6/28/23, 12:51 AM AZA-001 Pathway Feedback Search Clinical Topics Home Studies AZA 001 AZA 001 Disease Myelodysplastic syndrome 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 N = 358 70.0% male 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/m 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 24.5 https://web.pathway.md/studies/recurY9tid9NVwf5y 1/2 6/28/23, 12:51 AM AZA-001 Pathway 5 18.4 15 12.3 6.1 Significant increase 0.0 Azacitidine Conventional care 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. Open reference URL https://web.pathway.md/studies/recurY9tid9NVwf5y 2/2