The results from AZA-001 are published in The Lancet Oncology. The data indicate that Vidaza ( Azacitidine ) demonstrated a significant extension of overall survival compared to conventional care regimens ( CCR ) for patients with intermediate-2 and high-risk myelodysplastic syndromes ( MDS ) and acute myeloid leukemia ( AML ) with 20 to 30% bone marrow blasts.
The median overall survival for patients treated with Azacitidine ( n=179 ) was 24.5 months compared to 15 months for those receiving CCR treatment ( n=179 ), an improvement of 9.5 months ( p=0.0001 ).
CCR includes best supportive care, low-dose ARA-C, and standard chemotherapy.
There was a 42% reduction in the risk of death ( hazard ratio, HR=0.58 ). The two-year survival rate was nearly doubled at 50.8% for patients receiving Azacitidine versus 26.2% for patients receiving CCR ( p=0.0001 ). The extension of survival was seen across the relevant patient subgroups including those greater than 65 years of age and those with poor cytogenetics, a poor prognostic factor. In addition, 34% of patients with AML as classified by the World Health Organization ( WHO ) saw a survival benefit.
In addition, 45% of patients achieved red blood cell transfusion independence versus 11% of patients receiving CCR treatment ( p less than 0.0001 ) and 85.3% of patients treated with Azacitidine who were red-blood-cell transfusion independent at baseline remained so. Investigators aimed to treat patients until disease progression; the median number of cycles of Azacitidine was nine.
The clinical data from this international study comparing Azacitidine to conventional care regimens demonstrated for the first time a marked improvement in overall survival for intermediate-2 and high-risk MDS patients.
As previously reported with Azacitidine, the most common haematologic adverse events were neutropenia, thrombocytopenia and anaemia. The most common non-haematoligic events were injection site reactions, nausea, vomiting, fatigue and diarrhea. The rates of bleeding complications and infections were similar with best supportive care and risk of infection requiring intravenous antibiotic use was reduced by one-third with Azacitidine compared to conventional care regimens. ( Xagena )
Source: Celgene, 2009