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Vasopressin promotes progression of autosomal dominant polycystic kidney disease (ADPKD). Previously, the vasopressin-receptor antagonist tolvaptan slowed decline in renal function in ADPKD patients with creatinine clearance ≥60 mL/minute (N Engl J Med 2012; 367: 2407). Although side effects were relatively common in that study, the drug was approved for patients with ADPKD in Europe and Canada but not in the U.S. Now, in another industry-sponsored study, 1370 ADPKD patients (age range, 18–65) with estimated glomerular filtration rates (GFRs) of 25 to 65 mL/minute/1.73 m2 were randomized to tolvaptan or placebo. During a blinded run-in phase, about 5% of potential participants were withdrawn because of drug side effects.
During 1 year of tre…