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.
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.
For some people, early treatment may slow the progression of ADPKD and reduce the problems it causes.
For other people, some treatments may not work as well or might cause too many side effects.
This research will help doctors identify the people who will benefit from each treatment.
This means patients can start to benefit from the right treatment sooner.
ADPKD doesn’t only affect the kidneys — it can affect other parts of the body too, such as the liver.
This means people with the disease usually need to see a range of doctors specialising in different parts of the body during their lives.
Some people with ADPKD tell us that they experience inconsistencies or gaps in their care.
This can leave people frustrated and unsure whether they are getting the best care.
This research question aims to work out the best ways that different specialists providing care to people with ADPKD can work together consistently to support their patients’ health.
We do not know much about how pregnancy affects the bodies of women with ADPKD.
For example, does carrying a baby cause extra damage to the kidneys of women with ADPKD?
Can it make their liver cysts grow faster?
Based on this research, doctors will be able to give women with ADPKD a clearer picture of any risks of pregnancy to them and their babies.
This research may also help to work out which treatments could reduce the chance of these problems occurring.
There are a number of treatments to help manage ADPKD. For patients to be able to choose between the different treatments they are offered, they need to know the possible benefits and side
effects of each.
Yet there is not much research on risks and benefits, making it hard for healthcare professionals to give detailed advice on this.
This research will give patients and healthcare professionals a clearer picture of the benefits and risks of each treatment so they can be confident in selecting the right one together.
Kidney pain caused by ADPKD can be disabling and have a big impact on people’s lives. ADPKD pain can be hard to treat.
Sometimes the source of the pain (such as a cyst infection) is difficult to find or treat.
If researchers can discover which treatments are most effective for ADPKD pain, they can make a massive difference to some people’s lives.
Many people with ADPKD want to know what steps they can take to stop their disease worsening. We know that a healthy lifestyle and certain changes to diet (for example, not eating too much salt) can help.
We don’t have all the answers yet though, such as whether drinking a lot of water helps to keep the kidneys healthy.
This research can provide those answers, and so help people with PKD to take more active steps to protect their health and be in control of their disease.
A diagnosis of ADPKD can turn your life upside down and trigger a wide range of emotions.
Getting the right information and support can make an enormous difference. It can help you to cope, feel in control, and plan positively for the future.
People with ADPKD tell us that we still have more to do in this area.
This research will give people with ADPKD the chance to be heard. The results will mean better information and support for all patients, whether newly diagnosed or living with ADPKD for some time.
Tests are available to check for ADPKD in people who might have inherited the condition. This is called screening.
Screening for ADPKD in children who might have inherited the condition has some clear benefits. For example, children found to have ADPKD can begin to receive any treatments that might help. However, screening can also cause harm, such as making children and their parents anxious about their health and future.
This research will explore the benefits and harms of screening children. It will help us to understand which benefits and harms are most important to children, young people, and their families. The results will help similar people in the future choose whether or not they want to have screening tests.
ADPKD can increase the risk of getting a swollen blood vessel (an aneurysm), particularly in the brain.
Brain aneurysms often don’t cause a problem, but in some people they burst and bleed, which can cause a stroke or death. This makes aneurysms a source of great fear for some people with ADPKD, especially if other family members have had one. Some procedures can treat aneurysms before they burst.
This research will help us to understand more about aneurysms: why they occur in people with ADPKD, how they can be spotted early (through screening), and how they can be treated.
This research could save lives, reduce disability and reduce the anxiety that aneurysms cause.
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.
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