Arsenic trioxide as a treatment for myelodysplastic syndrome.
Myelodysplastic syndrome (MDS) is a heterogeneous bone marrow disorder primarily affecting older adults, for whom the only curative therapy, bone marrow transplantation, is rarely an option. New therapies, or novel applications of historical therapies, are desperately needed. Arsenic trioxide (ATO), which acts through pro-apoptotic, antiproliferative, and anti-angiogenesis mechanisms, has been used successfully to treat a variety of hematologic malignancies, including MDS. As monotherapy or in combination with other agents, it can effect hematologic improvement in 22% to 26% of patients, with tolerable side effects. MDS patients whose cells express the EVI1 mutation in particular may derive benefit from this therapy.
Sekeres
Mikkael A
MA
Department of Hematology and Medical Oncology, Cleveland Clinic Foundation, 9500 Euclid Avenue, Cleveland, OH 44195,USA. sekerem@ccf.org
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D017209:Apoptosis / Q000187:drug effects; D000077237:Arsenic Trioxide; D001152:Arsenicals / Q000008:administration & dosage / Q000494:pharmacology / Q000627:therapeutic use*; D001205:Ascorbic Acid / Q000008:administration & dosage / Q000627:therapeutic use; D002455:Cell Division / Q000187:drug effects; D002986:Clinical Trials as Topic; D004268:DNA-Binding Proteins / Q000235:genetics; D003907:Dexamethasone / Q000008:administration & dosage / Q000627:therapeutic use; D004359:Drug Therapy, Combination; D005978:Glutathione / Q000378:metabolism; D019337:Hematologic Neoplasms / Q000188:drug therapy; D006801:Humans; D015473:Leukemia, Promyelocytic, Acute / Q000188:drug therapy; D000074008:MDS1 and EVI1 Complex Locus Protein; D015337:Multicenter Studies as Topic; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000235:genetics; D009389:Neovascularization, Pathologic / Q000188:drug therapy; D010087:Oxides / Q000008:administration & dosage / Q000494:pharmacology / Q000627:therapeutic use*; D011519:Proto-Oncogenes / Q000235:genetics; D014157:Transcription Factors / Q000235:genetics
D001152:Arsenicals; D004268:DNA-Binding Proteins; D000074008:MDS1 and EVI1 Complex Locus Protein; C063575:MECOM protein, human; D010087:Oxides; D014157:Transcription Factors; D003907:Dexamethasone; D005978:Glutathione; D001205:Ascorbic Acid; D000077237:Arsenic Trioxide