According to a press release by Pharmion, Vidaza™ (azacitidine) significantly improves survival compared with conventional care in the treatment of myelodysplastic syndromes.

Myelodysplastic syndromes (MDS) are a group of blood (hematologic) disorders that are diagnosed in 10,000 to 20,000 individuals annually in the U.S. 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.

Vidaza was the first agent approved for the treatment of MDS in 2004. It applies its anticancer effects by targeting aspects of expression of DNA, ultimately killing cancer cells. Vidaza has demonstrated the ability to reduce or prevent the need for blood transfusions in MDS; however, its effects on survival have not been clearly established.

Researchers from several institutions recently conducted another clinical trial to further evaluate Vidaza in the treatment of MDS. This Phase III trial included 350 patients with MDS who were at a high risk of disease progression. Patients were treated with one of four treatment options: Vidaza, best supportive cancer, low-dose chemotherapy including cytarabine plus best supportive care, or standard chemotherapy plus best supportive care.

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  • Median survival was 24.4 months for patients treated with Vidaza compared with 15 months for those receiving other regimens.
  • At two years survival was 51% for patients treated with Vidaza compared with 26.2% for those treated with other therapies.
  • The researchers stated that survival benefits of Vidaza were “consistent regardless of the conventional care treatment option” and that “survival should now be the standard by which we evaluate treatment options for higher-risk MDS.”

The researchers concluded that Vidaza improves survival compared with standard treatment options for patients with MDS. Vidaza is the only agent to date that has demonstrated a survival benefit for MDS compared with conventional care therapies. Patients with MDS may wish to speak with their physician regarding their individual risks and benefits of treatment with Vidaza.

Reference: Pharmion. Vidaza significantly extends overall survival by 74% in Phase 3 trial in myelodysplastic syndromes (MDS). Accessed August 2007. Available