Nuove opzioni terapeutiche per la malattia renale policistica

Autosomal dominant polycystic kidney disease (ADPKD) is the most common renal genetic disorder being responsible for ff10% of cases of end-stage renal disease. At present, ADPKD therapy is essentially supportive, limited to blood pressure reduction and to symptomatic treatment of disease complications. However, recent findings on the pathophysiology of the disease have stimulated the research on new therapeutic strategies in an attempt to stop ADPKD progression. Mammalian target of rapamycin (mTOR) inhibitors, sirolimus and everolimus, seem to have some effects in ADPKD patients, but the therapeutic role of these drugs remains uncertain. The main candidates for the treatment of renal and hepatic cysts are the somatostatin analogues lanreotide and octreotide. The most promising therapy for the treatment of progressive ADPKD is the Vasopressin receptor (V2) antagonist tolvaptan. Other therapeutic strategies are currently under investigation but data are still not sufficient to establish if these approaches may provide consistent benefits in decelerating the progression of ADPKD in the next future.