A survival prognostic model for high-risk myelodysplastic syndrome patients treated with decitabine

Decitabine is widely used in China for high-risk myelodysplastic syndromes (MDS). However, the prognostic factors associated with overall survival (OS) remain incompletely defined.

We retrospectively analyzed 130 high-risk MDS patients treated with decitabine. Clinical characteristics and laboratory parameters were collected. Treatment response was assessed, and survival outcomes were evaluated using Cox regression models. A prognostic nomogram was developed to improve risk stratification beyond the conventional IPSS/IPSS-R systems.

The median age was 60.5 years, with 57.7% male patients. Median OS was 15.5 months. Univariate analysis showed that age ≥50 years, bone marrow blasts ≥10%, and iron overload (serum ferritin ≥500 µg/L) were significantly associated with inferior OS. Multivariate Cox regression confirmed age ≥50 (HR = 2.31, 95% CI: 1.21-4.40, p = 0.011) and elevated bone marrow blasts (HR = 1.92, 95% CI: 1.16-3.17, p = 0.012) as independent adverse prognostic factors. Poor karyotype also conferred poor outcomes. Next-generation sequencing data were available for 92 patients. Given the low mutation rate, the mutational analysis was conducted exploratorily; results suggested that TP53 mutations were significantly associated with adverse prognosis. A prognostic nomogram integrating age, ECOG score, bone marrow blasts, cytogenetics, hemoglobin, platelet count, absolute neutrophil count, ring sideroblasts, and ferritin demonstrated improved risk stratification, though validation was limited to internal cohorts.

Decitabine provides short-term hematologic benefits in high-risk MDS, but overall prognosis remains poor. Age, bone marrow blasts, iron overload, and high-risk cytogenetics are key prognostic factors. Incorporating clinical, cytogenetic, and mutational variables into new prognostic models may enhance individualized treatment decision-making for high-risk MDS patients.