Myelodysplastic syndromes: recent advancements in risk stratification and unmet therapeutic challenges

Am Soc Clin Oncol Educ Book. 2013. doi: 10.14694/EdBook_AM.2013.33.e256.

Abstract

Significant advances have been achieved in understanding and treating myelodysplastic syndromes (MDS) in the past decade. For the first time, three drugs were approved specifically for this disease. Novel sequencing techniques have expanded our understanding of the molecular basis of MDS. Several clinically significant recurrent gene mutations have been identified. The classification and risk stratification of MDS continues to evolve in light of such advances. However, treatment options remain limited and novel therapeutic strategies are needed. In this review we address key questions for management of MDS. How do we better classify and risk stratify MDS, tailoring treatment accordingly? How do we diagnose and manage the challenging group of patients with MDS/myeloproliferative neoplasms (MPN) overlap? And finally, what is on the horizon for novel therapies?

Publication types

  • Research Support, Non-U.S. Gov't
  • Review
  • Video-Audio Media

MeSH terms

  • Antineoplastic Agents / therapeutic use*
  • Biomarkers, Tumor / genetics
  • Decision Support Techniques
  • Drug Design
  • Genetic Predisposition to Disease
  • Humans
  • Molecular Diagnostic Techniques
  • Molecular Targeted Therapy
  • Mutation
  • Myelodysplastic Syndromes / diagnosis
  • Myelodysplastic Syndromes / drug therapy*
  • Myelodysplastic Syndromes / epidemiology
  • Myelodysplastic Syndromes / genetics
  • Phenotype
  • Predictive Value of Tests
  • Risk Assessment
  • Risk Factors
  • Treatment Outcome

Substances

  • Antineoplastic Agents
  • Biomarkers, Tumor