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New strategies for relapsed acute myeloid leukemia: fertile ground for translational research

Dinner, Shira N.; Giles, Francis J.; Altman, Jessica K.

Current Opinion in Hematology: March 2014 - Volume 21 - Issue 2 - p 79–86
doi: 10.1097/MOH.0000000000000018
MYELOID DISEASE: Edited by Martin S. Tallman

Purpose of review Although frontline treatment of acute myeloid leukemia (AML) achieves high remission rates, approximately 75–80% of patients will either not respond to or relapse after initial therapy. Some patients, generally those who are younger, can be successfully salvaged with second-line chemotherapy followed by allogeneic stem cell transplantation. There is a great need for novel therapies in AML.

Recent findings Advances in molecular technology recently identified recurrent mutations including mutations of DNMT3A, IDH1/2, and TET2. These mutations represent a major advance in the understanding of leukemogenesis and prognosis, and have enabled the development of targeted therapies.

Summary Improved knowledge of the molecular pathogenesis of AML has allowed development of therapies targeting epigenetic modulation, intracellular signaling pathways, prosurvival proteins, and the tumor microenvironment.

Division of Hematology/Oncology and Northwestern Medicine Developmental Therapeutics Institute, Robert H. Lurie Comprehensive Cancer Center, Northwestern University-Feinberg School of Medicine, Chicago, Illinois, USA

Correspondence to Shira Dinner, MD, 676 N. St. Clair St., Suite 850, Chicago, Illinois 60611, USA. Tel: +1 312 695 2120; e-mail:

© 2014 Wolters Kluwer Health | Lippincott Williams & Wilkins