CAR-T（CAR-T）细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T（CAR-T）细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
儿童和年轻成人 B 系急性白血病 CAR-T 治疗综述：新加坡临床视角
英文原题：A Review of CAR-T Therapy in Pediatric and Young Adult B-Lineage Acute Leukemia: Clinical Perspectives in Singapore.
查看英文原题
A Review of CAR-T Therapy in Pediatric and Young Adult B-Lineage Acute Leukemia: Clinical Perspectives in Singapore.
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中文摘要
约10%–15%的儿童B细胞急性淋巴细胞白血病（B-ALL）患者确诊时为高危或属于复发/难治性疾病。在新加坡及该地区尚无CAR-T 细胞疗法时，此类儿童和年轻成人的治疗选择为常规挽救化疗或化学免疫治疗方案，作为桥接治疗，随后接受基于全身放疗的异基因造血干细胞移植（allo-HSCT）。这会导致明显的急性和长期毒性，生存结局也不理想。若能找到长期毒性更少的治愈性挽救疗法，将有助于提高这些儿童和年轻成人的质量调整生命年。本综述聚焦复发/难治性儿童B-ALL疾病负担、现有策略局限、CAR-T 治疗r/r B-ALL的新兴模式，以及我们对儿童CAR-T 疗法卫生经济学和未来方向的本地视角。
展开英文摘要原文
Approximately 10-15% of pediatric B-cell acute lymphoblastic leukemia (B-ALL) are high risk at diagnosis or relapsed/ refractory. Prior to the availability of chimeric antigen receptor T-cell (CAR-T) in Singapore and the region, the treatment options for these paediatric and young adults are conventional salvage chemotherapy or chemo-immunotherapy regimens as a bridge to allogeneic total body irradiation-based hematopoietic stem cell transplantation (allo-HSCT).
This results in significant acute and long-term toxicities, with suboptimal survival outcomes. Finding a curative salvage therapy with fewer long-term toxicities would translate to improved quality-adjusted life years in these children and young adults. In this review, we focus on the burden of relapsed/refractory pediatric B-ALL, the limitations of current strategies, the emerging paradigms for the role of CAR-T in r/r B-ALL, our local perspectives on the health economics and future direction of CAR-T therapies in pediatric patients.
论文信息
- 作者
- Seng MS、Meierhofer AC、Lim FL、Soh SY、Hwang WYK
- 第一作者单位
- Department of Paediatric Hematology and Oncology, KK Women's and Children's Hospital, Singapore.Singapore
- 通讯作者单位
- Duke-NUS Medical School, Singapore.Singapore
- 文献类型
- 综述
- 期刊
- OncoTargets and therapy2023
- 原文标识
- PubMed 36941828 · DOI 10.2147/OTT.S271373
在 PubMed 查看 → 出版商原文（DOI） 全文 PDF（PMC）· 可下载 治疗专题与资料阅读指南 资料来源与翻译说明 报告译文或资料问题 →