PURPOSE: The aim of this study was to assess the predictive role of FOXO1 expression changes in determining the response to hypomethylation therapy in patients with high-risk myelodysplastic syndrome (MDS).

METHODS: FOXO1 mRNA levels were measured using real-time PCR in 62 newly diagnosed MDS patients undergoing decitabine treatment. The study analyzed the relationship between FOXO1 expression and clinical indicators, treatment outcomes, and prognosis.

RESULTS: Responders exhibited significantly elevated FOXO1 levels, which were associated with improved peripheral blood counts, decreased bone marrow blasts, and enhanced T-cell immunity and polarization. Increased FOXO1 expression after four cycles of decitabine was associated with a more favorable treatment response. Univariate and multivariate Cox analyses revealed that elevated FOXO1 expression was linked to prolonged overall survival and leukemia-free survival.

CONCLUSIONS: Elevated FOXO1 expression in high-risk MDS patients undergoing decitabine treatment enhances patient prognosis and survival.