Childhood nephrotic syndrome: clinical associations and response to therapy

The concept of nephrotic syndrome has changed from that of a single disease entity in children to that of a complex association of distinct types of glomerular histopathology and clinical courses. Nephrotic syndrome is steroid-responsive in the majority of children. The prognosis for steroid-responsive children is good, even for those who have frequent relapses requiring maintenance therapy or for those who become steroid-dependent. The disease rarely progresses to renal insufficiency or death in these patients. Cyclophosphamide and chlorambucil have proved effective only in steroid-responsive patients with minimal-lesion disease. Because of their toxicity, these agents should be limited to use in patients with severe steroid side effects. The effectiveness of these drugs in steroid-resistant diseases is unclear and still under investigation.