The biological mechanisms underlying the ineffective haematopoiesis and increased risk of leukaemic evolution in MDS is largely known. A careful clinical and cytogenetical assessment is required to correctly classify and risk stratify the patients. A curative approach with allogeneic stem cell transplantation should always be considered at the initial assessment. In low-risk patient receiving chronic RBC transfusions, symptomatic iron-overload is a reality and carries a significant mortality, and therefore, the iron status should be monitored and chelators used when indicated. The first line treatment of anaemia is erythroid growth factors. Lenalidomide is a highly potent therapy in low-risk MDS with del 5q, however, the treatment carries a high risk of rapidly developing neutropenia and thrombocytopenia. Several promising drugs are currently under investigation in low-risk MDS including thrombopoietin analogues and epigenetically active drugs.
- myelodysplastic syndromes (MDS)