Targeted therapy

Similar to immunotherapeutic approaches, the use of targeted therapy attempts to circumvent the severe toxicity of traditional chemotherapy by employing agents that target leukemia-specific variants and/or their abnormal present or missing byproducts. While randomized clinical trials have not yet demonstrated that targeted therapies improve outcomes in children with newly diagnosed AML, single-arm trials have demonstrated a survival benefit, such as the sorafenib trial described below. Because most data on the use of targeted agents are from adult clinical trials, the adult experience is initially described, followed by a description of the more limited experience in children.