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估算 CAR-T 细胞治疗相关细胞因子释放综合征和神经系统事件管理成本的模型

英文原题:A Model to Estimate Cytokine Release Syndrome and Neurological Event Management Costs Associated With CAR T-Cell Therapy.

查看英文原题

A Model to Estimate Cytokine Release Syndrome and Neurological Event Management Costs Associated With CAR T-Cell Therapy.

PubMed 2022/10/19(内容时间) Transplant Cell Ther Q1 · IF 4.7(JCR 2025)

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中文摘要

嵌合抗原受体(CAR)T细胞疗法在复发/难治性大B细胞淋巴瘤(LBCL)中显示出疗效,但与细胞因子释放综合征(CRS)和神经系统事件(NE)相关。

我们旨在估算在接受lisocabtagene maraleucel(liso-cel)、axicabtagene ciloleucel(axi-cel)或tisagenlecleucel(tisa-cel)治疗的复发/难治性LBCL患者中,三线或更后线治疗背景下CRS和NE管理的总成本。

我们开发了一个经济决策树模型,使用临床和经济学数据,从美国医疗保健系统角度以2020年美元估算每例患者不良事件(AE)管理的加权平均成本。在2项预设分析中,通过蒙特卡洛模拟估算平均预期成本和平均治疗患者95%置信区间,并按AE和分级进一步分层计算每种CAR-T 细胞疗法的每例患者成本。在基础病例中,liso-cel、axi-cel和tisa-cel的总体加权平均每例患者成本分别为$18,718、$47,665和$42,538。liso-cel、axi-cel和tisa-cel每次CRS事件的加权平均每例患者成本分别为$8213、$20,442和$26,009;每次NE的加权平均每例患者成本分别为$10,505、$27,223和$16,528。

在基础病例情景中,liso-cel与axi-cel和tisa-cel相比,估算总平均成本差异分别为-$28,947和-$23,819。在情景分析(替代成本输入)中,liso-cel与axi-cel相比估算总平均成本差异为-$24,498,liso-cel与tisa-cel相比为-$19,326。在基础案例和情景分析中,与axi-cel和tisa-cel相比,liso-cel由于较低的发病率和症状严重程度,每位接受治疗患者的加权平均CRS和NE成本最低。这些发现强调了CAR-T 细胞疗法之间安全性差异的经济影响。

展开英文摘要原文

Chimeric antigen receptor (CAR) T-cell therapies demonstrated efficacy in relapsed/refractory large B-cell lymphoma (LBCL) but are associated with cytokine release syndrome (CRS) and neurological events (NE).

We wanted to estimate the total cost of CRS and NE management among patients with relapsed/refractory LBCL treated with lisocabtagene maraleucel (liso-cel), axicabtagene ciloleucel (axi-cel), or tisagenlecleucel (tisa-cel) in the third- or later-line setting. An economic decision tree model was developed using clinical and economic data to estimate a weighted average per-patient adverse event (AE) management cost from a United States health care system perspective in 2020 dollars. In 2 predefined analyses, mean expected cost and 95% confidence intervals of the average treated patient were estimated via Monte Carlo simulations, with per-patient costs for each CAR T-cell therapy further stratified by AE and grade. In the base case, the overall weighted average per-patient cost was $18,718, $47,665, and $42,538 for liso-cel, axi-cel, and tisa-cel, respectively.

The weighted average per-patient cost per CRS event was $8213, $20,442, and $26,009 for liso-cel, axi-cel, and tisa-cel, respectively; the weighted average per-patient cost per NE was $10,505, $27,223, and $16,528, respectively. Differences in the base-case scenario estimated total mean costs for liso-cel were -$28,947 and -$23,819 compared with axi-cel and tisa-cel, respectively.

In the scenario analysis (alternative cost input), differences in the estimated total mean costs were -$24,498 for liso-cel versus axi-cel, and -$19,326 for liso-cel versus tisa-cel. Across the base case and scenario analysis, liso-cel had the lowest weighted average CRS and NE costs per treated patient compared with axi-cel and tisa-cel owing to lower incidence rates and symptom severity.

These findings highlight the economic implications of differences in safety among CAR T-cell therapies.

论文信息

作者
Badaracco J、Gitlin M、Keating SJ
第一作者单位
BluePath Solutions, Los Angeles, California.United States
通讯作者单位
Bristol Myers Squibb, Princeton, New Jersey. Electronic address: scott.j.keating@gmail.com.
文献类型
非美国政府资助研究
期刊
Transplantation and cellular therapy2023 Jan
原文标识
PubMed 36272529 · DOI 10.1016/j.jtct.2022.10.009