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抗 BCMA 嵌合抗原受体 T 细胞疗法治疗复发/难治性多发性骨髓瘤的成本效果分析。

Cost-Effectiveness of Anti-BCMA Chimeric Antigen Receptor T Cell Therapy in Relapsed/Refractory Multiple Myeloma.

机构信息

Division of Hematology, Department of Medicine, Jichi Medical University, Tochigi, Japan.

Division of Hematology, Department of Medicine, Jichi Medical University, Tochigi, Japan; Division of Cell Transplantation and Transfusion, Jichi Medical University, Tochigi, Japan.

出版信息

Transplant Cell Ther. 2024 Jan;30(1):118.e1-118.e15. doi: 10.1016/j.jtct.2023.10.001. Epub 2023 Oct 5.


DOI:10.1016/j.jtct.2023.10.001
PMID:37802181
Abstract

Despite its promising outcomes, anti-BCMA chimeric antigen receptor T cell therapy (CAR-T) is the most expensive myeloma treatment developed to date, and its cost-effectiveness is an important issue. This study aimed to assess the cost-effectiveness of anti-BCMA CAR-T compared to standard antimyeloma therapy in patients with relapsed/refractory multiple myeloma. The model included myeloma patients in Japan and the United States who have received ≥3 prior lines of antimyeloma therapy, including immunomodulatory drugs, proteasome inhibitors, and anti-CD38 monoclonal antibodies. A Markov model was constructed to compare the CAR-T strategy, in which patients receive either idecabtagene vicleucel (ide-cel) or ciltacabtagene autoleucel (cilta-cel) followed by 3 lines of multiagent chemotherapy after relapse, and the no CAR-T strategy, in which patients receive only chemotherapy. Data from the LocoMMotion, KarMMa, and CARTITUDE-1 trials were extracted. Several assumptions were made regarding long-term progression-free survival (PFS) with CAR-T. Extensive scenario analyses were made regarding regimens for no CAR-T strategies. The outcome was an incremental cost-effectiveness ratio (ICER) with willingness-to-pay thresholds of ¥7,500,000 in Japan and $150,000 in the United States. When a 5-year PFS of 40% with cilta-cel was assumed, the ICER of the CAR-T strategy versus the no CAR-T strategy was ¥7,603,823 per QALY in Japan and $112,191 per QALY in the United States over a 10-year time horizon. When a 5-year PFS of 15% with ide-cel was assumed, the ICER was ¥20,388,711 per QALY in Japan and $261,678 per QALY in the United States over a 10-year time horizon. The results were highly dependent on the PFS assumption with CAR-T and were robust to changes in most other parameters and scenarios. Although anti-BCMA CAR-T can be cost-effective even under current pricing, a high long-term PFS is necessary.

摘要

尽管嵌合抗原受体 T 细胞疗法(CAR-T)具有广阔的前景,但它是迄今为止开发的治疗多发性骨髓瘤最昂贵的药物,其成本效益是一个重要问题。本研究旨在评估抗 BCMA CAR-T 与标准抗骨髓瘤疗法相比,在复发/难治性多发性骨髓瘤患者中的成本效益。该模型包括日本和美国接受过≥3 线抗骨髓瘤治疗(包括免疫调节剂、蛋白酶体抑制剂和抗 CD38 单克隆抗体)的骨髓瘤患者。构建了一个马尔可夫模型,比较了 CAR-T 策略,即患者在复发后接受 idecabtagene vicleucel(ide-cel)或 cilta-cel,然后接受 3 线联合化疗,以及无 CAR-T 策略,即患者仅接受化疗。从 LocoMMotion、KarMMa 和 CARTITUDE-1 试验中提取数据。对 CAR-T 的长期无进展生存期(PFS)进行了大量假设。对无 CAR-T 策略的方案进行了广泛的情景分析。结果是一个增量成本效益比(ICER),在日本的意愿支付阈值为 750 万日元,在美国为 15 万美元。当假设 cilta-cel 的 5 年 PFS 为 40%时,CAR-T 策略与无 CAR-T 策略相比,在日本的 ICER 为每 QALY7603823 日元,在美国为每 QALY112191 美元,在 10 年时间内。当假设 ide-cel 的 5 年 PFS 为 15%时,在日本的 ICER 为每 QALY20388711 日元,在美国的 ICER 为每 QALY261678 美元,在 10 年时间内。结果高度依赖于 CAR-T 的 PFS 假设,对大多数其他参数和方案的变化具有稳健性。尽管抗 BCMA CAR-T 即使在当前价格下也具有成本效益,但需要有较高的长期 PFS。

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Cost-Effectiveness of Anti-BCMA Chimeric Antigen Receptor T Cell Therapy in Relapsed/Refractory Multiple Myeloma.

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引用本文的文献

[1]
T cell redirecting therapy for relapsed multiple myeloma.

Discov Oncol. 2025-8-18

[2]
Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review.

Healthcare (Basel). 2025-8-2

[3]
Knowledge-map and bibliometric analysis of scientific research on FDA-approved Chimeric Antigen Receptor T cell products (2015-2024).

Discov Oncol. 2025-8-3

[4]
Role of Chimeric Antigen Receptor T-Cells in the Evolving Therapeutic Landscape of Multiple Myeloma: A Literature Review.

Cureus. 2025-3-5

[5]
CAR-T cell therapies: patient access and affordability solutions.

Future Sci OA. 2025-12

[6]
Navigating the Economic Burden of Multiple Myeloma: Insights into Cost-effectiveness of CAR-T and Bispecific Antibody Therapies.

Curr Hematol Malig Rep. 2025-1-4

[7]
CAR T Cell Nanosymbionts: Revealing the Boundless Potential of a New Dyad.

Int J Mol Sci. 2024-12-7

[8]
Cost-per-responder analysis of patients with lenalidomide-refractory multiple myeloma receiving ciltacabtagene autoleucel in CARTITUDE-4.

Front Immunol. 2024

[9]
Review of CAR T-Cell Therapy in Multiple Myeloma: A Canadian Perspective.

Curr Oncol. 2024-7-6

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