Autosomal Dominant Polycystic Kidney Disease (ADPKD) Clinical Trials: A Critical Appraisal

Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic hereditary kidney disease in humans, with a prevalence of 1 out of every 800–1,000 individuals, and is the cause of end-stage renal disease (ESRD) in 5–10 % of the prevalent patients on renal replacement therapy (RRT) worldwide [1]. The disease is characterized by the development, growth, and expansion of multiple renal cysts, leading to destruction of normal renal parenchyma, massively enlarged kidneys, and subsequent kidney function loss [2–4]. The natural course of ADPKD is often of progressive nature, eventually leading to ESRD in approximately 50 % of patients afflicted.