Combining DNA Methyltransferase and Histone Deacetylase Inhibition to Treat Acute Myeloid Leukemia/Myelodysplastic Syndrome: Achievements and Challenges

摘要

In the first randomized trial of older patients with myelodysplastic syndrome/acute myeloid leukemia (adaptive randomization) to receive treatment with intravenous decitabine (5-day schedule) either alone or in combination with the oral histone deacetylase (HDAC) inhibitor valproic acid (7-day schedule), Issa and colleagues do not detect an improved response rate or overall survival with the combined treatment. The various factors potentially affecting clinical outcome in studies combining decitabine or 5-azacytidine with valproic acid or other HDAC inhibitors are discussed.