![[logo] HealthTree Foundation](https://static.healthtree.org/icons/icon_spinner.svg){kind=icon}
![[logo] HealthTree Foundation](https://static.healthtree.org/logo_icon_only.svg){kind=icon}
How it is administered
Azacitidine is given as an injection, either under the skin (subcutaneous) or into a vein (intravenous infusion). It comes as a sterile powder that is mixed with sterile water before use. The typical starting dose for adults is 75 mg/m² once daily for 7 days, repeated every 4 weeks. Your healthcare provider will determine the exact dose and schedule based on your individual needs and how you respond to treatment. Azacitidine is not available as a pill for this use.
How it works
Azacitidine is a type of medicine called a nucleoside metabolic inhibitor. It works by affecting the DNA inside cells, especially abnormal blood cells found in the bone marrow in conditions like chronic myelomonocytic leukemia (CMML). Azacitidine is believed to work in two main ways: it reduces abnormal DNA methylation (a chemical change that can turn genes on or off) and directly kills abnormal blood cells that are growing out of control. By reducing DNA methylation, azacitidine may help restore normal function to genes that control how blood cells grow and mature. Its direct cytotoxic effect helps to eliminate rapidly dividing cancer cells, while having less effect on normal, non-dividing cells. This dual action helps improve blood counts and can reduce the need for blood transfusions in many patients.
Common side effects
Common side effects of azacitidine include:
- Nausea
- Vomiting
- Diarrhea or constipation
- Fatigue or weakness
- Fever (pyrexia)
- Low blood counts (anemia, neutropenia, thrombocytopenia)
- Injection site reactions (redness, pain, swelling, bruising)
- Loss of appetite
- Headache
- Dizziness
- Rash or itching
Some side effects, like low blood counts, can increase your risk of infections or bleeding. Your healthcare team will monitor your blood counts regularly during treatment.
Who Should take it
Azacitidine is approved for adults with certain types of myelodysplastic syndromes (MDS), including chronic myelomonocytic leukemia (CMML). It is used when patients have low blood counts, need frequent transfusions, or have other signs of abnormal blood cell production. Your doctor may recommend azacitidine if you have CMML or another MDS subtype, especially if you are not eligible for a stem cell transplant or need to control symptoms and improve blood counts. It is also used in patients with higher-risk MDS to help prolong survival and reduce the need for transfusions. Treatment is usually continued as long as you are benefiting from it and not experiencing unacceptable side effects.
Who should not take it
You should not take azacitidine if you have advanced malignant liver tumors or if you are allergic to azacitidine or mannitol (an ingredient in the medication). People with severe liver problems are at higher risk for serious side effects. If you have severe kidney problems, your doctor will monitor you closely, as the medication is mainly removed from the body by the kidneys. Azacitidine can cause harm to unborn babies, so it should not be used during pregnancy unless absolutely necessary, and effective birth control is recommended during treatment. Always tell your doctor about any allergies or serious medical conditions before starting azacitidine.
Commonly used with
Azacitidine is often used alongside supportive care measures such as blood transfusions, antibiotics, and medications to manage nausea or infections. It may also be used with other treatments for MDS or CMML, depending on your specific situation, but it is most commonly used as a single agent for these conditions.
Commonly tested with
In clinical studies, azacitidine has been compared to best supportive care (such as transfusions and antibiotics), low-dose cytarabine, and combination chemotherapy regimens. It is sometimes studied in combination with other agents in clinical trials, but for CMML and related blood cancers, it is most often used alone or with supportive care.
