Marcus Elena, Stone Patrick, Thorburn Douglas, Walmsley Martine, Vivat Bella
Population Health Research Institute, St George's University of London, Cranmer Terrace, London, SW17 0RE, UK.
Marie Curie Palliative Care Research Department, Division of Psychiatry, University College London, London, UK.
J Patient Rep Outcomes. 2022 Jul 15;6(1):76. doi: 10.1186/s41687-022-00484-5.
Primary sclerosing cholangitis (PSC) is a rare incurable disease of the bile ducts and liver which can significantly impair quality of life (QoL). No existing QoL tools are entirely suitable for people living with PSC (PwPSC). We aimed to develop a measure of QoL for PwPSC in the UK, beginning by identifying relevant QoL issues. This paper describes our approach to this first stage, and discusses related benefits and limitations.
Scientific consensus on how to reliably stage PSC is lacking, due to its rarity and heterogeneity. We initially hypothesised four categories for PSC severity. After beginning the study, these were revised to six. For such a rare disease, the study could not recruit sufficient participants in each of these categories, particularly the more severe, in the time available. We therefore modified the design, adapting standard methodology for identifying potentially relevant issues. We started by conducting a thematic analysis of data from a previous survey of PwPSC, and extracting QoL issues from a literature review of QoL questionnaires of relevance to PwPSC. We then conducted group and individual interviews with PwPSC and clinicians, investigating the relevance, importance, phrasing, and breadth of coverage of issues identified. We also explored the validity of our hypothesised categories for disease severity.
We identified 1,052 potentially relevant QoL issues from the survey and literature review and took 396 of these forwards for discussion with 28 PwPSC. We found 168/396 issues were considered relevant by ≥ 60% of these participants. We then discussed this subset of 168 issues with 11 clinicians. PSC and clinician participants identified some problematic phrasing with 19 issues, due to potential upset (n = 12) or problems with understanding (n = 7). We included one new issue from those suggested.
We identified a range of QoL issues relevant to PwPSC, with a good breadth of coverage, although lacking an in-depth understanding of the PSC experience. Our strategy effectively identified relevant QoL issues for people living with this rare condition, for which there is no consensus on stratifying for its severity. This strategy should however be considered specific to such circumstances, not a general recommendation for an alternative approach.
原发性硬化性胆管炎（PSC）是一种罕见的、无法治愈的胆管和肝脏疾病，会严重影响生活质量（QoL）。现有的生活质量评估工具都不完全适用于原发性硬化性胆管炎患者（PwPSC）。我们旨在为英国的原发性硬化性胆管炎患者开发一种生活质量评估方法，首先要确定相关的生活质量问题。本文描述了我们在这第一阶段的方法，并讨论了相关的益处和局限性。
由于原发性硬化性胆管炎的罕见性和异质性，目前缺乏关于如何可靠地对其进行分期的科学共识。我们最初假设了原发性硬化性胆管炎严重程度的四个类别。在研究开始后，这些类别被修订为六个。对于这样一种罕见疾病，该研究无法在可用时间内为每个类别招募到足够的参与者，尤其是病情更严重的类别。因此，我们修改了设计，采用标准方法来确定潜在的相关问题。我们首先对之前一项原发性硬化性胆管炎患者调查的数据进行了主题分析，并从与原发性硬化性胆管炎相关的生活质量问卷的文献综述中提取生活质量问题。然后，我们对原发性硬化性胆管炎患者和临床医生进行了小组和个人访谈，调查所确定问题的相关性、重要性、表述方式和涵盖范围。我们还探讨了我们假设的疾病严重程度类别是否有效。
我们从调查和文献综述中确定了1052个潜在的相关生活质量问题，并从中选取了396个问题与28名原发性硬化性胆管炎患者进行讨论。我们发现，这些参与者中≥60%认为168/396个问题是相关的。然后，我们与11名临床医生讨论了这168个问题的子集。原发性硬化性胆管炎患者和临床医生参与者发现19个问题的表述存在问题，原因是可能令人不安（n = 12）或理解困难（n = 7）。我们从建议的问题中纳入了一个新问题。
我们确定了一系列与原发性硬化性胆管炎患者相关的生活质量问题，涵盖范围较广，尽管对原发性硬化性胆管炎的体验缺乏深入了解。我们的策略有效地为患有这种罕见疾病的患者确定了相关的生活质量问题，对于这种疾病的严重程度分层尚无共识。然而，应认为这种策略特定于此类情况，而非对替代方法的一般建议。
