Holstein Sarah A
Department of Internal Medicine, University of Nebraska Medical Center, Omaha, Nebraska, USA.
Drugs Today (Barc). 2023 Jan;59(1):1-16. doi: 10.1358/dot.2023.59.1.3509751.
While treatment options for multiple myeloma (MM) are continuing to expand, this disease remains one characterized by requiring multiple lines of therapy, with generally decreasing effectiveness of each subsequent line. The development of B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T-cell therapy has proven an exception to this rule. In the trial that led to approval of the BCMA CAR T-cell therapy ciltacabtagene autoleucel (cilta-cel) by the U.S. Food and Drug Administration (FDA), deep and durable response rates were observed in heavily pretreated patients. In this review we summarize the available clinical trial data for cilta-cel, including discussion on notable adverse events, as well as discuss ongoing studies that are likely to lead to paradigm changes in the management of MM. In addition, we discuss the issues that currently surround the real-world utilization of cilta-cel.
虽然多发性骨髓瘤（MM）的治疗选择在不断增加，但这种疾病的特点仍然是需要多线治疗，而且通常后续每一线治疗的效果都会降低。以B细胞成熟抗原（BCMA）为靶点的嵌合抗原受体（CAR）T细胞疗法的出现已证明是这一规律的例外。在美国食品药品监督管理局（FDA）批准BCMA CAR T细胞疗法西达基奥仑赛（cilta-cel）的试验中，在经过大量预处理的患者中观察到了深度且持久的缓解率。在本综述中，我们总结了西达基奥仑赛的现有临床试验数据，包括对显著不良事件的讨论，并探讨可能导致MM管理模式改变的正在进行的研究。此外，我们还讨论了目前围绕西达基奥仑赛实际应用的问题。
