Epigenetic therapies in MDS and AML

Elizabeth A. Griffiths, Steven D. Gore

Research output: Chapter in Book/Report/Conference proceedingChapter

Abstract

The use of low dose hypomethylating agents for patients with myelodysplastic syndrome (MDS) and secondary acute myeloid leukemia (AML) has had made a significant impact. In the past, therapies for these diseases were limited and patients who elected to receive treatment were subject to highly toxic, inpatient chemotherapeutics, which were often ineffective. In the era of hypomethylating agents (azacitidine and decitabine), a patient with high grade MDS or AML with multilineage dysplasia can be offered the alternative of outpatient, relatively low-toxicity therapy. Despite the fact that CR (CR) rates to such agents remain relatively low at 15-20%, a much larger percentage of patients will have clinically significant improvements in hemoglobin, platelet, and neutrophil counts while maintaining good outpatient quality of life. As our clinical experience with azanucleotides expands, questions regarding patient selection, optimal dosing strategy, latency to best response and optimal duration of therapy following disease progression remain, but there is no question that for some patients these agents offer, for a time, an almost miraculous clinical benefit. Ongoing clinical trials in combination and in sequence with conventional therapeutics, with other epigenetically active agents, or in conjunction with bone marrow transplantation continue to provide promise for optimization of these agents for patients with myeloid disease. Although the mechanism(s) responsible for the proven efficacy of these agents remain a matter of some controversy, activity is thought to stem from induction of DNA hypomethylation, direct DNA damage, or possibly even immune modulation; there is no question that they have become a permanent part of the armamentarium against myeloid neoplasms.

Original languageEnglish (US)
Title of host publicationAdvances in Experimental Medicine and Biology
Pages253-283
Number of pages31
Volume754
DOIs
StatePublished - 2013

Publication series

NameAdvances in Experimental Medicine and Biology
Volume754
ISSN (Print)00652598

Fingerprint

Myelodysplastic Syndromes
Acute Myeloid Leukemia
Epigenomics
Proxy
decitabine
Outpatients
Therapeutics
Azacitidine
Poisons
Bone Marrow Transplantation
Platelet Count
Patient Selection
DNA Damage
Disease Progression
Inpatients
Hemoglobins
Neutrophils
DNA
Quality of Life
Platelets

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)
  • Medicine(all)

Cite this

Griffiths, E. A., & Gore, S. D. (2013). Epigenetic therapies in MDS and AML. In Advances in Experimental Medicine and Biology (Vol. 754, pp. 253-283). (Advances in Experimental Medicine and Biology; Vol. 754). https://doi.org/10.1007/978-1-4419-9967-2-13

Epigenetic therapies in MDS and AML. / Griffiths, Elizabeth A.; Gore, Steven D.

Advances in Experimental Medicine and Biology. Vol. 754 2013. p. 253-283 (Advances in Experimental Medicine and Biology; Vol. 754).

Research output: Chapter in Book/Report/Conference proceedingChapter

Griffiths, EA & Gore, SD 2013, Epigenetic therapies in MDS and AML. in Advances in Experimental Medicine and Biology. vol. 754, Advances in Experimental Medicine and Biology, vol. 754, pp. 253-283. https://doi.org/10.1007/978-1-4419-9967-2-13
Griffiths EA, Gore SD. Epigenetic therapies in MDS and AML. In Advances in Experimental Medicine and Biology. Vol. 754. 2013. p. 253-283. (Advances in Experimental Medicine and Biology). https://doi.org/10.1007/978-1-4419-9967-2-13
Griffiths, Elizabeth A. ; Gore, Steven D. / Epigenetic therapies in MDS and AML. Advances in Experimental Medicine and Biology. Vol. 754 2013. pp. 253-283 (Advances in Experimental Medicine and Biology).
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