Introduction to Therapies in MDS

Therapeutic strategies in MDS range from watchful waiting to allogeneic stem cell transplantation. Most patients with lower risk MDS die from the complications related to progressive bone marrow failure and worsening cyopenias, whereas in high-risk MDS leukemic progression represents the main cause of death. Symptoms of anemia, the most common peripheral cytopenia, predominate in lower-risk MDS, and according to the available evidence-based therapeutic guidelines, current therapeutic interventions in these patients include erythropoietic stimulating agents, lenalidomide, RBC transfusion and iron chelation therapy, as well as immunosuppressive therapy in a subset of patients. Notably, a fraction of patients with low-risk MDS have a disease more aggressive than expected based on IPSS estimates and may deserve allogeneic stem cell transplantation early in the clinical course. Conversely, patients with high-risk MDS warrant disease-modifying treatments shortly after diagnosis or disease progression from a low-risk phase, and therapeutic strategies include hypomethylating agents (5-azacitidine and decitabine), intensive chemotherapy, allogeneic stem cell transplantation, or a combination of these approaches.