Statin Therapy in Patients with Early-Stage ADPKD

Autosomal dominant polycystic kidney ADPKD is the most common monogenic renal disorder accounting 8-10 percent of patients receiving renal replacement therapy for renal failure worldwide. At present tolvaptan Jynarque is the only FDA approved therapy to slow progression of renal disease in the U.S. However, high cost and side effects limit drug prescription. This underlines the need for alternative well-tolerated low cost therapies for ADPKD. Our group has previously shown that pravastatin therapy in children and young adults with ADPKD slowed the increase in total kidney volume due to cyst expansion. The primary goal of the current proposal is to test the efficacy of pravastatin therapy in decreasing renal cyst growth and improving renal function in adults with ADPKD. A secondary goal is to obtain initial insight into the mechanisms by which statins may improve kidney function and structure.