Objective Patient ownership of disease is vital in rare diseases like primary biliary cholangitis (PBC). This survey of UK members of the PBC foundation aimed to assess patients’ perception of their disease management, focusing on key biomarkers and problematic symptoms. Design Registered PBC foundation members were surveyed on their experiences on their most recent clinic visit, covering the type of hospital and clinician and whether biochemical response and symptom burden were discussed, including who initiated these conversations. Respondents were also asked about their willingness to initiate these conversations. Results Across 633 respondents, 42% remembered discussing alkaline phosphatase, the key biochemical response measure, and the majority of discussions were initiated by the healthcare provider. 56% of respondents remembered discussing itch, a key PBC symptom. There was no distinction between the grade of healthcare professional, but both patients and clinicians were significantly more likely to discuss symptoms over disease progression. Reassuringly, 84% of respondents felt willing to initiate conversations about their illness, regardless of the grade of managing clinician. Conclusions This work lays a positive foundation for patient education and empowerment projects, likely to improve clinical outcomes. Key aspects of management (biochemical response to treatment and symptom burden) should be emphasised as topics of discussion to both patients and clinicians managing PBC. We suggest a simple cue card to prompt patient-led discussion. All data relevant to the study are included in the article or uploaded as online supplementary information. Please contact JL for further data queries.

中文翻译：

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原发性胆汁性胆管炎长期管理的患者所有权

目的 患者对疾病的所有权对于原发性胆汁性胆管炎 (PBC) 等罕见疾病至关重要。这项针对 PBC 基金会英国成员的调查旨在评估患者对其疾病管理的看法，重点关注关键生物标志物和问题症状。设计 对注册的 PBC 基金会成员进行了调查，了解他们最近一次就诊的经历，包括医院和临床医生的类型以及是否讨论了生化反应和症状负担，包括谁发起了这些对话。受访者还被问及他们是否愿意发起这些对话。结果 在 633 名受访者中，42% 的人记得讨论过碱性磷酸酶，这是关键的生化反应指标，并且大部分讨论是由医疗保健提供者发起的。56% 的受访者记得讨论过瘙痒，这是 PBC 的一个关键症状。医疗保健专业人员的等级之间没有区别，但患者和临床医生更有可能讨论症状而不是疾病进展。令人欣慰的是，无论管理临床医生的级别如何，84% 的受访者都愿意就他们的疾病展开对话。结论 这项工作为患者教育和赋权项目奠定了积极的基础，可能会改善临床结果。管理的关键方面（对治疗和症状负担的生化反应）应作为患者和临床医生管理 PBC 的讨论主题加以强调。我们建议使用简单的提示卡来促进以患者为主导的讨论。所有与研究相关的数据都包含在文章中或作为在线补充信息上传。如需进一步的数据查询，请联系 JL。