New treatments for myelodysplastic syndromes

Over the past decade, there have been major advancements in the treatment of Myelodysplastic Syndromes (MDS). While best supportive care remains a cornerstone of MDS management, treatment strategies are now tailored based on the International Prognostic Scoring System (IPSS) risk category, cytogenetic profile, patient age, comorbidities, and treatment adherence. For patients with lower-risk MDS, hematopoietic growth factors such as high-dose erythropoiesis-stimulating agents and thrombopoietin receptor agonists play a key role. Supportive care should also include iron chelation therapy to prevent organ damage caused by iron overload in those requiring frequent transfusions. Biologic therapies like lenalidomide have become important, especially for lower-risk patients with a deletion 5q cytogenetic abnormality, many of whom achieve transfusion independence. In patients with intermediate-2 or high-risk MDS, hypomethylating agents have proven effective in delaying progression to acute leukemia and improving survival. Ongoing research is evaluating additional therapeutic agents, including histone deacetylase inhibitors, farnesyltransferase inhibitors, clofarabine, and ezatiostat.