title: Review of azacitidine trials in Intermediate-2-and High-risk myelodysplastic syndromes.
abstract: The efficacy of azacitidine, a cytosine nucleoside analog, has been demonstrated in two large phase III trials in myelodysplastic syndromes (MDS) patients, primarily in higher-risk patients. In the Cancer and Leukemia Group B (CALGB) study (lower-and higher-risk MDS patients; n = 191) azacitidine significantly prolonged median time-to-progression to acute myeloid leukemia or death (21 vs 13 months for supportive care; p = 0.007). The AZA-001 trial (Intermediate-2-and High-risk MDS patients; n = 358) showed azacitidine significantly prolonged median overall survival compared with conventional care regimens (24.5 vs 15.0 months; p = 0.0001). These results establish azacitidine as a reference first-line treatment in patients with Intermediate-2-and High-risk MDS who are not immediate candidates for allogeneic stem cell transplantation.
authors.affiliation: Service d'Hématologie Clinique, Hôpital Avicenne, Assistance Publique-Hôpitaux de Paris, Université Paris XIII, Bobigny, France. pierre.fenaux@avc.aphp.fr
authors.forename: Pierre
authors.initials: P
authors.lastname: Fenaux
authors.forename: Lionel
authors.initials: L
authors.lastname: Ades
mesh_terms: D000368:Aged; D000964:Antimetabolites, Antineoplastic / Q000627:therapeutic use*; D001374:Azacitidine / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D005260:Female; D006801:Humans; D008297:Male; D009190:Myelodysplastic Syndromes / Q000188:drug therapy*
chemical_list: D000964:Antimetabolites, Antineoplastic; D001374:Azacitidine