Key result
Voclosporin reduces UPCR ~61% vs vehicle in a rat model of nephrotic syndrome.
Why the study?
In nephrotic syndrome, the HDL2/HDL3 ratio is low despite normal total HDL concentration, and the authors hypothesized this is caused by urinary losses of lecithin-cholesterol acyltransferase leading to plasma deficiency.
Mean Difference: -6
Absolute Event Rate: 3.8% vs 9.8%
p-value: p=0.0374
Does not support clinical use in nephrotic syndrome; leaves open translation of antiproteinuric effects to humans.
Lecithin-cholesterol acetyltransferase (LCAT) is involved in the synthesis of plasma cholesteryl esters and is pivotal in the maturation of plasma high-density lipoprotein (HDL) and conversion of HDL3 to HDL2. In nephrotic syndrome (NS), the ratio of HDL2 to HDL3 is low even though the total concentration of HDL is generally normal. We hypothesize that the reduced HDL2/HDL3 ratio in NS is due to urinary losses of LCAT, leading to plasma LCAT deficiency. To test this hypothesis, Sprague-Dawley rats were randomized to NS (given 130 mg puromycin aminonucleoside on day 1 and 60 mg ip on day 14) or control groups and were studied on day 30. To dissect the effect of proteinuria from hypoalbuminemia, a group of Nagase rats with inherited hypoalbuminemia was included. Hepatic LCAT and glyceraldehyde-3-phosphate dehydrogenase (GAPDH) mRNA abundance and plasma and urine LCAT activity were measured. The NS group showed a fourfold rise in serum cholesterol and triglycerides, a fivefold rise in free cholesterol, and a fourfold fall in the HDL-to-total cholesterol ratio. Despite severe hypoalbuminemia, the Nagase rats showed only a mild elevation of serum cholesterol and triglycerides with a normal serum free cholesterol and HDL-to-total cholesterol ratio. The NS group exhibited a normal hepatic LCAT-to-GAPDH mRNA ratio, a marked reduction in plasma LCAT activity, and a significant increase in urinary LCAT excretion. LCAT/GAPDH mRNA and plasma and urine LCAT were normal in Nagase rats. Thus NS led to heavy urinary losses and reduced plasma concentration of LCAT, despite normal hepatic LCAT mRNA abundance. However, hypoalbuminemia, per se, without proteinuria as seen in the Nagase rats had no effect on plasma LCAT or the HDL-to-total cholesterol ratio. Therefore, proteinuria, not hypoalbuminemia, causes LCAT deficiency and a depressed HDL-to-total cholesterol ratio in NS.
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Vaziri et al. (2001) studied Puromycin aminonucleoside-induced nephrotic syndrome (PAN-NS) (n=28). Voclosporin (VCS) vs. Vehicle (vitamin E-TPGS, MCT oil, Tween 40, and 95% ethanol) was evaluated on Day 11 Urinary Protein-to-Creatinine Ratio (UPCR, mg/mg Cr) (MD -6.0 mg/mg Cr (61% reduction in median UPCR), p=0.0374). Voclosporin reduced Day 11 median urinary protein-to-creatinine ratio by 61% compared to vehicle (3.8 vs. 9.8 mg/mg Cr, P=0.0374) in rats with PAN-induced nephrotic syndrome.
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