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
英文原题:Cost-effectiveness of axicabtagene ciloleucel versus tisagenlecleucel for the treatment of 3L + relapsed/refractory large B-cell lymphoma in the United States: incorporating longer survival results.
Cost-effectiveness of axicabtagene ciloleucel versus tisagenlecleucel for the treatment of 3L + relapsed/refractory large B-cell lymphoma in the United States: incorporating longer survival results.
分数与星级只用于站内排序 —— 不代表疗效、安全性或个人适用性。
随着更长期的生存数据出现,从美国支付方的角度来看,对于既往接受过 2 线系统性治疗的 r/r LBCL 患者,axi-cel 相比 tisa-cel 仍是一种具有成本效益的选择。
提供关于嵌合抗原受体(CAR)T细胞疗法axicabtagene ciloleucel(axi-cel)和tisagenlecleucel(tisa-cel)用于治疗既往接受过2线系统性治疗的复发/难治性(r/r)大B细胞淋巴瘤(LBCL)患者的成本效益更新,使用更成熟的临床试验数据截止点(axi-cel总生存期[OS]为60个月,tisa-cel OS和无进展生存期[PFS]为45个月)。
采用由三个健康状态(进展前、进展后和死亡)组成的分区生存模型,估算axi-cel和tisa-cel在终生时间范围内的质量调整生命年(QALYs)和成本。axi-cel和tisa-cel的PFS和OS输入基于先前发表的匹配调整间接治疗比较(MAIC)。长期OS和PFS采用参数生存混合治愈模型(PS-MCMs)进行外推。CAR-T 细胞治疗药物获取和给药、预处理化疗、白细胞分离术、CAR-T 特异性监测、干细胞移植、住院、不良事件、常规护理和终末护理的成本来源于美国成本数据库。健康状态效用值来自既往发表文献。模型输入通过一系列敏感性分析和情景分析进行了变动。
与tisa-cel相比,axi-cel产生了2.51个额外QALYs和50,185美元的额外成本(增量成本效果比[ICER]为每获得一个QALY需19,994美元)。在概率敏感性分析(PSA)中,axi-cel相对于tisa-cel的ICER在99.4%的模拟中为50,000美元/QALY,在99%的模拟中为33,500美元。在一系列情景下,axi-cel相对于tisa-cel始终保持成本效果(假设支付意愿阈值为每QALY 150,000美元)。
A partitioned survival model consisting of three health states (pre-progression, post-progression and death) was used to estimate quality-adjusted life years (QALYs) and costs associated with axi-cel and tisa-cel over a lifetime horizon. PFS and OS inputs for axi-cel and tisa-cel were based on a previously published matching-adjusted indirect treatment comparison (MAIC). Long-term OS and PFS were extrapolated using parametric survival mixture cure models (PS-MCMs). Costs of CAR-T cell therapy drug acquisition and administration, conditioning chemotherapy, apheresis, CAR T-specific monitoring, stem cell transplant, hospitalization, adverse events, routine care, and terminal care were sourced from US cost databases. Health state utilities were derived from previous publications. Model inputs were varied using a range of sensitivity and scenario analyses.
Compared with tisa-cel, axi-cel resulted in 2.51 additional QALYs and $50,185 additional costs (an incremental cost-effectiveness ratio [ICER] of $19,994 per QALY gained). In probabilistic sensitivity analysis (PSA), the ICER for axi-cel versus tisa-cel was $50,000/QALY in 99.4% of simulations and $33,500 in 99% of simulations. Axi-cel remained cost-effective versus tisa-cel (assuming a willingness-to-pay threshold of $150,000 per QALY) across a range of scenarios.
With longer-term survival data, axi-cel continues to represent a cost-effective option versus tisa-cel for treatment of r/r LBCL among patients who have previously received 2 lines of systemic therapy, from a US payer perspective.
MEMBER ACCOUNT
登录成功会直接打开下一页。