ABSTRACT

Objective: To estimate the health and economic impact of the reduction in mortality and cardiovascular hospitalizations, associated with correct diagnosis of cardiac transthyretin amyloidosis (ATTR-CM), from the Spanish National Health System (NHS) perspective.

Methods: A costs and effects analysis were performed (probabilistic Markov model) with time horizons between 1 and 15 years, comparing the correct diagnosis of ATTR-CM versus the non-diagnosis. Transition probabilities were obtained from the ATTR-ACT study (placebo arm) and from the literature. Costs and healthcare resources were obtained from Spanish sources (€ 2019) and from a panel of Spanish clinical experts.

Results: After 1, 5, 10 and 15 years, the diagnosis of ATTR-CM would generate a gain of 0.031 (95%CI 0.025; 0.038); 0.387 (95%CI 0.329; 0.435); 0.754 (95%CI 0.678; 0.781) and 0.944 (95%CI 0.905; 0.983) life years per patient, respectively, with savings of € 212 (95%CI € −632; 633), € 2,289 (95%CI € 2,250; 2,517), € 2,859 (95%CI € 2,584; 3,149) and € 2,906 (95%CI € 2,669; 3,450) per patient, respectively, versus the non-diagnosis.

Conclusions: Just by correctly diagnosing ATTR-CM, years of life would be gained, cardiovascular hospitalizations would be avoided, and savings would be generated for the NHS, compared to the non-diagnosis of the disease.

摘要

目的：从西班牙国家卫生系统 (NHS) 的角度，评估与心脏转甲状腺素蛋白淀粉样变性 (ATTR-CM) 的正确诊断相关的死亡率和心血管住院率降低对健康和经济的影响。

方法：在 1 到 15 年的时间范围内进行成本和效果分析（概率马尔可夫模型），比较 ATTR-CM 的正确诊断与非诊断。转移概率从 ATTR-ACT 研究（安慰剂组）和文献中获得。成本和医疗保健资源来自西班牙来源（2019 欧元）和西班牙临床专家小组。

结果：1、5、10 和 15 年后，ATTR-CM 的诊断将产生 0.031（95%CI 0.025；0.038）的增益；0.387 (95% CI 0.329; 0.435); 每位患者分别为 0.754 (95%CI 0.678; 0.781) 和 0.944 (95%CI 0.905; 0.983) 生命年，节省 212 欧元（95%CI 0.632 欧元；633），2,289 欧元（95%CI 2,250 欧元） ; 2,517)、2,859 欧元（95%CI 2,584 欧元；3,149）和 2,906 欧元（95%CI 2,669 欧元；3,450）与非诊断患者相比。

结论：与未诊断疾病相比，只要正确诊断 ATTR-CM，就可以延长寿命，避免心血管住院，并为 NHS 节省开支。