Treatment Strategies and Issues in Low/Intermediate-1-Risk Myelodysplastic Syndrome (MDS) Patients

Myelodysplastic syndromes (MDS) are a heterogeneous group of progressive bone marrow neoplastic disorders associated with increased risk for transformation to acute leukemia. Hallmarks of MDS are peripheral blood cytopenias (especially anemia), frequently with hypercellular bone marrow, and dysplastic changes in one or more hematopoietic lineages. The wide variation in clinical presentation has confounded treatment strategies and hindered the development of new therapies. However, improved classification and prognostic systems are providing a more refined stratification of patients, helping to guide treatment and management decisions as well as to appropriately select patients for clinical trials. Patients with International Prognostic Scoring System classifications of low- and intermediate-1 (Low/Int-1) risk are considered to have “low-risk” MDS. These patients are primarily treated with low-intensity supportive care, especially red blood cell transfusions, to treat their symptoms and maintain their quality of life. In small subsets of Low/Int-1-risk patients with MDS, hematopoietic cytokines or antithymocyte globulin may reduce transfusion requirements. The drawbacks to these treatments are high failure rates, even with improved predictive models, and the high cost of cytokines. Regardless of risk category, a patient's age and existing comorbidities must be factored into treatment decisions. It is anticipated that trials with new and investigational agents may soon provide definitive treatments for patients with Low/Int-1-risk MDS when used alone or in conjunction with supportive measures.