How it is administered

Azacitidine is administered either as a subcutaneous injection (under the skin) or as an intravenous infusion (into a vein). It comes as a sterile powder that is reconstituted with sterile water before use. The recommended starting dose for adults with myelodysplastic syndromes (MDS) is 75 mg/m² daily for 7 days, repeated every 4 weeks. The medication is given in cycles, and treatment may continue as long as the patient benefits from it. Your healthcare provider will determine the exact dosing and method of administration based on your specific needs.

How it works

Azacitidine is a type of chemotherapy known as a nucleoside metabolic inhibitor. It works by interfering with the DNA in abnormal blood cells in the bone marrow. Specifically, azacitidine is a pyrimidine nucleoside analog of cytidine. It is believed to work in two main ways:

First, it causes hypomethylation of DNA, which can restore the normal function of genes that are important for cell differentiation and growth. This means it may help abnormal cells behave more like normal cells.

Second, azacitidine has a direct toxic effect on abnormal blood cells, particularly those that are dividing rapidly and are not responding to normal growth controls. This helps to reduce the number of abnormal cells in the bone marrow, allowing healthier blood cells to grow.

Overall, azacitidine helps improve blood counts, reduce the need for transfusions, and may prolong survival in people with certain types of MDS.

Who Should take it

Azacitidine is indicated for adults with specific subtypes of myelodysplastic syndromes (MDS), including:

• Refractory anemia (RA)
• Refractory anemia with ringed sideroblasts (RARS), if accompanied by low white blood cells, low platelets, or the need for transfusions
• Refractory anemia with excess blasts (RAEB)
• Refractory anemia with excess blasts in transformation (RAEB-T)
• Chronic myelomonocytic leukemia (CMMoL)

It is used in patients who have abnormal blood counts, require frequent transfusions, or have a higher risk of progression to acute myeloid leukemia (AML). Azacitidine can help improve blood counts, reduce transfusion needs, and may improve survival for these patients.

Who should not take it

Azacitidine should not be taken by:

• Patients with advanced malignant hepatic (liver) tumors
• Patients with a known hypersensitivity to azacitidine or mannitol (an ingredient in the formulation)

Caution is also needed in patients with severe pre-existing liver or kidney problems, as they may be at higher risk for side effects. Azacitidine can cause harm to unborn babies, so it should not be used during pregnancy unless absolutely necessary. Women of childbearing potential and men with partners of childbearing potential should use effective contraception during treatment and for a period after the last dose.

Commonly used with

Azacitidine is often used as a single agent for the treatment of MDS. However, supportive care measures such as blood transfusions, antibiotics, antiemetics (for nausea), and growth factors may be used alongside azacitidine to help manage symptoms and side effects. It is not typically combined with other chemotherapy agents for MDS, but your doctor may recommend additional supportive treatments based on your individual needs.

Commonly tested with

In clinical studies, azacitidine has been compared to best supportive care, low-dose cytarabine, and intensive chemotherapy regimens (such as cytarabine with anthracyclines) in patients with MDS. It has also been studied in combination with supportive care measures like transfusions and antibiotics. The main focus of these studies has been to evaluate its effectiveness in improving blood counts, reducing transfusion needs, and prolonging survival in patients with higher-risk MDS.