Somatostatin analogues for treatment of polycystic liver disease

The present review summarizes the existing knowledge on polycystic liver disease (PCLD) and highlights the progress made in medical treatment for this condition in the past year.

PCLD is associated with autosomal dominant polycystic kidney disease (ADPKD) and autosomal dominant PCLD. Signaling pathways of adenosine 3',5'-cyclic monophosphate (cAMP) and mammalian target of rapamycin (mTOR) are aberrantly regulated in polycystic livers and promote hepatic cystogenesis. Somatostatin analogues reduce intracellular cAMP, and this might prevent fluid accumulation in hepatic cysts. Several clinical trials published over the last year now show that somatostatin analogues when given for 6-12 months in patients with ADPKD and PCLD decrease total liver volume, attenuate polycystic kidney volume, and improve perception of health. In two recent studies mTOR inhibitors failed to halt the progression of ADPKD. It is still too early to recommend to start somatostatin analogues in PCLD and definitive answers should come from future clinical trials.

Somatostatin analogues are promising new medical drug options in the treatment of PCLD. However, more needs to be elucidated with regard to molecular mechanisms in hepatic cystogenesis, the uncertainty who will respond to therapy and long-term outcomes.