azacitidine (ay-zuh-SY-tih-deen) is a drug used to treat myelodysplastic syndromes. It is also being studied in the treatment of other conditions and types of cancer.
How does it work?
Liver safety of Azacitidine
Azacitidine is associated with a low rate of transient serum enzyme elevations during therapy and has only rarely been implicated in cases of clinically apparent acute liver injury with jaundice.
Mechanism of action of Azacitidine
Azacitidine (ay" za sye' ti deen: also spelled azacytidine) is a pyrimidine analogue (5-azacytidine) which is converted intracellularly to a triphosphate which becomes incorporated into RNA and DNA. While azacitidine has anticancer effects, it proved to have limited usefulness in solid tumors and lymphomas. In low doses, azacitidine inhibits methylation of DNA and results in the expression of silenced genes, including tumor suppressor genes. Studies done in vitro and in vivo have shown that azacitidine induces differentiation of bone marrow cells and results in normalization of bone marrow in a proportion of patients with myelodysplasia.
FDA approval information for Azacitidine
Azacitidine was approved for use in the United States in 2004 and the current single indication is for therapy of myelodysplastic syndromes. It is also under evaluation as therapy of acute myelogenous leukemia. Azacitidine is available as a powder for injection in 100 mg vials under the trade name of Vidaza. An oral formulation is currently under evaluation.
Dosage and administration for Azacitidine
The usual initial dosage regimen in adults is 75 mg per meter-squared body surface area subcutaneously each day for 7 days, with repeat courses after 28 days. A minimum of 4 courses is recommended and the dose can be increased based upon tolerance and response.
Side effects of Azacitidine