title: The podocyte as a target: cyclosporin A in the management of the nephrotic syndrome caused by WT1 mutations.
abstract: Children with steroid-resistant nephrotic syndrome secondary to WT1-associated glomerulopathies (WT1-GP) were considered unresponsive to cyclosporin A (CsA). This assumption is challenged by the findings of recent studies. The patients of these studies had different types of WT1 mutations and varying clinical presentations. However, all of them were of young age and the favourable response to CsA might be the result of treatment at an early stage of the disease. The additional administration of angiotensin-converting enzyme inhibitors may have contributed to the positive outcome. We review recent data on the role of WT1 in the development of WT1-GP and discuss putative therapeutic targets explaining the therapeutic effect of CsA.
authors.affiliation: Department of Pediatric Nephrology, P & A Kyriakou Children's Hospital of Athens, Goudi 111527, Athens, Greece. stefanid@hol.gr
authors.forename: Constantinos J
authors.initials: CJ
authors.lastname: Stefanidis
authors.forename: Uwe
authors.initials: U
authors.lastname: Querfeld
mesh_terms: D000367:Age Factors; D000806:Angiotensin-Converting Enzyme Inhibitors / Q000008:administration & dosage / Q000494:pharmacology; D002675:Child, Preschool; D016572:Cyclosporine / Q000008:administration & dosage* / Q000494:pharmacology; D030321:Denys-Drash Syndrome / Q000188:drug therapy / Q000235:genetics; D004351:Drug Resistance; D004359:Drug Therapy, Combination; D005260:Female; D052159:Frasier Syndrome / Q000188:drug therapy / Q000235:genetics; D016162:Genes, Wilms Tumor; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage* / Q000494:pharmacology; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D008775:Methylprednisolone / Q000008:administration & dosage / Q000494:pharmacology; D058990:Molecular Targeted Therapy; D009154:Mutation; D009404:Nephrotic Syndrome / Q000188:drug therapy* / Q000235:genetics* / Q000503:physiopathology; D050199:Podocytes / Q000187:drug effects
chemical_list: D000806:Angiotensin-Converting Enzyme Inhibitors; D007166:Immunosuppressive Agents; D016572:Cyclosporine; D008775:Methylprednisolone