Single-center phase 1-2 trial of omacetaxine and azacitidine for previously untreated myelodysplastic syndromes with excess blasts

Patients with myelodysplastic syndromes with excess blasts (MDS-EB) have poor long-term outcomes. Preclinical studies showed that MDS-EB stem cells are dependent upon protein synthesis. A phase 1-2 trial at the University of Colorado (Aurora) evaluated the protein synthesis inhibitor omacetaxine mepesuccinate in combination with the hypomethylating agent azacitidine for previously untreated MDS-EB. The overall response rate was 54% with complete remission in 17%. The median duration of response was 719 days. The median progression-free survival and overall survival were 92 days and 1.5 years, respectively. The authors conclude that the combination of omacetaxine and azacitidine could be further studied for patients with MDS-EB.