Infant acute myeloid leukemia: insights into the biology and therapy

Abstract Infant Acute Myeloid Leukemia (IAML), which manifests within the first two years of life, represents a distinct subtype of childhood acute leukemia characterized by unique clinical and biological features. The incidence of IAML is comparable to that of infant acute lymphoblastic leukemia (IALL), and represents approximately 10–25% of childhood AML cases. Despite improvements in overall survival (OS) rates achieved through risk stratification, stratified chemotherapy, and supportive care, the clinical efficacy of conventional treatment remains constrained. Challenges persist in enhancing the initial remission rate and mitigating relapse after remission. In recent years, the in-depth researches and integrated application of hematopoietic stem cell transplantation (HSCT), targeted therapy, and immunotherapy have introduced novel strategies for precise treatment and personalized clinical management of the disease. This review provides a comprehensive summary of the epidemiological, clinical, and biological characteristics of IAML, along with an exploration of the current stage of treatment research progress.