Novel therapies for myelodysplastic syndromes

Authors

  • Stefan Faderl M.D.,

    Corresponding author
    1. Department of Leukemia, The University of Texas M.D. Anderson Cancer Center, Houston, Texas
    • Department of Leukemia, Box 428. The University of Texas M.D. Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030
    Search for more papers by this author
    • Fax (713) 794-4297

  • Hagop M. Kantarjian M.D.

    1. Department of Leukemia, The University of Texas M.D. Anderson Cancer Center, Houston, Texas
    Search for more papers by this author

  • Dr. Armand Keating was Guest Editor for this article.

Abstract

BACKGROUND

The assessment of patients with myelodysplastic syndromes (MDS) and the choice of therapies remain challenging. New therapies are now emerging after the identification of molecular targets that result in improvement of hematologic parameters and may hold promise for the prevention of disease progression.

METHODS

A review of the English literature was performed that included original articles and related reviews from MEDLINE (PubMed) and abstracts based on published meeting material.

RESULTS

MDS is a heterogeneous group of disorders. Although current classification and prognostic schemes have proven valid to define subgroups, they are insufficient to take into consideration the significant biologic diversity of MDS. New molecular targets are identified as the mosaic of pathophysiologic pathways in MDS is being unraveled. Novel and targeted therapeutic agents, such as the inhibition of farnesyl transferases and receptor tyrosine kinases, more potent thalidomide analogs, and arsenic trioxide, have shown encouraging results and may offer durable benefit to patients with MDS.

CONCLUSIONS

Although progress has been made in the understanding of clinical manifestations and some of the molecular pathways underlying ineffective hematopoiesis and leukemic transformation in MDS, intensive clinical and laboratory research continues to 1) identify further relevant pathophysiologic pathways, 2) better define MDS subgroups, and 3) develop new drugs based on a clearer understanding of disease biology. Cancer 2004. © 2004 American Cancer Society.

Ancillary