CYCLOSPORINE A (CSA) MONOTHERAPY FOR PRIMARY STEROID RESISTANT NEPHROTIC SYNDROME (SRNS) OF CHILDHOOD

MJ Kemper. K Timmermann, H Altrogge, U Helmchen*, DE Müller-Wiefel

Pediatric Nephrology and *Pathology, Univ. Hospital Eppendorf, Hamburg, Germany

OBJECTIVE: Although regarded as a disease with unfavourable prognosis, treatment of primary SRNS is not standardised. We here report our single-centre experience with CSA monotherapy as first line treatment after establishment of SRNS.

PATIENTS AND METHODS: 19 patients presented with SRNS at a median of 3 (range 0.2-8) years according to the definitions of the ISKDC and APN. Renal biopsy showed focal-segmental glomerulosclerosis (FSGS) in 12 and minimal changes (MCNS) in 7 patients. CSA was begun at a dose of 150 mg/m²/d with a monoclonal trough level aimed at 150-200 ng/ml and steroids were discontinued over a period of 4-6 weeks. Median follow-up was 6 (range 0.25-12) years.

RESULTS: 5 patients (26%) did not show any benefit from CSA treatment (4 FSGS, 1 MCNS) and had persistent NS, while 5 patients (26%) had a partial response (serum albumin >25 g/1; 3 FSGS, 2 MCNS). 9 patients (47%) went into complete remission (5 FSGS, 4 MCNS) and even more importantly in 7 of these patients CSA could be discontinued for a median of 4 (range 1-6) years at latest follow-up (4 FSGS, 3 MCNS). In 2 patients steroid sensitive relapses relapses developed after stopping CSA.

CONCLUSIONS: Our data indicate, that not all patients with SRNS (FSGS and MCNS) have an unfavourable prognosis. A stepwise therapeutical approach seems to be justified in the treatment of this disorder, since in complete responders to CSA treatment, this immunosuppression can be discontinued successfully.