Role of Precision Medicine in Pediatric Oncology

Childhood cancer is the leading cause of nonaccidental death in children and adolescents. Over the past 50 years, development of novel therapies and improvements in supportive care have led to improvements in long-term survival rates. However, there remains great morbidity associated with cancer treatment among childhood cancer survivors, and the outcomes for patients who relapse remain poor. The introduction of precision medicine, an approach that uses the understanding of genetic and biochemical profiles of a disease (as enabled by next-generation sequencing) to tailor treatment to a patient, has quickly started to change the diagnostic and therapeutic landscape of pediatric oncology. With its use, a better understanding of tumor biology, improved classification systems for various cancers, and genetically and molecularly targeted therapeutic strategies have been developed. We review the implementation of precision medicine in pediatric oncology and its effect on diagnosis, management, and treatment of pediatric cancers. [Pediatr Ann. 2022;51(1):e8-e14.].