Centre for Health Economics and Medicines Evaluation, Bangor University, Bangor, England, UK.
The Walton Centre NHS Trust, Liverpool, England, UK.
Value Health. 2019 Nov;22(11):1257-1265. doi: 10.1016/j.jval.2019.05.011. Epub 2019 Aug 1.
Motor neurone disease (MND) places a significant burden on patients, their carers, and healthcare systems.
To estimate health utilities and costs of MND within the UK setting.
Patients with MND, recruited via 22 regional clinics, completed a postal questionnaire of a cost and quality-of-life survey. Health outcome assessment included the EuroQoL (EQ)-5D-5L, EQ-5D-visual analogue scale, Amyotrophic Lateral Sclerosis Utility Index, and the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised. Clinical staging was based on the Kings and Milano-Torino (MiToS) systems. The questionnaire asked about patients' use of primary, secondary, and community care services in the previous 3 months. Variability in total costs was examined using regression models.
595 patients were included in the health utility analysis, of whom 584 patients also completed a resource use questionnaire. Mean health utility decreased and costs increased between consecutive Kings stages, from 0.76 (95% CI 0.71-0.80) and £1096 (£757-£1240) in Kings stage 1, to 0.50 (0.45-0.54) and £3311 (£2666-£4151) in stage 4, respectively. The changes by MiToS stages were from 0.71 (0.69-0.73) and £1115 (£937-£1130) in MiToS stage 0, to 0.25 (0.07-0.42) and £2899 (£2190-£3840) in stage 2. Kings stages 3 and 4 and MiToS stages 1 and 2, respectively, were significant in explaining variability in total costs.
The impact of MND on health utilities and costs differs by disease severity. The data provided here can be used in cost-effectiveness analyses and to inform decision-making regarding healthcare provision for people with MND.
运动神经元病（MND）给患者、其护理人员和医疗保健系统带来了巨大负担。
在英国环境下，估算 MND 的健康效用和成本。
通过 22 个区域诊所招募 MND 患者，完成一份邮寄问卷，内容为成本和生活质量调查。健康结果评估包括 EuroQoL（EQ）-5D-5L、EQ-5D 视觉模拟量表、肌萎缩侧索硬化效用指数和肌萎缩侧索硬化功能评定量表修订版。临床分期基于 Kings 和 Milano-Torino（MiToS）系统。问卷询问了患者在过去 3 个月内使用初级、二级和社区保健服务的情况。使用回归模型检查总费用的变异性。
595 名患者纳入健康效用分析，其中 584 名患者还完成了资源使用问卷。Kings 分期每增加一期，健康效用逐渐降低，费用逐渐增加，从 Kings 分期 1 时的 0.76（95%CI 0.71-0.80）和 1096 英镑（757-1240 英镑），到 Kings 分期 4 时的 0.50（0.45-0.54）和 3311 英镑（2666-4151 英镑）。MiToS 分期的变化情况为，从 MiToS 分期 0 时的 0.71（0.69-0.73）和 1115 英镑（937-1130 英镑），到 MiToS 分期 2 时的 0.25（0.07-0.42）和 2899 英镑（2190-3840 英镑）。 Kings 分期 3 和 4 以及 MiToS 分期 1 和 2 分别是解释总费用变异性的显著因素。
MND 对健康效用和成本的影响因疾病严重程度而异。这里提供的数据可用于成本效益分析，并为 MND 患者的医疗保健提供决策提供信息。
