Recent advances in management of autosomal-dominant polycystic kidney disease.

Abstract

Promising developments in the search for effective pharmacotherapies for autosomal-dominant polycystic kidney disease (ADPKD) are reviewed. The formation and development of cysts characteristic of ADPKD result in inexorable renal and extrarenal manifestations that give rise to more rapid disease progression and more widespread complications than are seen with other forms of chronic kidney disease. To date, no agent has gained Food and Drug Administration marketing approval for use in patients with ADPKD, complicating efforts to meet the medical needs of this population. Although definitive ultrasonographic diagnostic strategies are available, molecular screening approaches lack sufficient evidence and patient outcomes data to support broad clinical application. Recently completed and ongoing clinical trials point to a number of encouraging platforms for evidence-based ADPKD management. Tolvaptan therapy significantly improved cyst burden and slowed disease progression among patients with early-stage ADPKD in a large-scale trial, while somatostatin therapies may also be useful in halting disease progression and managing comorbid polycystic liver disease. Stem cell research and nanomedicine might represent novel approaches to gaining comprehensive insights on ADPKD and, ultimately, to targeting the disease's origins, thereby making restoration of kidney function possible. A number of pharmacotherapy approaches to ADPKD management show promise but are unlikely to be curative, fueling interest among researchers in finding new applications for nanomedicine and stem cell technologies that can slow ADPKD progression and better control complications of the disease.