Among the diseases that require renal replacement therapy (RRT), ADPKD is the fourth for incidence and prevalence. In Italy, there are at least 32,000 patients affected by ADPKD, of which about 2900 in dialysis. The pure costs of dialysis treatment for the Italian National Health Service can be conservatively estimated at 87 million euros per year. Even a modest slowdown in the evolution of the disease would obtain an important result in terms of reduction of health expenditure. In recent years, many new or repurposed drugs have been evaluated in clinical trials for ADPKD. In this review we will mainly focus on advanced stage clinical trials (phase 2 and 3). We have grouped these studies according to the molecular pathway addressed by the experimental drug or the therapeutic strategy. More than 10 years after the start of the first Phase III clinical trials in ADPKD, the first drug active in slowing disease progression is finally available. It cannot be considered a goal but only the beginning of a journey because of the significant side effects and the high cost of Tolvaptan. An exuberant basic research activity in the field, together with the large number of ongoing protocols, keep the nephrologists and their patients positive with regard to the discovery of new and better therapies in a not-too-distant future.
Keywords: ADPKD; Clinical trial; Renal volume; Somatostatin; Tolvaptan.