New agents in myelodysplastic syndromes.
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 farnesyltransferases 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.
Jabbour
Elias
E
Department of Leukemia, Unit 428, MD Anderson Cancer Center, 1515 Holcombe Blvd, Houston, TX 77063, USA.
Giles
Francis J
FJ
D020533:Angiogenesis Inhibitors / Q000627:therapeutic use; D001803:Blood Transfusion; D002986:Clinical Trials as Topic; D003131:Combined Modality Therapy; D018450:Disease Progression; D015195:Drug Design; D015507:Drugs, Investigational / Q000627:therapeutic use*; D004791:Enzyme Inhibitors / Q000627:therapeutic use; D004921:Erythropoietin / Q000627:therapeutic use; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015337:Multicenter Studies as Topic; D009190:Myelodysplastic Syndromes / Q000145:classification / Q000188:drug therapy* / Q000235:genetics / Q000601:surgery; D011379:Prognosis; D012306:Risk; D014212:Tretinoin / Q000627:therapeutic use
D020533:Angiogenesis Inhibitors; D015507:Drugs, Investigational; D004791:Enzyme Inhibitors; D004921:Erythropoietin; D016179:Granulocyte Colony-Stimulating Factor; D014212:Tretinoin