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Permanent URL to this publication: http://dx.doi.org/10.5167/uzh-24522

Serra, A L; Kistler, A D; Poster, D; Krauer, F; Senn, O; Raina, S; Pavik, I; Rentsch, K; Regeniter, A; Weishaupt, D; Wüthrich, R P (2009). Safety and tolerability of sirolimus treatment in patients with autosomal dominant polycystic kidney disease. Nephrology, Dialysis, Transplantation, 24(11):3334-3342.

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BACKGROUND: We initiated a randomized controlled clinical trial to assess the effect of sirolimus on disease progression in patients affected by autosomal dominant polycystic kidney disease (ADPKD). Here we report the preliminary safety results of the first 6 months of treatment. METHOD: A total of 25 patients were randomized to sirolimus 2 mg/day and 25 patients to no treatment except standard care. Treatment adherence was monitored electronically. At baseline and at Month 6, laboratory parameters were analysed and the urinary protein profile in 24-h urine collections was determined. RESULTS: Both treatment groups were well balanced for age, sex and renal function. In 94.1 +/- 11.4% of the study days, patients in the sirolimus group were exposed to the drug when assuming a therapeutic efficacy duration of 30 h. At Month 6, the mean sirolimus dose and trough level were 1.28 +/- 0.71 mg/day and 3.8 +/- 1.9 microg/l, respectively. Glomerular (albumin, transferrin, IgG) and tubular (retinol-binding protein, alpha(1)-microglobulin) protein excretion remained unchanged. Glomerular filtration rate also did not change significantly. Haematological parameters were similar in both groups, except for a mild reduction of the mean corpuscular volume of erythrocytes in patients receiving sirolimus. Lipid levels were similar in both groups. Adverse events were transient and mild, and no grade 3 or 4 events occurred. The incidence of infections was similar in the sirolimus group (80%) and the standard group (88%). The most common gastrointestinal adverse events were mucositis (72% in the sirolimus group versus 16% in the standard group, P = 0.0001) and diarrhoea (36% in the sirolimus versus 20% in the standard group, P = 0.345). CONCLUSION: Treatment of ADPKD patients with sirolimus with a dose of 1-2 mg/day is safe and does not cause proteinuria or impairment of GFR. Treatment adherence was excellent. (ClinicalTrials.gov number, NCT00346918.).


13 citations in Web of Science®
15 citations in Scopus®
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Additional indexing

Item Type:Journal Article, refereed, original work
Communities & Collections:04 Faculty of Medicine > University Hospital Zurich > Clinic for Nephrology
04 Faculty of Medicine > University Hospital Zurich > Institute of Clinical Chemistry
04 Faculty of Medicine > University Hospital Zurich > Institute of General Practice
Dewey Decimal Classification:610 Medicine & health
540 Chemistry
Deposited On:30 Nov 2009 16:12
Last Modified:05 Apr 2016 13:34
Publisher:Oxford University Press
Publisher DOI:10.1093/ndt/gfp280
PubMed ID:19525519

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