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CARe 可及性:儿童、青少年和年轻成人获取嵌合抗原受体 (CAR) T 细胞治疗的真实世界医疗决策叙事

英文原题:Access to CARe: A Narrative of Real-World Medical Decision-Making to Access Chimeric Antigen Receptor (CAR) T-Cell Therapy in Children, Adolescents, and Young Adults.

查看英文原题

Access to CARe: A Narrative of Real-World Medical Decision-Making to Access Chimeric Antigen Receptor (CAR) T-Cell Therapy in Children, Adolescents, and Young Adults.

PubMed 2025/01/23(内容时间) Pediatr Blood Cancer Q2 · IF 2.4(JCR 2025)

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中文摘要

嵌合抗原受体(CAR)T细胞疗法对于复发/难治性B细胞血液系统恶性肿瘤患儿是一种可能挽救生命的治疗手段,并且对于其他儿童癌症仍是一种重要的研究性疗法。然而,通过商业使用和临床试验获得这类疗法的机会仍然不理想,尤其是在儿童、青少年和年轻成人中。通过一系列基于病例的讨论,我们概述了关于真实世界医学决策的指导,并提供了增强CAR-T 细胞疗法作为治疗方式可及性的潜在解决方案。

展开英文摘要原文

Chimeric antigen receptor (CAR) T-cell therapy is a potentially life-saving treatment for children with relapsed/refractory B-cell hematologic malignancies, and remains an important investigational therapy for other childhood cancers.

Yet, access to this class of therapies remains suboptimal through both commercial use and clinical trials, especially in children, adolescents, and young adults. Using a series of case-based discussions, we outline guidance on real-world medical decision-making, and offer potential solutions to enhancing access to CAR T-cell therapy as a treatment modality.

论文信息

作者
Steineck A、Chao K、Hall AG、Jacoby E、Leahy AB、Ligon JA、Luciani K、Schultz LM
第一作者单位
Department of Pediatrics, Medical College of Wisconsin, MACC Fund Center for Cancer and Blood Disorders, Milwaukee, Wisconsin, USA.United States
通讯作者单位
Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USA.United States
文献类型
综述 · 病例报告
期刊
Pediatric blood & cancer2025 Apr
原文标识
PubMed 39844726 · DOI 10.1002/pbc.31516