Molecularly Targeted Therapies in Pediatric Myelodysplastic Syndromes

  • Lia Gore


New technologies have improved our understanding of hematopoietic and leukemia biology, and expanding knowledge of underlying molecular pathways and genomic aberrations is facilitating the development of new therapies for relapsed and refractory disease (Bhojwani et al. 2006; Carroll et al. 2006). Myelodysplasia and associated syndromes constitute a broad range of bone marrow dysfunction, characterized by variable clinical features; cytopenias due to bone marrow production and/or dysfunction and insufficiencies are typical. Clinical symptoms are frequently due to anemia, neutropenia, and/or thrombocytopenia, and transfusion dependence is common although not universal. The progression from myelodysplastic syndromes (MDS) to leukemia, particularly in children, is unequivocal, although the time course over which this occurs is highly variable. Risk factors for progression and lability of disease have been identified, and great effort is being made to identify the molecular triggers in MDS that are both causative and clinically relevant.


Valproic Acid Acute Promyelocytic Leukemia HDAC Inhibitor Acute Myelogenous Leukemia Arsenic Trioxide 
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© Springer Science+Business Media, LLC 2010

Authors and Affiliations

  1. 1.The University of Colorado Cancer Center and The Center for Cancer and Blood Disorders, The Children’s Hospital, University of Colorado DenverAuroraUSA

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