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Mutation-Driven Therapy in MDS

  • Myelodysplastic Syndromes (M Savona, Section Editor)
  • Published:
Current Hematologic Malignancy Reports Aims and scope Submit manuscript

Abstract

Purpose of Review

Genetic sequencing in myelodysplastic syndrome (MDS) has provided an improved understanding of the complexity and heterozygosity of the disease. More importantly, our molecular understanding of MDS is leading to rapid advancements and personalized therapy for our patients. Herein, we review the current mutation-driven treatment landscape in MDS, first focusing on individual mutations. We then discuss the effect of specific gene mutations on response and outcomes to standard therapies as well as to cutting edge investigational therapies.

Recent Findings

Molecular annotation of MDS can predict response rates and outcomes to our current standard of care therapies including hypomethylating agents, lenalidomide, and allogeneic stem cell transplantation. Clinical trials targeting molecular subsets of MDS are underway with some in very early stages while others advancing to phase III trials. Targeting TP53 and IDH1/2 mutations appear to be promising targets with substantial efficacy seen in several trials to date. Furthermore, novel therapeutic strategies such as immuno-oncology agents are of significant interest with future investigation required to understand the molecular predictors of response.

Summary

Mutation-driven therapy in MDS is rapidly expanding and has tremendous potential in a disease where limited standard therapy options exist.

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  1. Department of Hematology and Oncology, H. Lee Moffitt Cancer Center, 12902 Magnolia Drive FOB 3rd Floor, Tampa, FL, 33612, USA

    David M. Swoboda & David A. Sallman

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Swoboda, D.M., Sallman, D.A. Mutation-Driven Therapy in MDS. Curr Hematol Malig Rep 14, 550–560 (2019). https://doi.org/10.1007/s11899-019-00554-4

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