Tolvaptan for improvement of hepatic edema: A phase 3, multicenter, randomized, double-blind, placebo-controlled trial

Aim Hepatic edema is manifested by ascites, lower limb edema and intolerable symptoms. Some patients insufficiently respond to the conventional diuretic therapy. Therefore, a novel therapeutic option is required. We conducted a phase 3 study to confirm therapeutic effect of tolvaptan on hepatic edema associated with liver cirrhosis. Methods In our multicenter, randomized, double‐blind, placebo‐controlled trial, liver cirrhosis patients who showed insufficient response to conventional diuretics were randomly assigned to 7‐day administration of either tolvaptan at 7.5 mg/day or placebo as an add‐on therapy to conventional diuretics. The primary outcome was change in bodyweight from baseline. Results Of 164 eligible patients, 84 were assigned to tolvaptan and 80 to placebo. Change in bodyweight from baseline on the final dosing day was −0.44 kg (standard deviation [SD], 1.93) in the placebo group and −1.95 kg (SD, 1.77) in the tolvaptan group (P