The longlist of 35 indicative questions* that appeared in the survey, grouped by theme and with shortlisted questions shown first
| Theme | Question | Shortlisted |
| Diagnosis | What are the benefits and harms of screening for and diagnosing ADPKD in children and young people (up to 18 years) at risk of having inherited this condition? | ✓ |
| When a person is found to have kidney cysts but they do not have a family history of ADPKD, what tests should be performed to confirm their diagnosis and check for ADPKD? | ✓ | |
| Symptoms, disease course and pathogenesis | What effect does pregnancy have on women with ADPKD including their pregnancy health, kidney function, and liver cysts? | ✓ |
| What causes enlarged blood vessels (aneurysms) in some people with ADPKD and what is the most effective way to screen for and treat aneurysms? | ✓ | |
| Why do the symptoms and severity of polycystic liver disease (PLD) vary between people? | ✓ | |
| How does ADPKD affect children and young people (up to 18 years), including those who might not have symptoms (eg, those diagnosed based on an ultrasound of their kidneys or genetic test). | ||
| In people with ADPKD, do kidney stones increase the risk of kidney function decline? | ||
| If a woman with ADPKD is pregnant, what effect does her condition have on her child before birth? | ||
| For women with PLD, does using oral contraceptives to prevent pregnancy or hormone replacement therapy to treat the menopause affect their PLD? | ||
| Management of ADPKD | Does early treatment of high blood pressure improve the long-term health of people with ADPKD and/or reduce the risk of thickened heart walls (left ventricular hypertrophy)? | ✓ |
| What are the most effective treatments for high blood pressure (hypertension) for people (children and adults) with ADPKD? | ✓ | |
| What symptoms are associated with cyst infections in people with ADPKD, and how are cyst infections best managed (investigated and treated)? | ✓ | |
| What causes severe (acute) and long-term (chronic) kidney pain in people with ADPKD? | ✓ | |
| For people with ADPKD experiencing pain, what treatments work best to reduce this pain? For example, treatments might include removing nerves in the kidney or draining the liquid out of kidney cysts. | ✓ | |
| What treatments can be developed that slow or prevent progression of ADPKD and so improve patients’ quality of life? | ✓ | |
| In which circumstances should removal of a kidney (known as nephrectomy) be considered in people with ADPKD, and are there alternative treatments? | ✓ | |
| Which people with ADPKD would benefit from early treatment and how can doctors identify them? | ✓ | |
| What are the benefits and harms of drugs that can be used for the management of ADPKD including PLD? | ✓ | |
| What are the benefits and harms of treating high cholesterol and/or high uric acid (known as hyperuricaemia) in people with ADPKD? | ||
| How common is it for people with ADPKD to have blood in their urine and how should this be assessed and treated according to severity and complications? | ||
| For people receiving medicines to reduce the risk of a blood clot during dialysis or transplantation, what is the risk of having blood in the urine and what problems can this cause? | ||
| How can other measures of kidney structure and function be used alongside commonly used measures such as kidney length or total kidney volume to monitor disease progression in people without symptoms or with early stage ADPKD? | ||
| Kidney failure | Are people with ADPKD and kidney failure who are receiving peritoneal dialysis more likely to suffer complications or failure of this treatment than people without ADPKD? | |
| What are the risks of complications of kidney transplantation in people with ADPKD (during transplantation and in the long term) compared with patients without ADPKD? | ||
| What proportion of people with ADPKD and kidney failure develop kidney cancer, and what tests and care should patients with signs of kidney cancer receive? | ||
| Practical integrated patient support | When people are newly diagnosed with ADPKD, how does this affect them psychologically and what impact does it have on their life? What information and support would help people at this time? | ✓ |
| What lifestyle, exercise and/or dietary changes (including amount of water drunk) are beneficial for people with ADPKD and PLD? | ✓ | |
| How should the care of people with ADPKD be organised to improve their outcomes (ie, reduce illness and death)? How can we ensure that this care includes appropriate access to consistent, specialist care provided by a team of experts? | ✓ | |
| What information resources are required to support family planning for people with ADPKD? | ||
| How does social inequality affect long-term outcomes in people with ADPKD? | ||
| When researching ADPKD, including new treatments, what measurements such as kidney size, kidney function, quality of life and other side effects reported by patients themselves can be used? | ||
| How can healthcare practitioners recognise the need for and provide a holistic approach to the care of people with ADPKD and family members? | ||
| Monitoring kidney disease | Can assessment of the way the blood is flowing through the kidney be used to see how much a person’s ADPKD has progressed (ie, how much damage it has caused to the kidney)? | |
| How useful is assessment of glomerular filtration rate (as a way to measure kidney function) in the early stages of ADPKD? | ||
| Is measuring levels of protein in a person’s urine helpful for assessing how far their ADPKD has progressed? |
*The questions are group by theme for ease of reference only. Indicative questions were presented in a random order in the survey.
ADPKD, autosomal dominant polycystic kidney disease.