How to Treat Myelodysplastic Syndromes: A Comprehensive Guide to Modern Therapies

Myelodysplastic syndromes (MDS) are a group of diverse bone marrow disorders characterized by the ineffective production of blood cells due to abnormal clonal proliferation of hematopoietic stem cells. These conditions fall under the category of hematologic malignancies and often lead to cytopenias—low blood cell counts—that increase the risk of infections, anemia, and bleeding. As our understanding of MDS has evolved, so have treatment strategies, which now rely heavily on accurate risk stratification to guide therapeutic decisions.

Understanding Risk Stratification in MDS

One of the most critical steps in managing myelodysplastic syndromes is determining the patient's risk level. The International Prognostic Scoring System (IPSS) has long been used to assess disease severity, but the revised version—IPSS-R—offers a more precise evaluation based on cytogenetic profiles, blast percentage in the bone marrow, and the degree of cytopenias. Patients are typically categorized into lower-risk and higher-risk groups, each requiring a distinct treatment approach.

Treatment for Lower-Risk MDS Patients

For individuals classified in the lower-risk category, the primary goal is to improve blood cell production and reduce the need for transfusions. Therapies often focus on stimulating the bone marrow and modulating the immune system. Commonly prescribed medications include erythropoiesis-stimulating agents such as erythropoietin, which help boost red blood cell levels. Additionally, immunosuppressive therapies like cyclosporine and corticosteroids may be used, particularly in patients with evidence of immune-mediated bone marrow suppression. Androgens are also occasionally employed to enhance hematopoiesis, especially in selected cases with hypoplastic MDS.

Advanced Therapies for Higher-Risk MDS

Patients with higher-risk MDS face a greater likelihood of disease progression to acute myeloid leukemia (AML), necessitating more aggressive interventions. The cornerstone of current treatment for this group is hypomethylating therapy. Drugs such as azacitidine and decitabine work by reversing abnormal DNA methylation patterns that silence tumor suppressor genes, thereby restoring more normal cellular function. Clinical studies have shown these agents can delay disease progression, improve survival, and, in some cases, induce remission.

Emerging Options and Supportive Care

Beyond pharmacological treatments, supportive care remains essential. This includes regular blood transfusions, iron chelation therapy to manage transfusion-related iron overload, and infection prevention strategies. For eligible patients, allogeneic stem cell transplantation—the only potentially curative option—is considered, especially in younger individuals with matched donors. Ongoing research into targeted therapies, immune checkpoint inhibitors, and combination regimens continues to expand the horizon for MDS treatment, offering renewed hope for improved outcomes.