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.

小儿急性髓性白血病 (AML) 由造血前体细胞的克隆扩增发展而来，其特征在于形态学和细胞分子异质性。尽管过去 40 年的总体生存率显着提高，但儿童 AML 的主要治疗挑战是预防复发和管理复发性疾病。大约 25% 患有 AML 的儿童和青少年会出现疾病复发并面临不良预后。我们对复发性 AML 的基因组、表观基因组、代谢组学和免疫病理生理学的更深入了解，有助于为儿科临床试验开发更好的治疗策略。生物合理药物的开发至关重要，因为传统的化学治疗抢救方案并非对所有患者都有效，并且对经过大量预处理的患者存在器官毒性风险。改善复发性儿科 AML 结局的另一个主要障碍是历史上缺乏可用性和参与临床试验。目前正在努力启动新兴疗法的多国临床试验。本综述的目的是总结目前可用和新开发的复发性儿科 AML 疗法。