The views of people affected by ADPKD are vital when deciding what to research. We asked the James Lind Alliance (who specialise in prioritising research) to help us prioritise the views of people affected by ADPKD (either directly or due to ADPKD in their family) and healthcare professionals.

To find the top 10 priorities, we funded a research Priority Setting Partnership (PSP), overseen by a steering group which included people living with and affected by ADPKD, healthcare professionals and the James Lind Alliance

We summarised 117 questions about ADPKD care and management that hadn't been answered by research into a shortlist of 35. We asked patients, family members, carers and healthcare professionals to rank which of the 35 questions were the most important to them. Over 700 people replied to our survey. The Top 10 research questions below are the result.

Read the ADPKD research priorities report (PDF, 737 KB) for more detail on each of these priorities and how we arrived at the Top 10.

Our next step is to encourage researchers to use the Top 10 list (and the other questions that didn’t make the top 10) to inform their research plans. 

The Top 10

1. What treatments can be developed that slow or prevent progression of ADPKD and improve patients’ quality of life?

ADPKD gets worse over time, causing damage to the kidneys. This is called progression.

Most treatments that people with ADPKD receive can reduce symptoms but don’t prevent the disease progressing.

For many people, damage to the kidneys eventually means they will need dialysis or a kidney transplant.

ADPKD can also cause other health problems as it progresses. Tolvaptan (Jinarc®) is the only treatment available that can help to slow ADPKD progression in some patients.

This research question aims to find new treatments that can slow or prevent ADPKD progression and so improve patients’ lives.

You can see the shortlist of 35 questions, with the underlying list of 117 research uncertainties, on the website of the James Lind Alliance.

Read the ADPKD PSP scope here.

Thank you!

We would like to thank everyone with ADPKD, family member, carer, and healthcare professional who took part in this PSP and the James Lind Alliance facilitators. Without their time and opinions, this PSP would not have been possible. To get involved or for more information about our research priorities, do get in touch here 

Current partners

Kidney Research UK The James Lind Alliance

Polycystic Kidney Disease Charity
91 Royal College St, London NW1 0SE
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