Department of Urology, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Department of Urology, Istanbul University, Istanbul, Turkey.
BJU Int. 2018 Sep;122(3):434-440. doi: 10.1111/bju.14220. Epub 2018 Apr 24.
To model the cost-effectiveness of a biomarker-based approach to select patients for neoadjuvant chemotherapy (NAC) before radical cystectomy (RC) in muscle-invasive bladder cancer (MIBC).
We obtained data from the most recent clinical studies on patients with locally advanced MIBC treated by RC, including stage distributions, overall survival (OS) estimates, associated costs, and utilisation/response to NAC. Additionally, we estimated the putative efficacy of three biomarkers to select patients for NAC: DNA-repair gene panel [ataxia telangiectasia mutated (ATM), retinoblastoma 1 (RB1), and Fanconi anaemia complementation group C (FANCC)], excision repair cross-complementation group 2 (ERCC2), and ribonucleic acid (RNA) subtypes. A decision analysis model was developed to evaluate the cost-effectiveness of biomarker-based approaches to select patients with MIBC for NAC. Comparison of cost-effectiveness included RC alone, unselected NAC plus RC, and NAC based on the three aforementioned biomarkers.
The DNA-repair gene panel-based approach to NAC was the most cost-effective strategy (mean OS of 3.14 years, $31 482/life year). Under this approach, 38% would undergo NAC, about twice the number of patients who are currently receiving NAC for MIBC. Such an approach would improve mean OS by 5.2, 1.6, and 4.4 months compared to RC alone, a hypothetical scenario where all patients received NAC, and compared to current estimates of NAC utilisation, respectively.
A biomarker-based strategy to identify patients with MIBC who should undergo NAC was more cost-effective than unselected use of NAC or RC alone. As further data becomes available, such a model may serve as a basis for incorporating biomarkers into clinical decision making.
构建生物标志物指导新辅助化疗（NAC）选择肌层浸润性膀胱癌（MIBC）患者的成本效果模型。
我们获取了最新的 MIBC 患者接受根治性膀胱切除术（RC）治疗的临床研究数据，包括各分期分布、总生存期（OS）估计、相关成本以及 NAC 的应用/反应情况。此外，我们还评估了三种生物标志物对 NAC 患者选择的潜在疗效：DNA 修复基因谱（共济失调毛细血管扩张突变基因（ATM）、视网膜母细胞瘤基因 1（RB1）和范可尼贫血互补组 C（FANCC））、切除修复交叉互补基因 2（ERCC2）和核糖核酸（RNA）亚型。建立决策分析模型以评估 MIBC 患者 NAC 选择的基于生物标志物的方法的成本效果。成本效果比较包括单独接受 RC、未经选择的 NAC 联合 RC 以及基于上述三种生物标志物的 NAC。
基于 DNA 修复基因谱的 NAC 方法是最具成本效益的策略（平均 OS 为 3.14 年，每生命年 31482 美元）。在此方法下，约 38%的患者将接受 NAC，是目前 MIBC 接受 NAC 治疗患者人数的两倍。与单独接受 RC、所有患者均接受 NAC 的假设情况以及当前 NAC 应用的估计值相比，这种方法可分别将 MIBC 患者的平均 OS 提高 5.2、1.6 和 4.4 个月。
基于生物标志物的策略可识别应接受 NAC 的 MIBC 患者，比未经选择的 NAC 或单独接受 RC 更具成本效果。随着更多数据的出现，此类模型可能成为将生物标志物纳入临床决策的基础。
