Hypomethylating agents (HMA) treatment for myelodysplastic syndromes: alternatives in the frontline and relapse settings

Natalie Uy, Abhay Singh, Steven D. Gore, Thomas Prebet

Research output: Contribution to journalReview articlepeer-review

2 Scopus citations


Introduction: Hypomethylating agents (HMA) have played a pivotal role for treating myelodysplastic syndromes (MDS) over the past decade, inducing sustained hematological responses and delaying progression to leukemia. However, a vast majority of patients will experience treatment failure within 2 years, with poor prognoses and limited options, and management of this growing patient population remains unclear. Areas covered: With the introduction of new agents in the MDS field, a better understanding of the biology of MDS, and updated information on standard of care options (including allogeneic transplantation), we re-evaluate the global treatment strategy in MDS via novel agents, focusing in particular on investigational approaches for patients who fail to respond to HMA when applicable. This review aims to address two questions: what are reasonable alternatives to HMA in MDS, and what strategies can be used for patients experiencing HMA failure. Expert opinion/commentary: HMA therapy remains a mainstay of treatment, even if additional research is still warranted to maximize its benefits for the different groups of patients. The outcome of patients experiencing HMA failure remains grim, without standard of care, but several new approaches seem promising, as there is an increasing focus on studying treatments for patients refractory to HMA treatment.

Original languageEnglish (US)
Pages (from-to)1213-1224
Number of pages12
JournalExpert opinion on pharmacotherapy
Issue number12
StatePublished - Aug 13 2017
Externally publishedYes


  • Disease management
  • IPSS
  • epigenetic modification
  • hypomethylating agent failure
  • hypomethylating agents
  • investigational agents
  • myelodysplastic syndrome
  • treatment failure

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


Dive into the research topics of 'Hypomethylating agents (HMA) treatment for myelodysplastic syndromes: alternatives in the frontline and relapse settings'. Together they form a unique fingerprint.

Cite this