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阿基仑赛和替雷利珠单抗嵌合抗原受体T细胞疗法治疗复发或难治性滤泡性淋巴瘤的疗效、安全性及成本最小化分析

Efficacy, safety, and cost-minimization analysis of axicabtagene ciloleucel and tisagenlecleucel CAR T-Cell therapies for treatment of relapsed or refractory follicular lymphoma.

作者信息

Ghanem Buthainah

机构信息

Department of Pharmaceutical Economics and Policy, School of Pharmacy, Chapman University, Irvine, CA, USA.

出版信息

Invest New Drugs. 2023 Oct;41(5):710-718. doi: 10.1007/s10637-023-01389-w. Epub 2023 Aug 12.

Abstract

Axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel) are chimeric antigen receptor (CAR) T-cell therapies used to treat adult patients with relapsed or refractory follicular lymphoma (rrFL) after two or more lines of systemic therapy. In the absence of head-to-head clinical trials, this study aimed to compare the efficacy, safety, and cost of axi-cel and tisa-cel in the treatment of rrFL after at least two lines of treatment. Overall response rate (ORR) and safety signals were compared using reporting odds ratios (RORs) with 95% confidence intervals (CIs) at p < 0.05. Progression-free survival (PFS), duration of response (DoR), and overall survival (OS) were compared using the Kaplan?Meier method with a log-rank test. Cost and cost-minimization analyses of drug acquisition, drug administration, serious adverse events (AEs), and relapsed management were calculated. Costs were extracted from the IBM-Micromedex Red Book, Centers for Medicare and Medicaid Services, and existing literature. Statistical analyses were conducted using Microsoft Excel and R version 4.0.5. No statistically significant differences were observed between axi-cel and tisa-cel in terms of ORR, DoR, and OS (p > 0.05). PFS was significantly better with tisa-cel (p < 0.05). Axi-cel was significantly associated with higher incidences of CRS, neurologic events, and grade 3-4 AEs than tisa-cel (ROR > 1, p < 0.05). Axi-cel and tisa-cel cost $512,021 and $450,885 per patient, respectively, resulting in savings of US$61,136 with tisa-cel over axi-cel. Tisa-cel appears to have a better safety profile, fewer serious AEs, lower mortality rate, and lower cost than axi-cel.

摘要

阿基仑赛注射液(axi-cel)和替雷利珠单抗注射液(tisa-cel)是嵌合抗原受体(CAR)T细胞疗法,用于治疗经两种或更多线全身治疗后复发或难治性滤泡性淋巴瘤(rrFL)的成年患者。在缺乏头对头临床试验的情况下,本研究旨在比较axi-cel和tisa-cel在至少两线治疗后治疗rrFL的疗效、安全性和成本。使用报告比值比(ROR)和95%置信区间(CI)在p<0.05时比较总缓解率(ORR)和安全信号。使用Kaplan-Meier方法和对数秩检验比较无进展生存期(PFS)、缓解持续时间(DoR)和总生存期(OS)。计算了药物采购、药物给药、严重不良事件(AE)和复发管理的成本及成本最小化分析。成本数据来自IBM-Micromedex Red Book、医疗保险和医疗补助服务中心以及现有文献。使用Microsoft Excel和R 4.0.5版本进行统计分析。在ORR、DoR和OS方面,axi-cel和tisa-cel之间未观察到统计学显著差异(p>0.05)。tisa-cel的PFS显著更好(p<0.05)。与tisa-cel相比,axi-cel与CRS、神经系统事件和3-4级AE的发生率显著更高相关(ROR>1,p<0.05)。axi-cel和tisa-cel每位患者的成本分别为512,021美元和450,885美元,tisa-cel比axi-cel节省61,136美元。与axi-cel相比,tisa-cel似乎具有更好的安全性、更少的严重AE、更低的死亡率和更低的成本。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/df8d/10560186/ff1612e271ef/10637_2023_1389_Fig1_HTML.jpg

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