Supplementary material: Evaluation of emerging NASH therapies: the impact of treatment efficacy profiles on long-term health outcomes

These are peer-reviewed supplementary materials for the article 'Evaluation of emerging NASH therapies: the impact of treatment efficacy profiles on long-term health outcomes' published in the Journal of Comparative Effectiveness Research.Model validationSupplementary Table 1: Validation of predicted outcomesSupplemental referencesAim: Evaluations of nonalcoholic steatohepatitis (NASH) treatments require predicting lifetime outcomes from short-term clinical trials. Materials & methods: A Markov model with NASH fibrosis stages F0–F3, NASH resolution, compensated cirrhosis (F4/CC), and liver-related complication (LRC) states was developed using literature-based standard of care (SoC) data. Hypothetical efficacy profiles were defined affecting resolution (100%-increase), fibrosis improvement (100% increase), or fibrosis worsening (50% decrease). Results: For the SoC, 10-year LRC rates increased with baseline fibrosis stage (F1: 3.0%; F2: 9.8%; F3: 27.2%; F4/CC: 64.9%). The fibrosis worsening profile reduced predicted 10-year LRC rates (F1: 1.9%; F2: 6.5%; F3: 19.1%; F4/CC: 55.0%) more than the resolution and fibrosis improvement profiles (F1: 2.6%/2.6%; F2: 8.5%/8.3%; F3: 23.3%/23.0%; F4/CC: NA/59.0%). Scenario analyses considered alternative SoC progression, treatment efficacy and treatment-stopping rules. Conclusion: Potential NASH efficacy profiles have differing impacts on predicted long-term outcomes, providing insights for future stakeholders.

本数据集为发表于《比较疗效研究杂志》的论文《新兴非酒精性脂肪性肝炎（NASH）治疗评估：治疗有效性对长期健康结果的影响》的同行评审补充材料。模型验证补充表1：预测结果的验证补充参考文献目标：非酒精性脂肪性肝炎（NASH）治疗的评估需要预测从短期临床试验中获得的终身结果。材料与方法：基于文献中的标准治疗（SoC）数据，构建了一个包含NASH纤维化阶段F0-F3、NASH缓解、代偿性肝硬化（F4/CC）和肝相关并发症（LRC）状态的马尔可夫模型。定义了假设的治疗有效性特征，影响缓解（100%增加）、纤维化改善（100%增加）或纤维化恶化（50%减少）。结果：对于SoC，10年LRC发生率随基线纤维化阶段增加（F1：3.0%；F2：9.8%；F3：27.2%；F4/CC：64.9%）。纤维化恶化特征相较于缓解和纤维化改善特征，降低了预测的10年LRC发生率（F1：1.9%；F2：6.5%；F3：19.1%；F4/CC：55.0%）更多（F1：2.6%/2.6%；F2：8.5%/8.3%；F3：23.3%/23.0%；F4/CC：未提供/59.0%）。情景分析考虑了替代SoC进展、治疗有效性和治疗停止规则。结论：潜在的NASH治疗效果特征对预测的长期结果产生不同影响，为未来的利益相关者提供了见解。