On May 19, 2004 the U.S. Food and Drug Administration approved azacitidine as injectable suspension (Vidaza) for treatment of patients with the following MDS subtypes: RA or RARS (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), RAEB, RAEB-T, and CMML. Azacitidine is the first agent approved for treatment of myelodysplastic syndrome. It is now in trials for acute meyelogenous leukemia – AML. Subset analysis of the international phase III trial (AZA-001) demonstrating that the overall survival benefit observed in higher-risk MDS patients extended to patients with acute myeloid leukemia (AML).
Almost one third of the patients (113 of 358) enrolled in the AZA-001 study met the WHO criteria for AML (median 23% bone marrow blasts), which has a poor prognosis and does not respond well to conventional chemotherapy. This subset analysis of the AZA-001 study showed the median overall survival was 24.5 months with VIDAZA compared to 16.0 months with CCR (p=0.005). Additionally, 50 percent of the AML patients who were treated with VIDAZA survived at least two years, compared to only 16 percent of AML patients treated with CCR.
NCCN recommends low intensity maintenance with drugs that include azacytidine as an option on p. AML-11.
Gemtuzumab ozogamicin (GO) (Mylotarg, CMA-676) is a novel chemotherapeutic agent consisting of an anti-CD33 monoclonal antibody linked to calicheamicin, and is associated with a 30% response rate in patients with CD33-positive acute myeloid leukemia (AML) in first relapse.
nccn, AML 2012
Edlin R, Connock M, Tubeuf S, Round J, Fry-Smith A, Hyde C, Greenheld W. zacitidine for the treatment of myelodysplastic syndrome, chronic myelomonocytic leukaemia and acute myeloid leukaemia. Health Technol Assess. 2010 May;14 Suppl 1:69-74.