Azacitidine for the treatment of myelodysplastic syndromes (MDS).pub

Azacitidine is a pyrimidine nucleoside analogue with multiple mechanisms involved in its antineoplastic action. There is evidence that RNA metabolism is the primary target of this antimetabolite, although its inhibition of DNA methylation has been proposed as the main effect responsible for its clinical efficacy in myelodysplastic syndromes. In patients with myelodysplastic syndromes (MDS), azacitidine appears to restore normal growth and differentiation of bone marrow cells by causing hypomethylation of DNA and direct cytotoxicity on abnormal haematopoietic cells in the bone marrow. Hypomethylation may permit the normal functioning of genes that regulate differentiation and proliferation.