在墨西哥,用 blinatumomab 治疗 B 前体急性淋巴细胞白血病儿科高危首次复发患者的成本效益

IF 2.1 4区 医学 Q3 HEMATOLOGY Leukemia research Pub Date : 2024-08-22 DOI:10.1016/j.leukres.2024.107560
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引用次数: 0

摘要

背景blinatumomab是一种CD3/CD19定向双特异性T细胞吞噬分子,能吸引T细胞裂解表达CD19的B细胞。基于一项多中心、开放标签、3期随机临床试验(临床试验编号:NCT02393859),我们旨在从墨西哥医疗支付方的角度评估blinatumomab与标准巩固化疗(SC)相比治疗高风险初治复发费城染色体阴性B细胞前体急性淋巴细胞白血病(B-ALL)儿科患者的成本效益(CE)。方法 采用决策分析模型--分区生存模型来估算患者一生中的生命年数(LY)和成本。我们假设存活超过 5 年的患者均已治愈。为了考虑癌症治疗的残留影响,我们在模型中加入了超额死亡率。无事件生存期(EFS)和总生存期(OS)是通过将混合治愈和标准参数生存分布拟合到 3 期试验的事件时间数据来估算的。该模型考虑了治疗成本、不良事件成本、随访成本、后续异基因造血干细胞移植(alloHSCT)成本和后续治疗成本。结果相对于SC,Blinatumomab的终生获益期为5.11年,增量成本为621,111美元。在基础病例中,blinatumomab与标准治疗相比的ICER估计为121,526美元。成本效益对时间跨度的变化很敏感。在墨西哥确定的支付意愿阈值下,相对于SC,Blinatumomab的成本效益概率为99%。结论相对于SC,Blinatumomab在OS和EFS方面具有更大的获益。概率、确定性和情景分析表明,blinatumomab 具有最佳性价比。因此,在高危首次复发的B-ALL儿童患者中,作为巩固治疗的一部分使用blinatumomab是一种经济有效的选择。
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Cost-effectiveness of blinatumomab for the treatment of B‑precursor acute lymphoblastic leukemia pediatric patients with high‑risk first‑relapse in Mexico

Background

Blinatumomab is a CD3/CD19-directed bispecific T-cell engager molecule that engages T cells to lyse CD19-expressing B cells. Based on a multicenter, open-label, phase 3, randomized clinical trial (Clinical Trials ID: NCT02393859), we aimed to evaluate the cost-effectiveness (CE) of blinatumomab compared to standard consolidation chemotherapy (SC) for the treatment of pediatric patients with high-risk first-relapsed Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (B-ALL) from a Mexico healthcare payer perspective.

Methods

A decision-analytic model, a partitioned survival model, was used to estimate the life-years (LYs) and costs over a lifetime horizon. We assumed that patients who remained alive beyond a 5-year period were cured. To account for the lingering impacts of cancer treatment, an excess mortality rate was incorporated into the model. Event-free survival (EFS) and overall survival (OS) were estimated by fitting mixture-cure and standard parametric survival distributions to the time-to-event data from the phase 3 trial. The model accounted for treatment costs, adverse event costs, follow-up costs, subsequent allogeneic hematopoietic stem cell transplantation (alloHSCT) costs, and subsequent treatment costs.

Results

Blinatumomab was associated with a lifetime gained of 5.11 years at an incremental cost of $621,111 MXN, relative to SC. The ICER for blinatumomab vs Standard of care was estimated to be $121,526 MXN/LY gained in the base case. Cost-effectiveness was sensitive to varying the time horizon. Blinatumomab had a probability of 99 % of being cost-effective, relative to SC, at the willingness to pay threshold defined in Mexico.

Limitations

Health-related quality of life values were not included in the analysis and therefore we did not estimate the quality-adjusted life-years gained.

Conclusions

Blinatumomab was associated with greater benefit in terms of OS and EFS relative to SC. Probabilistic, deterministic, and scenario analyses indicate that blinatumomab represents the best value for money. Therefore, blinatumomab administered as part of consolidation therapy in B-ALL pediatric patients with high-risk first relapse is a cost-effective option.

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来源期刊
Leukemia research
Leukemia research 医学-血液学
CiteScore
4.00
自引率
3.70%
发文量
259
审稿时长
1 months
期刊介绍: Leukemia Research an international journal which brings comprehensive and current information to all health care professionals involved in basic and applied clinical research in hematological malignancies. The editors encourage the submission of articles relevant to hematological malignancies. The Journal scope includes reporting studies of cellular and molecular biology, genetics, immunology, epidemiology, clinical evaluation, and therapy of these diseases.
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