{"title":"西他他烯自体醇治疗多发性骨髓瘤。","authors":"Sarah A Holstein","doi":"10.1358/dot.2023.59.1.3509751","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":72848,"journal":{"name":"","volume":"59 1","pages":"1-16"},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Ciltacabtagene autoleucel for the treatment of multiple myeloma.\",\"authors\":\"Sarah A Holstein\",\"doi\":\"10.1358/dot.2023.59.1.3509751\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":72848,\"journal\":{\"name\":\"\",\"volume\":\"59 1\",\"pages\":\"1-16\"},\"PeriodicalIF\":0.0,\"publicationDate\":\"2023-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1358/dot.2023.59.1.3509751\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1358/dot.2023.59.1.3509751","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
摘要
虽然多发性骨髓瘤(MM)的治疗方案不断扩大,但这种疾病仍然需要多种治疗方案,每一种后续治疗方案的有效性普遍下降。b细胞成熟抗原(BCMA)导向嵌合抗原受体(CAR) t细胞疗法的发展已被证明是这一规则的例外。在美国食品和药物管理局(FDA)批准BCMA CAR - t细胞疗法ciltacabtagene autoleuel (cilta-cel)的试验中,在大量预处理的患者中观察到深度和持久的缓解率。在这篇综述中,我们总结了cilta-cel的现有临床试验数据,包括对值得注意的不良事件的讨论,以及对可能导致MM管理范式改变的正在进行的研究的讨论。此外,我们讨论了目前围绕cilta-cel的实际应用的问题。
Ciltacabtagene autoleucel for the treatment of multiple myeloma.
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.