Blinatumomab 是一种 CD3/CD19 导向的双特异性 T 细胞接合分子，可接合 T 细胞来裂解表达 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 ID：NCT02393859），我们旨在评估 blinatumomab 与标准巩固化疗 (SC) 相比用于治疗儿科患者的成本效益 (CE)从墨西哥医疗保健付款人的角度来看，患有高风险首次复发的费城染色体阴性 B 细胞前体急性淋巴细胞白血病 (B-ALL)。使用决策分析模型（分区生存模型）来估计生命年（ LYs）和整个生命周期的成本。我们假设存活超过 5 年的患者已被治愈。为了考虑癌症治疗的持续影响，模型中纳入了超额死亡率。通过将混合治愈和标准参数生存分布与 3 期试验的事件发生时间数据进行拟合来估计无事件生存期 (EFS) 和总生存期 (OS)。该模型考虑了治疗费用、不良事件费用、随访费用、随后的同种异体造血干细胞移植 (alloHSCT) 费用以及随后的治疗费用。Blinatumomab 与增加的 621,111 墨西哥比索的成本增加了 5.11 年的寿命相关，相对于SC。在基本情况下，blinatumomab 与标准护理相比的 ICER 估计为 121,526 MXN/LY。成本效益对时间范围的变化很敏感。在墨西哥规定的支付意愿阈值下，相对于 SC，Blinatumomab 具有成本效益的概率为 99%。分析中未包括与健康相关的生活质量值，因此我们没有估计质量调整后的质量获得的生命年数。相对于 SC，Blinatumomab 在 OS 和 EFS 方面具有更大的益处。概率性、确定性和情景分析表明，blinatumomab 具有最佳性价比。因此，对于首次复发高风险的 B-ALL 儿科患者，将 blinatumomab 作为巩固治疗的一部分进行治疗是一种经济有效的选择。版权所有 © 2024 作者。由爱思唯尔有限公司出版。保留所有权利。

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.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.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.Health-related quality of life values were not included in the analysis and therefore we did not estimate the quality-adjusted life-years gained.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.Copyright © 2024 The Authors. Published by Elsevier Ltd.. All rights reserved.