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Eur J Pediatr. 2011 Mar;170(3):389-91. doi: 10.1007/s00431-010-1278-4. Epub 2010 Sep 17.

Effect of cyclosporin A on proteinuria in the course of glomerulopathy associated with WT1 mutations.

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Department of Paediatrics and Nephrology, Medical University of Białystok, ul. Waszyngtona 17, 15-274 Białystok, Poland.


Denys-Drash syndrome (DDS) is characterized by progressive glomerulopathy caused by diffuse mesangial sclerosis (DMS), genitourinary defects, and a higher risk of developing Wilms' tumor. It is commonly assumed that the DMS is unresponsive to any medications. In this report, we present a patient with Denys-Drash syndrome, in whom the cyclosporine A (CsA) was found to induce total remission. This observation and observations of other authors confirm that in genetic forms of nephrotic syndrome, the proteinuric effect of CsA may be due to a non-immunologic mechanism. We confirm the beneficial effect of CsA treatment in DDS; however, the potential nephrotoxicity of this drug will probably not allow long-term use.

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