Andreas L Serra, Diane Poster, Andreas D Kistler, Fabienne Krauer, Shagun Raina, James Young, Katharina M Rentsch, Katharina S Spanaus, Oliver Senn, Paulus Kristanto, Hans Scheffel, Dominik Weishaupt, Rudolf P Wüthrich
BACKGROUND: In autosomal dominant polycystic kidney disease (ADPKD), aberrant activation of the mammalian target of rapamycin (mTOR) pathway is associated with progressive kidney enlargement. The drug sirolimus suppresses mTOR signaling. METHODS: In this 18-month, open-label, randomized, controlled trial, we sought to determine whether sirolimus halts the growth in kidney volume among patients with ADPKD. We randomly assigned 100 patients between the ages of 18 and 40 years to receive either sirolimus (target dose, 2 mg daily) or standard care...
August 26, 2010: New England Journal of Medicine