Update on myelodysplastic syndromes: New approaches to classification and therapy

Abstract

Myelodysplastic syndromes have long provided hematologists with difficult therapeutic challenges, and until recently treatment options beyond supportive care were limited. Recent advances in our understanding of hematopoiesis, immunology, and genetics have led to a better understanding of the natural history of these disorders and have facilitated development of more rational and targeted treatment approaches. A number of promising agents are in various phases of study, including arsenic trioxide, CC5013, the farnesyltransferase inhibitors, and DNA methyltransferase inhibitors. In addition, less intensive strategies for allogeneic stem cell transplantation now permit us to offer potentially curative therapy to a larger proportion of patients. Optimal management of an individual patient requires consideration of the disease and its expected course, available treatment options, the patient’s age and condition, and an ongoing assessment of the goals of therapy.