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Permanent URL to this publication: http://dx.doi.org/10.5167/uzh-34854

Serra, A L; Poster, D; Kistler, A D; Krauer, F; Raina, S; Young, J; Rentsch, K M; Spanaus, K S; Senn, O; Kristanto, P; Scheffel, H; Weishaupt, D; Wüthrich, R P (2010). Sirolimus and kidney growth in autosomal dominant polycystic kidney disease. New England Journal of Medicine, 363(9):820-829.

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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. All patients had an estimated creatinine clearance of at least 70 ml per minute. Serial magnetic resonance imaging was performed to measure the volume of polycystic kidneys. The primary outcome was total kidney volume at 18 months on blinded assessment. Secondary outcomes were the glomerular filtration rate and urinary albumin excretion rate at 18 months. RESULTS: At randomization, the median total kidney volume was 907 cm(3) (interquartile range, 577 to 1330) in the sirolimus group and 1003 cm(3) (interquartile range, 574 to 1422) in the control group. The median increase over the 18-month period was 99 cm(3) (interquartile range, 43 to 173) in the sirolimus group and 97 cm(3) (interquartile range, 37 to 181) in the control group. At 18 months, the median total kidney volume in the sirolimus group was 102% of that in the control group (95% confidence interval, 99 to 105; P=0.26). The glomerular filtration rate did not differ significantly between the two groups; however, the urinary albumin excretion rate was higher in the sirolimus group. CONCLUSIONS: In adults with ADPKD and early chronic kidney disease, 18 months of treatment with sirolimus did not halt polycystic kidney growth. (ClinicalTrials.gov number, NCT00346918.)


204 citations in Web of Science®
225 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
Date:June 2010
Deposited On:15 Jul 2010 09:19
Last Modified:05 Apr 2016 14:11
Publisher:Massachusetts Medical Society
Additional Information:Comment in: N Engl J Med. 2010 Aug 26;363(9):879-81.
Publisher DOI:10.1056/NEJMoa0907419
PubMed ID:20581391

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