Alternative 5-Azacitidine 5-Day 100 mg/m

Introduction: 5-Azacitidine (AZA) is a major treatment option for myelodysplastic neoplasms (MDS) and myelodysplastic/myeloproliferative neoplasms (MDS/MPN). Here we evaluate the efficacy and toxicity of an alternative AZA regimen (100 mg/m Results: Median patient age was 66 years, with most patients (98%) having intermediate or high-risk disease. Overall response rate (ORR) was 62% with 22% complete responses (CR). Median OS and median PFS were 22.5 and 18.2 months, respectively. Inferior response rates were calculated in therapy-related MDS (t-MDS) and MDS with excess blast II, with t-MDS having also statistically worse OS and PFS. MDS/MPN patients showed 73.6% ORR with 31.5% CR. Transfusion independence (TI) for red blood cells (RBC) was achieved in 45.9% of transfusion-dependent patients and in 30% for platelets. CR patients showed longer mOS and mPFS (70.6 and 64.7 months, respectively). Longer mOS was also correlated with allogeneic transplantation (48.8 vs. 16.9 months, Conclusion: This study demonstrates that this alternative AZA regimen has comparable efficacy and safety to the standard regimen, compared with historical data. Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.