Document Detail

Safety and tolerability of sirolimus treatment in patients with autosomal dominant polycystic kidney disease.
MedLine Citation:
PMID:  19525519     Owner:  NLM     Status:  MEDLINE    
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. ( number, NCT00346918.).
Andreas L Serra; Andreas D Kistler; Diane Poster; Fabienne Krauer; Oliver Senn; Shagun Raina; Ivana Pavik; Katharina Rentsch; Axel Regeniter; Dominik Weishaupt; Rudolf P W??thrich
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Publication Detail:
Type:  Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't     Date:  2009-06-13
Journal Detail:
Title:  Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association     Volume:  24     ISSN:  1460-2385     ISO Abbreviation:  Nephrol. Dial. Transplant.     Publication Date:  2009 Nov 
Date Detail:
Created Date:  2009-10-16     Completed Date:  2010-01-07     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  8706402     Medline TA:  Nephrol Dial Transplant     Country:  England    
Other Details:
Languages:  eng     Pagination:  3334-42     Citation Subset:  IM    
Division of Nephrology, University Hospital Z??rich, Institute of Physiology, Switzerland.
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MeSH Terms
Erythrocyte Indices / drug effects
Immunosuppressive Agents / adverse effects*
Kidney / drug effects,  physiology
Lipids / blood
Medication Adherence
Polycystic Kidney, Autosomal Dominant / drug therapy*
Sirolimus / adverse effects*,  blood
Reg. No./Substance:
0/Immunosuppressive Agents; 0/Lipids; 53123-88-9/Sirolimus

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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