Pediatric acute myeloid leukemia (AML) develops from clonal expansion of hematopoietic precursor cells and is characterized by morphologic and cytomolecular heterogeneity. Although the past 40 years have seen significant improvements in overall survival, the prevailing treatment challenges in pediatric AML are the prevention of relapse and the management of relapsed disease. Approximately 25% of children and adolescents with AML suffer disease relapse and face a poor prognosis. Our greater understanding of the genomic, epigenomic, metabolomic, and immunologic pathophysiology of relapsed AML allows for better therapeutic strategies that are being developed for pediatric clinical trials. The development of biologically rational agents is critical as conventional chemotherapeutic salvage regimens are not effective for all patients and pose risk of organ toxicity in heavily pretreated patients. Another major barrier to improvement in outcomes for relapsed pediatric AML is the historic lack of availability and participation in clinical trials. There are ongoing efforts to launch multinational clinical trials of emerging therapies. The purpose of this review is to summarize currently available and newly developed therapies for relapsed pediatric AML.
© 2022. The Author(s), under exclusive licence to Springer Nature Limited.