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Opinion|Videos|December 23, 2025

Understanding MDS: Disease Biology, Risk Stratification, and Treatment Goals

Myelodysplastic syndromes (MDS) are a group of clonal bone marrow disorders driven by ineffective hematopoiesis, genetic mutations, and progressive marrow failure. These underlying disease processes lead to chronic cytopenias, increased infection and bleeding risk, transfusion dependence, and, in some patients, progression to acute myeloid leukemia—significantly impacting both survival and quality of life. Risk stratification plays a central role in guiding management, with tools such as prognostic scoring systems used to categorize patients as lower- or higher-risk based on cytogenetics, marrow blasts, and blood counts. Treatment goals differ across these groups: in lower-risk MDS, the focus is on improving cytopenias, reducing transfusion burden, and maintaining quality of life, while higher-risk disease emphasizes altering disease course, delaying progression to AML, and extending survival.

Myelodysplastic syndromes (MDS) are a group of clonal bone marrow disorders driven by ineffective hematopoiesis, genetic mutations, and progressive marrow failure. These underlying disease processes lead to chronic cytopenias, increased infection and bleeding risk, transfusion dependence, and, in some patients, progression to acute myeloid leukemia—significantly impacting both survival and quality of life. Risk stratification plays a central role in guiding management, with tools such as prognostic scoring systems used to categorize patients as lower- or higher-risk based on cytogenetics, marrow blasts, and blood counts. Treatment goals differ across these groups: in lower-risk MDS, the focus is on improving cytopenias, reducing transfusion burden, and maintaining quality of life, while higher-risk disease emphasizes altering disease course, delaying progression to AML, and extending survival.

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