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 for relapsed or refractory large B-cell lymphoma: a systematic review and meta-analysis.
Cost-effectiveness of chimeric antigen receptor T-cell therapy for relapsed or refractory large B-cell lymphoma: a systematic review and meta-analysis.
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在终生时间范围内,CAR-T 治疗 r/r LBCL 不具有显著的成本效益,尽管正的 INB 表明在 HICs 中作为二线或三线治疗时存在成本效益趋势。应进一步开展成本效益分析,以比较用于 r/r LBCL 患者治疗的不同 CAR-T 产品,以及比较 CAR-T 产品与用于 r/r LBCL 治疗的新兴疗法,因为本研究未涉及这些比较。
CAR-T 治疗复发/难治性大B细胞淋巴瘤(r/r LBCL)的成本效果证据仍存在争议,因为CAR-T 具有治愈患者的潜力,但治疗费用高昂。本研究旨在通过系统综述和meta分析,综合CAR-T 治疗成人r/r LBCL的成本效果数据。
通过数据库检索(MEDLINE、EMBASE 和 CENTRAL)识别了从建库至 2024 年 11 月关于 CAR-T 治疗 r/r LBCL 的成本效果分析。成本使用购买力平价转换为 2023 美元。增量净效益(INB)使用国家特定的支付意愿阈值计算,并采用随机效应模型进行合并。结果按国家收入水平和治疗线数进行分层。偏倚风险使用 ECOBIAS 清单进行评估。方案注册于 PROSPERO #CRD42024602683。
本系统综述共纳入34项研究。26项研究比较了CAR-T 与铂类化疗联合自体干细胞移植(ASCT)治疗r/r LBCL,8项研究比较了不同CAR-T 疗法。作为LBCL的二线治疗,在高收入国家(HICs)中,与铂类化疗联合ASCT相比,CAR-T 的INB为$28,846(95%置信区间[CI]:-$43,265至$100,957;I²=0%)(n=9)。作为针对相同对照的三线治疗,在HICs中,CAR-T 的INB为$48,838(95%CI:-$156,625至$254,302;I²=79%)(n=9)。
Cost-effectiveness analyses of CAR-T for the treatment of r/r LBCL from inception through November 2024 were identified through database searches (MEDLINE, EMBASE, and CENTRAL). Costs were converted to 2023 US dollars using purchasing power parity. Incremental net benefit (INB) was calculated using country-specific willingness-to-pay thresholds and pooled using a random-effects model. Results were stratified by country income level and line of therapy. The risk of bias was assessed using the ECOBIAS checklist. Protocol registered at PROSPERO #CRD42024602683.
Thirty-four studies were included in this systematic review. Twenty-six studies compared CAR-T to platinum-based chemotherapy with autologous stem cell therapy (ASCT) for r/r LBCL and eight studies compared between CAR-T therapies. As 2nd-line therapy for LBCL, CAR-T was found to have an INB of $28,846 (95% Confidence Interval [CI]: -$43,265 to $100,957; I 2 = 0%) ( n = 9) compared to platinum-based chemotherapy with ASCT in high-income countries (HICs). As 3rd-line therapy against the same comparator, CAR-T had an INB of $48,838 (95% CI: -$156,625 to $254,302; I 2 = 79%) ( n = 9) for HICs.
Over a lifetime horizon, CAR-T was not significantly cost-effective for the treatment of r/r LBCL, though positive INBs suggest a trend toward cost-effectiveness as 2nd or 3rd-line therapy in HICs. Further cost-effective analyses should be conducted to compare between CAR-T products for the treatment of patients with r/r LBCL, as well as between CAR-T products and emerging novel therapies for r/r LBCL treatment, as this study did not address these comparisons.
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