Practical approach to patient therapy affected by Autosomal Dominant Autosomic Polycystic Kidney Disease


The Autosomal Dominant Polycystic Kidney Disease(ADPKD) is the most frequent renal genetic condition and involves 7 to 10% of subjects undergoing renal replacement therapy. It is estimated that between 24,000 and 34,000 subjects in Italy are affected by this condition. For an illness that has long been neglected due to a lack of treatment options, an attractive treatment possibility is now available: tolvaptan has shown clinical efficacy regarding disease progression in two clinical trials (ADPKD patients with mild renal failure and ADPKD patients with advanced renal failure). The possible liver toxicity expressed in about 4% of the subjects exposed to the drug and an important aquaretic effect suggest prudence and attention in the use of this new molecule. Based on these critical points, some clinicians with direct experience in the use of the drug have briefly collected in the pages to follow the main clinical recommendations for the treatment of ADPKD patients. The recommendations concern the general approach to the patient affected by ADPKD but with particular attention to the aspects related to the new treatment. The delicate task of introducing the opportunities and limitations of the offered therapy to the patient will be deepened. Finally, the document wants to suggest how best to organize a clinic dedicated to this condition.

Keywords: Autosomal Dominant Polycystic Kidney Disease, Renal failure, Cyst, Aneurysm, tolvaptan

Sorry, this entry is only available in Italian.


Riccardo Magistroni


Il Rene Policistico Autosomico Dominante (ADPKD) è la più frequente patologia genetica di interesse nefrologico e coinvolge dal 7 al 10% dei soggetti in trattamento sostitutivo renale (13).


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