Pediatric Blood Cancer and Childhood Leukemia: Disease Burden, Molecular Pathogenesis, Treatment-Related Complications and Emerging Precision Therapeutics: A Review

Pediatric blood cancers, particularly childhood leukemia, constitute a major proportion of malignancies affecting children worldwide and remain a significant global health concern. Leukemia originates in the bone marrow and is characterized by the uncontrolled proliferation of abnormal white blood cells, leading to impaired hematopoiesis and immune dysfunction. The most common subtypes include acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), with ALL being the predominant form in pediatric populations. The global epidemiology of pediatric leukemia demonstrates substantial variation in incidence, survival rates and access to treatment across different regions. Survival outcomes are significantly higher in developed countries compared to low and middle-income regions, largely due to disparities in healthcare infrastructure, early diagnosis and availability of advanced therapies. Advances in molecular biology have greatly enhanced the understanding of leukemogenesis, highlighting the roles of chromosomal abnormalities, gene mutations, dysregulated signaling pathways, and epigenetic modifications. These insights have contributed to improved diagnostic accuracy and the development of targeted therapeutic strategies. Conventional treatment approaches, including chemotherapy, radiation therapy and hematopoietic stem cell transplantation, have significantly improved survival rates. However, these therapies are often associated with considerable short-term and long-term complications such as immunosuppression, organ toxicity and secondary malignancies. Recent advances in precision therapeutics, including targeted therapy, immunotherapy (notably CAR-T cell therapy) and gene-based approaches, offer improved efficacy with reduced toxicity. These innovations mark a shift toward personalized medicine, enhancing prognosis and quality of life. This review provides a comprehensive overview of disease burden, molecular pathogenesis, treatment-related complications and emerging precision therapeutic strategies in pediatric blood cancer and childhood leukemia.