Das Ananya, Callenberg Keith M, Styn Mindi A, Jackson Sara A
Arizona Center for Digestive Health, Gilbert, Arizona, USA.
University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, USA; Interpace Diagnostics Corporation (formerly RedPath Integrated Pathology), Pittsburgh, Pennsylvania, USA.
Endosc Int Open. 2016 May;4(5):E549-59. doi: 10.1055/s-0042-103415. Epub 2016 Apr 15.
The surveillance of patients with nondysplastic Barrett's esophagus (NDBE) has a high cost and is of limited effectiveness in preventing esophageal adenocarcinoma (EAC). Ablation for NDBE remains expensive and controversial. Biomarkers of genomic instability have shown promise in identifying patients with NDBE at high risk for progression to EAC. Here, we evaluate the cost-effectiveness of using such biomarkers to stratify patients with NDBE by risk for EAC and, subsequently, the cost-effectiveness of ablative therapy.
A Markov decision tree was used to evaluate four strategies in a hypothetical cohort of 50-year old patients with NDBE over their lifetime: strategy I, natural history without surveillance; strategy II, surveillance per current guidelines; strategy III, ablation for all patients; strategy IV, risk stratification with use of a biomarker panel to assess genomic instability (i. e., mutational load [ML]). Patients with no ML underwent minimal surveillance, patients with low ML underwent standard surveillance, and patients with high ML underwent ablation. The incremental cost-effectiveness ratio (ICER) and incremental net health benefit (INHB) were assessed.
Strategy IV provided the best values for quality-adjusted life years (QALYs), ICER, and INHB in comparison with strategies II and III. RESULTS were robust in sensitivity analysis. In a Monte Carlo analysis, the relative risk for the development of cancer in the patients managed with strategy IV was decreased. Critical determinants of strategy IV cost-effectiveness were the complete response rate, cost of ablation, and surveillance interval in patients with no ML.
The use of ML to stratify patients with NDBE by risk was the most cost-effective strategy for preventive EAC treatment. Targeting ablation toward patients with high ML presents an opportunity for a paradigm shift in the management of NDBE.
对非发育异常性巴雷特食管(NDBE)患者进行监测成本高昂,且在预防食管腺癌(EAC)方面效果有限。NDBE的消融治疗成本依然高昂且存在争议。基因组不稳定性生物标志物在识别有进展为EAC高风险的NDBE患者方面显示出前景。在此,我们评估使用此类生物标志物按EAC风险对NDBE患者进行分层的成本效益,以及随后消融治疗的成本效益。
采用马尔可夫决策树来评估一组假设的50岁NDBE患者一生中的四种策略:策略I,不进行监测的自然病程;策略II,按照现行指南进行监测;策略III,对所有患者进行消融治疗;策略IV,使用生物标志物组合评估基因组不稳定性(即突变负荷[ML])进行风险分层。无ML的患者接受最少监测,低ML的患者接受标准监测,高ML的患者接受消融治疗。评估增量成本效益比(ICER)和增量净健康效益(INHB)。
与策略II和III相比,策略IV在质量调整生命年(QALY)、ICER和INHB方面提供了最佳值。敏感性分析结果稳健。在蒙特卡洛分析中,采用策略IV管理的患者发生癌症的相对风险降低。策略IV成本效益的关键决定因素是完全缓解率、消融成本以及无ML患者的监测间隔。
使用ML按风险对NDBE患者进行分层是预防性EAC治疗中最具成本效益的策略。针对高ML患者进行消融治疗为NDBE的管理带来了模式转变的机会。