Molecular therapeutic approaches for pediatric acute myeloid leukemia.

摘要: Approximately two thirds of children with acute myeloid leukemia (AML) are cured intensive multi-agent chemotherapy. However, primary chemorefractory and relapsed AML remains a significant source childhood cancer mortality, highlighting the need for new therapies. Further therapy intensification traditional cytotoxic agents is not feasible given potential toxicity to normal tissues conventional chemotherapy risk long-term end-organ dysfunction. Significant emphasis has been placed upon development molecularly targeted therapeutic approaches adults high-risk subtypes goal improving remission induction minimizing relapse. Several promising currently in clinical testing or late preclinical AML, including monoclonal antibodies against cell surface proteins, kinase inhibitors, proteasome epigenetic agents, chimeric antigen receptor engineered T immunotherapies. Many these therapies have specifically tested relapsed/refractory via phase 1 2 trials smaller number under 3 evaluation de novo AML. Although successful identification implementation drugs formidable challenge, enthusiasm novel molecular great benefit who will otherwise fail standard therapy.