Kroep Sonja, Lansdorp-Vogelaar Iris, van der Steen Alex, Inadomi John M, van Ballegooijen Marjolein
Department of Public Health, Erasmus Medical Center University, Rotterdam, the Netherlands (SK, ILV, AVDS, MVB)
Division of Gastroenterology, Department of Medicine, University of Washington, Seattle, Washington, USA (JMI)
Med Decis Making. 2015 Aug;35(6):726-33. doi: 10.1177/0272989X14551640. Epub 2014 Oct 2.
Estimates for the annual progression rate from Barrett's esophagus (BE) to esophageal adenocarcinoma (EAC) vary widely. In this explorative study, we quantified how this uncertainty affects the estimates of effectiveness and efficiency of screening and treatment for EAC.
We developed 3 versions of the University of Washington / Microsimulation Screening Analysis-EAC model. The models differed with respect to the annual progression rate from BE to EAC (0.12% or 0.42%) and the possibility of spontaneous regression of dysplasia (yes or no). All versions of the model were calibrated to the observed Surveillance, Epidemiology, and End Results esophageal cancer incidence rates from 1998 to 2009. To identify the impact of natural history, we estimated the incidence and deaths prevented as well as numbers needed to screen (NNS) and treat (NNT) of a one-time perfect screening at age 65 years that detected all prevalent BE cases, followed by a perfect treatment intervention.
Assuming a perfect screening and treatment intervention for all patients with BE, the maximum EAC mortality reduction (64%-66%) and the NNS per death prevented (470-510) were similar across the 3 model versions. However, 3 times more people needed to be treated to prevent 1 death (24 v. 8) in the 0.12% regression model compared with the 0.42% progression model. Restricting treatment to those with dysplasia or only high-grade dysplasia resulted in smaller differences in NNT (2-3 to prevent one EAC case) but wider variation in effectiveness (mortality reduction of 15%-24%).
The uncertainty in the natural history of the BE to EAC sequence influenced the estimates of effectiveness and efficiency of BE screening and treatment considerably. This uncertainty could seriously hamper decision making about implementing BE screening and treatment interventions.
巴雷特食管（BE）进展为食管腺癌（EAC）的年进展率估计差异很大。在这项探索性研究中，我们量化了这种不确定性如何影响EAC筛查和治疗的有效性和效率估计。
我们开发了3个版本的华盛顿大学/微观模拟筛查分析-EAC模型。这些模型在BE进展为EAC的年进展率（0.12%或0.42%）以及发育异常自发消退的可能性（是或否）方面存在差异。所有版本的模型均根据1998年至2009年观察到的监测、流行病学和最终结果食管癌发病率进行校准。为了确定自然史的影响，我们估计了65岁时一次性完美筛查（检测到所有现患BE病例）并随后进行完美治疗干预所预防的发病率和死亡人数，以及筛查所需人数（NNS）和治疗所需人数（NNT）。
假设对所有BE患者进行完美的筛查和治疗干预，3个模型版本的最大EAC死亡率降低（64%-66%）和预防每例死亡的NNS（470-510）相似。然而，与0.42%进展模型相比，0.12%消退模型中预防1例死亡需要治疗的人数多3倍（24比8）。将治疗限制在发育异常或仅高级别发育异常的患者中，NNT的差异较小（预防1例EAC病例为2-3），但有效性的差异较大（死亡率降低15%-24%）。
BE向EAC转变自然史的不确定性对BE筛查和治疗的有效性和效率估计有很大影响。这种不确定性可能严重阻碍关于实施BE筛查和治疗干预的决策。
