Year: 2026
Type: article
Study design: Observational Study
Abstract: Background and aim Myelodysplastic neoplasms (MDS) comprise a heterogeneous group of clonal disorders with variable prognosis and risk progression to acute myeloid leukemia (AML). This study aims to evaluate clinical and laboratory parameters and outcomes in a cohort of patients with de novo MDS treated at a single academic center in São Paulo and compare with those described on previous Brazilian cohorts. Materials and methods This retrospective single-center, observational study included 427 patients with de novo MDS followed between 2010 and 2025. Demographic, clinical, laboratory, cytogenetic, and treatment data were obtained from the Institutional Myelodysplastic Syndromes Registry (REDCap). Statistical analyses were performed using R software (version 4.5.3). The study was approved by the Institutional Ethics Committee (CAAE 63245922.1.0000.0068). Results and discussion The median age of the cohort was 70 years, 58% were male, and median follow-up was 1.74 years. Cytogenetic analysis was performed in 98% of patients, with abnormal karyotypes identified in 39% During follow-up, 20% received azacitidine and 8.5% underwent allogeneic hematopoietic cell transplantation (allo-HCT) According to the Revised International Prognostic Scoring System (IPSS-R), 56% of patients were classified as low risk (≤3.5). The IPSS-R significantly discriminated against overall survival (3.9 vs. 1.2 years for patients with IPSS-R ≤3.5 and >3.5, respectively; p<0.001). AML transformation occurred in 28% of patients, with a 3-year cumulative incidence of 23%. Median leukemia-free survival was 2.5 years, with an estimated 5-year rate of 31%. Patients with IPSS-R >3.5 experienced significantly higher leukemic transformation rates than those with IPSS-R ≤3.5 (87% vs. 52%; p< 0.0001) Compared with previous Brazilian MDS registries, this cohort showed a similar clinical profile, including age at diagnosis, male predominance (58% vs. 49–56%), and comparable cytopenias. Cytogenetic analysis was performed more frequently (98% vs. 62.4–89.6%), whereas the prevalence of abnormal karyotypes remained similar (39% vs. 39.8–50.5%). The proportion of low-risk patients was comparable to the first Brazilian registry (56% vs. 55.9%) but lower than in subsequent national series (75.6–83.2%), likely reflecting the greater complexity of a tertiary referral center. The proportion of patients undergoing allo-HCT was like previous Brazilian cohorts (8.5% vs. 3.4–6.4%), whereas azacitidine use was higher (20% vs. 7.5%), reflecting improved drug availability and access. Conclusion Limited advances in access to diagnostic studies and treatment were identified, without improvement in overall survival compared with historical cohorts. Brazilian MDS registry, the incorporation of molecular sequencing, and the inclusion of additional drugs in the Unified Health System (SUS) are desirable.
Institution: Universidade de São Paulo
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: diamond
OpenAlex ID: W7223139827