Idiopathic nephrotic syndrome in children: a new treatment strategy and future perspective

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  • Iijima Kazumoto
    Department of Pediatrics, Kobe University Graduate School of Medicine

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  • 小児特発性ネフローゼ症候群―新たな治療戦略と今後の課題―

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At least 20% of children with idiopathic nephrotic syndrome develop frequent relapses or steroid dependence under or after the treatment with immunosuppressive agents such as cyclosporine (complicated FRNS/SDNS). To evaluate the efficacy and safety of rituximab in childhood-onset, complicated FRNS/SDNS, a multicenter, double-blind, randomized, placebo-controlled trial was carried out by the Research Group of Childhood-onset Refractory Nephrotic Syndrome (RCRNS) in Japan (RCRNS01). RCRNS01 showed that rituximab is safe and effective for the treatment of childhood-onset, complicated FRNS/SDNS. In 2014, the use of rituximab for patients with complicated FRNS/SDNS was approved, first in the world, by the Ministry of Health, Labour and Welfare, Japan. However, further modification of rituximab therapy, including repeated courses and adjunct immunosuppressive therapies, may be necessary to extend the relapse-free period, as this drug does not cure nephrotic syndrome. In addition, the pathogenesis of idiopathic nephrotic syndrome in children should be clarified to develop a specific and definitive therapy for this disease.

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