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Positive effects of single-daily high-dose mizoribine therapy after cyclophosphamide in young children with steroid-dependent nephrotic syndrome

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Abstract

Background

Mizoribine (MZR) therapy after cyclophosphamide (CPM) therapy may be an attractive option in patients with steroid-dependent nephrotic syndrome (SDNS) for the purpose of maintaining remission. This is because CPM is administered only once due to its severe side effects such as gonadal toxicity. However, the long-term prognosis after the treatment regimen remains unknown.

Methods

We retrospectively analyzed the clinical course (median follow-up, 5.9 years) of 54 young children with SDNS (43 boys; age < 10 years) who had undergone 12-week CPM therapy. The patients were classified into two groups: group A, undergoing MZR therapy for > 12 months for maintaining remission after CPM therapy (N = 36), and group B, undergoing CPM monotherapy (N = 18).

Results

For 2 years after CPM therapy, 21 of the 36 group A patients were in sustained remission, whereas only 4 of the 18 group B patients had maintained remission (58% vs. 22%, p < 0.05). Furthermore, the rate of regression to SDNS after CPM was significantly lower in group A than in group B (6% vs. 39%, p < 0.05). At the last follow-up (mean age, 10.9 years), 27 of the 36 group A patients (75%) had not received any steroid-sparing agent after the treatment regimen.

Conclusions

Single daily high-dose MZR therapy after CPM therapy may have positive outcomes in young children with SDNS in the long term.

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Funding

SF received clinical research funding B at Saitama Children’s Medical Center.

Author information

Authors and Affiliations

  1. Division of Nephrology, Saitama Children’s Medical Center, 1-2 Shintoshin, Chuo-Ku, Saitama-city, Saitama, 330 8777, Japan

    Akira Mizutani, Shuichiro Fujinaga & Koji Sakuraya

  2. Department of Pediatrics and Adolescent Medicine, Juntendo University Graduate School of Medicine, Tokyo, Japan

    Akira Mizutani & Toshiaki Shimizu

  3. Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan

    Daishi Hirano

Authors
  1. Akira Mizutani
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  2. Shuichiro Fujinaga
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  3. Koji Sakuraya
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  4. Daishi Hirano
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  5. Toshiaki Shimizu
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Corresponding author

Correspondence to Shuichiro Fujinaga.

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Conflict of interest

The authors declare no conflicts of interest.

Ethical standards

All procedures performed in studies involving human participants were in accordance with the ethical standards of the Institutional Research Committee and/or National Research Committee at which the study was conducted (approval number 2018-02-19) with the 1964 Helsinki Declaration and its later amendments or comparable ethical standards.

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Informed consent was obtained from all participants included in this study.

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Mizutani, A., Fujinaga, S., Sakuraya, K. et al. Positive effects of single-daily high-dose mizoribine therapy after cyclophosphamide in young children with steroid-dependent nephrotic syndrome. Clin Exp Nephrol 23, 244–250 (2019). https://doi.org/10.1007/s10157-018-1628-5

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  • DOI: https://doi.org/10.1007/s10157-018-1628-5

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