Research priorities for autosomal dominant polycystic kidney disease: a UK priority setting partnership
Objectives 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 pri...
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| Format: | Article |
| Language: | English |
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BMJ Publishing Group
2022-06-01
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| Series: | BMJ Open |
| Online Access: | https://bmjopen.bmj.com/content/12/6/e055780.full |
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| author | Tess Harris Albert Ong Paul J D Winyard Richard N Sandford Maryrose Tarpey Hannah R Bridges Wendy D Brown Natasha L O’Brien Ann C Daly Bharat K Jindal Gillian S Mundy Albert J Power John Sayer Roslyn J Simms Patricia D Wilson |
| author_facet | Tess Harris Albert Ong Paul J D Winyard Richard N Sandford Maryrose Tarpey Hannah R Bridges Wendy D Brown Natasha L O’Brien Ann C Daly Bharat K Jindal Gillian S Mundy Albert J Power John Sayer Roslyn J Simms Patricia D Wilson |
| author_sort | Tess Harris |
| collection | DOAJ |
| description | Objectives 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.Design 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.Setting UK.Participants 747 survey respondents (76% patients, 13% carers, 11% healthcare professionals); 23 workshop attendees.Results 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.Conclusions 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. |
| format | Article |
| id | doaj-art-2092f08f2e0a4bed98b4eb0a38ddcf19 |
| institution | Kabale University |
| issn | 2044-6055 |
| language | English |
| publishDate | 2022-06-01 |
| publisher | BMJ Publishing Group |
| record_format | Article |
| series | BMJ Open |
| spelling | doaj-art-2092f08f2e0a4bed98b4eb0a38ddcf192025-01-28T09:55:09ZengBMJ Publishing GroupBMJ Open2044-60552022-06-0112610.1136/bmjopen-2021-055780Research priorities for autosomal dominant polycystic kidney disease: a UK priority setting partnershipTess Harris0Albert Ong1Paul J D Winyard2Richard N Sandford3Maryrose Tarpey4Hannah R Bridges5Wendy D Brown6Natasha L O’Brien7Ann C Daly8Bharat K Jindal9Gillian S Mundy10Albert J Power11John Sayer12Roslyn J Simms13Patricia D Wilson14Head Office, PKD International, Geneva, SwitzerlandAcademic Nephrology Unit, The Henry Wellcome Laboratories for Medical Research, University of Sheffield Medical School, Sheffield, UKUCL Great Ormond Street Institute of Child Health, London, UKDepartment of Medical Genetics, University of Cambridge, Cambridge, UKJames Lind Alliance, Southampton, UKHB Health Comms Ltd, Orpington, UKLondon Kidney Network, London, UKADPKD patient representative, Reading, UKBirmingham Women’s and Children’s Hospitals NHS Foundation Trust, Birmingham, UKRoyal College of General Practitioners Yorkshire Faculty, Huddersfield, UKADPKD Patient Representative, Newcastle, UKNorth Bristol NHS Trust, Bristol, UKUniversity of Newcastle upon Tyne, Newcastle upon Tyne, UKNorthern General, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UKDepartment of Renal Medicine, Royal Free NHS Foundation Trust, University College London, London, UKObjectives 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.Design 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.Setting UK.Participants 747 survey respondents (76% patients, 13% carers, 11% healthcare professionals); 23 workshop attendees.Results 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.Conclusions 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.https://bmjopen.bmj.com/content/12/6/e055780.full |
| spellingShingle | Tess Harris Albert Ong Paul J D Winyard Richard N Sandford Maryrose Tarpey Hannah R Bridges Wendy D Brown Natasha L O’Brien Ann C Daly Bharat K Jindal Gillian S Mundy Albert J Power John Sayer Roslyn J Simms Patricia D Wilson Research priorities for autosomal dominant polycystic kidney disease: a UK priority setting partnership BMJ Open |
| title | Research priorities for autosomal dominant polycystic kidney disease: a UK priority setting partnership |
| title_full | Research priorities for autosomal dominant polycystic kidney disease: a UK priority setting partnership |
| title_fullStr | Research priorities for autosomal dominant polycystic kidney disease: a UK priority setting partnership |
| title_full_unstemmed | Research priorities for autosomal dominant polycystic kidney disease: a UK priority setting partnership |
| title_short | Research priorities for autosomal dominant polycystic kidney disease: a UK priority setting partnership |
| title_sort | research priorities for autosomal dominant polycystic kidney disease a uk priority setting partnership |
| url | https://bmjopen.bmj.com/content/12/6/e055780.full |
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