Abstract
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 proapoptotic, antiproliferative, and antiangiogenesis 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.
MeSH terms
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Angiogenesis Inhibitors / administration & dosage
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Angiogenesis Inhibitors / therapeutic use
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Antineoplastic Agents / therapeutic use
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Apoptosis / drug effects
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Arsenic Trioxide
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Arsenicals / administration & dosage
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Arsenicals / pharmacology
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Arsenicals / therapeutic use*
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Clinical Trials as Topic
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DNA-Binding Proteins / genetics
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Drug Therapy, Combination
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Humans
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Leukemia, Promyelocytic, Acute / drug therapy
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MDS1 and EVI1 Complex Locus Protein
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Multicenter Studies as Topic
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Myelodysplastic Syndromes / drug therapy*
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Myelodysplastic Syndromes / genetics
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Oxides / administration & dosage
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Oxides / pharmacology
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Oxides / therapeutic use*
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Proto-Oncogenes / genetics
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Salvage Therapy
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Thalidomide / administration & dosage
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Thalidomide / therapeutic use
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Transcription Factors / genetics
Substances
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Angiogenesis Inhibitors
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Antineoplastic Agents
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Arsenicals
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DNA-Binding Proteins
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MDS1 and EVI1 Complex Locus Protein
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MECOM protein, human
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Oxides
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Transcription Factors
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Thalidomide
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Arsenic Trioxide