The Evolution of Research and Therapy With Hypomethylating Agents in Acute Myeloid Leukemia and Myelodysplastic Syndrome: New Directions for Old Drugs

Nicholas J. Short, Hervé Dombret, Lionel Adès, Hagop M Kantarjian

Research output: Contribution to journalReview articlepeer-review

5 Scopus citations

Abstract

Azacitidine and decitabine are cytosine analogs that function as DNA methyltransferase inhibitors. These agents, commonly referred to as “hypomethylating agents,” are widely used for the treatment of myelodysplastic syndrome and acute myeloid leukemia (AML). In this review, we discuss the clinical development of these drugs, including the early studies that led to the optimization of their doses and schedules, and the pivotal studies that led to their regulatory approval, both as monotherapy and in combination with venetoclax for older adults with AML who are unfit for intensive chemotherapy. We also review the more recent development of oral hypomethylating agent formulations and the novel oral strategies being developed in myelodysplastic syndrome and AML.

Original languageEnglish (US)
Pages (from-to)29-36
Number of pages8
JournalCancer Journal (United States)
Volume28
Issue number1
DOIs
StatePublished - 2022

Keywords

  • Azacitidine
  • Decitabine
  • DNA methyltransferase inhibitors
  • Epigenetics
  • Venetoclax

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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