Indications
Azacitidine is a nucleoside metabolic inhibitor. It is used to treat patients with the following FAB subtypes of myelodysplastic syndrome (MDS): refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS), if the patient also has neutropenia or thrombocytopenia or needs transfusions. It is also used for refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukemia (CMMoL).
⚠️ Always consult a registered physician before taking any medicine.
Pharmacology
Azacitidine is a pyrimidine nucleoside analog of cytidine, and it works by inhibiting DNA and RNA methyltransferases. Once it enters the cell, enzymes convert it into nucleotide triphosphates, and these are then built into DNA and RNA.
In laboratory studies on cancer cells, including acute myeloid leukemia cells, azacitidine that had been incorporated into DNA inhibited DNA methyltransferases. This reduced DNA methylation and altered gene expression, including re-expression of genes that regulate tumor suppression and cell differentiation. When azacitidine was incorporated into the RNA of cancer cells, including leukemic cells, it inhibited RNA methyltransferases, reduced RNA methylation, decreased RNA stability and decreased protein synthesis.
Antileukemic activity of azacitidine was shown in AML cell lines in vitro, where it reduced cell viability and induced apoptosis. In leukemic tumor models in vivo, azacitidine decreased tumor burden and increased survival.
Pregnancy & Breastfeeding
Pregnancy: Azacitidine can cause harm to an unborn baby when given to a pregnant woman. This is based on how the medicine works and on what was seen in animals. There are no data on Azacitidine use in pregnant women, so the risk linked to the medicine can't be properly assessed. In animals, Azacitidine was teratogenic and caused embryo-fetal lethality at doses lower than the recommended human daily dose of oral Azacitidine on a mg/m2 basis. Pregnant women should be told about the possible risk to the baby. The background risk of major birth defects and miscarriage for the population this medicine is given to is unknown. Every pregnancy carries some background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.
Lactation: There are no data on whether Azacitidine passes into human milk, or on its effects on a breastfed child or on milk production. Because the breastfed child could have serious adverse reactions, women should be advised not to breastfeed during treatment with Azacitidine and for 1 week after the last dose.
⚠️ Talk to your doctor before taking any medicine during pregnancy or while breastfeeding.
Dosage Reference
⚠️ Reference ranges from official sources only. Consult your doctor for personalized dosing.