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Evaluation of continuous infusion low‐dose 5‐azacytidine in the treatment of myelodysplastic syndromes
Author(s) -
Chitambar Christopher R.,
Libnoch Joseph A.,
Matthaeus William G.,
Ash Robert C.,
Ritch Paul S.,
Anderson Tom
Publication year - 1991
Publication title -
american journal of hematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.456
H-Index - 105
eISSN - 1096-8652
pISSN - 0361-8609
DOI - 10.1002/ajh.2830370207
Subject(s) - medicine , myelodysplastic syndromes , drug , drug holiday , platelet , azacitidine , chemotherapy , continuous infusion , gastroenterology , pharmacology , bone marrow , immunology , biochemistry , gene expression , chemistry , dna methylation , human immunodeficiency virus (hiv) , gene
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/m 2 /day to 35 mg/m 2 /day, with most patients receiving 16.5 mg/m 2 /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.