Key clinical point: Oral azacitidine (CC-486) was associated with a significantly higher red blood cell (RBC) transfusion independence (TI) rate in patients with low-risk myelodysplastic syndrome (MDS) and high-risk disease features. The rate of early death was higher in the CC-846 arm.
Major finding: A significantly higher proportion of patients achieved RBC TI for 56 consecutive days in CC-486 vs. placebo arms (30.8% vs. 11.1%; odds ratio, 3.6; P = .0002). Overall, the death rate was similar, but death within the first 56 days was higher with CC-486 vs. placebo (16 vs. 6), most related to infections in patients with significant pretreatment neutropenia.
Study details : Findings are from AZA-MDS-003, a phase 3 trial involving 216 patients with low-risk MDS and RBC transfusion-dependent anemia and thrombocytopenia who were randomly allocated to either CC-486 (n=107) or placebo (n=109) for 21 days/28-day cycle.
Disclosures: The study was supported by Celgene Corporation, a Bristol-Myers Squibb company. Some of the authors declared receiving research funding, honoraria, travel and accommodation expenses, consulting/advisory role, being an employee of and/or stock/other ownership interest in various pharmaceutical companies including Celgene.
Source: Garcia-Manero G et al. J Clin Oncol. 2021 Mar 25. doi: 10.1200/JCO.20.02619.