Risk-Stratified Therapy for Pediatric Acute Myeloid Leukemia

SIMPLE SUMMARY: Owing to the 40-year worldwide efforts for improving diagnosis and therapy for acute myeloid leukemia (AML), the second most common type of leukemia in children, overall survival rates of children with AML have now reached 70% to 80% in developed countries. This review article comprehensively describes the history and advances in the current state-of-the-art risk-stratified therapy for AML in children. However, it is likely that the traditional approaches have already reached their limits, and therefore, novel approaches are absolutely essential. The current state and future directions for incorporating novel molecular-targeted drugs into contemporary therapy through international collaboration are also extensively discussed. These aspects present key solutions for further improvements in outcomes of children with AML. ABSTRACT: Acute Myeloid Leukemia (AML) is the second most common type of leukemia in children. Recent advances in high-resolution genomic profiling techniques have uncovered the mutational landscape of pediatric AML as distinct from adult AML. Overall survival rates of children with AML have dramatically improved in the past 40 years, currently reaching 70% to 80% in developed countries. This was accomplished by the intensification of conventional chemotherapy, improvement in risk stratification using leukemia-specific cytogenetics/molecular genetics and measurable residual disease, appropriate use of allogeneic hematopoietic stem cell transplantation, and improvement in supportive care. However, the principle therapeutic approach for pediatric AML has not changed substantially for decades and improvement in event-free survival is rather modest. Further refinements in risk stratification and the introduction of emerging novel therapies to contemporary therapy, through international collaboration, would be key solutions for further improvements in outcomes.