眼咽型肌营养不良症相关的社会经济成本与健康相关生活质量
Socioeconomic costs and health-related quality of life associated with oculopharyngeal muscular dystrophy.
文献信息
| PMID | 42935765 |
|---|---|
| 原文 | 在 PubMed 查看原文 ↗ |
| 发表日期 | 2026 |
| 作者 | Ian C Smith |
| 作者单位 | The Ottawa Hospital Research Institute, Ottawa, ON, Canada. |
| 期刊 | Frontiers in public health |
| SCI 分区 | Q1 |
| IF | 4.3 |
| 研究类型 | 临床研究 · 临床 |
| 所属专科 | 咽喉科 |
中文摘要
背景： 眼咽型肌营养不良症（OPMD）是一种进行性遗传性神经肌肉疾病。OPMD通过限制吞咽、降低活动能力显著影响生活质量，并可因窒息导致死亡。我们开展本研究以从患者角度量化OPMD的疾病负担。这些数据对于为在研治疗的成本效果研究提供信息以及为公共政策提供依据是必要的。
方法： 作为全国遗传性与炎症性神经肌肉疾病负担研究调查的一部分，加拿大OPMD患者被询问OPMD对其健康相关生活质量（健康效用指数第3版；HUI3）、就业状况、家庭财务、生产力（工作生产力与活动受损问卷）以及为帮助管理OPMD而用于辅助设备和医疗保健的自付费用的影响。社会人口学和流行病学数据被用于将参与者回答外推为加拿大OPMD患者自付总成本的估计值。
结果： OPMD患者的健康相关生活质量较低（n=139，平均年龄66±10岁，52%为女性），HUI3评分为0.398±0.323，而常模人群为0.826。较低的HUI3评分与更高的辅助设备成本和更大的经济困难相关。29%的OPMD受访者处于有偿就业状态，而常模人群为41%。总工作生产力和非工作活动的受损分别为32±27%和61±29%。有偿就业与更多获得私人保险相关（比值比4.2），而后者又与就诊相关医疗保健提供者的可能性呈正相关（比值比2.5）。加拿大OPMD患者自付总成本估计为7840万加元（2026年加元）。间接成本占总成本的84%；其中53%为非工作活动受损，18%为收入能力丧失，12%为带病出勤，2%为缺勤。直接成本占总成本的16%；其中7%为医疗保健就诊，9%为辅助设备。
讨论： OPMD造成巨大的直接和间接经济负担，主要由于对工作和生产力的影响，而对设备或卫生专业人员成本的影晌较小。OPMD的高间接成本应成为经济评估和未来针对新兴治疗干预措施的卫生政策决策的考虑因素。
英文摘要
BACKGROUND: Oculopharyngeal muscular dystrophy (OPMD) is a progressive genetic neuromuscular disease. OPMD substantially impacts quality of life by limiting swallowing, decreasing mobility and can cause death by choking. We conducted this study to quantify the burden of OPMD from the patient perspective. These data are needed to inform cost-effectiveness studies for treatments in development and to inform public policy.
METHODS: As part of the national Burden of Inherited and Inflammatory Neuromuscular Disease study survey, Canadians with OPMD were asked about the impact of OPMD on their health-related quality of life (Health Utilities Index Mark 3; HUI3), employment status, household finances, productivity (Work Productivity and Activity Impairment questionnaire), and out-of-pocket costs for assistive equipment and healthcare to help manage OPMD. Sociodemographic and epidemiological data were used to extrapolate participant responses into an estimate of the total patient-borne cost of OPMD for Canada.
RESULTS: Health-related quality of life was low in OPMD (n = 139, mean age 66 ± 10 years, 52% female) with HUI3 scores of 0.398 ± 0.323, compared to 0.826 for the normative population. Low HUI3 scores were associated with higher costs for assistive equipment and greater financial hardship. 29% of respondents with OPMD were in paid employment compared to 41% in the normative population. Impairments in total work productivity and non-work activity were 32 ± 27% and 61 ± 29%, respectively. Paid employment was associated with greater access to private insurance (odds ratio 4.2) which was in turn positively associated with the likelihood of visiting allied healthcare providers (odds ratio 2.5). The total patient-borne cost of OPMD for Canada was estimated to be $78.4 million (2026 Canadian dollars). Indirect costs accounted for 84% of the total; 53% for non-work activity impairment, 18% for loss of earning capacity, 12% for presenteeism and 2% for absenteeism. Direct costs comprised 16% of the total; 7% for healthcare visits and 9% for assistive equipment.
DISCUSSION: OPMD imposes substantial direct and indirect economic burden, primarily due to impact on work and productivity and less on equipment or health professional costs. The high indirect costs of OPMD should be a consideration in economic evaluations and future health policy decisions for emerging therapeutic interventions.