AI Summary & Key Takeaways
AI InsightAzacitidine, a hypomethylating agent, improves overall survival (OS) in higher-risk MDS; however, azacitidine-specific prognostic indicators, including the treatment schedule, remain poorly defined.
📌 3 Key Takeaways
Abstract
Introduction: Myelodysplastic syndromes (MDS) are clonal hematopoietic stem cell neoplasms associated with ineffective hematopoiesis and peripheral cytopenias. Azacitidine, a hypomethylating agent, improves overall survival (OS) in higher-risk MDS; however, azacitidine-specific prognostic indicators, including the treatment schedule, remain poorly defined. Methods: In this retrospective cohort study, 70 MDS patients treated with azacitidine were evaluated. Clinical, laboratory, treatment, and outcome data were analyzed. Survival was estimated using the Kaplan-Meier method, and independent prognostic factors were assessed by multivariable Cox proportional hazards regression. Results: Median OS was 46.9 months (95% CI, 10.2–83.5). On multivariable Cox analysis, hemoglobin ≤9.05 g/dL (HR 3.79, 95% CI 1.57–9.15, p=0.003), LDH ≥300.5 U/L (HR 2.65, 95% CI 1.08–6.52, p=0.033), and high/very high Revised International Prognostic Scoring System (IPSS-R) category (HR 5.61, 95% CI 1.31–24.05, p=0.020) were independently associated with inferior OS. Patients receiving a 7-day azacitidine regimen had shorter OS (HR 4.59, 95% CI 1.02–20.50, p=0.046); however, this group had worse baseline renal function and laboratory parameters and a numerically higher-risk disease profile, and the finding is likely affected by confounding by indication. Elevated ferritin did not retain independent significance after adjustment. Conclusion: Baseline hemoglobin, LDH, and IPSS-R risk category are independent prognostic factors for OS in MDS patients treated with azacitidine. The observed survival difference between the 5-day and 7-day schedules should not be interpreted causally, given the imbalance in baseline risk between the groups. These simple clinical and laboratory parameters may help guide individualized risk stratification and treatment planning.