A randomized controlled trial evaluated the effect of pravastatin on kidney disease outcomes in adult patients with early-stage autosomal dominant polycystic kidney disease.

INTRODUCTION Whether treatment with pravastatin mitigates kidney dysfunction in adult patients with early autosomal dominant polycystic kidney disease (ADPKD) is unknown. To answer this, we performed a prospective, randomized, controlled, double-blind clinical trial in 150 patients (52 males and 98 females; 25-60 years) with ADPKD, and an estimated glomerular filtration rate (eGFR) of 60 ml/min or more per 1.73m2. METHODS Patients were randomly assigned to receive 40 mg of pravastatin or a placebo for two years. The primary outcome was the annual change in height-adjusted total kidney volume (HtTKV). Secondary outcomes were the annual change in kidney blood flow by magnetic resonance angiography and the annual change in measured GFR (mGFR). RESULTS Among the participants who underwent randomization, the annual rate of increase in HtTKV median (interquartile range) was: 3.1% (1.4, 6.8) in the placebo and the pravastatin 4.3% (3.0, 6.6) group. The annual rate of decline in kidney blood flow (ml/min per 1.73m2) was -32.7 (-62.1, -0.8) in placebo vs. -15.1 (-50.7, 14.4) in the pravastatin group. The median change in mGFR (ml/min per 1.73m2) -2.3 (-5.1, 1.6) for placebo and -1.4 (-6.4, 2.0) for the pravastatin group. Overall, no parameter differed significantly between treatment groups. There was a similar trend in patients with faster disease progression (Mayo Imaging Class 1C, D, and E). CONCLUSIONS Among adult patients with ADPKD and preserved kidney function, pravastatin did not slow the increase in HtTKV and had no effect on slowing the decline in kidney blood flow and kidney function compared to placebo.