Real-World Outcome and Prognostic Factors in MDS Patients Treated with Azacitidine—A Retrospective Analysis.

Simple Summary: Azacitidine (AZA) is an essential drug in the treatment of myelodysplastic syndromes (MDS) that has made it possible to extend patients' survival and improve their quality of life. Unfortunately, despite the widespread use of AZA, its prognostic factors for response still remain unknown. Here, we retrospectively analyzed the efficacy and safety of AZA therapy in 79 patients with MDS in a real-life setting. Furthermore, we provided some potential biomarkers of response and survival. The study confirmed that the achievement of response to AZA is not mandatory for obtaining a survival benefit in patients with MDS. Unfavorable cytogenetic risk was determined to be the most negative prognostic factor for both response and survival. Moreover, older age, a complex or monosomal karyotype, higher IPSS or IPSS-R risk and a higher level of serum ferritin level were associated with significantly shorter survival. Azacitidine (AZA) is recognized as a vital drug used in the therapy of myelodysplastic syndromes (MDS) due to its beneficial effect on survival and quality of life. Nevertheless, many patients fail to respond to AZA treatment, as prognostic factors still are not identified. The present retrospective analysis included 79 patients with MDS treated with AZA as first-line therapy in a real-life setting. The percentage of patients with good, intermediate, and poor cytogenetics was 46.8%, 11.4%, and 34.2%, respectively. The overall response rate (complete remission [CR], partial remission [PR], and hematological improvement [HI]) was 24%. The CR, PR, and HI rates were 13.9%, 2.5%, and 7.6%, respectively. Stable disease (SD) was documented in 40.5% of patients. The median overall survival (OS) and progression-free survival (PFS) were 17.6 and 14.96 months, respectively. Patients with ORR and SD had a significantly longer median OS (23.8 vs. 5.7 months, p = 0.0005) and PFS (19.8 vs. 3.5 months, p < 0.001) compared to patients who did not respond to AZA. In univariate analysis, only an unfavorable cytogenetic group was a prognostic factor of a lower response rate (p = 0.03). In a multivariate model, older age (p = 0.047), higher IPSS (International Prognostic Scoring System) risk (p = 0.014), and higher IPSS-R cytogenetic risk (p = 0.004) were independent factors of shorter OS. Independent prognostic factors for shorter PFS were age (p = 0.001), IPSS risk (p = 0.02), IPSS cytogenetic risk (p = 0.002), and serum ferritin level (p = 0.008). The safety profile of AZA was predictable and consistent with previous studies. In conclusion, our study confirms the efficacy and safety of AZA in a real-world population and identifies potential biomarkers for response and survival. [ABSTRACT FROM AUTHOR]