BACKGROUND. Aberrant DNA methylation, which results in leukemogenesis, is frequent in patients with myelodysplastic syndromes (MDS) and is a potential target for pharmacologic therapy. Decitabine indirectly depletes methylcytosine and causes hypomethylation of target gene promoters. METHODS. A total of 170 patients with MDS were randomized to receive either decitabine at a dose of 15 mg/m2 given intravenously over 3 hours every 8 hours for 3 days (at a dose of 135 mg/m2 per course) and repeated every 6 weeks, or best supportive care. Response was assessed using the International Working Group criteria and required that response criteria be met for at least 8 weeks. RESULTS. Patients who were treated with decitabine achieved a significantly higher overall response rate (17%), including 9% complete responses, compared with supportive care (0%) (P
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Kantarjian, H., Issa, J. J., Rosenfeld, C. S., Bennett, J. M., Albitar, M., DiPersio, J., … Saba, H. (2006). Decitabine improves patient outcomes in myelodysplastic syndromes. Cancer, 106(8), 1794–1803. https://doi.org/10.1002/cncr.21792
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