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复发性/难治性儿童急性髓系白血病的精准医学和免疫治疗方法

英文原题:Precision medicine and immunotherapeutic approaches for relapsed/refractory paediatric acute myeloid leukaemia.

查看英文原题

Precision medicine and immunotherapeutic approaches for relapsed/refractory paediatric acute myeloid leukaemia.

PubMed 2025/06/19(内容时间) Curr Opin Pharmacol Q1 · IF 5.5(JCR 2025)

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中文摘要

尽管采用现代风险适应一线治疗的儿童和青少年/年轻成人急性髓系白血病(AML)患者的临床结局有所改善,但仍有约三分之一的患者会复发。通过临床试验数据,全面的细胞分子遗传学诊断及其与生存结局相关性的显著进展,已识别出原发化疗耐药和/或随后复发风险最高的儿童AML患者,这些患者需要新的治疗方法。这篇简短的北美视角文章重点介绍了在特定高危儿童AML亚型中已显示或 emerging 活性的有前景的小分子抑制剂、抗体-based和细胞免疫疗法,并概述了针对复发儿童和青少年/年轻成人AML的潜在治疗策略。

展开英文摘要原文

Despite improved clinical outcomes amongst children and adolescents/young adults with acute myeloid leukaemia (AML) treated with modern risk-adapted frontline therapies, approximately one third of patients will relapse. Significant advances in comprehensive cytomolecular genetic diagnostics and correlation with survival outcomes via clinical trial data have identified paediatric patients with AML at highest risk of primary chemoresistance and/or subsequent relapse for whom new treatment approaches are needed.

This brief North American-based perspective highlights promising small molecule inhibitors and antibody-based and cellular immunotherapies with demonstrated or emerging activity in specific high-risk subtypes of paediatric AML and outlines potential treatment strategies for children and adolescents/young adults with relapsed AML.

论文信息

作者
Smith CC、Tasian SK
第一作者单位
Children's Medical Center Dallas, Dallas, Texas, United States; University of Texas Southwestern Medical Center, Dallas, Texas, United States. Electronic address: Caroline.Smith@UTSouthwestern.edu.United States
通讯作者单位
Children's Hospital of Philadelphia, Division of Oncology and Center for Childhood Cancer Research, Philadelphia, Pennsylvania, United States; University of Pennsylvania School of Medicine, Department of Pediatrics, Philadelphia, Pennsylvania, United States; Prinses Máxima Center for Pediatric Oncology, Utrecht, the Netherlands. Electronic address: tasians@chop.edu.United States
文献类型
综述
期刊
Current opinion in pharmacology2025 Aug
原文标识
PubMed 40633478 · DOI 10.1016/j.coph.2025.102546