Pediatric acute leukemia therapies informed by molecular analysis of high-risk disease

The acute leukemias are the most common cancer of children, adolescents, and young adults. These diseases are characterized by a tremendous variability in clinical course, prompting a continuing search for accurate predictors of outcome. Using algorithms based on clinical features at presentation, response to therapy, and several molecular analyses, some patients are diagnosed with features of high-risk disease and comparatively greater risk for relapse. Molecular analyses of patients with high-risk acute leukemias have resulted in an improved understanding of how dysregulated cellular signaling can affect resistance to conventional therapy. Whereas exciting discoveries continue to be made in the identification of relevant molecular biomarkers and targeted therapies, the challenges and opportunities associated with these findings remain to be clearly defined in future clinical trials.