[Efficacy and Safety of Venetoclax Combined with Azacitidine versus Azacitidine Monotherapy in the Treatment of Higher-Risk Myelodysplastic Syndromes].

Abstract

Objective: To compare the efficacy and safety of venetoclax (VEN) combined with azacitidine (AZA) regimen versus AZA monotherapy regimen in the treatment of patients with higher-risk myelodysplastic syndromes (HR-MDS).

Methods: The clinical data of 62 patients with HR-MDS who were treated at the Affiliated Hospital of Xuzhou Medical University from April 2020 to June 2024 were retrospectively collected. According to the treatment regimen, the patients were divided into the combined therapy group (VEN plus AZA) and the monotherapy group (AZA alone). The efficacy, adverse reactions, and survival outcomes of the two groups were compared.

Results: The overall response rate (ORR) of the combination therapy group was 79.2% (19/24), which was significantly higher than that of the monotherapy group (39.5%, 15/38) (P＜0.05). Among these responses, 45.8% (11/24) of patients in the combination therapy group achieved marrow complete remission (mCR), which was significantly higher than the 18.4% (7/38) in the monotherapy group (P＜0.05). The median number of cycles to response in the combination therapy group was 1 (range, 1-2), which was significantly shorter than the 2 (range, 1-3) cycles in the monotherapy group (P＜0.05). Regarding safety, the incidence rates of grade≥3 anemia and neutropenia in the combination therapy group were significantly higher than those in the monotherapy group (P＜0.05). Additionally, there were no statistically significant differences in the incidence rates of non-hematological adverse events (including gastrointestinal reactions, secondary infections, liver and kidney dysfunction, and allergic reactions) between the two groups (P＞0.05). Survival analysis revealed a median overall survival (OS) of 20.5 months in the combination therapy group, significantly longer than the 13.6 months in the monotherapy group (P＜0.05). Furthermore, the median progression-free survival (PFS) was 16.0 months in the combination therapy group, significantly longer than the 8.7 months in the monotherapy group (P＜0.05).

Conclusion: The VEN combined with AZA regimen not only significantly improves treatment response rates in patients with HR-MDS but also effectively extends patient survival, with a favorable safety profile and overall manageable adverse reactions.