嵌合抗原受体T细胞（CAR T）疗法用于复发或难治性儿童及年轻成人B细胞急性淋巴细胞白血病的潜力。
The potential of CAR T therapy for relapsed or refractory pediatric and young adult B-cell ALL.
作者信息
Forsberg Matthew H, Das Amritava, Saha Krishanu, Capitini Christian M
机构信息
Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA,
Wisconsin Institute for Discovery, University of Wisconsin, Madison, WI, USA,
出版信息
Ther Clin Risk Manag. 2018 Sep 3;14:1573-1584. doi: 10.2147/TCRM.S146309. eCollection 2018.
Recent advancements in immunooncology have resulted in the generation of novel therapies such as chimeric antigen receptor (CAR) T cells, which have revolutionized the treatment of pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia. The journey of tisagenlecleucel (formerly CTL019) from early preclinical success to the US Food and Drug Administration approval is summarized in this review. Strategies that are currently being investigated to improve the efficacy and safety profile of CAR T-cells are also explored, as well as the factors contributing to the present state of patient access to CAR T therapy.
免疫肿瘤学的最新进展催生了嵌合抗原受体（CAR）T细胞等新型疗法，这些疗法彻底改变了复发或难治性B细胞急性淋巴细胞白血病儿科患者的治疗方式。本综述总结了tisagenlecleucel（原CTL019）从早期临床前成功到获得美国食品药品监督管理局批准的历程。同时还探讨了目前正在研究的提高CAR T细胞疗效和安全性的策略，以及影响患者获得CAR T治疗现状的因素。
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