Myelodysplastic syndrome is a heterogeneous category of hematologic disorders that has been recognized primarily by abnormal morphology and number of blood cells originating from hematopoietic stem cells in the bone marrow based on the genetic as well as epigenetic changes in the hematopoietic stem cells. Its frequency is increased by aging, emphasizing the role genetic and epigenetic factors in its occurrence and most of the cases have been diagnosed at the age of 65 and higher. Marked genetic abnormalities as the cause of the syndrome and the old age of the patients make it a difficult category of hematologic disorders from therapeutic point of view. However, the disease is not limited to the elder adults and pediatric patients with Myelodysplastic disorders have been diagnosed with the median age of 6 years with a significantly less frequency. Chemotherapy has been applied as the only therapeutic option in these patients with variable response rate and duration of response. However, further basic investigation addressing the molecular aspects and the pathogenesis of MDS categories has clarified its association with certain genetic mutations and signaling pathways; hence opening new opportunities in MDS treatment. . In this paper, we will discuss the current concept and trends of targeted therapies for Myelodysplastic syndrome.
Key words: Myelodysplastic syndrome; Immunotherapy; Refractory anemia; Targeted therapy.Download Pdf