[Advances in the management of myelodysplastic neoplasms]

Myelodysplastic syndromes/neoplasms (MDS) are clonal myeloid malignancies arising from hematopoietic stem cell abnormalities. Treatment is risk-adapted: in lower-risk MDS, the primary goals are hematologic improvement and reduction of progression risk, and agents such as lenalidomide, erythropoiesis-stimulating agents, and luspatercept are commonly used. In contrast, higher-risk MDS requires disease-modifying approaches aimed at preventing leukemic transformation and prolonging survival, with allogeneic hematopoietic stem cell transplantation and hypomethylating agents forming the therapeutic backbone. Recent advances in the pathophysiological understanding of clonal hematopoiesis have revealed its role in MDS-associated autoinflammatory syndromes. In addition, indolent myeloid hematopoietic disorders that have not yet progressed to myeloid neoplasms have been increasingly characterized, shifting attention toward early intervention strategies targeting clonal hematopoiesis. Furthermore, the identification of asymptomatic carriers of pathogenic germline variants has raised new clinical challenges regarding surveillance and management. This review summarizes current therapeutic approaches to MDS in the context of these emerging insights.