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ASCO Educational Book; 2008
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Genetically Defined Prognostic Factors and Novel Therapeutics for AML

Richard M. Stone, MD

From the Dana-Farber Cancer Institute, Boston, Massachusetts

Author's disclosure of potential conflicts of interest is found at the end of this article.

Address reprint requests to Richard M. Stone, MD, Dana-Farber Cancer Institute, 44 Binney Street, Boston, MA 02115; E-mail: rstone{at}partners.org

Overview: Although the basic therapeutic approach to acute myeloid leukemias (AML; except for acute promyelocytic leukemia [APL]) has changed little in the past several decades, an increasing understanding of the molecular pathophysiology in this heterogeneous disease suggests that targeted therapies may be on the horizon. The basic approach to non-APL/AML for adults younger than age 60 is induction chemotherapy with an anthracycline plus cytarabine followed by either dose-intensive postremission therapy (high-dose ara-C or high-dose chemotherapy with autologous peripheral blood stem cell rescue) or allogeneic stem cell transplantation. AML in older adults is a common and vexing problem because of the challenging host biology and the multiplicity of resistance mechanisms in myeloblasts that arise in such patients. The genetic underpinnings of the disease are being exploited to define prognosis, and in some cases, to guide therapy; it is hoped that these features also will be used to develop useful targeted therapies that will provide a more effective and less toxic approach for the treatment of this disease.