How it Works

In acute myeloid leukemia (AML), the bone marrow produces abnormal cells that cannot mature and do not function properly. Resulting in abnormal cell build-up in the bone marrow and blood. 

Azacitidine is an antimetabolite, which means that it works by disrupting the DNA production of these abnormal cells (hypomethylation), the genetic material that controls the growth and function of these cells. Causing the death of rapidly dividing cells, including cancer cells that are no longer responsive to normal growth control mechanisms. This could also restore normal function to genes critical for differentiation and proliferation.

How it’s Administered

Oral pills: The pills are usually taken once a day, and the patient should take them at the same time each day. It is important to swallow the pills whole and not crush or chew them.

Who Should Take Azacitidine

• Patients with acute myeloid leukemia that have already had intensive chemotherapy and have reached a point of complete or almost complete remission, and are not fit for intensive therapy or stem cell transplantation.
• Patients with refractory anemia or refractory anemia with ringed sideroblasts, red blood cells with abnormal amounts of iron (if accompanied by low neutrophil or platelets counts requiring transfusions)
• Refractory anemia with excess blasts (immature cells)
• Refractory anemia with excess blasts in transformation (now classified as acute myelogenous leukemia with multilineage dysplasia)
• Patients with chronic myelomonocytic leukemia 

Who Shouldn’t Take Azacitidine 

• Pregnant women. It is important to inform your healthcare provider if you are pregnant, or planning to become pregnant before starting treatment, as it may cause harm to the fetus.
• Patients in reproductive age must use a contraceptive method during treatment and after 3 months of concluding the therapy. 
• Patients with advanced malignant tumors in the liver
• Breastfeeding women should suspend breastfeeding if they start taking this medication. 
• People who are allergic to the medication or any of its components.

The Most Common Side Effects of Taking Azacitidine Include:

• Decreased blood cell counts: Your levels of platelets, white and red blood cells may temporarily drop. Which may cause, fatigue, weakness, shortness of breath, increased risk of infections, bleeding or bruising. 
• Gastrointestinal effects like nausea, vomit, constipation, abdominal pain
• Injection site reactions 
• Appetite loss
• Dyspnea (difficulty breathing) 
• Lung infections, sore throat or fever

Commonly Used in Acute Myeloid Leukemia With

• Venetoclax
• ​​Gemtuzumab ozogamicin
• Low-dose cytarabine
• As a stand-alone single-drug therapy for maintenance.

TAKE A LOOK AT THE AVAILABLE CLINICAL TRIALS FOR AML

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Date last updated: 12/22/22