Long-term prognosis of children with steroid-dependent nephrotic syndrome following high-dose mizoribine therapy

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  • Yamada Akifumi
    Division of nephrology, Saitama Children’s Medical Center Department of Pediatrics, The Jikei University School of Medicine
  • Fujinaga Shuichiro
    Division of nephrology, Saitama Children’s Medical Center
  • Hara Taichi
    Division of nephrology, Saitama Children’s Medical Center
  • Urushihara Yasuko
    Division of nephrology, Saitama Children’s Medical Center
  • Hirano Daishi
    Department of Pediatrics, The Jikei University School of Medicine
  • Ohtomo Yoshiyuki
    Department of Pediatrics, Juntendo Nerima Hospital
  • Ida Hiroyuki
    Department of Pediatrics, The Jikei University School of Medicine

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  • シクロスポリン投与前に高用量ミゾリビン療法を施行したステロイド依存性ネフローゼ症候群の長期的検討

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Background: Recent studies showed that high-dose mizoribine (MZR) therapy may be effective in the management of children with steroid-dependent nephrotic syndrome (SDNS). However, long-term prognosis of the patients following the immunosuppressive agent remains unclear. Patients and methods: We retrospectively analyzed 12 patients with SDNS who received high-dose MZR prior to cyclosporine (CsA) administration. MZR was initiated at a single daily dose of 5 mg/kg administered after breakfast and adjusted to maintain 2-h post-dose MZR levels of approximately 3-5 μg/mℓ. Results: In all but one patient, treatment with MZR (mean MZR dose, 8.6 mg/kg/day; mean C2, 3.4 μg/mℓ) resulted in significant reduction of the mean prednisolone dose from 0.45 to 0.12 mg/kg/day and the mean relapse rate from 1.86 to 0.40 episodes/12 months. Of six patients who were able to discontinue high-dose MZR therapy, only one patient returned to SDNS and was re-treated with MZR during follow-up periods (median 4.2 years). Conclusion: High-dose MZR therapy may allow many patients with SDNS to avoid further toxic immunosuppressive agents such as CsA in the long term.

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