What are the most promising new agents in myelodysplastic syndromes?

Namrata S. Chandhok, Prajwal C. Boddu, Steven D. Gore, Thomas Prebet

Research output: Contribution to journalReview articlepeer-review


Purpose of reviewMyelodysplastic syndromes (MDS) are a diverse group of clonal disorders of hematopoietic stem or progenitor cells that represent the most common class of acquired bone marrow failure syndromes in adults. Despite significant improvement in the pathologic insight into this group of disorders, therapeutic options remain limited and allogeneic hematopoietic stem-cell transplantation is the only treatment that can induce long-term remission in patients with MDS. The goals of therapy for MDS are based on disease prognostication, with a focus of minimizing transfusion dependence and preserving quality of life in low-risk groups and preventing progression of disease to acute myeloid leukemia in high-risk groups. Given the dearth of approved treatment options, there is a marked need for novel therapies across the board, and there are several novel agents currently in the pipeline.Recent findingsAmong the promising agents with preclinical and early phase efficacy in higher risk MDS, apoptosis targeting with BCL-2 inhibitors have been a standout. There is also a keen interest in immunotherapy, and targeted agents (genetic, signaling pathways, bispecific antibodies, antibody-drug conjugates, and others described in this review).SummaryIn this review, we will highlight some of the promising new agents currently under investigation for the management of MDS.

Original languageEnglish (US)
Pages (from-to)77-87
Number of pages11
JournalCurrent Opinion in Hematology
Issue number2
StatePublished - Mar 1 2019
Externally publishedYes


  • acute myeloid leukemia
  • apoptosis inhibition
  • cytotoxic therapy
  • disease management
  • erythropoiesis
  • hypomethylating agent
  • immune checkpoint inhibitors
  • investigational agents
  • lenalidomide
  • luspatercept

ASJC Scopus subject areas

  • Hematology


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