REPRISE
Trial question
What is the effect of tolvaptan in patients with later-stage autosomal dominant polycystic kidney disease?
Study design
Multi-center
Double blinded
RCT
Population
Characteristics of study participants
50.0% female
50.0% male
N = 1370
1370 patients (690 female, 680 male)
Inclusion criteria: patients with later-stage autosomal dominant polycystic kidney disease
Key exclusion criteria: pregnancy or lactation; need for chronic diuretic use; hepatic impairment or liver function abnormalities other than that expected for ADPKD; advanced diabetes; contraindications to required trial assessments
Interventions
N=683 tolvaptan (daily morning and after doses of 90 mg and 30 mg; 60 mg and 30 mg; or 45 mg and 15 mg, or less for 12 months)
N=687 placebo (mock morning and afternoon doses of 90 mg and 30 mg; 60 mg and 30 mg; or 45 mg and 15 mg, or less for 12 months)
Primary outcome
Decline in estimated GFR at 1 year
-2.34
-3.61
0.0
-0.9
-1.8
-2.7
-3.6
Tolvaptan
Placebo
Significant
increase ▲
Significant increase in decline in estimated GFR at 1 year (-2.34 vs. -3.61 ; difference 1.27, 95% CI 0.86 to 1.68)
Secondary outcomes
Significant increase in slopes of the change in the estimated GFR at 1 year (-3.16 vs. -4.17 ; difference 1.01, 95% CI 0.62 to 1.4)
Safety outcomes
No significant difference in the rates of new or worsening adverse events.
Conclusion
In patients with later-stage autosomal dominant polycystic kidney disease, tolvaptan was superior to placebo with respect to decline in estimated GFR at 1 year.
Reference
Torres VE, Chapman AB, Devuyst O et al. Tolvaptan in Later-Stage Autosomal Dominant Polycystic Kidney Disease. N Engl J Med. 2017 Nov 16;377(20):1930-1942.
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