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:Comparative Economic Evaluations of CAR-T Therapies for Relapsed or Refractory Diffuse Large B-Cell Lymphoma: A Systematic Review.
Comparative Economic Evaluations of CAR-T Therapies for Relapsed or Refractory Diffuse Large B-Cell Lymphoma: A Systematic Review.
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在这项针对复发/难治性大 B 细胞淋巴瘤的 CAR-T 细胞疗法头对头比较的经济学评价的系统综述中,当前证据提示 axi-cel 可能是最具成本效益的选择。然而,鉴于这些研究依赖于间接比较,且缺乏任何在低至中等收入国家开展的研究,在开展更多评价或临床试验之前,必须谨慎解读这些结果。
CAR-T 细胞疗法改变了复发/难治性大B细胞淋巴瘤的治疗格局,但属于最昂贵的治疗手段之一。此外,其在复发/难治性大B细胞淋巴瘤成人患者中的比较经济价值仍不确定。本研究的目的是了解这些疗法在该人群中的比较价值,以及影响哪种干预措施被认为比其他措施更具成本效果这一结论的主要因素。
为评估CAR-T 细胞疗法的比较成本效果及成本效果结果的驱动因素,对Embase、Scopus和PubMed进行了从建库至2024年12月的系统性文献检索,并于2025年10月更新。若研究为针对复发/难治性大B细胞淋巴瘤的CAR-T 细胞疗法头对头比较的完整经济学评价,则纳入研究。两名评价者独立提取人群特征和模型结构等关键信息的数据。结果以原始格式报告,成本效果结论基于各国特定的支付意愿阈值进行评估。纳入研究的报告质量采用综合卫生经济学评价报告标准(CHEERS)2022清单和Drummond 10项清单进行评估。
本系统综述纳入了来自美国、西班牙、法国和日本的八项完整经济学评价。所有研究均模拟了成人人群或既往接受过两线及以上治疗失败的复发/难治性大B细胞淋巴瘤患者,采用终身时间 horizon,且大多采用支付方视角(n = 6)。所有研究均使用三状态分区生存模型,但严重依赖间接比较方法,如匹配调整间接比较。评估了三种CAR-T 细胞疗法:axicabtagene ciloleucel(axi-cel)、tisagenlecleucel(tisa-cel)和lisocabtagene maraleucel(liso-cel)。Axi-cel是最常报告具有成本效益的选择(n = 7),其中两项研究认为其是优势策略。由于缺乏每个治疗组的个体患者水平数据且依赖间接比较,模型结果存在很大不确定性。
Chimeric antigen receptor T-cell therapies have changed the treatment paradigm of relapsed or refractory large B-cell lymphoma but are among the most expensive treatments. Moreover, their comparative economic value remains uncertain in adults with relapsed or refractory large B-cell lymphoma. The objective of this study was to understand the comparative value of these therapies in this population and the main factors that influenced conclusions on which intervention was considered more cost effective than others.
To assess the comparative cost effectiveness of chimeric antigen receptor T-cell therapies and the drivers of cost-effectiveness results, a systematic literature search of Embase, Scopus, and PubMed was conducted from inception to December 2024 and updated in October 2025. Studies were selected if they were full economic evaluations of head-to-head comparisons of chimeric antigen receptor T-cell therapies for relapsed or refractory large B-cell lymphoma. Two reviewers independently extracted data on key information such as population characteristics and model structure. Results were reported in their original format, and conclusions on cost effectiveness were evaluated based on country-specific willingness-to-pay thresholds. The reporting quality of the included studies was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 checklist and the Drummond 10-Item Checklist.
Eight full economic evaluations across the USA, Spain, France, and Japan were included in this systematic review. All studies modeled an adult population or patients with relapsed or refractory large B-cell lymphoma who had failed two or more lines of prior therapy, were conducted over a lifetime horizon, and mostly used the payer perspective (n = 6). All studies utilized three-state partitioned survival models but relied heavily on indirect comparison methods such as matching-adjusted indirect comparison. Three chimeric antigen receptor T-cell therapies were evaluated: axicabtagene ciloleucel (axi-cel), tisagenlecleucel (tisa-cel), and lisocabtagene maraleucel (liso-cel). Axi-cel was the most frequently reported cost-effective option (n = 7) with two studies concluding it was the dominant strategy. There was substantial uncertainty in the model results given the lack of individual patient-level data for each arm and reliance on indirect comparisons.
In this systematic review of economic evaluations of head-to-head comparisons of chimeric antigen receptor T-cell therapies for relapsed or refractory large B-cell lymphoma, current evidence suggests that axi-cel may be the most cost-effective option. However, given the studies' reliance on indirect comparisons and the absence of any study conducted in low- to middle-income countries, these results must be carefully interpreted until additional evaluations or clinical trials are conducted.
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