Ghanem Buthainah
Department of Pharmaceutical Economics and Policy, School of Pharmacy, Chapman University, Irvine, CA, USA.
Invest New Drugs. 2023 Oct;41(5):710-718. doi: 10.1007/s10637-023-01389-w. Epub 2023 Aug 12.
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.
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。