The clinical use of DNA methyltransferase inhibitors in myelodysplastic syndromes

Abdallah Abou Zahr, Ehab Saad Aldin, Lisa Barbarotta, Nikolai Podoltsev, Amer M. Zeidan

Research output: Contribution to journalReview articlepeer-review

19 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)1019-1036
Number of pages18
JournalExpert review of anticancer therapy
Issue number9
StatePublished - Sep 2 2015
Externally publishedYes


  • DNA methyltransferase inhibitors
  • allogeneic hematopoietic stem cell transplant
  • azacitidine
  • azanucleosides
  • decitabine
  • epigenetics
  • hypomethylating agents
  • myelodysplastic syndromes

ASJC Scopus subject areas

  • Oncology
  • Pharmacology (medical)


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