Autosomal dominant polycystic kidney disease (ADPKD) – targets of pharmacotherapy and extrarenal complications
Journal Title: Postępy Nauk Medycznych - Year 2015, Vol 28, Issue 10
Abstract
Autosomal dominant polycystic kidney disease (ADPKD) is hereditary cystic disorderwith predominantly renal manifestation (large kidneys, flank pain, haematuria, hypertensionand renal insufficiency). Some patients also present extrarenal symptoms, includinghepatic cysts, cerebral and aortic aneurysms and colonic diverticula. The underlying geneticbackground of ADPKD is related to mutation of the PKD1 and PKD2 genes, codingspecific protein products, known as polycystin 1 and 2. Renal failure develops mainlyabout the 6th decade of life. Apart from classic, symptomatic treatment of chronic kidneydisease, several specific therapies, aimed to several pathways of disease mechanism,have been conducted in clinical trials, including use of ACE, mTOR, V2 receptor and cAMPinhibitors. None of those was universally effective in terms of complete stopping cystsgrowth and slowing deterioration of renal function or appeared to be not widely acceptable,due to high incidence of specific adverse events. Further investigation is required fordeveloping effective and acceptable and friendly specific therapies of ADPKD.
Authors and Affiliations
Ryszard Grenda
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