Mancuso Katia, Talarico Marco, Manzato Enrica, Barbato Simona, Tacchetti Paola, Zamagni Elena, Cavo Michele
IRCCS Azienda Ospedaliero-Universitaria di Bologna, Istituto di Ematologia "Seràgnoli", 40138 Bologna, Italy.
Dipartimento di Scienze Mediche e Chirurgiche, Università di Bologna, 40138 Bologna, Italy.
Cancers (Basel). 2025 Apr 30;17(9):1514. doi: 10.3390/cancers17091514.
The introduction of chimeric antigen receptor T (CAR-T) cell and bispecific antibody (BsAb) therapies has revolutionized multiple myeloma (MM) treatment, offering exceptional efficacy, and culminating in recent regulatory approval. However, these therapies have brought unique toxicity challenges, manifesting not only with the well-established cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), but also with the emergence of other common and equally distinctive toxicities, including cytopenias, hypogammaglobulinemia, infections, and the rare but life-threatening immune effector cell-like lymphohistiocytosis syndrome (IEC-HS). These adverse events are characterized by unique mechanisms of action that differ from those of previous treatments for MM, thereby requiring specialized knowledge to optimize day-to-day management and ultimately maximize therapeutic benefits while ensuring patient safety. Additionally, the toxicity profiles of these T-cell engager therapies are becoming increasingly important in treatment decisions, with implications for patient selection and therapy sequencing. In this review, we provide a comprehensive overview of the current state-of-the-art regarding the incidence, etiopathogenetic mechanisms, and clinical manifestations of these increasingly less non-prototypical but still lesser-known side effects than CRS and ICANS, in order to offer clear and actionable insights into their effective management, while emphasizing critical points for future improvement, in view of the increasing number of MM patients who will benefit from the newly approved and upcoming immunotherapies.
嵌合抗原受体T(CAR-T)细胞疗法和双特异性抗体(BsAb)疗法的引入彻底改变了多发性骨髓瘤(MM)的治疗方式,展现出卓越的疗效,并最终获得了近期的监管批准。然而,这些疗法带来了独特的毒性挑战,不仅表现为已为人熟知的细胞因子释放综合征(CRS)和免疫效应细胞相关神经毒性综合征(ICANS),还出现了其他常见且同样独特的毒性反应,包括血细胞减少、低丙种球蛋白血症、感染,以及罕见但危及生命的免疫效应细胞样淋巴组织细胞增生综合征(IEC-HS)。这些不良事件具有独特的作用机制,与先前用于MM的治疗方法不同,因此需要专业知识来优化日常管理,并最终在确保患者安全的同时最大化治疗益处。此外,这些T细胞衔接器疗法的毒性特征在治疗决策中变得越来越重要,对患者选择和治疗顺序具有影响。在本综述中,我们全面概述了这些目前比CRS和ICANS越来越不典型但仍鲜为人知的副作用的发生率、病因发病机制和临床表现的最新情况,以便为其有效管理提供清晰且可操作的见解,同时鉴于将受益于新批准和即将推出的免疫疗法的MM患者数量不断增加,强调未来改进的关键点。