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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成人患者的成本效果分析。

Cost-effectiveness of chimeric antigen receptor T-cell therapy in adults with relapsed or refractory follicular lymphoma.

机构信息

Yale School of Medicine, Yale University, New Haven, CT.

Department of Internal Medicine, Yale School of Medicine, Yale University, New Haven, CT.

出版信息

Blood Adv. 2023 Mar 14;7(5):801-810. doi: 10.1182/bloodadvances.2022008097.

Abstract

Follicular lymphoma (FL) is traditionally considered treatable but incurable. In March 2021, the US Food and Drug Administration approved the use of chimeric antigen receptor (CAR) T-cell therapy in patients with relapsed or refractory (R/R) FL after ≥2 lines of therapy. Priced at $373 000, CAR T-cell therapy is potentially curative, and its cost-effectiveness compared with other modern R/R FL treatment strategies is unknown. We developed a Markov model to assess the cost-effectiveness of third-line CAR T-cell vs standard of care (SOC) therapies in adults with R/R FL. We estimated progression rates for patients receiving CAR T-cell and SOC therapies from the ZUMA-5 trial and the LEO CReWE study, respectively. We calculated costs, discounted life years, quality-adjusted life years (QALYs), and the incremental cost-effectiveness ratio (ICER) of CAR T-cell vs SOC therapies with a willingness-to-pay threshold of $150 000 per QALY. Our analysis was conducted from a US payer's perspective over a lifetime horizon. In our base-case model, the cost of the CAR T-cell strategy was $731 682 compared with $458 490 for SOC therapies. However, CAR T-cell therapy was associated with incremental clinical benefit of 1.50 QALYs, resulting in an ICER of $182 127 per QALY. Our model was most sensitive to the utilities associated with CAR T-cell therapy remission and third-line SOC therapies and to the total upfront CAR T-cell therapy cost. Under current pricing, CAR T-cell therapy is unlikely to be cost-effective in unselected patients with FL in the third-line setting. Both randomized clinical trials and longer term clinical follow-up can help clarify the benefits of CAR T-cell therapy and optimal sequencing in patients with FL.

摘要

滤泡性淋巴瘤 (FL) 传统上被认为是可治疗但不可治愈的疾病。2021 年 3 月,美国食品和药物管理局批准嵌合抗原受体 (CAR) T 细胞疗法用于至少接受过 2 线治疗后复发或难治性 (R/R) FL 患者。CAR T 细胞疗法的价格为 373000 美元,具有潜在的治愈效果,但其与其他现代 R/R FL 治疗策略相比的成本效益尚不清楚。我们开发了一个马尔可夫模型来评估三线 CAR T 细胞与 R/R FL 成人标准护理 (SOC) 治疗相比的成本效益。我们分别从 ZUMA-5 试验和 LEO CReWE 研究中估计接受 CAR T 细胞和 SOC 治疗的患者的进展率。我们使用愿意支付每 QALY 150000 美元的阈值,计算了 CAR T 细胞与 SOC 治疗的成本、贴现生命年、质量调整生命年 (QALY) 和增量成本效益比 (ICER)。我们的分析是从美国支付者的角度在终身时间范围内进行的。在我们的基本模型中,CAR T 细胞策略的成本为 731682 美元,而 SOC 疗法的成本为 458490 美元。然而,CAR T 细胞治疗与额外的 1.50 QALY 临床获益相关,导致每 QALY 的 ICER 为 182127 美元。我们的模型对与 CAR T 细胞治疗缓解和三线 SOC 治疗相关的效用以及 CAR T 细胞治疗的总前期成本最为敏感。根据目前的定价,在三线环境中,CAR T 细胞疗法不太可能对未经选择的 FL 患者具有成本效益。随机临床试验和更长时间的临床随访可以帮助阐明 CAR T 细胞疗法对 FL 患者的益处和最佳治疗顺序。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/342d/10011202/7d3fb211a26b/BLOODA_ADV-2022-008097-fx1.jpg

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