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

Purpose: Promising developments in the search for effective pharmacotherapies for autosomal-dominant polycystic kidney disease (ADPKD) are reviewed.

Summary: 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.