Everolimus in Patients with Autosomal Dominant Polycystic Kidney Disease

In this 2-year, double-blind trial, patients with ADPKD were randomly assigned to receive either placebo or the mTOR inhibitor everolimus, since the mTOR pathway is important in cyst growth. Although everolimus slowed the increase in kidney volume, as assessed by means of magnetic resonance imaging, it did not slow the progression of renal impairment. Autosomal dominant polycystic kidney disease (ADPKD) affects approximately 1 of every 1000 persons in the general population 1 and develops, by means of slowly progressive renal-cyst growth, to end-stage renal disease in over 50% of patients. Hepatic and pancreatic cysts, as well as cerebral and abdominal aneurysms, contribute to ADPKD-associated morbidity and mortality. Arterial hypertension, recurrent urinary tract infection, nephrolithiasis, and abdominal pain are frequently the presenting symptoms. 2 Approximately 85% of patients with ADPKD have mutations in the polycystic kidney disease 1 gene ( PKD1 ), whereas most of the remaining 15% have polycystic kidney disease 2 gene ( PKD2 ) . . .