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Societal costs and burden of hereditary transthyretin amyloidosis polyneuropathy.
Amyloid ( IF 5.5 ) Pub Date : 2019-12-19 , DOI: 10.1080/13506129.2019.1701429
Mónica Inês 1 , Teresa Coelho 2, 3 , Isabel Conceição 1, 4 , Filipa Landeiro 5 , Mamede de Carvalho 1, 4 , João Costa 1, 6
Affiliation  

Background: Hereditary transthyretin amyloidosis polyneuropathy (ATTRv-PN) is a rare life-threatening disease that imposes considerable mortality and morbidity associated with increased costs, high social support and productivity losses. This study aims to estimate the societal costs and burden of ATTRv-PN.

Methods: A cost-of-illness (COI) and burden of disease model were specified from a societal perspective, using a prevalence-based approach. Direct and indirect costs were included. Healthcare resource use was retrieved from public databases, previous Portuguese studies and the literature. The burden of disease was expressed in terms of disability-adjusted life years (DALYs), as defined by the World Health Organisation.

Results: In 2016, the total annual COI of ATTRv-PN in Portugal was 52,502,796€ and the mean cost per patient was 28,152€ (79% direct; 21% indirect costs). Treatments accounted for 52% of total costs, while 0.18% were devoted to disease prevention. A total of 2056 DALYs were lost, 26% due to disability and 74% due to death.

Conclusions: Annual costs and burden of ATTRv-PN were considerable but within the range of other rare diseases. Policies and public interventions to prevent and reduce the burden of disease should be prioritised, since patients experience excess morbidity, mortality and total costs will likely increase in the future.



中文翻译:

遗传性运甲状腺素蛋白淀粉样变性多发性神经病的社会成本和负担。

背景:遗传性甲状腺素转运蛋白淀粉样变性多发性神经病(ATTRv-PN)是一种罕见的威胁生命的疾病,伴随着成本增加,高社会支持和生产力损失,造成相当大的死亡率和发病率。这项研究旨在估计ATTRv-PN的社会成本和负担。

方法:采用基于流行的方法,从社会角度确定了疾病成本(COI)和疾病负担模型。直接和间接费用都包括在内。从公共数据库,先前的葡萄牙研究和文献中检索了医疗保健资源的使用。根据世界卫生组织的定义,疾病负担以残疾调整生命年(DALYs)表示。

结果: 2016年,葡萄牙ATTRv-PN的年度总COI为52,502,796欧元,每位患者的平均费用为28,152欧元(直接费用占79%;间接费用占21%)。治疗占总费用的52%,而0.18%用于疾病预防。总共损失了2056个DALY,其中26%因残疾而死亡,74%因死亡。

结论: ATTRv-PN的年度成本和负担相当可观,但在其他罕见疾病的范围内。应优先考虑采取预防和减轻疾病负担的政策和公共干预措施,因为患者的发病率,死亡率和总费用未来都可能增加。

更新日期:2019-12-19
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