occur in some patients. Tell your doctor or nurse if you develop these or any other problems.

FDA approval

Yes – first approved in 2004

Disclaimer: This information does not cover all possible uses, actions, precautions, side effects, or interactions. It is not intended as medical advice, and should not be relied upon as a substitute for talking with your doctor, who is familiar with your medical needs.

Last Medical Review: 02/16/2012
Last Revised: 02/16/2012