Background: C3-glomerulopathy (C3G) is a rare pediatric kidney disease characterised by dysregulation of the alternative complement pathway, with glomerular deposition of C3. C3G may often present as a steroid-resistant nephrotic syndrome (SRNS), and there is no established effective therapy: the usual treatment involves corticosteroids and immunosuppressive drugs. Pioglitazone, a PPAR-γ agonist with a protective action on podocytes, was reported in a few cases as helpful in reducing proteinuria when combined with steroids. Case-diagnosis/treatment: We report the case of a 13-year-old girl with silent past medical history who presented with SRNS. A kidney biopsy showed findings indicative of C3G. A low sodium diet and angiotensin-converting enzyme inhibitor were started; immunosuppressive treatment with mycophenolate mofetil (MMF) was administered due to the cortico-resistance. Because of poor response to the immunosuppressant, a trial with eculizumab was attempted without significant response and persistence of proteinuria in the nephrotic range. A further therapeutic trial was performed with tacrolimus with no disease remission. Due to a severe deterioration in her condition, the girl was hospitalized and treated with high-dose steroid bolus. A daily dose of oral prednisone and MMF were re-started without benefit with persistent levels of nephrotic range proteinuria. The administration of pioglitazone consistently lowered proteinuria levels for the first time since the onset of the disease, with a maintenance of the effect and normalization (< 0.15 g/24 h) at the 10-month follow-up. Conclusions: In this patient affected by C3G, pioglitazone proved effective in reducing proteinuria levels.

Pioglitazone, a PPAR-y agonist, as one of the new therapeutic candidates for C3 glomerulopathy / Balestra, E; Barbi, E; Ceconi, V; Di Maso, V; Conversano, E; Pennesi, M. - In: PEDIATRIC NEPHROLOGY. - ISSN 0931-041X. - (2024). [10.1007/s00467-023-06088-5]

Pioglitazone, a PPAR-y agonist, as one of the new therapeutic candidates for C3 glomerulopathy

Conversano E;
2024

Abstract

Background: C3-glomerulopathy (C3G) is a rare pediatric kidney disease characterised by dysregulation of the alternative complement pathway, with glomerular deposition of C3. C3G may often present as a steroid-resistant nephrotic syndrome (SRNS), and there is no established effective therapy: the usual treatment involves corticosteroids and immunosuppressive drugs. Pioglitazone, a PPAR-γ agonist with a protective action on podocytes, was reported in a few cases as helpful in reducing proteinuria when combined with steroids. Case-diagnosis/treatment: We report the case of a 13-year-old girl with silent past medical history who presented with SRNS. A kidney biopsy showed findings indicative of C3G. A low sodium diet and angiotensin-converting enzyme inhibitor were started; immunosuppressive treatment with mycophenolate mofetil (MMF) was administered due to the cortico-resistance. Because of poor response to the immunosuppressant, a trial with eculizumab was attempted without significant response and persistence of proteinuria in the nephrotic range. A further therapeutic trial was performed with tacrolimus with no disease remission. Due to a severe deterioration in her condition, the girl was hospitalized and treated with high-dose steroid bolus. A daily dose of oral prednisone and MMF were re-started without benefit with persistent levels of nephrotic range proteinuria. The administration of pioglitazone consistently lowered proteinuria levels for the first time since the onset of the disease, with a maintenance of the effect and normalization (< 0.15 g/24 h) at the 10-month follow-up. Conclusions: In this patient affected by C3G, pioglitazone proved effective in reducing proteinuria levels.
2024
C3-glomerulopathy; Children; Pioglitazone; Proteinuria; Steroid-resistant nephrotic syndrome.
01 Pubblicazione su rivista::01a Articolo in rivista
Pioglitazone, a PPAR-y agonist, as one of the new therapeutic candidates for C3 glomerulopathy / Balestra, E; Barbi, E; Ceconi, V; Di Maso, V; Conversano, E; Pennesi, M. - In: PEDIATRIC NEPHROLOGY. - ISSN 0931-041X. - (2024). [10.1007/s00467-023-06088-5]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/1726855
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