Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disease worldwide. It is a chronic, progressive condition characterised by the development and growth of cysts in the kidneys and other organs, notably in the liver in most patients and, less commonly in the seminal vesicles, pancreas, arachnoid membrane and spinal meninges.

ADPKD patients have a higher risk of cardiovascular and cerebrovascular complications, including hypertension, cardiac valvular abnormalities and intracranial aneurysms. Two in three ADPKD patients will experience pain - both acute and chronic - in their lifetime. Pain relief is often inadequate. Kidney and liver cystic complications such as ruptures and infections are frequent and may need surgical intervention if antibiotics are not effective.

Most ADPKD patients develop end-stage renal disease (ESRD), usually before the age of 60 years. ADPKD accounts for approximately one in ten of patients requiring renal replacement therapy (RRT) by kidney transplantation or dialysis. (Data: Renal Registry 31.12.2015). Some patients will require nephrectomies before or after transplant, owing to size and cyst burden. Approximately 40* patients a year will require liver transplants owing to massive bulk, pain and severely impaired quality of life (QoL). Lifelong physical and psychological effects of ADPKD can harm overall QoL and affect the ability to work and function socially.

Until recently, ADPKD was considered a disease of adulthood owing to the late onset of most symptoms. However, studies of children diagnosed with ADPKD reveal that one in three has hypertension.

* NHSBT data for 2018 shows 23 liver transplants for polycystic liver disease although numbers are expected to rise due to new allocation policy for deceased liver donation.