Effects of azacitidine compared with conventional care regimens in elderly (≥ 75 years) patients with higher-risk myelodysplastic syndromes

Abstract

This analysis compared azacitidine (AZA) to conventional care regimens (CCR) and their associated overall survival (OS) and tolerability in the subset of 87 elderly (≥ 75 years) patients with higher-risk MDS (FAB: RAEB, RAEB-t, CMML and IPSS: Int-2 or High) from the AZA-001 trial. Patients were randomized to AZA (75 mg/m(2)/daysubcutaneously × 7 days every 28 days) (n=38) or CCR (n=49) and had median ages of 78 and 77 years, respectively. AZA significantly improved OS vs CCR (HR: 0.48 [95%CI: 0.26, 0.89]; p=0.0193) and 2-year OS rates were 55% vs 15% (p<0.001), respectively. AZA was generally well tolerated compared with CCR, which was primarily best supportive care (67%). Grade 3-4 anemia, neutropenia, and thrombocytopenia with AZA vs CCR were 13% vs 4%, 61% vs 17%, and 50% vs 30%, respectively. Given this efficacy and tolerability, AZA should be considered the treatment of choice in patients aged ≥ 75 years with good performance status and higher-risk MDS.

Trial registration: ClinicalTrials.gov NCT00071799.

Copyright © 2010 Elsevier Ireland Ltd. All rights reserved.

Figures

Fig 1

Patient disposition showing preselection and randomization to treatment.

Fig 2

Overall survival azacitidine (AZA) vs CCR.

Fig 3

Hematologic improvement azacitidine vs CCR (HI-E = hematologic improvement in erythroid lineage; HI-P = platelet lineage; HI-N = neutrophil lineage).

Fig 4

Nadir Hgb, platelet, and ANC values over time: azacitidine vs CCR and preselection to LDAC comparisons.