VINCRISTINE AND FOCAL SEGMENTAL SCLEROSIS - DO WE NEED A MULTICENTER TRIAL

Over the last 10 years, eight children have received vincristine for t he treatment of steroid-and cyclophosphamide-resistant nephrotic syndr ome at Great Ormond Street Hospital for Children, London. We present o ur experience of these eight cases and put forward a case for reassess ing the effectiveness of vincristine in this disorder. In our series, two children treated with vincristine achieved complete remission with preserved renal function, including relapses in one. Both had primary steroid-and cyclophosphamide-resistant focal segmental glomerulo scle rosis (FSGS). Of the other cases, four also had primary FSGS, one fami lial FSGS and one mesangioproliferative glomerulonephritis. We discuss in general the pros and cons of vincristine therapy in nephrotic synd rome versus the cytotoxic agents that are currently used and the diffe rences in clinical features among the responders and non-responders in this small group. In addition, we explore why this may have occurred and summarise the literature over the last 25 years, where vincristine appeared to have been beneficial, especially in secondary forms of ne phrotic syndrome associated with malignancy. We conclude that vincrist ine therapy warrants re-examination as it could be a valuable alternat ive therapeutic agent in some cases of FSGS with relatively minor side effects.