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Therapeutic spectrum in the treatment of myelodysplastic syndromes

During the last 15 years, important progress has been made in the understanding of the biology and prognosis of myelodysplastic syndromes (MDS). It is a clonal disorder, characterised by ineffective haematopoiesis, which can lead to either fatal cytopenias or acute myelogenous leukaemias. Risk-adapted treatment strategies were established, due to the high median age (60 – 75 years) of the MDS patients and the individual history of the disease (i.e. number of cytopenias, cytogenetic changes, transfusion requirements). Allogeneic bone marrow transplantation currently offers the only potentially curative treatment, but this form of therapy is not available for the ‘typical’ MDS patient, who is > 60 years of age. Therapy with erythropoietin and granulocyte colony-stimulating factor has improved the quality of life of selected patients. The development of target-specific therapies, including antibodies and small molecules directed against specific molecular alterations in MDS, with minimal adverse effects, is the hope for the future. Furthermore, the innovative use of immunomodulatory agents and the optimising of cytotoxic treatment should continue to help in the treatment of MDS.