Novel therapies in myelodysplastic syndromes

PURPOSE OF REVIEW: Currently, there is a rapid expansion of novel, efficacious therapies for the treatment of patients with myelodysplastic syndromes (MDS) at a rate never seen to date. In this review, we will outline new treatment strategies in MDS focusing on novel hypomethylating agents (HMA) and combinations in addition to targeted and immune-based therapies. RECENT FINDINGS: Large-scale gene sequencing and immune-based research has given us a great deal of information regarding the complexity and heterogeneity of MDS. This rapid improvement in our knowledge has provided a framework for development of novel therapies with specific gene and immune-based targets. Additionally, expanding and optimizing our current HMA-based strategies has led us to potentially not only ease administration but also improve outcomes. SUMMARY: Novel therapies in MDS are greatly needed is a disease state where few options are currently available, particularly in the HMA failure setting. Fortunately, through comprehensive genetic profiling, characterization of novel underlying pathogenic drivers, and understanding of the immune microenvironment, the treatment paradigm of patients with MDS is encouraging.