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嵌合抗原受体 T 细胞疗法:临床疗效、毒性及门诊管理最佳实践的全面综述。

Chimeric Antigen Receptor T Cell Therapy: A Comprehensive Review of Clinical Efficacy, Toxicity, and Best Practices for Outpatient Administration.

机构信息

Department of Pharmacy, University of North Carolina Medical Center, Chapel Hill, North Carolina.

Department of Pharmacy, Vanderbilt University Medical Center, Nashville, Tennessee.

出版信息

Transplant Cell Ther. 2021 Jul;27(7):558-570. doi: 10.1016/j.jtct.2021.01.014. Epub 2021 Jan 20.

DOI:10.1016/j.jtct.2021.01.014
PMID:33910041
Abstract

Chimeric antigen receptor T cell (CAR T) therapy has been integrated into treatment algorithms for acute leukemia, lymphoma, and, most recently, multiple myeloma. The number of clinical trials in both hematologic and solid tumor malignancies for new products and potential indications continues to grow. The clinical toxicities of CAR T therapy include cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome, which often warrant inpatient admission for close monitoring and treatment. Consequently, many centers have built processes around the administration of these cells in the inpatient setting. As new products gain Food and Drug Administration approval with more manageable toxicity profiles, and as institutions gain experience with the management of these toxicities, outpatient administration and monitoring should be expected. In addition, payor reimbursements for inpatient treatment have put the sustainability of inpatient CAR T therapy in jeopardy, especially for centers with a payor mix that includes a high proportion of Medicare patients. This has the serious potential to limit access to care. As the use of CAR T therapy continues to expand, changes in payment models, care settings, or both are needed to ensure the sustainability of safe, efficient, and cost-effective treatment. This review outlines the efficacy and toxicity of currently approved products, as well as best practices to optimize the management of CAR T cell therapy in the outpatient setting.

摘要

嵌合抗原受体 T 细胞(CAR T)疗法已被纳入急性白血病、淋巴瘤以及最近的多发性骨髓瘤的治疗方案中。在血液系统恶性肿瘤和实体瘤恶性肿瘤中,针对新产品和潜在适应证的临床试验数量持续增长。CAR T 疗法的临床毒性包括细胞因子释放综合征和免疫效应细胞相关神经毒性综合征,这通常需要住院密切监测和治疗。因此,许多中心已经建立了在住院环境下管理这些细胞的流程。随着新的产品获得食品和药物管理局(FDA)批准,具有更可管理的毒性特征,并且随着机构在管理这些毒性方面获得经验,预计将可以进行门诊管理和监测。此外,住院 CAR T 治疗的支付方报销使住院 CAR T 治疗的可持续性受到威胁,特别是对于支付方组合中包括大量医疗保险患者的中心。这有可能严重限制获得治疗的机会。随着 CAR T 疗法的使用不断扩大,需要改变支付模式、护理环境或两者兼而有之,以确保安全、高效和具有成本效益的治疗的可持续性。本综述概述了目前批准的产品的疗效和毒性,以及优化门诊环境下 CAR T 细胞治疗管理的最佳实践。

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