Recent advances in the understanding of the molecular mechanisms underlying autosomal dominant polycystic kidney disease (ADPKD) set the stage for the development of various treatments aimed to arrest or delay disease progression. In particular, clinical trials showed that the use of somatostatin analogues in patients with ADPKD is able to slow down the increase in total kidney volume and the progressive decline in renal function over the long-term. Treatment with these agents is generally well tolerated and it also enables to control cyst growth in the liver in patients with this extra-renal manifestation of the disease. By contrast, mammalian target of rapamycin (mTOR) inhibitors, although promising in different experimental models of kidney and liver disease, in most clinical studies they were not effective in slowing cystic kidney growth and/or improving the progressive renal function decline in patients with ADPKD. Therapy with mTOR inhibitors is also limited by the elevated incidence of adverse events, which frequently requires drug withdrawal or dose reduction, eventually nullifying the potential renoprotective effect.
Full text of the article is available in Italian.