title: New agents in myelodysplastic syndromes.
abstract: Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic disorders characterized by ineffective hematopoiesis resulting in peripheral cytopenia and by increased progression to acute myeloid leukemia (AML). Therapeutic interventions for MDS other than allogeneic stem cell transplantation have been palliative. Novel and targeted therapeutic agents such as the inhibition of farnesyl transferases and receptor tyrosine kinases, more potent thalidomide analogs, arsenic trioxide, immunomodulating agents, hypomethylating agents, and histone deacetylase inhibitors have shown encouraging results and may offer durable benefit to patients with MDS. Further development of rational therapies and improvements in the outcome of patients with MDS are likely to emerge from an increased understanding of the pathophysiology of these diseases.
authors.affiliation: Department of Leukemia, MD Anderson Cancer Center, 1515 Holcombe Blvd, Houston, TX 77063, USA.
authors.forename: Elias J
authors.initials: EJ
authors.lastname: Jabbour
authors.forename: Francis J
authors.initials: FJ
authors.lastname: Giles
mesh_terms: D019883:Alkyl and Aryl Transferases / Q000037:antagonists & inhibitors; D020533:Angiogenesis Inhibitors / Q000627:therapeutic use; D017209:Apoptosis; D001152:Arsenicals / Q000627:therapeutic use; D001374:Azacitidine / Q000627:therapeutic use; D001549:Benzamides; D002869:Chromosome Aberrations; D002986:Clinical Trials as Topic; D016207:Cytokines / Q000502:physiology; D019175:DNA Methylation; D004791:Enzyme Inhibitors / Q000627:therapeutic use; D051231:Farnesyltranstransferase; D006133:Growth Substances / Q000627:therapeutic use; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D000068877:Imatinib Mesylate; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D009190:Myelodysplastic Syndromes / Q000145:classification / Q000188:drug therapy* / Q000235:genetics / Q000401:mortality / Q000503:physiopathology / Q000628:therapy; D016376:Oligonucleotides, Antisense / Q000494:pharmacology; D010879:Piperazines / Q000627:therapeutic use; D011379:Prognosis; D011743:Pyrimidines / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D020533:Angiogenesis Inhibitors; D001152:Arsenicals; D001549:Benzamides; D016207:Cytokines; D004791:Enzyme Inhibitors; D006133:Growth Substances; D007166:Immunosuppressive Agents; D016376:Oligonucleotides, Antisense; D010879:Piperazines; D011743:Pyrimidines; D000068877:Imatinib Mesylate; D019883:Alkyl and Aryl Transferases; D051231:Farnesyltranstransferase; D001374:Azacitidine