Established and emerging targeted therapies in the myelodysplastic syndromes

Therapy for the myelodysplastic syndromes (MDS) is an evolving area of research which has made significant use of the increased understanding of the complex biology of these disorders. Novel agents targeting multiple pathogenic pathways are being actively tested in preclinical and clinical settings and hold the potential to be available to clinicians before long.

Herein we provide an historical framework for understanding the current use of hypomethylating agents in MDS and discuss recent developments in the field of targeted therapy in MDS including data from published and ongoing clinical studies with oral hypomethylating agents, PI3/polo-like kinase inhibitors, TGF-β inhibitor/ligand traps, and immune checkpoint inhibitors. A comprehensive review of recent literature was undertaken using PubMed and Medline. Expert commentary: Management of MDS patients will evolve substantially in the near future with the incorporation of molecular data into patient stratification models and with the introduction of novel targeted agents.