Molecular-Targeted Therapy of Pediatric Acute Myeloid Leukemia

Acute myeloid leukemia (AML) accounts for approximately 15-20% of all childhood leukemia cases. The overall survival of children with acute myeloid leukemia does not exceed 82%, and the 5-year event-free survival rates range from 46% to 69%. Such suboptimal outcomes are the result of numerous mutations and epigenetic changes occurring in this disease that adversely affect the susceptibility to treatment and relapse rate. We describe various molecular-targeted therapies that have been developed in recent years to meet these challenges and were or are currently being studied in clinical trials. First introduced in adult AML, novel forms of treatment are slowly beginning to change the therapeutic approach to pediatric AML. Despite promising results of clinical trials investigating new drugs, further clinical studies involving greater numbers of pediatric patients are still needed to improve the outcomes in childhood AML.

Automated summary

Acute myeloid leukemia (AML) comprises 15-20% of childhood leukemia cases, with a survival rate of not exceeding 82% and 5-year event-free survival rates ranging from 46% to 69%. The disease is influenced by multiple mutations and epigenetic changes, affecting treatment susceptibility and relapse rate. Molecular-targeted therapies have been developed and studied in clinical trials to address these challenges, originally introduced in adult AML and now gradually changing the therapeutic approach to pediatric AML. Research involving larger pediatric populations is needed to further improve outcomes.