• Liver
• Drug Development

Liver cysts are a frequent finding on radiological imaging. Prevalence rates depend on the technique, but with CT or MRI, simple hepatic cysts are seen in approximately one-fifth of the population.1 Usually no more than one or two small (<3 cm) cysts are present. These cysts are innocuous and do not require immediate clinical attention. The situation is different when there are multiple and/or large liver cysts such as in polycystic liver disease (PLD). Though PLD is infrequent, patients are seen in every gastroenterology practice. Many patients give a positive family history of PLD. The majority of PLD patients will also have multiple renal cysts and suffer from autosomal dominant polycystic kidney disease (ADPKD). This is a progressive disorder that leads to end-stage renal disease. By contrast, some PLD patients do not have that renal phenotype and they are affected by isolated autosomal dominant polycystic liver disease (PCLD). While the genetic background of ADPKD and PCLD is dissimilar, there is great analogy in their phenotypical behaviour. PLD is characterised by an age-dependent increase of number and size of the liver cysts.1 Within this context, the physiological function of the liver is usually unaffected, but because of the unrelenting growth, significant hepatomegaly develops over time. This is associated with onset of symptoms, such as abdominal pain, early satiety, and dyspnoea which causes a compromised quality of life.2 Thus far, the primary endpoint of treatment …