Myelodysplastic Syndromes–Many New Drugs, Little Therapeutic Progress.
Posted by rob on September 24, 2010 under Uncategorized | 
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Myelodysplastic Syndromes–Many New Drugs, Little Therapeutic Progress.
Mayo Clin Proc. 2010 Sep 22;
Authors: Tefferi A
Myelodysplastic syndromes (MDS) constitute a form of blood cancer that primarily affects the elderly and is characterized by anemia (or other cytopenias) and a high risk of leukemic transformation. The World Health Organization (WHO) system has formally classified MDS as 1 of 5 myeloid malignancies that also include acute myeloid leukemia (AML) and myeloproliferative neoplasms. All myeloid malignancies, including MDS, are clonal stem cell diseases. Unlike the case with BCR-ABL1-positive chronic myelogenous leukemia, the disease-causing mutation in MDS is currently unknown. It is generally believed that the MDS phenotype is additionally affected by secondary mutations, genetic haploinsufficiency, epigenetic changes, and altered host response that result in cytokine, immune, and bone marrow stromal changes. Unfortunately, none of this information has successfully been translated into a robust pathogenetic framework or effective targeted therapy.
PMID: 20861462 [PubMed - as supplied by publisher]