title: Evaluation of continuous infusion low-dose 5-azacytidine in the treatment of myelodysplastic syndromes.
abstract: The treatment of myelodysplastic syndromes (MDS) is both difficult and controversial. In the current study, we evaluated the efficacy of low-dose 5-azacytidine in the treatment of this disorder. Fifteen patients with MDS were entered on study to be treated with 5-azacytidine by continuous intravenous infusion for 14 days. Doses of the drug ranged from 10 mg/m2/day to 35 mg/m2/day, with most patients receiving 16.5 mg/m2/day. In two patients, the drug was stopped early in the course of treatment because of thrombocytopenia. Thirteen patients completed therapy according to protocol. Three of 13 patients demonstrated a partial response to therapy. Of these three patients, one had an increase in platelet and absolute neutrophil counts, while the other two no longer required support with red cell transfusions. The drug was well tolerated and significant myelosuppression did not occur in most patients. Low-dose 5-azacytidine appears to have activity in the treatment of primary MDS and future studies should consider evaluation of this drug in combination with hematopoietic growth factors.
authors.affiliation: Department of Medicine, Medical College of Wisconsin, Milwaukee 53226.
authors.forename: C R
authors.initials: CR
authors.lastname: Chitambar
authors.forename: J A
authors.initials: JA
authors.lastname: Libnoch
authors.forename: W G
authors.initials: WG
authors.lastname: Matthaeus
authors.forename: R C
authors.initials: RC
authors.lastname: Ash
authors.forename: P S
authors.initials: PS
authors.lastname: Ritch
authors.forename: T
authors.initials: T
authors.lastname: Anderson
mesh_terms: D000328:Adult; D000368:Aged; D001374:Azacitidine / Q000009:adverse effects / Q000627:therapeutic use*; D001803:Blood Transfusion; D001853:Bone Marrow / Q000187:drug effects; D017707:Erythrocyte Transfusion; D005260:Female; D006801:Humans; D007262:Infusions, Intravenous; D007958:Leukocyte Count / Q000187:drug effects; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000628:therapy; D009504:Neutrophils / Q000473:pathology; D010976:Platelet Count / Q000187:drug effects
chemical_list: D001374:Azacitidine