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Sirolimus and Kidney Growth in Autosomal Dominant Polycystic Kidney Disease
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Sirolimus and Kidney Growth in Autosomal Dominant Polycystic Kidney Disease
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Journal Article
Sirolimus and Kidney Growth in Autosomal Dominant Polycystic Kidney Disease
2010
Overview
In autosomal dominant polycystic kidney disease (ADPKD), aberrant activation of the mammalian target of rapamycin (mTOR) pathway is associated with progressive kidney enlargement. Sirolimus (rapamycin) suppresses mTOR signaling and was studied in this 18-month open-label, randomized, controlled trial involving adults with ADPKD and early chronic kidney disease. Sirolimus at a daily target dose of 2 mg did not halt polycystic kidney growth.
Autosomal dominant polycystic kidney disease (ADPKD) is the most frequent hereditary kidney disease and the cause of end-stage renal disease in 7 to 10% of all patients undergoing dialysis.
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–
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The disease is characterized by the growth of numerous kidney cysts, which leads to progressive destruction of the adjacent renal parenchyma and massive enlargement of the kidneys.
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Renal function is often preserved until the age of 40 years because functioning nephrons undergo compensatory hypertrophy.
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Subsequently, the glomerular filtration rate (GFR) decreases, and end-stage renal disease ensues in many patients by the fifth decade. As yet, no treatment is available to . . .
Publisher
Massachusetts Medical Society
Subject
/ Antibiotics. Antiinfectious agents. Antiparasitic agents
/ Biological and medical sciences
/ Cysts
/ Female
/ Humans
/ Intracellular Signaling Peptides and Proteins - antagonists & inhibitors
/ Kidney Failure, Chronic - prevention & control
/ Kidneys
/ Male
/ Malformations of the urinary system
/ Nephrology. Urinary tract diseases
/ NMR
/ Pharmacology. Drug treatments
/ Polycystic Kidney, Autosomal Dominant - drug therapy
/ Polycystic Kidney, Autosomal Dominant - pathology
/ Polycystic Kidney, Autosomal Dominant - physiopathology
/ Protein-Serine-Threonine Kinases - antagonists & inhibitors
