Polycystic Kidney Disease Charity, London, UK
HB Health Comms Ltd, Orpington, UK.
BMJ Open. 2022 Jun 15;12(6):e055780. doi: 10.1136/bmjopen-2021-055780.
Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney condition, accounting for 7%-10% of patients with kidney failure. Fundamental basic science and clinical research on ADPKD is underway worldwide but no one has yet considered which areas should be prioritised to maximise returns from limited future funding. The Polycystic Kidney Disease Charity began a priority setting partnership with the James Lind Alliance (JLA) in the UK in 2019-2020 to identify areas of uncertainty in the ADPKD care pathway and allow patients, carers and healthcare professionals to rank the 10 most important questions for research.
The scope covered ADPKD diagnosis and management, identifying new treatments to prevent/slow disease progression and practical, integrated patient support (https://pkdcharity.org.uk/research/for-researchers/adpkd-research-priorities). We used adapted JLA methodology. Initially, an independent information specialist collated uncertainties in ADPKD care from recent consensus conference proceedings and additional literature. These were refined into indicative questions with Steering Group oversight. Finally, the 10 most important questions were established via a survey and online consensus workshop.
UK.
747 survey respondents (76% patients, 13% carers, 11% healthcare professionals); 23 workshop attendees.
117 uncertainties in ADPKD care were identified and refined into 35 indicative questions. A shortlist of 17 questions was established through the survey. Workshop participants reached agreement on the top 10 ranking. The top three questions prioritised by patients, carers and healthcare professionals centred around slowing disease progression, identifying persons for early treatment and organising care to improve outcomes.
Our shortlist reflects the varied physical, psychological and practical challenges of living with and treating ADPKD, and perceived gaps in knowledge that impair optimal care. We propose that future ADPKD research funding takes these priorities into account to focus on the most important areas and to maximise improvements in ADPKD outcomes.
常染色体显性遗传多囊肾病（ADPKD）是最常见的遗传性肾病，占肾衰竭患者的 7%-10%。全世界都在进行 ADPKD 的基础科学和临床研究，但尚未有人考虑应优先考虑哪些领域，以最大限度地从有限的未来资金中获得回报。多囊肾病慈善基金会（PKD Charity）于 2019-2020 年在英国与詹姆斯林德联盟（JLA）建立了优先事项设定伙伴关系，以确定 ADPKD 护理途径中的不确定领域，并使患者、照顾者和医疗保健专业人员能够对研究的 10 个最重要问题进行排名。
研究范围涵盖 ADPKD 的诊断和管理，确定预防/减缓疾病进展的新治疗方法以及实用的综合患者支持（https://pkdcharity.org.uk/research/for-researchers/adpkd-research-priorities）。我们使用了经过改编的 JLA 方法。最初，一名独立的信息专家从最近的共识会议记录和其他文献中整理了 ADPKD 护理中的不确定性。在指导小组的监督下，这些不确定性被细化为指示性问题。最后，通过调查和在线共识研讨会确定了 10 个最重要的问题。
英国。
747 名调查受访者（76%为患者，13%为照顾者，11%为医疗保健专业人员）；23 名研讨会参与者。
确定了 117 个 ADPKD 护理中的不确定性，并将其细化为 35 个指示性问题。通过调查确定了 17 个问题的候选名单。研讨会参与者就前 10 名排名达成一致。患者、照顾者和医疗保健专业人员最关心的前三个问题集中在减缓疾病进展、确定早期治疗对象和组织护理以改善结果上。
我们的候选名单反映了治疗和治疗 ADPKD 所带来的各种身体、心理和实际挑战，以及知识差距，这些差距会影响最佳护理。我们建议未来的 ADPKD 研究资金考虑这些优先事项，关注最重要的领域，最大限度地提高 ADPKD 结果的改善。
