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CAR-T 细胞治疗多发性骨髓瘤:现状与未来潜力更新

英文原题:CAR-T cell therapy for multiple myeloma: An update on the current state and future potential.

查看英文原题

CAR-T cell therapy for multiple myeloma: An update on the current state and future potential.

PubMed 2025/10/04(内容时间) Best Pract Res Clin Haematol Q2 · IF 3.4(JCR 2025)

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中文摘要

多发性骨髓瘤(MM)领域在过去十年中取得了显著的治疗进展,包括近期出现的CAR-T 细胞疗法。目前有两种FDA批准的用于MM的CAR-T 产品,均靶向B细胞成熟抗原(BCMA)。这些药物在晚期疾病患者中显示出显著的治疗效果,现已获批用于更早期的复发阶段。CAR-T 疗法与独特的不良反应相关,包括细胞因子释放综合征、神经毒性、低丙种球蛋白血症、感染风险和血细胞减少。尽管缓解率显著,但大多数患者最终仍会出现疾病进展,原因可能是由于CAR-T 细胞持久性不足和耗竭、抗原阴性变异株的出现以及免疫抑制性肿瘤微环境所导致的治疗耐药。该领域的重大努力致力于提高CAR-T 细胞疗法的疗效并减轻其毒性。在本综述中,我们回顾了已获批和正在研究中的用于MM的CAR-T 模型。

展开英文摘要原文

The field of multiple myeloma (MM) has seen significant therapeutic advances over the last decade including the recent advent of Chimeric Antigen Receptor T cell (CAR-T) therapy. Currently there are two FDA approved CAR-T products for MM, both targeting B cell maturation antigen (BCMA). These agents have demonstrated striking therapeutic efficacy in patients with advanced disease and are now also approved in earlier relapse. CAR-T therapy is associated with unique toxicities including cytokine release syndrome, neurotoxicity, hypogammaglobulinemia, risk of infections and cytopenias.

While response rates are dramatic, most patients ultimately experience disease progression due to therapeutic resistance potentially arising from lack of persistence and exhaustion of CAR-T cells, emergence of antigen negative variants, and the immunosuppressive tumor microenvironment. Significant efforts in the field are dedicated towards improving the efficacy and mitigating the toxicity of CAR-T cell therapy. In this publication, we review the approved and investigational CAR-T models for MM.

论文信息

作者
Pophali P、Rosenblatt J、Avigan D
第一作者单位
Division of Hematology and Oncology, Beth Israel Deaconess Medical Center, 330 Brookline Avenue, Boston, MA, 02215, USA. Electronic address: ppophali@bidmc.harvard.edu.Israel
通讯作者单位
Division of Hematology and Hematologic Malignancies, Beth Israel Deaconess Medical Center, 330 Brookline Avenue, Boston, MA, 02215, USA. Electronic address: davigan@bidmc.harvard.edu.Israel
文献类型
综述
期刊
Best practice & research. Clinical haematology2025 Dec
原文标识
PubMed 41320446 · DOI 10.1016/j.beha.2025.101659