CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T(CAR-T)细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Cost-effectiveness of chimeric antigen receptor T-cell therapy in adults with relapsed or refractory follicular lymphoma.
Cost-effectiveness of chimeric antigen receptor T-cell therapy in adults with relapsed or refractory follicular lymphoma.
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滤泡性淋巴瘤(FL)传统上被认为可治疗但不可治愈。2021年3月,美国食品药品监督管理局批准对接受过2线治疗后的复发/难治性(R/R)FL患者使用嵌合抗原受体(CAR)T细胞疗法。CAR-T 细胞疗法定价为373 000美元,具有潜在治愈性,但其与其他现代R/R FL治疗策略相比的成本效益尚不明确。
我们建立了一个Markov模型,以评估三线CAR-T 细胞疗法与标准治疗(SOC)在成人R/R FL患者中的成本效益。我们分别根据ZUMA-5试验和LEO CReWE研究估算了接受CAR-T 细胞疗法和SOC治疗患者的疾病进展率。
我们计算了CAR-T 细胞疗法与SOC治疗的成本、贴现生命年、质量调整生命年(QALYs)以及增量成本效益比(ICER),支付意愿阈值为每QALY 150 000美元。
我们的分析从美国支付方角度出发,采用终生时间跨度。在我们的基础病例模型中,CAR-T 细胞策略的成本为731 682美元,而SOC治疗为458 490美元。
然而,CAR-T 细胞疗法与1.50 QALYs的增量临床获益相关,导致ICER为每QALY 182 127美元。我们的模型对与CAR-T 细胞疗法缓解和三线SOC治疗相关的效用值以及CAR-T 细胞疗法总前期成本最为敏感。在当前定价下,CAR-T 细胞疗法在三线治疗环境下用于未经选择的FL患者中不太可能具有成本效益。随机临床试验和更长期的临床随访均可有助于阐明CAR-T 细胞疗法在FL患者中的获益及最佳序贯治疗。
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.
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