Management and orphan drug development for acute myeloid leukemia

Introduction: Acute myeloid leukemia (AML) is a genetically heterogeneous disease with blocked differentiation of myeloid precursor cells. Genetic alterations have been identified in AML patients over the past several decades, which significantly improved the characterization and diagnosis of this disease. However, the therapeutic options for the treatment of AML are still limited. Areas covered: This review summarizes the diagnosis and prognosis of AML and current strategies for AML treatment. The new treatment agents that are in use and under development are focused on fms-related tyrosine kinase 3 tyrosine kinase inhibitors, heat shock protein 90 inhibitors, hypomethylating agents, immunotherapy of AML and other potential strategies. Expert opinion: Although the heterogeneity of AML significantly influences the outcomes in the treatment of AML patients, the application of high-throughput technology (such as next-generation sequencing), which helps to identify new genetic events and understand the molec...