Supplementary materials: Estimating the health benefits of timely diagnosis and treatment of transthyretin amyloid cardiomyopathy

These are peer-reviewed supplementary tables for the article 'Estimating the health benefits of timely diagnosis and treatment of transthyretin amyloid cardiomyopathy' published in the Journal of Comparative Effectiveness Research.Supplementary material file S1. Sensitivity and scenario analysis results.Supplementary material file S2. Technical appendix.o S2.1 Model overviewo S2.2 NYHA class (disease) progression profileso S2.3 Disease-related mortalitySupplementary Figure S1. Parametric models of overall survival – Pooled tafamidis arms (On-treatment).Supplementary Figure S2. Parametric models of overall survival – Placebo arm (Off-treatment).Summary: Aim: Delayed diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) represents a missed opportunity for intervention. This study estimates the health benefits of timely diagnosis and treatment with tafamidis. Methods: A disease simulation model was developed to predict health outcomes under scenarios of timely and delayed diagnosis and treatment. Efficacy and quality of life (QoL) profiles were derived from the pivotal tafamidis trial and diagnostic delay durations from the literature. Results: Timely diagnosis and treatment were predicted to extend mean life expectancy by 5.46 and 7.76 years, relative to delayed diagnosis, for wild-type and hereditary ATTR-CM, respectively. Corresponding QALY gains were 4.50 and 6.22. Conclusion: Timely diagnosis and treatment with tafamidis are predicted to significantly improve survival and QoL for ATTR-CM patients.

本数据集为发表于《比较疗效研究杂志》的论文《评估及时诊断和治疗转甲状腺素蛋白淀粉样心肌病（ATTR-CM）的健康益处》的同行评审补充表格。补充材料文件S1：敏感性分析和情景分析结果。补充材料文件S2：技术附录。其中S2.1为模型概述，S2.2为纽约心脏协会（NYHA）分级（疾病）进展曲线，S2.3为疾病相关死亡率。补充图表S1：总生存期的参数模型——汇总tafamidis治疗组（治疗中）。补充图表S2：总生存期的参数模型——安慰剂组（非治疗中）。总结：目的：转甲状腺素蛋白淀粉样心肌病（ATTR-CM）的延迟诊断代表着干预机会的丧失。本研究旨在评估及时诊断和治疗tafamidis的健康益处。方法：开发了一种疾病模拟模型，以预测及时和延迟诊断及治疗下的健康结果。疗效和生存质量（QoL）曲线图来源于关键性tafamidis试验，诊断延迟时间来源于文献。结果：预测及时诊断和治疗可分别使野生型和遗传型ATTR-CM患者的平均寿命延长5.46年和7.76年，相对于延迟诊断，相应的质量调整生命年（QALY）增益分别为4.50和6.22。结论：预测及时使用tafamidis进行诊断和治疗将显著改善ATTR-CM患者的生存率和生存质量。