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拟行移植的复发/难治性大 B 细胞淋巴瘤患者中二线 Liso-Cel 与自体造血干细胞移植每临床结局成本的估算

英文原题:Estimating the Cost per Clinical Outcome of Second-Line Liso-Cel Versus Autologous Stem Cell Transplantation in Patients with Transplantation-Intended Relapsed/Refractory Large B Cell Lymphoma.

查看英文原题

Estimating the Cost per Clinical Outcome of Second-Line Liso-Cel Versus Autologous Stem Cell Transplantation in Patients with Transplantation-Intended Relapsed/Refractory Large B Cell Lymphoma.

PubMed 2023/08/05(内容时间) Transplant Cell Ther Q1 · IF 4.7(JCR 2025)

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中文摘要

在考虑复发/难治性(R/R)大B细胞淋巴瘤(LBCL)治疗时,必须同时考量与治疗相关的总护理成本(TCOC)及临床获益。

我们利用TRANSFORM研究(ClinicalTrials.gov NCT03575351)的数据,估算了接受二线lisocabtagene maraleucel(liso-cel)治疗与自体干细胞移植(ASCT)治疗的R/R LBCL患者1年TCOC及每项临床结局的成本。采用蒙特卡洛模拟方法进行了每项临床结局的成本分析。成本输入基于对医疗资源利用(HCRU)的回顾性微观成本分析生成。使用中期分析(2021年3月)的患者水平数据推导HCRU和临床输入。临床输入包括中位无事件生存期(EFS)、中位无进展生存期(PFS)、客观缓解率和完全缓解(CR)率。在意向治疗分析中,liso-cel组每例患者的平均(标准差)TCOC为$550,864($173,087),ASCT组为$413,200($290,802)。

每项临床结局的成本模型估计,liso-cel与ASCT相比,每EFS月的平均成本为$57,295 vs $186,369,每PFS月为$40,949 vs $78,797,每总体缓解者为$653,965 vs $881,804,每完全缓解者为$828,045 vs $1,063,822。该经济模型显示,由于liso-cel的疗效优于ASCT,liso-cel相比ASCT降低了每EFS月、每PFS月、每总体缓解者和每完全缓解者的平均估计TCOC。尽管接受liso-cel治疗的患者前期成本较高,但需要后续治疗的患者较少,且累积的下游成本较低。这些结果强调了在评估总成本时考虑缓解持续时间和临床获益的重要性。

展开英文摘要原文

It is important to consider the total cost of care (TCOC) associated with a therapy and clinical benefit for relapsed or refractory (R/R) large B cell lymphoma (LBCL).

We estimated the 1-year TCOC and cost per clinical outcome for patients with R/R LBCL treated with second-line lisocabtagene maraleucel (liso-cel) versus autologous stem cell transplantation (ASCT) using data from the TRANSFORM study (ClinicalTrials. gov NCT03575351). A cost per clinical outcome analysis using a Monte Carlo simulation approach was conducted. Cost inputs were generated from a retrospective microcosting analysis of healthcare resource utilization (HCRU). Patient-level data from an interim analysis (March 2021) were used to derive HCRU and clinical inputs. Clinical inputs included median event-free survival (EFS), median progression-free survival (PFS), objective response rate, and complete response (CR) rate. In the intention-to-treat analysis, the mean (standard deviation) TCOC per patient was $550,864 ($173,087) for liso-cel and $413,200 ($290,802) for ASCT.

The cost per clinical outcome model estimated a mean cost for liso-cel versus ASCT per EFS month of $57,295 versus $186,369, per PFS month of $40,949 versus $78,797, per overall responder of $653,965 versus $881,804, and per complete responder of $828,045 versus $1,063,822. This economic model shows reductions in mean estimated TCOC per EFS month, PFS month, overall responder, and complete responder with liso-cel versus ASCT owing to the superior efficacy of liso-cel.

Although liso-cel-treated patients incurred greater upfront costs, fewer required subsequent therapy, and they accumulated less downstream costs. These results underscore the importance of considering the durability of response and clinical benefit when assessing total costs.

论文信息

作者
Saeedian M、Badaracco J、Botros A、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: afraim.botros@bms.com.
期刊
Transplantation and cellular therapy2023 Nov
原文标识
PubMed 37544410 · DOI 10.1016/j.jtct.2023.08.001