Autosomal Dominant Polycystic Kidney Disease Top 10 (priority setting in association with the JLA)

  1. What treatments can be developed that slow or prevent progression of ADPKD and improve patients’ quality of life?
  2. Which people with ADPKD would benefit from early treatment and how can doctors identify them?
  3. What are the best ways to organise the care of people with ADPKD to improve their outcomes?
  4. What effect does pregnancy have on women with ADPKD including their pregnancy health, kidney function, and liver cysts?
  5. What are the benefits and harms of drugs that can be used for the management of ADPKD including polycystic liver disease (PLD)?
  6. For people with ADPKD experiencing pain, what treatments (such as treatments for cyst infections, draining fluid from cysts or removing nerves) work best to reduce this pain?
  7. What changes to lifestyle, exercise and/or diet (including amount of water drunk) benefit people with ADPKD and polycystic liver disease (PLD)?
  8. 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?
  9. 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?
  10. What causes enlarged blood vessels (aneurysms) in some people with ADPKD and what is the most effective way to screen for and treat aneurysms?

The following questions were also discussed and put in order of priority at the final workshop

  1. What symptoms are associated with cyst infection in people with ADPKD, and how are cyst infections best managed (investigated and treated)?
  2. What causes severe (acute) and long-term (chronic) kidney pain in people with ADPKD?
  3. What are the most effective treatments for high blood pressure (hypertension) for people (children and adults) with ADPKD?
  4. In which circumstances should removal of a kidney (known as nephrectomy) be considered in people with ADPKD, and are there alternative treatments?
  5. 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)?
  6. 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?
  7. Why do the symptoms and severity of PLD vary between people?

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    For full details of all of the questions identified by this PSP, please see the document below.