Efficacy and safety of decitabine combined with arsenic trioxide in elderly high-risk myelodysplastic neoplasm patients: a retrospective study.

[OBJECTIVES] Elderly patients with high-risk myelodysplastic neoplasm (MDS) face poor outcomes with limited treatment options, often progressing to acute myeloid leukemia (AML). This study investigates the efficacy and safety of combining decitabine (DAC) with arsenic trioxide (ATO) as a novel therapeutic approach.

[METHODS] A retrospective analysis was conducted on 120 elderly high-risk MDS patients, with 52 receiving ATO-DAC (ATO-DAC group) and 68 receiving DAC monotherapy (DAC group). Treatment outcomes were assessed through overall response rate (ORR), progression-free survival (PFS), and overall survival (OS). Adverse events were recorded and compared between the two groups.

[RESULTS] The ATO-DAC group demonstrated a significantly higher ORR of 78.85% compared to 52.94% in the DAC group (= 0.026). Median PFS was 7.5 months for the ATO-DAC group versus 5.0 months for the DAC group (= 0.021), and median OS was 14.5 months compared to 11.5 months, respectively (= 0.034). Although adverse events were more frequent in the ATO-DAC group, the safety profile remained manageable. These findings suggest that the ATO-DAC combination provides superior efficacy compared to DAC monotherapy.

[DISCUSSION] The combination of DAC and ATO offers a promising and innovative treatment option for elderly high-risk MDS patients, enhancing response rates and survival outcomes while maintaining a manageable safety profile.

[CONCLUSION] This study underscores the clinical relevance of this regimen, warranting further investigation in prospective trials.