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接受 cilta-cabtagene autoleucel 治疗的 lenalidomide 难治性多发性骨髓瘤患者的成本-反应者分析。在 CARTITUDE-4 研究中。

Cost-per-responder analysis of patients with lenalidomide-refractory multiple myeloma receiving ciltacabtagene autoleucel in CARTITUDE-4.

机构信息

Department of Blood and Marrow Transplant and Cellular Immunotherapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL, United States.

Janssen Scientific Affairs, LLC, a Johnson & Johnson company, Horsham, PA, United States.

出版信息

Front Immunol. 2024 Aug 21;15:1408892. doi: 10.3389/fimmu.2024.1408892. eCollection 2024.

Abstract

INTRODUCTION

Ciltacabtagene autoleucel (cilta-cel) is a chimeric antigen receptor T-cell therapy approved for patients with relapsed/refractory multiple myeloma (RRMM). In the phase 3 trial, CARTITUDE-4 (NCT04181827), cilta-cel demonstrated improved efficacy vs. standard of care (SOC; daratumumab plus pomalidomide and dexamethasone [DPd] or pomalidomide plus bortezomib and dexamethasone [PVd]) with a ≥ complete response (≥CR) rate of 73.1% vs. 21.8%.

METHODS

A cost-per-responder model was developed to assess the value of cilta-cel and SOC (87% DPd and 13% PVd) based on the CARTITUDE-4 trial data from a US mixed payer perspective (76.7% commercial, 23.3% Medicare). The model was developed using progression-free survival (PFS), overall survival (OS), and ≥CR endpoints from CARTITUDE-4 over a period of 25.4 months. Inpatient stays, outpatient visits, drug acquisition, administration, and monitoring costs were included. The base-case model assumed an inpatient setting for each cilta-cel infusion; another scenario included 30% outpatient and 70% inpatient infusions. Costs of managing grade 3-4 adverse events (AEs) and grade 1-4 cytokine release syndrome and neurotoxicity were included. Subsequent therapy costs were incurred after disease progression; terminal care costs were considered upon death events. Outcomes included total cost per treated patient, total cost per complete responder, and cost per month in PFS between cilta-cel and SOC. Costs were adjusted to 2024 US dollars.

RESULTS

Total cost per treated patient, total cost per complete responder, and total cost per month in PFS were estimated at $704,641, $963,941, and $30,978 for cilta-cel, respectively, and $840,730, $3,856,559, and $42,520 for SOC over the 25.4-month period. Cost drivers included treatment acquisition costs before progression and subsequent treatment costs ($451,318 and $111,637 for cilta-cel; $529,795 and $265,167 for SOC). A scenario analysis in which 30% of patients received an outpatient infusion (assuming the same payer mix) showed a lower cost per complete responder for cilta-cel ($956,523) than those with an infusion in the inpatient setting exclusively.

DISCUSSION

This analysis estimated that cost per treated patient, cost per complete responder, and cost per month in PFS for cilta-cel were remarkably lower than for DPd or PVd, highlighting the substantial clinical and economic benefit of cilta-cel for patients with RRMM.

摘要

简介

Ciltacabtagene autoleucel(cilta-cel)是一种嵌合抗原受体 T 细胞疗法,已获批准用于治疗复发/难治性多发性骨髓瘤(RRMM)患者。在 3 期临床试验 CARTITUDE-4(NCT04181827)中,cilta-cel 与标准治疗(SOC;daratumumab 联合泊马度胺和地塞米松[DPd]或泊马度胺联合硼替佐米和地塞米松[PVd])相比,疗效更佳,完全缓解率(≥CR)为 73.1%,而 21.8%。

方法

基于 CARTITUDE-4 试验数据,从美国混合支付者的角度(76.7%商业,23.3%医疗保险),建立了一个基于每个反应者的成本模型,以评估 cilta-cel 和 SOC(87% DPd 和 13% PVd)的价值。该模型使用了 CARTITUDE-4 试验的无进展生存期(PFS)、总生存期(OS)和≥CR 终点,时间为 25.4 个月。包括住院和门诊就诊、药物获得、管理和监测成本。基础模型假设每个 cilta-cel 输注均为住院设置;另一种方案包括 30%的门诊和 70%的住院输注。包括 3-4 级不良事件(AE)和 1-4 级细胞因子释放综合征和神经毒性的管理成本。疾病进展后会产生后续治疗成本;在发生死亡事件时会考虑临终护理成本。结果包括每例治疗患者的总成本、每例完全缓解者的总成本和 PFS 期间每月的成本。成本调整为 2024 年的美元。

结果

在 25.4 个月的时间内,cilta-cel 的每例治疗患者总成本、每例完全缓解者总成本和 PFS 期间每月总成本分别估计为 704641 美元、963941 美元和 30978 美元,SOC 分别为 840730 美元、3856559 美元和 42520 美元。成本驱动因素包括治疗前进展的治疗获取成本和后续治疗成本(cilta-cel 为 451318 美元和 111637 美元;SOC 为 529795 美元和 265167 美元)。在一项 30%的患者接受门诊输注的方案分析中(假设支付者组合相同),cilta-cel 的每例完全缓解者成本低于完全住院输注的成本(956523 美元)。

讨论

本分析估计,cilta-cel 的每例治疗患者总成本、每例完全缓解者总成本和 PFS 期间每月总成本明显低于 DPd 或 PVd,这突出了 cilta-cel 为 RRMM 患者带来的显著临床和经济效益。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/cff2/11372240/5b6e6438670f/fimmu-15-1408892-g001.jpg

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