研究动态
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复发性儿童急性髓性白血病:2023年的现状与技术水平。

Relapsed pediatric acute myeloid leukaemia: state-of-the-art in 2023.

发表日期:2023 Mar 02
作者: Grace Egan, Sarah K Tasian
来源: HAEMATOLOGICA

摘要:

尽管新诊断急性髓性白血病(AML)的儿童和青少年的预后在过去二十年中有了显著改善,但仍有超过三分之一的患者继续复发并经历亚优质的长期结果。考虑到患有复发性AML的患者数量很少,历史上国际协作存在物流障碍,包括试验资金和药物可用性不足,AML复发的管理在儿童肿瘤学合作组之间存在差异,几种挽救疗法被利用并且缺乏普遍定义的治疗反应指标。然而,随着国际AML社区利用共同的知识和资源来描述复发疾病的基因和免疫表型异质性、识别特定AML亚型中感兴趣的生物学靶点、开发新的精准医疗方法进行早期临床试验的合作,以及解决全球范围内药物的普遍获得问题,复发儿童AML治疗的现状正在迅速改变。本次综述全面总结了迄今为止在治疗复发AML的儿科患者方面取得的进展,并强调了在各种积极和新兴的临床研究中正在进行国际协作促进的现代、领先的治疗方法,这些方法是通过学术儿科肿瘤学家、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实现的。
Although outcomes for children and adolescents with newly-diagnosed acute myeloid leukaemia (AML) have improved significantly over the past two decades, more than one-third of patients nonetheless continue to relapse and experience suboptimal long-term outcomes. Given the small numbers of patients with relapsed AML and historic logistical barriers to international collaboration including poor trial funding and drug availability, management of AML relapse has varied amongst paediatric oncology cooperative groups with several utilised salvage regimens and a lack of universally-defined response criteria. The landscape of relapsed paediatric AML treatment is rapidly changing, however, as the international AML community harnesses collective knowledge and resources to characterize the genetic and immunophenotypic heterogeneity of relapsed disease, identify biologic targets of interest within specific AML subtypes, develop new precision medicine approaches for collaborative investigation in earlyphase 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 paediatric 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 amongst academic paediatric oncologists, laboratory scientists, regulatory agencies, pharmaceutical partners, cancer research sponsors, and patient advocates.