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Erschienen in:
01.09.2015 | Original Article
Response to cyclosporine in steroid-resistant nephrotic syndrome: discontinuation is possible
Erschienen in: Pediatric Nephrology | Ausgabe 9/2015
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Background
Steroid-resistant nephrotic syndrome (SRNS) is still regarded as a serious disease although treatment with cyclosporine (CSA) has improved outcome. However, the duration of treatment in responders is unclear, and treatment of patients with genetic causes is a matter of debate.
Methods
Thirty-six patients with SRNS were studied retrospectively. Median age at presentation was 3.2 (range, 0.06–15.0) and median follow-up 15.5 years (range, 1.8–27.7), respectively; 23 (64 %) had focal segmental glomerulosclerosis (FSGS) on biopsy. In 33/36 patients (92 %), genetic testing was performed for at least three most common genes known to be mutated in SRNS.
Results
Nineteen patients (53 %), especially those with minimal change nephrotic syndrome (MCNS) at initial biopsy (p < 0.002), entered complete remission with CSA monotherapy, including one patient with compound heterozygous NPHS1 and dominant ACTN4 mutation, respectively. Ten patients entered partial remission (28 %, all FSGS), including two with NPHS2 mutations. Seven patients (six FSGS, one MCNS) did not respond to treatment. In 15 of 19 responders to CSA, treatment was stopped after a median of 3.1 years (range, 0.5–14) and no further relapses occurred in 11/15 (73 %) patients with median follow-up of 9.7 years.
Conclusions
CSA monotherapy is effective in SRNS. Discontinuation of CSA is possible in many patients with complete remission.