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平衡 CAR T:CAR T 细胞疗法在血液恶性肿瘤中的疗效和安全性观点。

Balancing the CAR T: perspectives on efficacy and safety of CAR T-cell therapy in hematologic malignancies.

机构信息

Memorial Sloan Kettering Cancer Center, New York, NY. Email:

出版信息

Am J Manag Care. 2021 Aug;27(13 Suppl):S243-S252. doi: 10.37765/ajmc.2021.88736.

Abstract

Chimeric antigen receptor (CAR) T-cell therapy is an example of how immunotherapy is revolutionizing the treatment of hematologic malignancies with unprecedented response rates in patients with relapsed/refractory lymphoma, multiple myeloma, and acute lymphoblastic leukemia. The process for administering CAR T-cell therapy is complex, with multiple steps including CAR T-cell manufacturing, lymphodepleting chemotherapy, cellular therapy infusion, and management of short-term and long-term toxicities. While effective, broad use of CAR T-cell therapies is limited by potential for life-threatening toxicities, challenges related to manufacturing a patient-specific product, high costs and inadequate reimbursement, and incomplete or unsustained disease response. Pharmacists are intricately involved in the process of providing CAR T-cell therapy both at the organizational level of budgeting and coordinating therapy and in direct patient care roles providing counseling and support for adverse effect management. Research in CAR T-cell therapy is expected to improve tolerability and expand indications to more types of malignancies and earlier phases of disease. Managed care professionals should have an understanding of the clinical trial data and place in therapy in lymphoma, myeloma, and acute lymphoblastic leukemia as well as guideline recommendations for adverse effect management associated with CAR T-cell therapies.

摘要

嵌合抗原受体 (CAR) T 细胞疗法是免疫疗法如何彻底改变血液系统恶性肿瘤治疗的一个例子,在复发/难治性淋巴瘤、多发性骨髓瘤和急性淋巴细胞白血病患者中取得了前所未有的反应率。CAR T 细胞疗法的给药过程非常复杂,包括 CAR T 细胞制造、淋巴细胞耗竭化疗、细胞疗法输注以及短期和长期毒性的管理。尽管有效,但由于潜在的致命毒性、制造患者特异性产品的相关挑战、高昂的成本和不足的报销以及不完全或不可持续的疾病反应,CAR T 细胞疗法的广泛应用受到限制。药剂师在提供 CAR T 细胞疗法的过程中都有深入的参与,包括在预算和协调治疗的组织层面,以及在直接为患者提供咨询和支持以管理不良反应的角色。CAR T 细胞疗法的研究预计将提高耐受性并扩大适应证,涵盖更多类型的恶性肿瘤和疾病的早期阶段。管理式医疗保健专业人员应了解临床试验数据以及 CAR T 细胞疗法在淋巴瘤、骨髓瘤和急性淋巴细胞白血病中的治疗地位,以及与 CAR T 细胞疗法相关的不良反应管理的指南建议。

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