Abstract

In recent years, researches on the mechanisms of methylation and demethylation treatment strategies have brought new ideas for treatment of myelodysplastic syndrome (MDS) . Azacitidine and decitabine were approved by United States Food and Drug Administration(FDA) for the treatment of MDS. Chinese researchers have a preliminary understanding of the clinical applications of decitabine after 5 years exploration. Azacitidine can improve the quality of life of high-risk patients with MDS, and prolong overall survival (OS) and the median time of acute myeloid leukemia(AML) transformation, especially in high-risk MDS patients over 65 years old compared with decitabine. Azacitidine maintenance therapy after hematopoietic stem cell transplantation(HSCT) can delay the recurrence. In addition, application of low-dose azacitidine for low-risk MDS patients is still in exploring. Azacitidine has been combined with other novel agents such as histone deacetylase(HDAC) inhibitors, lenalidomide and infliximab monoclonal antibody are very promising in clinical researches. However, azacitidine has not been approved by China Food and Drug Administration (CFDA) and lacks clinical experience. This article reviews literatures on exploring optimal dose, efficacy of different subtypes of MDS and prospects of azacitidine combination therapy. Key words: Azacitidine; Myelodysplastic syndromes; Drug therapy

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