Abstract

Acute myeloid leukemia (AML) is a highly heterogeneous blood cancer. The risk stratification and treatment decisions are mainly based on recurrent genetic and molecular biologic characteristics, which represent the natural targets of targeted therapies of AML. In recent years, new targeted agents have been rapidly developed. However, the majority of existed targeted agents have been failed to show survival improvement in multicenter randomized clinical trials. Drugs with promising effects on overall survival (OS) or event-free survival (EFS) mainly comprise all-trans retinoic acid, arsenic trioxide, midostaurin, enasidenib, decitabine, azacitidine and gemtuzumab. This article reviews the necessity, limitations and development of targeted therapy for AML and the agents which have the potential to improve the prognosis of AML. Key words: Leukemia, myeloid, acute; Targeted therapy; Survival

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