A subset analysis of the international phase III trial ( AZA-001 ) has demonstrated that the overall survival benefit observed in higher-risk myelodysplastic syndromes ( MDS ) patients extended to patients with acute myeloid leukemia ( AML ). The data, presented at the 50th Annual Meeting of the American Society of Hematology ( ASH ), reported that patients with WHO-defined AML who were treated with Azacitidine ( Vidaza ) achieved significantly improved overall survival compared to those treated with a conventional care regimen.
The AZA-001 trial recently showed that treatment with Vidaza provides an unprecedented survival benefit in patients with higher-risk myelodysplastic syndromes. Based on these results, Vidaza received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use ( CHMP ) for certain patients with higher-risk myelodysplastic syndromes and WHO-defined acute myeloid leukemia and gained an approval from the FDA ( Food and Drug Administration ) for an expanded label to include the survival data in higher-risk myelodysplastic syndromes.
Almost one third of the patients ( 113 of 358 ) enrolled in the AZA-001 study met the WHO criteria for acute myeloid leukemia ( 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 Azacitidine compared to 16.0 months with conventional care regimen ( p=0.005 ). Additionally, 50 percent of the acute myeloid leukemia patients who were treated with Azacitidine survived at least two years, compared to only 16 percent of AML patients treated with conventional care regimen.
The subset analysis also showed that patients treated with Azacitidine had fewer infections requiring intravenous antibiotics and reduced rates of hospitalization and red blood cell transfusions.
In the AZA-001 study, the most commonly occurring adverse reactions were thrombocytopenia ( 69.7% ), neutropenia ( 65.7% ) and anemia ( 51.4% ). ( Xagena )
Source: Celgene, 2008