Clinical Proof-Of-Concept Trial To Assess The Therapeutic Effect Of Sirolimus In Patients With Autosomal Dominant Polycystic Kidney Disease: SUISSE ADPKD Study

Andreas L Serra; Andreas D Kistler; Diane Poster; Marian Struker; Rudolf P Wüthrich; Dominik Weishaupt; Frank Tschirch

Disclosures

BMC Nephrology 

In This Article

Discussion

The SUISSE ADPKD study seeks to determine if sirolimus halts kidney volume growth in patients with ADPKD early in the disease course. Study participation is restricted to young patients with preserved kidney function because any effective treatment of ADPKD needs to be started early in the course of the disease to have an impact on long term kidney function. Our trial is the first clinical study addressing this question. If sirolimus treatment can reduce or stop volume growth in patients with maintained kidney function and prior documented kidney volume progression, these ADPKD patients could benefit the most from a treatment with an anti-proliferative agent like sirolimus. Similar studies using specific mTOR inhibitors to halt disease progression have been announced by others (Mayo Clinic, USA; Certican® trial, Novartis, Germany). We anticipate that sirolimus might be an effective therapeutic option for ADPKD patients that are prone to progress to end-stage renal disease.

Pre-publication History

The pre-publication history for this paper can be accessed here: https://www.biomedcentral.com/1471-2369/8/13/prepub

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