西班牙大疱性表皮松解症患者的经济负担和健康相关生活质量。

Economic burden and health-related quality of life in patients with epidermolysis bullosa in Spain.

机构信息

Economic Analysis and Finance Department, Faculty of Social Sciences and Information Technologies, University of Castilla-La Mancha, Talavera de la Reina, 45600, Spain.

Economic Analysis and Finance Department, Faculty of Law and Social Sciences, University of Castilla-La Mancha, Toledo, 45071, Spain.

出版信息

Orphanet J Rare Dis. 2024 Sep 23;19(1):352. doi: 10.1186/s13023-024-03328-1.

DOI:10.1186/s13023-024-03328-1

PMID:39313783

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11421099/

Abstract

BACKGROUND

. Epidermolysis bullosa (EB) is a rare genetic skin disorder characterized by fragility of skin with appearance of acute and chronic wounds. The aim of this study was to determine the economic burden and the health-related quality of life (HRQoL) of patients with epidermolysis bullosa (EB) in Spain from a societal perspective.

METHODS

. We conducted a cross-sectional, retrospective study including 62 patients with EB (62% dystrophic, 9.6% junctional, 3.2% Kindler syndrome, and 26% with simplex EB). Data were collected from questionnaires completed by patients or their caregivers. The costs were estimated, including not only direct healthcare costs but also direct non-healthcare costs and productivity losses. We compared severe EB (Dystrophic, Junctional EB and Kindler syndrome) to non-severe EB (simplex EB) using as reference year 2022. HRQoL was measured by generic (EQ-5D) and specific (QoLEB) questionnaires.

RESULTS

The average annual cost for an EB patient was €31,352. Direct healthcare costs represented 17.2% of the total cost, direct non-healthcare costs (mainly informal care costs) 71.3% and productivity losses 11.5% of the total cost. Participants in the severe EB group had a slightly higher average cost than participants in the non-severe EB group (€31,706 vs. €30,337). Direct healthcare costs and non-healthcare costs were higher in the severe EB group (€6,205 vs. €3,024 and €23,148 vs. €20,113) while productivity losses were higher in the non-severe EB group (€7,200 vs. €2,353). The mean utility index score, where the maximum value possible is one, was 0.45 for patients with severe EB (0.76 for their caregivers) and 0.62 for those with non-severe EB (0.77 for their caregivers).

CONCLUSIONS

. The social economic burden of EB, resulting from the high direct non-healthcare cost of informal care, and from the loss of productivity, accentuates the importance of not restricting cost analysis to direct healthcare costs. This substantiates that EB, particularly severe EB represents a significant hidden cost that should be revealed to society and should be considered in the support programmes for people who suffer from this disease, and in the economic evaluation of new treatments.

摘要

背景

. 大疱性表皮松解症（EB）是一种罕见的遗传性皮肤疾病，其特征是皮肤脆弱，容易出现急性和慢性伤口。本研究旨在从社会角度确定西班牙大疱性表皮松解症（EB）患者的经济负担和健康相关生活质量（HRQoL）。

方法

. 我们进行了一项横断面、回顾性研究，纳入了 62 名 EB 患者（62%为营养不良型，9.6%为交界型，3.2%为 Kindler 综合征，26%为单纯型 EB）。数据来自患者或其护理人员完成的问卷调查。估计了成本，不仅包括直接医疗保健成本，还包括直接非医疗保健成本和生产力损失。我们将严重 EB（营养不良型、交界型 EB 和 Kindler 综合征）与非严重 EB（单纯型 EB）进行了比较，参考年份为 2022 年。使用通用（EQ-5D）和特定（QoLEB）问卷衡量 HRQoL。

结果

EB 患者的年平均费用为 31352 欧元。直接医疗保健成本占总成本的 17.2%，直接非医疗保健成本（主要是非正式护理成本）占 71.3%，生产力损失占 11.5%。严重 EB 组参与者的平均费用略高于非严重 EB 组参与者（31706 欧元比 30337 欧元）。严重 EB 组的直接医疗保健成本和非医疗保健成本较高（6205 欧元比 3024 欧元和 23148 欧元比 20113 欧元），而非严重 EB 组的生产力损失较高（7200 欧元比 2353 欧元）。严重 EB 患者的平均效用指数得分（可能的最大值为 1）为 0.45（其护理人员为 0.76），非严重 EB 患者为 0.62（其护理人员为 0.77）。