2022 Volume 35 Issue 2 Pages 125-132
Galloway-Mowat syndrome (GAMOS) is a disorder characterized by mental retardation with microcephaly and steroid-resistant nephrotic syndrome (SRNS). According to previous reports, SRNS defines the prognosis in patients with GAMOS because of its resistance to treatment. We herein describe a patient with GAMOS who achieved long-term remission on cyclosporine. A 1-year-old girl was noted to have mental retardation and microcephaly at a health checkup. She developed proteinuria at 2 years of age; she thereafter met the criteria for SRNS at 5 years of age and was diagnosed with GAMOS based on clinical symptoms. After the introduction of cyclosporine A for SRNS, the proteinuria gradually decreased, and nephrotic remission was achieved at 7 years of age. When the cyclosporine was discontinued after remission, the proteinuria worsened again, suggesting that the cyclosporine had contributed to the decrease in proteinuria. To reduce nephrotoxicity, the cyclosporine was changed to once-daily administration at 8 years of age. Renal biopsy at 14 years of age showed no obvious nephrotoxicity, and remission was maintained. Once-daily administration of cyclosporine may be effective in reducing nephrotoxicity and improving the prognosis in patients with hereditary SRNS such as GAMOS nephropathy.
