转化生物医学

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Acute myeloid leukaemia - therapy - past, present and future

Paul Faduola, Alan Hakim, Juli Mansnerus, Atsuko Imai, Rob O Neill

Acute myeloid leukemia (AML) is characterized by genetic aberrations and a variable response to therapy which has made treatment of AML challenging. The objective of this paper is to review conventional treatments and their development, phase I-III clinical trials of new agents, novel pathways where future interventions may have therapeutic potential, and clinical trial assessment in AML. This study showed that a detailed understanding of the molecular changes associated with chromosomal and genetic abnormalities is necessary to pilot new therapy design. Although several deregulated proteins and genes have been identified, their diversity among AML patients have made it difficult to identify a single substance that can hit these diverse targets . New agents have shown promise but there remains a huge need to be met for effective and targeted therapies to be successful.