Redefining pediatric leukemia care – innovations in risk assessment and targeted treatment: a narrative review

Pediatric leukemia, primarily comprising acute lymphoblastic leukemia (ALL) and acute myeloid leukemia, represents approximately 30% of childhood cancers worldwide. Over the past decade, advances in molecular and genetic profiling, including next-generation sequencing and minimal residual disease monitoring, have refined risk stratification and enabled individualized treatment strategies. Integrating these approaches into clinical practice has improved survival rates, particularly for high-risk patients, by guiding therapy intensity and informing targeted interventions. Targeted therapies, such as tyrosine kinase inhibitors for Philadelphia chromosome-positive ALL, Chimeric Antigen Receptor T cell therapy for relapsed or refractory cases, and monoclonal antibodies like blinatumomab and inotuzumab ozogamicin, have transformed treatment outcomes while reducing chemotherapy-related toxicity. Despite these advances, challenges remain, including the development of therapy resistance (e.g., BCR-ABL1 and FLT3 mutations) and long-term adverse effects such as cardiotoxicity and secondary malignancies. This narrative review summarizes recent innovations in risk assessment and targeted therapies, highlights current challenges, and discusses future directions to optimize personalized pediatric leukemia care.