Management of lower-risk myelodysplastic syndromes: the art and evidence

Abstract

Myelodysplastic syndromes (MDS) represent a spectrum of bone marrow failure with variable outcome. Patients with "lower-risk" disease have an expected median survival measured in years, and a low risk of leukemia progression. Patients with "higher-risk" MDS, on the other hand, have expected survival measured in months without treatment and rapid leukemia progression. The outcome of those distinct groups can be explained by different underlying disease biology. In clinical practice, patients are stratified into risk groups based on prognostic models, most commonly the International Prognostic Scoring System (IPSS). In higher-risk disease, the standard of care is hypomethylating agents to extend survival and suppress leukemia potential, and consideration of allogeneic stem cell transplantation, which remains the only curative option. Patients classified as having lower-risk disease begin treatment with management focused on ameliorating hematologic deficits, related symptoms, or both. This review of lower-risk MDS highlights the biology of the disease and models for risk stratification. We use a case-based format to discuss current options for treatment, including erythropoiesis-stimulating agents, hypomethylating agents, lenalidomide, immunosuppressive therapy, supportive care, and investigational agents.