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According to the results of a Phase III clinical trial, treatment with Vidaza® (azacitidine) results in better survival than conventional care among patients with higher-risk myelodysplastic syndromes (MDS). These results were published in Lancet Oncology.

Myelodysplastic syndromes are a group of blood (hematologic) disorders that are diagnosed in 10,000 to 20,000 individuals annually in the United States. MDS occurs when immature blood cells do not mature properly and cannot perform their intended function. They instead crowd out normal blood cells in the bone marrow, often keeping other cells from performing their intended functions. Furthermore, approximately one-third of patients with MDS will have their disease progress to acute myeloid leukemia (AML).

Vidaza was first approved for the treatment of MDS in 2004. The approval was based on improved response rates and reduced need for transfusions; prior to the current study there was not conclusive evidence that Vidaza improved survival.[1]

The current study (AZA-001) involved 358 patients with higher-risk MDS.[2] Patients were treated with either Vidaza or conventional care (best supportive care, low-dose cytarabine, or intensive chemotherapy). Patients have now been followed for a median of 21.1 months.

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  • Median overall survival was 24.5 months for patients treated with Vidaza compared with 15 months for those treated with conventional care.
  • At two years survival was nearly 51% for patients treated with Vidaza compared with 26.2% for those treated with conventional care.

The researchers concluded that treatment with Vidaza significantly improves survival compared with conventional therapy in higher-risk MDS.


[1] Garcia-Manero G. Improving survival in myelodysplastic syndromes. Lancet Oncology. 2009;10:200-201.

[2] Fenaux P, Mufti GJ, Hellstrom-Lindberg E et al. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncology. 2009;10:223-32.