[Pediatric acute lymphoblastic leukemia: update on pathophysiology and management].

The prognosis of pediatric acute lymphoblastic leukemia (ALL) has dramatically improved, both basic research and clinical studies are continuously conducted in pursuit of further improvement. Recent advances in genomic analysis technology have enabled us to comprehensively identify genomic alterations in leukemic cells and thus have contributed to the better understanding of the molecular pathogenesis underlying ALL development. These genomic alterations can be applied not only as prognostic factors but also as therapeutic targets. Although somatically acquired genomic alterations in leukemic cells have long been in the focus of molecular analysis for ALL, inherited genetic (germline) variations are now acknowledged as factors involved in the interpretation of ALL susceptibility, therapy response, and toxicities of ALL therapy. Integrated understanding of leukemia and host biology is thus required. In terms of treatment, stratified treatment based on minimal residual disease status and the adaptation of novel therapeutic agents, such as small molecule agents and immunotherapy, appears to be a promising strategy to improve relapsed/refractory ALL without excess complications. Clinical trials should focus on incorporating genome-based diagnostics/stratification and the use of these novel agents into standard therapy in the future.