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In this trial, patients with autosomal dominant polycystic kidney disease were randomly assigned to tolvaptan, a vasopressin V
2
-receptor antagonist, or placebo. Over 3 years, the increase in total kidney volume in the tolvaptan group was half that in the placebo group.
Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic kidney disease and the fourth leading cause of end-stage kidney disease in adults worldwide.
1
,
2
It results in the progressive development of kidney cysts, kidney pain, hypertension, and, ultimately, kidney failure. Effective treatment for ADPKD has been lacking.
Studies of animal models implicate the antidiuretic hormone arginine vasopressin and its second messenger adenosine-3′,5′-cyclic monophosphate (cAMP) as promoters of kidney-cyst cell proliferation and luminal fluid secretion. The suppression of vasopressin release by means of high water intake, genetic elimination of vasopressin, and vasopressin V
2
-receptor blockade all reduce the . . .