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:Health State Utilities for Adverse Events Associated with Chimeric Antigen Receptor T-Cell Therapy in Large B-Cell Lymphoma.
Health State Utilities for Adverse Events Associated with Chimeric Antigen Receptor T-Cell Therapy in Large B-Cell Lymphoma.
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更严重的 AE 与更大的负效用相关。本研究中估计的健康状态效用可能有助于评估 CAR-T 细胞疗法对 LBCL 患者价值的成本效果模型。
嵌合抗原受体(CAR)T细胞疗法为大B细胞淋巴瘤(LBCL)提供了有效治疗。评估和比较这些治疗价值的成本-效用分析需要代表关键特征的健康状态效用,以区分不同疗法。本研究估计了与CAR-T 细胞疗法相关的不良事件(AEs)的效用,包括细胞因子释放综合征(CRS)和神经系统事件(NEs)。
健康状态情景描述基于文献综述、CAR-T 细胞治疗试验的不良事件报告以及临床医生意见起草。健康状态通过在英国普通人群参与者中进行的时间权衡访谈进行赋值。第一个情景描述的是接受CAR-T 细胞治疗且未发生AE的复发/难治性LBCL。另外五个情景具有相同的LBCL和治疗描述,但增加了AE。与这些AE相关的负效用(即效用下降)通过从其他健康状态的效用中减去无AE健康状态的效用计算得出。
共完成218名参与者的访谈(50%为男性;平均年龄49岁)。无AE的CAR-T 细胞疗法的平均(标准差[SD])效用值为0.73(0.30)。与CRS相关的平均(SD)负效用为:1级-0.01(0.04),2级-0.05(0.09),3/4级-0.23(0.24)。与NE相关的平均(SD)负效用为:1/2级-0.04(0.07),3/4级-0.18(0.22)。
Chimeric antigen receptor (CAR) T-cell therapy provides effective treatment for large B-cell lymphoma (LBCL). Cost-utility analyses examining and comparing the value of these treatments require health state utilities representing key characteristics to differentiate among therapies. This study estimated utilities for adverse events (AEs) associated with CAR T-cell therapy, including cytokine release syndrome (CRS) and neurological events (NEs).
Health state vignettes were drafted based on literature review, AE reports from a trial of CAR T-cell therapy, and clinician input. Health states were valued in time trade-off interviews with general population participants in the UK. The first vignette described relapsed/refractory LBCL treated with CAR T-cell therapy without AEs. Five other vignettes had the same LBCL and treatment description, with the addition of an AE. Disutilities (i.e., utility decrease) associated with these AEs were calculated by subtracting the utility of the health state without AEs from those of the other health states.
Interviews were completed with 218 participants (50% male; mean age 49 years). Mean (standard deviation [SD]) utility for CAR T-cell therapy without AEs was 0.73 (0.30). Mean (SD) disutilities associated with CRS were -0.01 (0.04) for grade 1, -0.05 (0.09) for grade 2, and -0.23 (0.24) for grade 3/4. Mean (SD) disutilities associated with NEs were -0.04 (0.07) for grade 1/2 and -0.18 (0.22) for grade 3/4.
More severe AEs were associated with greater disutilities. Health state utilities estimated in this study may be useful in cost-effectiveness models examining the value of CAR T-cell therapy in patients with LBCL.
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