Combined Therapy with Mycophenolate and Cyclosporine for the Treatment of Steroid-Dependent/Resistant Nephrotic Syndrome in Children: A 9-Case Analysis—Dual Therapy in Nephrotic Syndrome

Introduction: Steroid-resistant nephrotic syndrome (SRNS) represents a severe and challenging form of pediatric nephrotic syndrome and is associated with a high risk of progression to end-stage kidney disease. Calcineurin inhibitors (CNIs) are the standard second-line therapy; however, their use is limited by frequent relapses and long-term nephrotoxicity. Mycophenolate mofetil (MMF) offers a more favorable safety profile and a complementary mechanism of action; however, the clinical utility of combining MMF with CNIs remains largely under-explored in this population. Case Presentation: We describe a series of nine pediatric patients with steroid-resistant or steroid-dependent nephrotic syndrome who were refractory to cyclosporine monotherapy. These patients were treated with a combination regimen of cyclosporine (4–5 mg/kg/day; target trough levels of 75–150 ng/mL) and MMF (600 mg/m2/day). This therapeutic approach was associated with a reduction in corticosteroid dosage and a decrease in the annual number of relapses in most patients. Conclusions: In this small case series of pediatric patients with corticosteroid-dependent or steroid-resistant nephrotic syndrome refractory to cyclosporine monotherapy, the addition of mycophenolate mofetil was associated with a reduction in relapse frequency and corticosteroid requirements. Despite the limited sample size, these findings suggest that combination therapy may be a therapeutic option in difficult-to-treat pediatric nephrotic syndrome and warrant further evaluation in controlled studies.

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Publication Details

Journal
International Journal of Translational Medicine
Published
2026-05-27
DOI
https://doi.org/10.3390/ijtm6020024
Primary Topic
Renal Diseases and Glomerulopathies
Type
article
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article

Combined Therapy with Mycophenolate and Cyclosporine for the Treatment of Steroid-Dependent/Resistant Nephrotic Syndrome in Children: A 9-Case Analysis—Dual Therapy in Nephrotic Syndrome

Carolina Ochoa-García, Natalia Osorio, Luisa Fernanda Rojas-Rosas, María Carolina Isaza López et al.
International Journal of Translational Medicine
Renal Diseases and Glomerulopathies
article

Combined Therapy with Mycophenolate and Cyclosporine for the Treatment of Steroid-Dependent/Resistant Nephrotic Syndrome in Children: A 9-Case Analysis—Dual Therapy in Nephrotic Syndrome

Carolina Ochoa-García, Natalia Osorio, Luisa Fernanda Rojas-Rosas, María Carolina Isaza López, Esteban Villegas-Arbeláez, Miguel Ángel Restrepo, Richard Baquero-Rodriguez, Lina Maria Serna-Higuita, Mayra Estevez, Melissa Navarro
article en

Abstract

Introduction: Steroid-resistant nephrotic syndrome (SRNS) represents a severe and challenging form of pediatric nephrotic syndrome and is associated with a high risk of progression to end-stage kidney disease. Calcineurin inhibitors (CNIs) are the standard second-line therapy; however, their use is limited by frequent relapses and long-term nephrotoxicity. Mycophenolate mofetil (MMF) offers a more favorable safety profile and a complementary mechanism of action; however, the clinical utility of combining MMF with CNIs remains largely under-explored in this population. Case Presentation: We describe a series of nine pediatric patients with steroid-resistant or steroid-dependent nephrotic syndrome who were refractory to cyclosporine monotherapy. These patients were treated with a combination regimen of cyclosporine (4–5 mg/kg/day; target trough levels of 75–150 ng/mL) and MMF (600 mg/m2/day). This therapeutic approach was associated with a reduction in corticosteroid dosage and a decrease in the annual number of relapses in most patients. Conclusions: In this small case series of pediatric patients with corticosteroid-dependent or steroid-resistant nephrotic syndrome refractory to cyclosporine monotherapy, the addition of mycophenolate mofetil was associated with a reduction in relapse frequency and corticosteroid requirements. Despite the limited sample size, these findings suggest that combination therapy may be a therapeutic option in difficult-to-treat pediatric nephrotic syndrome and warrant further evaluation in controlled studies.

International Journal of Translational MedicineVol. 6(2)
Hospital de Sant Pau (ES), Hospital Universitario de San Vicente Fundación (CO), Instituto del Corazón de Bucaramanga (CO), Municipality of Medellín (CO), Clínica Somer (CO), Hospital Pablo Tobon Uribe (CO), University Children's Hospital Tübingen (DE), Clínica CES (CO), Institut de Recerca Sant Pau (ES), University of Tübingen (DE)
Good health and well-being
Openalex Percentile: Top 7%
Renal Diseases and Glomerulopathies
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