title: The clinical use of DNA methyltransferase inhibitors in myelodysplastic syndromes.
abstract: Myelodysplastic syndromes (MDS) include a heterogeneous group of acquired hematopoietic malignancies characterized by ineffective hematopoiesis, peripheral cytopenias, and a varying propensity for progression to acute myeloid leukemia. Patients with higher risk MDS have dismal outcomes and treatment options are very limited. Aside from allogeneic hematopoietic cell transplantation, the only potentially curative treatment, DNA methyltransferase inhibitors (DNMTIs) are the only intervention that prolongs overall survival in patients with higher risk MDS. The clinical use of DNMTIs in MDS was one of the earliest successful attempts at epigenetic reprogramming of cancer. In this review, we discuss the clinical use of DNMTIs in MDS highlighting the current challenges and controversies and future directions of research needed to explore the full therapeutic potential of these agents.
authors: a 1 Division of Hematology Oncology, Mount Sinai Beth Israel, New York, NY, USA.
authors: Abdallah
authors: A
authors: Abou Zahr
authors: Ehab
authors: E
authors: Saad Aldin
authors: Lisa
authors: L
authors: Barbarotta
authors: Nikolai
authors: N
authors: Podoltsev
authors: Amer M
authors: AM
authors: Zeidan
mesh_terms: D004248:DNA (Cytosine-5-)-Methyltransferases / Q000037:antagonists & inhibitors*; D018450:Disease Progression; D004791:Enzyme Inhibitors / Q000494:pharmacology / Q000627:therapeutic use*; D044127:Epigenesis, Genetic; D006801:Humans; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000201:enzymology / Q000503:physiopathology; D015996:Survival Rate
chemical_list: D004791:Enzyme Inhibitors; D004248:DNA (Cytosine-5-)-Methyltransferases
keywords: DNA methyltransferase inhibitors; allogeneic hematopoietic stem cell transplant; azacitidine; azanucleosides; decitabine; epigenetics; hypomethylating agents; myelodysplastic syndromes