Saha Sanjib, Gerdtham Ulf-Göran, Sjödahl Gottfrid, Häggström Christel, Catto James W F, Kelly John D, Ullén Anders, Holmberg Lars, Liedberg Fredrik
Health Economics Unit, Department of Clinical Sciences (Malmö), Lund University, Lund, Sweden.
Department of Economics, Lund University, Lund, Sweden.
Front Oncol. 2025 Apr 2;15:1556881. doi: 10.3389/fonc.2025.1556881. eCollection 2025.
Guidelines recommend neoadjuvant chemotherapy (NAC) and radical cystectomy (RC) for muscle-invasive bladder cancer (MIBC). Current recommendations do not consider genomic profiles, although the Basal/Squamous (Ba/Sq) subtype is less likely to respond to NAC compared to Urothelial-like (Uro) and Genomically Unstable (GU) subtypes. The aim of this study is to perform cost-effectiveness analyses of a de-escalated use of NAC in patients with Ba/Sq tumors and MIBC.
A cost-effectiveness analysis was performed using a decision analytic Markov model using a healthcare provider perspective. Treatment and prognosis probabilities originated from the Bladder Cancer Data Base, Sweden (BladderBaSe) 2.0. Information on molecular subtype and outcomes was retrieved from published studies, and quality-adjusted life year (QALY) data were obtained from the iROC trial. Costs were collected from the regional healthcare registers in Sweden, utility values were obtained from the literature, and outcomes are presented as incremental cost-effectiveness ratio (ICER). Scenario analyses, along with several one-way and probabilistic sensitivity analyses were performed to capture uncertainties.
At a 5-year time horizon, the model predicts that molecular subtype-based treatment has an ICER of 4,964 Euro/QALY (66,766 Swedish Krona/QALY), which is deemed cost-effective in the Swedish setting. At €7,427 (100,000 SEK) willingness-to-pay threshold, the molecular subtype-based treatment has a 65% probability of being cost-effective. The results were not sensitive to uncertainty analyses.
Molecular subtype-based treatment of MIBC, i.e., refraining from administering NAC to patients with Ba/Sq tumors, is cost-effective compared to the current treatment practices in Sweden.
指南推荐对肌层浸润性膀胱癌(MIBC)采用新辅助化疗(NAC)和根治性膀胱切除术(RC)。尽管基底/鳞状(Ba/Sq)亚型与尿路上皮样(Uro)和基因组不稳定(GU)亚型相比对NAC反应较小,但目前的推荐并未考虑基因组特征。本研究的目的是对Ba/Sq肿瘤和MIBC患者减少使用NAC进行成本效益分析。
从医疗服务提供者的角度,使用决策分析马尔可夫模型进行成本效益分析。治疗和预后概率源自瑞典膀胱癌数据库(BladderBaSe)2.0。分子亚型和结果的信息从已发表的研究中检索,质量调整生命年(QALY)数据从iROC试验中获得。成本从瑞典的地区医疗登记处收集,效用值从文献中获得,结果以增量成本效益比(ICER)表示。进行了情景分析以及多项单因素和概率敏感性分析以捕捉不确定性。
在5年的时间范围内,该模型预测基于分子亚型的治疗的ICER为4,964欧元/QALY(66,766瑞典克朗/QALY),在瑞典的情况下被认为具有成本效益。在7,427欧元(100,000瑞典克朗)的支付意愿阈值下,基于分子亚型的治疗具有65%的成本效益概率。结果对不确定性分析不敏感。
与瑞典目前的治疗方法相比,基于分子亚型的MIBC治疗,即不给Ba/Sq肿瘤患者使用NAC,具有成本效益。