, Volume 5, Issue 2, pp 71-80
Date: 09 Aug 2012

Management of Transfusion-Dependent Myelodysplastic Syndromes

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Abstract

The clinical and biological heterogeneity of the myelodysplastic syndromes (MDS) has engendered new expectations that therapy must be individualized. As a consequence, anemia management strategies for patients with MDS have evolved from a long-standing reliance upon supportive measures to active treatment guided by the risks posed by the disease. Median survival for a patient with MDS ranges from several months to ≥5 years with differing treatment goals, such as the promotion of hematopoiesis with recombinant cytokines to reducing the risk of leukemic transformation or death with agents such as azacitidine. As further insight into the molecular pathogenesis of these disorders has emerged, significant progress has been made in identifying new drug targets. Among these promising new agents are the farnesyl transferase inhibitors, immunosuppressive therapy, small-molecule tyrosine kinase inhibitors, and angiogenesis inhibitors. Phase I and II trials have shown encouraging activity with these agents and larger randomized trials are expected to define their place in the management of MDS.