西班牙脊髓性肌萎缩症（SMA）患者的社会/经济成本和与健康相关的生活质量。
Social/economic costs and health-related quality of life in patients with spinal muscular atrophy (SMA) in Spain.
机构信息
Faculty of Occupational Therapy, Speech Therapy and Nursing, University of Castilla-La Mancha, Talavera de la Reina. Toledo, Spain.
Faculty of Law and Social Sciences, University of Castilla-La Mancha, Toledo, Spain.
出版信息
Orphanet J Rare Dis. 2017 Aug 18;12(1):141. doi: 10.1186/s13023-017-0695-0.
BACKGROUND
The aim of this study was to determine the economic burden and health-related quality of life (HRQOL) of patients with Spinal Muscular Atrophy (SMA) and their caregivers in Spain.
METHODS
This was a cross-sectional and retrospective study of patients diagnosed with SMA in Spain. We adopted a bottom up, prevalence approach design to study patients with SMA. The patient's caregivers completed an anonymous questionnaire regarding their socio-demographic characteristics, use of healthcare services and non-healthcare services. Costs were estimated from a societal perspective (including healthcare costs and non-healthcare costs), and health-related quality of life (HRQOL) was assessed using the EQ-5D questionnaire. The main caregivers also answered a questionnaire on their characteristics and on their HRQOL.
RESULTS
A total of 81 caregivers of patients with different subtypes of SMA completed the questionnaire. Based on the reference unitary prices for 2014, the average annual costs per patient were € 33,721. Direct healthcare costs were € 10,882 (representing around 32.3% of the total cost) and the direct non-healthcare costs were € 22,839 (67.7% of the total cost). The mean EQ-5D social tariff score for patients was 0.16, and the mean score of the EQ-5D visual analogue scale was 54. The mean EQ-5D social tariff score for caregivers was 0.49 and their mean score on the EQ-5D visual analogue scale was 69.
CONCLUSION
The results highlight the burden that SMA has in terms of costs and decreased HRQOL, not only for patients but also for their caregivers. In particular, the substantial social/economic burden is mostly attributable to the high direct non-healthcare costs.
背景
本研究旨在确定西班牙脊髓性肌萎缩症（SMA）患者及其照顾者的经济负担和健康相关生活质量（HRQOL）。
方法
这是一项在西班牙进行的 SMA 患者的横断面和回顾性研究。我们采用自下而上的流行率方法设计来研究 SMA 患者。患者的照顾者完成了一份关于他们的社会人口统计学特征、医疗保健服务和非医疗保健服务使用情况的匿名问卷。成本是从社会角度（包括医疗保健成本和非医疗保健成本）估算的，健康相关生活质量（HRQOL）是使用 EQ-5D 问卷评估的。主要照顾者还回答了一份关于他们的特征和他们的 HRQOL 的问卷。
结果
共有 81 名患有不同亚型 SMA 的患者照顾者完成了问卷。根据 2014 年的参考单位价格，每位患者的平均年费用为 33721 欧元。直接医疗保健成本为 10882 欧元（占总成本的 32.3%），直接非医疗保健成本为 22839 欧元（占总成本的 67.7%）。患者的平均 EQ-5D 社会关税评分为 0.16，平均 EQ-5D 视觉模拟评分（VAS）为 54。照顾者的平均 EQ-5D 社会关税评分为 0.49，他们的 EQ-5D 视觉模拟评分（VAS）平均得分为 69。
结论
研究结果强调了 SMA 在成本和 HRQOL 下降方面给患者及其照顾者带来的负担。特别是，主要是由于高直接非医疗保健成本导致了巨大的社会/经济负担。
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