• Roser Torra.
• Enfermedades Renales Hereditarias, Fundación Puigvert, Institut d'Investigació Biomèdica Sant Pau, Universidad Autónoma de Barcelona, Barcelona, España. Electronic address: rtorra@fundacio-puigvert.es.
• Med Clin (Barc). 2014 Jan 21; 142 (2): 73-9.

AbstractAutosomal dominant polycystic kidney disease is the most frequent hereditary kidney disease. However it lacks a specific treatment. Its prevalence is 1/800 and causes the need for renal replacement therapy in 8-10% of patients on dialysis or kidney transplant. It is caused by mutations in the PKD1 and PKD2 genes, which cause a series of alterations in the polycystic cells, which have become therapeutic targets. There are many molecules that are being tested to counteract the alterations of these therapeutic targets. There are studies in all phases of research, from phase i to phase iv. Some of the molecules being tested are tolvaptan, mTOR inhibitors and, among many other, somatostatin analogues. These drugs are extensively reviewed in this article. Based on the accumulated experience the primary objective of the trials is the slowing of the increase in renal volume. Yet other renal end points such as renal function and hypertension are necessary. It is expected that in the coming years we can have specific, well tolerated, effective and affordable drugs for the treatment of autosomal dominant polycystic kidney disease. Copyright © 2013 Elsevier España, S.L. All rights reserved.

45 keywords...

hide…