Relapsed pediatric acute myeloid leukaemia: state-of-the-art in 2023.
Relapsed pediatric acute myeloid leukaemia: state-of-the-art in 2023.
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DOI：
10.3324/haematol.2022.281106
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发表时间：
2023-09-01
期刊：
影响因子：
10.1
通讯作者：
Tasian, Sarah K.
中科院分区：
文献类型：
--
作者：
Egan, Grace;Tasian, Sarah K.
尽管新诊断的急性髓系白血病 (AML) 儿童和青少年的预后在过去二十年中已显着改善，但超过三分之一的患者继续复发并经历次优的长期结果。鉴于复发性 AML 患者数量较少以及国际合作历史上的后勤障碍（包括试验资金和药物供应不足），儿科肿瘤合作小组对 AML 复发的管理各不相同，采用了多种挽救方案，并且缺乏普遍定义的反应标准。然而，随着国际 AML 界利用集体知识和资源来描述复发性疾病的遗传和免疫表型异质性、确定特定 AML 亚型中感兴趣的生物靶标、开发新的精准医学方法以在早期临床试验中进行合作研究，并应对全球普遍药物可及性的挑战，复发性儿科 AML 治疗的格局正在迅速发生变化。本综述全面概述了迄今为止在复发性 AML 儿科患者的治疗方面取得的进展，并重点介绍了在活跃和新兴的临床研究中现代、最先进的治疗方法，这些方法是由学术儿科肿瘤学家、实验室科学家、监管机构、制药合作伙伴、癌症研究赞助商和患者倡导者之间的国际合作所促进的。
Although outcomes of children and adolescents with newly diagnosed acute myeloid leukemia (AML) have improved significantly over the past two decades, more than one-third of patients continue to relapse and experience suboptimal long-term outcomes. Given the small numbers of patients with relapsed AML and historical logistical barriers to international collaboration including poor trial funding and drug availability, the management of AML relapse has varied among pediatric oncology cooperative groups with several salvage regimens utilized and a lack of universally defined response criteria. The landscape of relapsed pediatric AML treatment is changing rapidly, however, as the international AML community harnesses collective knowledge and resources to characterize the genetic and immunophenotypic heterogeneity of relapsed disease, identify biological targets of interest within specific AML subtypes, develop new precision medicine approaches for collaborative investigation in early-phase clinical trials, and tackle challenges of universal drug access across the globe. This review provides a comprehensive overview of progress achieved to date in the treatment of pediatric patients with relapsed AML and highlights modern, state-of-the-art therapeutic approaches under active and emerging clinical investigation that have been facilitated by international collaboration among academic pediatric oncologists, laboratory scientists, regulatory agencies, pharmaceutical partners, cancer research sponsors, and patient advocates.
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