Background & Aims

There have been few high-quality studies of the costs, preference-based health-related quality of life (HRQoL) and cost effectiveness of treatments for primary biliary cholangitis (PBC). We aimed to estimate the marginal effects of PBC complications and symptoms, accounting for treatment, on HRQoL and the annual cost of health care in the United Kingdom (UK). These are essential components for evaluation of cost effectiveness and this information will aid in evaluation of new treatments.

Methods

Questionnaires were mailed to 4583 participants in the UK-PBC research cohort and data were collected on HRQoL and use of the National Health Service (NHS) in the UK from 2015 through 2016. HRQoL was measured using the EQ-5D-5L instrument. The annual cost of resource use was calculated using unit costs obtained from NHS sources. We performed econometric analyses to determine the effects of treatment, symptoms, complications, liver transplantation status, and patient characteristics on HRQoL and annual costs.

Results

In an analysis of data from 2240 participants (over 10% of all UK PBC patients), we found that PBC symptoms have a considerable effect on HRQoL. Ursodeoxycholic acid therapy was associated with significantly higher HRQoL regardless of response status. Having had a liver transplant and ascites were also independently associated with reduced HRQoL. Having had a liver transplant (US$4294) and esophageal varices (US$3401) were the factors with the two greatest mean annual costs to the NHS. Symptoms were not independently associated with cost but were associated with reduction in HRQoL for patients, indicating the lack of effective treatments for PBC symptoms.

Conclusions

In an analysis of data from 2240 participants in the UK PBC, we found that HRQoL and cost estimates provide greater insight into the relative importance of PBC-related symptoms and complications. These findings provide estimates for health technology assessments of new treatments for PBC.

中文翻译：

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原发性胆汁性胆管炎对英国生活质量和医疗费用的影响

 背景与目标

关于原发性胆汁性胆管炎 (PBC) 治疗的成本、基于偏好的健康相关生活质量 (HRQoL) 和成本效益的高质量研究很少。我们的目的是估计英国 (UK) PBC 并发症和症状（考虑治疗）对 HRQoL 和年度医疗费用的边际影响。这些是评估成本效益的重要组成部分，这些信息将有助于评估新疗法。

 方法

向 UK-PBC 研究队列的 4583 名参与者邮寄了问卷，并收集了 2015 年至 2016 年英国 HRQoL 和国民医疗服务 (NHS) 使用情况的数据。HRQoL 使用 EQ-5D-5L 仪器进行测量。年度资源使用成本是使用从 NHS 来源获得的单位成本计算的。我们进行了计量经济学分析，以确定治疗、症状、并发症、肝移植状态和患者特征对 HRQoL 和年度费用的影响。

 结果

在对 2240 名参与者（超过所有英国 PBC 患者的 10%）的数据分析中，我们发现 PBC 症状对 HRQoL 有相当大的影响。无论反应状态如何，熊去氧胆酸治疗均与显着较高的 HRQoL 相关。接受肝移植和腹水也与 HRQoL 降低独立相关。肝移植（4294 美元）和食管静脉曲张（3401 美元）是 NHS 每年平均费用的两个最大因素。症状与费用并不独立相关，但与患者 HRQoL 的降低相关，表明缺乏针对 PBC 症状的有效治疗方法。

 结论

在对英国 PBC 2240 名参与者的数据分析中，我们发现 HRQoL 和成本估算可以更深入地了解 PBC 相关症状和并发症的相对重要性。这些发现为 PBC 新疗法的卫生技术评估提供了估计。