Division of Gastroenterology and Hepatology, University of Colorado Anschutz Medical Campus, Aurora, Colorado, USA.
Center for Clinical Epidemiology and Biostatistics, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Inflamm Bowel Dis. 2019 Apr 11;25(5):925-936. doi: 10.1093/ibd/izy321.
Corticosteroids (CS) and anti-TNF drugs are used to treat Crohn's disease (CD). In this study, we assessed the net health benefit of initiating anti-TNF therapy relative to additional CS use in CD using a novel combination of a retrospective cohort study and a simulation model.
Using Medicaid data from 2001 to 2005 and Medicare data from 2006 to 2013, beneficiaries were identified with CD and CS use who subsequently received either an anti-TNF or reached a cumulative dose of >3000 mg CS during the year. By using overall and latent class-specific remission-time equivalent (RTE) estimates derived from discrete-choice experiments, mean 12-month cumulative RTEs were calculated after propensity score adjustment for baseline characteristics. A Markov model was constructed using transition probabilities derived from the retrospective cohort to perform additional sensitivity analyses of RTE estimates, analytic assumptions, and transition probabilities. Cumulative RTEs were calculated via Monte Carlo simulation in this model.
In the retrospective cohort, 1563 new anti-TNF initiators and 1563 propensity score-matched prolonged CS users were identified. Anti-TNF use was associated with greater mean RTEs at the end of 1 year (5.34 vs 4.54, incremental benefit: 0.79; 95% CI, 0.53-1.07). This benefit persisted in all latent classes. In the Markov model, anti-TNF therapy was the preferred strategy, and the results were robust in multiple sensitivity analysis and latent class analysis.
In both a retrospective cohort study and a simulation model, anti-TNF use was associated with improved quality of life, measured as RTEs, when compared with continued CS utilization for CD.
皮质类固醇（CS）和抗 TNF 药物被用于治疗克罗恩病（CD）。在这项研究中，我们使用一种新的回顾性队列研究和模拟模型相结合的方法，评估了与额外 CS 应用相比，起始抗 TNF 治疗对 CD 的净健康获益。
使用 2001 年至 2005 年的 Medicaid 数据和 2006 年至 2013 年的 Medicare 数据，确定了患有 CD 和 CS 使用者，他们随后在当年接受了抗 TNF 治疗或累积 CS 剂量>3000mg。通过使用来自离散选择实验的总体和潜在类别特定缓解时间等效（RTE）估计值，在对基线特征进行倾向评分调整后，计算了 12 个月的平均累积 RTE。使用从回顾性队列中得出的转移概率构建了一个 Markov 模型，以对 RTE 估计值、分析假设和转移概率进行额外的敏感性分析。在该模型中，通过蒙特卡罗模拟计算累积 RTE。
在回顾性队列中，确定了 1563 名新的抗 TNF 起始剂和 1563 名匹配的延长 CS 使用者。与延长 CS 应用相比，抗 TNF 治疗与 1 年末更高的平均 RTE 相关（5.34 比 4.54，增量获益：0.79；95%CI，0.53-1.07）。这种获益在所有潜在类别中均持续存在。在 Markov 模型中，抗 TNF 治疗是首选策略，且在多次敏感性分析和潜在类别分析中结果稳健。
在回顾性队列研究和模拟模型中，与继续使用 CS 治疗 CD 相比，抗 TNF 治疗与改善生活质量（以 RTE 衡量）相关。
