Thursday, July 15, 2010


Deficiency of Factor H can lead to two diseases that we can think of : Dense Deposit Disease and Atypical HUS.
As Nephrologists, we share a lot with the immunology and the hematology world and have to know basics of those disclipines to understand certain diseases.
Factor H is a regulator of the alternative pathway.  If deficient, one can have uncontrolled C3 activation and leading to secondary C3 deficiency and C3 deposition in the kidney as well. When Factor H knockout mice were developed, they got secondary C3 deficiency, and a renal disease that showed massive accumulation of C3 along the glomerular basement membrane.  A recent mice study in Kidney international( link below) showed that when these knockout mice were given infusion of factor H, there was rapid normalization of the C3 levels in the plasma and resolution of the renal disease.  
Interestingly, they couldn't treat the mice long term as they developed an immune response to the factor H itself.  
This is a nice study that showed a proof of concept of replacing just Factor H in these patients.
Take a look at the Pediatric Nephrology Blog regarding another review on current available therapies and all updates from Dr.Sethi( HUS expert)


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