Validation of Models Used to Inform Colorectal Cancer Screening Guidelines: Accuracy and Implications.
Validation of Models Used to Inform Colorectal Cancer Screening Guidelines: Accuracy and Implications.
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DOI：
10.1177/0272989x15622642
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发表时间：
2016-07
期刊：
影响因子：
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通讯作者：
Lansdorp-Vogelaar I
中科院分区：
文献类型：
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作者：
Rutter CM;Knudsen AB;Marsh TL;Doria-Rose VP;Johnson E;Pabiniak C;Kuntz KM;van Ballegooijen M;Zauber AG;Lansdorp-Vogelaar I
微观模拟模型综合了有关疾病过程和干预措施的证据，为预测预防、筛查和治疗策略的长期益处和危害提供了一种方法。由于模型通常需要对不可观察的过程进行假设，因此评估模型的预测准确性非常重要。我们验证了三种结直肠癌（CRC）显微模拟模型与英国柔性乙状结肠镜筛查（UKFSS）试验的结果，UKFSS试验是一项随机对照试验，旨在检查一次性柔性乙状结肠镜筛查降低CRC死亡率的有效性。这些模型结合了关于从腺瘤开始到临床前和症状性CRC发展的时间的不同假设。分析将模型预测与一系列结果的研究估计进行比较，以深入了解模型假设的准确性。所有三种模型都准确预测了筛查后10年CRC死亡率的相对降低（预测风险比，95%百分位数区间：0.56（0.44，0.71），0.63（0.51，0.75），0.68（0.53，0.83）; 95%置信区间估计：0.56（0.45，0.69））。两个具有较长平均临床前持续时间的模型准确地预测了十年CRC发病率的相对降低。两个平均逗留时间较长的模型准确预测了筛查检测到的癌症数量。所有三种模型都预测了结肠镜检查患者中有太多的近端腺瘤。模型准确性只能通过外部验证分析来确定，因此对于任何决策模型都是必不可少的。结果支持以下假设：从腺瘤开始到临床前癌症发展的平均时间较长（长达25年），平均逗留时间接近4年，这表明通过筛查进行早期检测和干预的窗口相对较长。留置时间的变化仍然不确定，可能具有重要的临床和政策意义。
Microsimulation models synthesize evidence about disease processes and interventions, providing a method for predicting long-term benefits and harms of prevention, screening, and treatment strategies. Because models often require assumptions about unobservable processes, assessing a model’s predictive accuracy is important. We validated three colorectal cancer (CRC) microsimulation models against outcomes from the United Kingdom Flexible Sigmoidoscopy Screening (UKFSS) Trial, a randomized controlled trial that examined the effectiveness of one-time flexible sigmoidoscopy screening to reduce CRC mortality. The models incorporate different assumptions about the time from adenoma initiation to development of preclinical and symptomatic CRC. Analyses compare model predictions to study estimates across a range of outcomes to provide insight into the accuracy of model assumptions. All three models accurately predicted the relative reduction in CRC mortality ten years after screening (predicted hazard ratios, with 95% percentile intervals: 0.56 (0.44,0.71), 0.63 (0.51,0.75), 0.68 (0.53,0.83); estimated with 95% confidence interval: 0.56 (0.45,0.69). Two models with longer average preclinical duration accurately predicted the relative reduction in ten-year CRC incidence. Two models with longer mean sojourn time accurately predicted the number of screen-detected cancers. All three models predicted too many proximal adenomas among patients referred to colonoscopy. Model accuracy can only be established through external validation analyses such as these and are therefore essential for any decision model. Results supported the assumptions that the average time from adenoma initiation to development of preclinical cancer is long (up to 25 years), and mean sojourn time is close to 4 years, suggesting the window for early detection and intervention by screening is relatively long. Variation in dwell time remains uncertain and could have important clinical and policy implications.