5-AZACITIDINE IN HIGH RISK MYELODYSPLASTIC SYNDROME-SINGLE CENTER EXPERIENCE

Abstract

Introduction

Myelodysplastic syndrome (MDS) represents a group of malignant clonal hematological disorders characterized by dysplasia in one or more hematopoietic lineages in the bone marrow, leading to cytopenias and an increased risk of developing acute myeloid leukemia. The only treatment option that has shown benefit in PFS and OS for high-risk MDS is 5-azacitidine (5-AZA).

Methods

The aim of this study was to determine the impact of 5-AZA on the treatment outcome of patients with high-risk MDS from January 1, 2013, to December 31, 2021, at the Clinical Hospital Merkur in Zagreb, Croatia. Retrospective analysis was performed on data from patients with high-risk MDS. The study included 38 patients (M:F=25:13), at time of study conclusion, 34 patients died. Laboratory data and overall survival of the patients were analyzed. Data were analyzed using the Kaplan-Meier method, Log-rank test, and Mann-Whitney U test.

Results

A longer overall survival was observed in patients treated with more than 12 cycles of 5-AZA. The median survival of the group receiving more than 12 cycles of 5-AZA was 24 months, while the median survival of patients receiving less than 12 cycles was 11 months (p=0.023). Higher creatinine levels at diagnosis and lower LDH levels before the first cycle were observed in the group of surviving patients.

Conclusions

The results of this study highlight the efficacy of 5-AZA in the first-line treatment of high-risk MDS patients, with a statistically significant difference in overall survival observed in group of HR MDS patients receiving at least 12 cycles of 5-AZA.