Sirolimus therapy to halt the progression of ADPKD

N Perico, L Antiga, A Caroli, P Ruggenenti… - Journal of the …, 2010 - journals.lww.com
Activation of mammalian target of rapamycin (mTOR) pathways may contribute to
uncontrolled cell proliferation and secondary cyst growth in patients with autosomal
dominant polycystic kidney disease (ADPKD). To assess the effects of mTOR inhibition on
disease progression, we performed a randomized, crossover study (The SIRENA Study)
comparing a 6-month treatment with sirolimus or conventional therapy alone on the growth
of kidney volume and its compartments in 21 patients with ADPKD and GFR≥ 40 ml/min per …