November 4, 2012

Tolvaptan in Patients with Autosomal Dominant Polycystic Kidney Disease

This long awaited clinical trial was presented on November 3, 2012 at the American Society of Nephrology Kidney Week.

This multicenter, double-blind, placebo-controlled trial enrolled patients with autosomal dominant polycystic kidney disease who were age 18 to 50 years, had total kidney volume of ≥ 750 ml and  an estimated CrCl of  ≥ 60 ml per min.  In a 2:1 ratio, patients received the V2-receptor antagonist tolvaptan or placebo.

There was a significant reduction in the primary outcome (annual rate of change in total kidney volume), with the tolvaptan group increasing at  2.8% per year versus 5.5% per year in the placebo group (P<0.001).

A secondary end point of a composite of time to clinical progression (worsening kidney function, kidney pain, hypertension, and albuminuria) was seen less often in the intervention group  with 44 vs. 50 events per 100 follow-up-years (P=0.01).

Tolvaptan was associated with a slower decline in kidney function (1/serum creatinine) at −2.61 [mg/ml]−1 per year vs. −3.81 [mg/ml]−1 per year, (P<0.001).

The tolvaptan group had higher rates of symptoms such thirst, polyuria and transaminitis which contributed to a higher discontinuation rate (23%, vs. 14% in the placebo group).  No patients in the trial reached end-stage renal disease.

See the accompanying editorial.

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