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.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
不适合移植的复发/难治性弥漫大 B 细胞淋巴瘤治疗选择的成本效果分析——效率前沿法的经验
英文原题：Cost-effectiveness analysis of transplant-ineligible relapsed or refractory diffuse large B-cell lymphoma treatment options-Experience of the efficiency frontier approach.
查看英文原题
Cost-effectiveness analysis of transplant-ineligible relapsed or refractory diffuse large B-cell lymphoma treatment options-Experience of the efficiency frontier approach.
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研究概要
采用 EF 方法，识别出 R/R DLBCL 适应症中目前最具成本效益的干预措施（基于成本效益比），以指导国际报销决策。
中文摘要
目的
自欧洲药品管理局批准CAR-T（CAR-T）细胞疗法用于三线及后续（3+L）治疗以来，复发/难治性（R/R）弥漫性大 B 细胞淋巴瘤（DLBCL）的治疗发生显著变化。获批疗法包括 axicabtagene ciloleucel（axi-cel）、lisocabtagene maraleucel（liso-cel）和 tisagenlecleucel（tisa-cel）；二线及后续治疗还可使用靶向疗法 polatuzumab vedotin-bendamustine-rituximab（pola-BR）和 tafasitamab-lenalidomide（Tafa-L）。相关治疗费用不断上升，带来经济负担。本研究从德国医疗支付方角度，采用效率前沿（EF）方法评估不适合移植的 R/R DLBCL 患者治疗方案的成本效益。
方法
通过系统文献综述确定 bendamustine-rituximab（BR）、rituximab-gemcitabine-oxaliplatin（R-GemOx）、axi-cel、liso-cel、tisa-cel、pola-BR 和 Tafa-L 的临床获益，以中位总生存期（OS）衡量。计算第一年治疗费用，包括药物和医疗服务费用，并将结果汇总于二维图表，分别展示二线和 3+L 的 EF。
结果
二线 EF 由 BR（中位 OS 11.49 个月，费用 23,958）和 Tafa-L（45.7 个月，费用 104,541）构成；3+L EF 由 R-GemOx（12.0 个月，29,080）、Tafa-L（15.5 个月，104,541）和 axi-cel（18.69 个月，308,516）构成。这些治疗方案构成新疗法的相应成本效益阈值。
结论
采用 EF 方法，本研究识别了 R/R DLBCL 适应证下当前成本效益比最高的治疗方案，可为国际报销决策提供指导。
展开英文摘要原文
OBJECTIVES
The treatment of relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) changed remarkably since the European Medicines Agency-approved chimeric antigen receptor T-cell (CAR-T) therapies (axicabtagene ciloleucel [axi-cel], lisocabtagene maraleucel [liso-cel], tisagenlecleucel [tisa-cel]) for the third-line onwards (3+L), and targeted therapies (polatuzumab vedotin-bendamustine-rituximab [pola-BR], tafasitamab-lenalidomide [Tafa-L]) for the second-line (2L) onwards. As associated rising treatment costs represent an economic burden, the cost-effectiveness of transplant-ineligible R/R DLBCL interventions was assessed from a German healthcare payer's perspective, using the efficiency frontier (EF) approach.
METHODS
A systematic literature review was performed to determine the clinical benefit concerning median overall survival (OS) of bendamustine-rituximab (BR), rituximab-gemcitabine-oxaliplatin (R-GemOx), axi-cel, liso-cel, tisa-cel, pola-BR, and Tafa-L. First-year treatment costs (drug and medical services costs) were calculated. Results were merged on two-dimensional graphs illustrating 2L and 3+L EFs.
RESULTS
Second-line EF is formed by BR (median OS 11.49 months, 23 958) and Tafa-L (45.7, 104 541), 3+L EF is formed by R-GemOx (12.0, 29 080), Tafa-L (15.5, 104 541), and axi-cel (18.69, 308 516). These interventions build the respective cost-effectiveness thresholds for novel interventions.
CONCLUSIONS
Using the EF approach, the currently most cost-effective interventions (based on cost-effectiveness ratios) in the indication of R/R DLBCL were identified to guide international reimbursement decisions.
论文信息
- 作者
- Kurte MS、Siefen AC、Jakobs F、von Tresckow B、Reinhardt HC、Kron F
- 第一作者单位
- Faculty of Medicine, University of Duisburg-Essen, Essen, Germany.Germany
- 通讯作者单位
- VITIS Healthcare Group, Cologne, Germany.Germany
- 文献类型
- 系统综述
- 期刊
- European journal of haematology2023 Dec
- 原文标识
- PubMed 37644352 · DOI 10.1111/ejh.14095