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Response to cyclosporine in steroid-resistant nephrotic syndrome: discontinuation is possible

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Abstract

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.

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Acknowledgments

This work is dedicated to Prof. Dirk E. Müller-Wiefel on occasion of his 70th birthday.

Conflict of interest

The authors declare no conflict of interest

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Author notes

  1. Carolin E. Sadowski

    Present address: Department of Gynecology and Obstetrics, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany

Authors and Affiliations

  1. Pediatric Nephrology, University Medical Center Hamburg-Eppendorf, Martinistr. 52, 20246, Hamburg, Germany

    Ilka Klaassen, Bünyamin Özgören, Kristina Möller, Michael van Husen, Anja Lehnhardt, Kirsten Timmermann, Folke Freudenberg, Jun Oh & Markus J. Kemper

  2. Institute of Pathology, University Medical Center Hamburg-Eppendorf, Martinistr. 52, 20246, Hamburg, Germany

    Udo Helmchen

  3. Division of Nephrology, Department of Medicine, Boston Children’s Hospital, Harvard Medical School, Boston, MA, USA

    Carolin E. Sadowski

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  1. Ilka Klaassen
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  2. Bünyamin Özgören
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Correspondence to Markus J. Kemper.

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Ilka Klaassen and Bünyamin Özgören contributed equally to this work.

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Klaassen, I., Özgören, B., Sadowski, C.E. et al. Response to cyclosporine in steroid-resistant nephrotic syndrome: discontinuation is possible. Pediatr Nephrol 30, 1477–1483 (2015). https://doi.org/10.1007/s00467-015-3109-3

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  • DOI: https://doi.org/10.1007/s00467-015-3109-3

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