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儿童急性髓系白血病的新兴 CAR-T 疗法

英文原题:Emerging CART Therapies for Pediatric Acute Myeloid Leukemia.

查看英文原题

Emerging CART Therapies for Pediatric Acute Myeloid Leukemia.

PubMed 2024/10/29(内容时间) J Pediatr Hematol Oncol Q3 · IF 0.9(JCR 2025)

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中文摘要

过去几十年中,儿童急性髓系白血病(AML)的预后逐步改善。然而,疾病复发后,总生存率为40%至50%;对化疗难治性患者而言,生存率甚至更低。亟需为这些儿童开发有效且毒性更低的疗法。近年来,嵌合抗原受体(CAR)T细胞等免疫靶向疗法开始应用,并在B细胞恶性肿瘤中显示出卓越的临床活性。基于其在其他血液系统恶性肿瘤中的研究结果,针对AML的CAR-T 疗法也正在开发中。AML CAR-T 疗法面临的最大挑战是确定特异性靶抗原,因为AML细胞表达的抗原通常也存在于健康造血干细胞上。本文概述CAR-T 在儿童AML中的应用前景,重点讨论AML CAR-T 已测试或正在研究的常见靶抗原。

展开英文摘要原文

The prognosis of children with acute myeloid leukemia (AML) has improved incrementally over the last decades.

However, at relapse, overall survival (OS) 40% to 50% and is even lower for patients with chemorefractory disease. Effective and less-toxic therapies are urgently needed for these children. In the last years, immune-directed therapies such as chimeric antigen receptor (CAR)-T cells were introduced, which showed outstanding clinical activity against B-cell malignancies. CART therapies are being developed for AML on the basis of the results obtained for other hematologic malignancies.

The biggest challenge of CART therapy for AML is to identify a specific target antigen, since antigens expressed in AML cells are usually shared with healthy hematopoietic stem cells. An overview of prospects of CART in pediatric AML, focused on the common antigens targeted by CART in AML that have been tested or are currently under investigation, is provided in this manuscript.

论文信息

作者
Ceolin V、Spadea M、Apolito V、Saglio F、Fagioli F
单位
Department of Pediatric Oncology/Hematology, Regina Margherita Children's Hospital.
文献类型
综述
期刊
Journal of pediatric hematology/oncology2024 Nov 1
原文标识
PubMed 39469946 · DOI 10.1097/MPH.0000000000002956