Tolvaptan in Later-Stage Autosomal Dominant Polycystic Kidney Disease: An Analysis of the REPRISE Trial

Abstract

In the past, Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease (ADPKD) and
its Outcomes (TEMPO) trial showed Tolvaptan, a vasopressin V2-receptor antagonist, slowed the decline in the estimated
Glomerular Filtration Rate (eGFR) in patients with estimated creatinine clearance of ≥60 ml per minute.
With this in consideration, Replicating Evidence of Preserved Renal Function: Investigation of Tolvaptan Safety and Efficacy
(REPRISE) trial was conducted to assess the efficacy and safety of Tolvaptan in patients with later stage ADPKD. In this trial,
1370 patients with later-stage ADPKD were randomized. These patients were either 18 to 55-years-old with eGFR of 25 to 65
mL per minute per 1.73 m2
of body surface area or 56 to 65-years-old with eGFR of 25 to 44 mL per minute per 1.73 m2
. The
primary end-point of this trial was the change in eGFR between baseline and end of trial period, with adjustments made for
exact duration that each patient participated for, interpolated to 1 year.
The change from baseline that was noted in the Tolvaptan group was -2.34 mL per minute per 1.73 m2 vs. -3.61 mL per minute
per 1.73 m2
in the placebo group. A difference of 1.27 mL per minute per 1.73 m2
(95% CI, p<0.001) was noted.
Based on the data collected by the REPRISE trial over a 1-year period, it can be concluded that Tolvaptan slows the decline in
eGFR than placebo in patients with later stage ADPKD.