Multinational phase 3 study of oral azacitidine for lower-risk MDS

The multinational phase 3 study AZA-MDS-003 evaluated CC-486 (oral azacitidine) versus placebo for lower-risk myelodysplastic syndromes with red blood cell tranfusion-dependent anemia and thrombocytopenia. CC-486 resulted in improved transfusion independence and induced durable improvements in hemoglobin concentrations and platelet counts. More early deaths occurred in the CC-486 arm, most related to infections in patients with significant pretreatment neutropenia.