Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney
disease, affecting 1 in 400 to 1000 individuals and accounting for 4% of end-stage renal
disease in the United States and 8-10% in Europe. The condition is characterized by
progressive development of kidney cysts with kidney enlargement and associated loss of kidney
function. High blood pressure and cardiovascular disease are common in patients with ADPKD.
Although the condition is often thought to affect primarily adults, it is clear that the
disease can be present in the fetus and young children.

This study was designed to determine if treatment with the medicine pravastatin can slow the
progression of kidney and heart disease when initiated early in life in patients with ADPKD.
The Investigators will assess differences between pravastatin and placebo study groups over
the three-year study period with respect to: 1) total kidney volume as assessed by magnetic
resonance imaging (MRI); 2) left ventricular mass index as assessed by MRI; 3) urinary
albumin excretion; and 4) endothelial-dependent vasodilation as assessed by brachial
ultrasound. A total of 110 subjects were enrolled in this research study. This study involved
pediatric subjects because the Investigators believe that early intervention is critical if
they are to decrease the morbidity and mortality associated with this condition. If
pravastatin is shown to be effective in ameliorating progression of renal and cardiovascular
disease in this study, routine management of people with this condition will be drastically
altered.

Inclusion Criteria:

- Age 8-22 years

- Autosomal dominant polycystic kidney disease

- Normal kidney function

Exclusion Criteria:

- Abnormal kidney function

- Past allergic history to medications used in study

- Liver disease

- Muscle disease/dystrophy

- Pregnancy, planned pregnancy, or lactation within study period

- Inability to cooperate with or clinical contraindication for magnetic resonance
imaging