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在接受 B 细胞成熟抗原靶向 CAR-T 细胞治疗的多发性骨髓瘤患者中平衡疗效与安全性

英文原题:Balancing Efficacy and Safety in Multiple Myeloma Patients Receiving B cell Maturation Antigen-Directed CAR T-Cell Therapy.

PubMed 2026/05/15(内容时间) BioDrugs Q1 · IF 8.5(JCR 2025)

研究概要

靶向 B 细胞成熟抗原(BCMA)的 CAR T 细胞疗法已成为复发/难治性多发性骨髓瘤患者的一种创新且有效的治疗,显示出高缓解率和持久缓解。

中文摘要

靶向 B 细胞成熟抗原(BCMA)的 CAR-T 细胞疗法已成为复发/难治性多发性骨髓瘤患者的一种创新且有效的治疗方式,可带来较高应答率和持久缓解。但其使用伴随广泛毒性,既有特征明确的常见事件,也有较罕见、描述较少的并发症。全面了解常见和罕见毒性,对于及时识别和处理、预防非复发死亡至关重要。常见毒性包括细胞因子释放综合征、免疫效应细胞相关神经毒性综合征(ICANS)、免疫效应细胞相关血液毒性和感染。此外也报告了发生率较低的不良事件,包括非 ICANS 神经毒性(如帕金森样运动障碍)、免疫介导性肠结肠炎、噬血细胞性淋巴组织细胞增多症和继发恶性肿瘤。毒性发生时间和严重程度各异,可能受 CAR-T 细胞扩增和持续程度及患者个体因素影响。本综述总结目前已获批 BCMA 靶向 CAR-T 疗法安全性特征的证据,重点介绍常见和罕见毒性、可能机制及管理策略。

展开英文摘要原文

B-cell maturation antigen (BCMA) directed CAR T-cell therapy has emerged as an innovative and effective treatment for patients with relapsed/refractory multiple myeloma, demonstrating high response rates and durable remissions. However, its use is associated with a broad spectrum of toxicities, ranging from well characterized common events to rarer, less well described complications. A comprehensive understanding of both common and rare toxicities is essential for timely recognition and management to prevent non-relapse mortality. Frequently observed toxicities include cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome (ICANS), immune effector cell-associated hematotoxicity, and infections. In addition, less frequent adverse events have been reported, including non-ICANS neurotoxicity such as parkinsonian-like movement disorders, immune-mediated enterocolitis, hemophagocytic lymphohistiocytosis, and secondary malignancies. The timing and severity of these toxicities is variable and may be influenced by the extent of CAR T-cell expansion and persistence, as well as patient-specific factors. In this review, we summarize currently available evidence with respect to the safety profile of approved BCMA-targeted CAR T-cell therapies, emphasizing both common and rare toxicities, their possible underlying mechanisms, and management strategies.

论文信息

作者
Kuipers MT、Migchelbrink J、Kramer AM、Kouwenhoven MCM、Zweegman S、Groen K、van de Donk NWCJ
第一作者单位
Department of Hematology, Amsterdam UMC, Vrije Universiteit Amsterdam, De Boelelaan 1117, 1081 HV, Amsterdam, The Netherlands.Netherlands
通讯作者单位
Department of Hematology, Amsterdam UMC, Vrije Universiteit Amsterdam, De Boelelaan 1117, 1081 HV, Amsterdam, The Netherlands. n.vandedonk@amsterdamumc.nl.Netherlands
文献类型
综述
期刊
BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy2026 Jul
原文标识
PubMed 42141360 · DOI 10.1007/s40259-026-00784-y