文章：

儿童急性髓系白血病风险分层治疗

Risk-Stratified Therapy for Pediatric Acute Myeloid Leukemia

原文发布日期：18 August 2023

DOI: 10.3390/cancers15164171

类型: Article

开放获取: 是

英文摘要：

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.

摘要翻译： 

急性髓系白血病是儿童中第二常见的白血病类型。近期高分辨率基因组分析技术的进展揭示了儿童急性髓系白血病与成人不同的突变谱系。过去40年间，儿童急性髓系白血病患者的总体生存率显著提高，目前在发达国家已达到70%至80%。这一成果得益于传统化疗方案的强化、基于白血病特异性细胞遗传学/分子遗传学及可测量残留病灶的风险分层改进、异基因造血干细胞移植的合理应用以及支持治疗的进步。然而，数十年来儿童急性髓系白血病的主要治疗策略并未发生根本性改变，无事件生存率的提升相对有限。通过国际合作进一步完善风险分层体系，并将新兴疗法引入当代治疗方案，将成为改善预后的关键途径。

原文链接：

Risk-Stratified Therapy for Pediatric Acute Myeloid Leukemia