Study co-authors include Norberto Perico, MD, Luca Antiga, PhD, Anna Caroli, Nadia Rubis, Olimpia Diadei, Giulia Gherardi, Silvia Prandini, Andrea Panozo, MD, Rodolfo Flores Bravo, MD, Sergio Carminati, Felipe Rodriguez De Leon, MD, Flavio Gaspari, Monica Cortinovis, Nicola Motterlini, Bogdan Ene-Iordache (Mario Negri Institute for Pharmacological Research, in Bergamo, Italy); Piero Ruggenenti, MD (Mario Negri Institute for Pharmacological Research and Azienda Ospedaliera Ospedali Riuniti Bergamo); Giorgio Fasolini, MD, Mariateresa Cafaro, MD, Patrizia Ondei, MD (Azienda Ospedaliera Ospedali Riuniti Bergamo); and Andrea Remuzzi (Mario Negri Institute for Pharmacological Research and the University of Bergamo).

In reviewing the results of Dr. Remuzzi's study in an accompanying editorial, Robert Schrier, MD (University of Colorado Denver) stated that they give hope for the treatment of patients with ADPKD. He noted, however, that six months of sirolimus treatment was associated with mildly abnormal liver function tests, as well as increased cholesterol, decreased red blood cells, and increased protein excretion in the urine. "This is potentially bothersome regarding possible lifelong therapy for ADPKD," he wrote. "Thus, results of larger and longer follow-up studies in ADPKD patients with mTOR inhibitors will be very important."

SOURCE Journal of the American Society Nephrology

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