AZA-001
Trial question
What is the effect of azacitidine in patients with higher-risk myelodysplastic syndromes?
Study design
Multi-center
Open label
RCT
Population
Characteristics of study participants
30.0% female
70.0% male
N = 358
358 patients (107 female, 251 male)
Inclusion criteria: patients with higher-risk myelodysplastic syndromes
Key exclusion criteria: therapy-related myelodysplastic syndrome, previous azacitidine treatment, or planned allogeneic stem-cell transplantation
Interventions
N=179 azacitidine (75 mg/m² per day for 7 days every 28 days)
N=179 conventional care (best supportive care, low-dose cytarabine, or intensive chemotherapy)
Primary outcome
Overall survival
24.5
15
24.5
18.4
12.3
6.1
0.0
Azacitidine
Conventional care
Significant increase ▲
Significant increase in overall survival (24.5 vs. 15 ; HR 1.72, 95% CI 1.3 to 2.33)
Secondary outcomes
Significant increase in median time to acute myeloid leukemia transformation (17.8 vs. 11.5 ; HR 2, 95% CI 1.43 to 2.86)
Safety outcomes
Significant differences in grade 3 or 4 toxicity, including neutropenia (91% vs. 76%), thrombocytopenia (85% vs. 80%) and anemia (57% vs. 68%).
Conclusion
In patients with higher-risk myelodysplastic syndromes, azacitidine was superior to conventional care with respect to overall survival.
Reference
Fenaux P, Mufti GJ, Hellstrom-Lindberg E et al. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32.
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