INTRODUCTION: The literature on the clinical effectiveness of treatments for steroid-resistant nephrotic syndrome (SRNS) is very limited. The available evidence suggests a beneficial effect of cyclosporine on remission rates. Mycophenolate mofetil (MMF) represents a promising therapeutic alternative without nephrotoxicity. The purpose of the present study was to evaluate the efficacy and tolerance of MMF therapy in children with SRNS. METHODS: Six patients with SRNS were treated with MMF combined with oral prednisolone at a dosage of 1 mg/kg per day. The initial dosage of MMF was 600 mg/m² per 12 hours, adjusted to maintain levels of mycophenolic acid at 2.5-5 µg/mL. The planned duration of study to assess treatment efficacy was 12 weeks. All patients had a pathological renal study. RESULTS: The treatment with MMF was started at a median age of 11 years (range 9-13). Pathological patterns were 4 patients with focal segmental glomerulosclerosis (FSGS), 1 patient with minimal change disease (MCD) and 1 patient with diffuse mesangial proliferation glomerulonephritis (DMP). Only 1 patient, who had MCD, achieved complete remission. One patient went into partial remission. However, the subgroup with no response to MMF appeared to have a reduction in proteinuria and an increase of serum albumin. There were no significant beneficial effects on the level of glomerular filtration. CONCLUSIONS: In this small, single-center study, a therapeutic response to MMF was obtained only in one third of patients. This response rate, though it is low, encourages us to use MMF in some patients who are resistant to conventional therapy for SRNS.
INTRODUCTION: The literature on the clinical effectiveness of treatments for steroid-resistant nephrotic syndrome (SRNS) is very limited. The available evidence suggests a beneficial effect of cyclosporine on remission rates. Mycophenolate mofetil (MMF) represents a promising therapeutic alternative without nephrotoxicity. The purpose of the present study was to evaluate the efficacy and tolerance of MMF therapy in children with SRNS. METHODS: Six patients with SRNS were treated with MMF combined with oral prednisolone at a dosage of 1 mg/kg per day. The initial dosage of MMF was 600 mg/m² per 12 hours, adjusted to maintain levels of mycophenolic acid at 2.5-5 µg/mL. The planned duration of study to assess treatment efficacy was 12 weeks. All patients had a pathological renal study. RESULTS: The treatment with MMF was started at a median age of 11 years (range 9-13). Pathological patterns were 4 patients with focal segmental glomerulosclerosis (FSGS), 1 patient with minimal change disease (MCD) and 1 patient with diffuse mesangial proliferation glomerulonephritis (DMP). Only 1 patient, who had MCD, achieved complete remission. One patient went into partial remission. However, the subgroup with no response to MMF appeared to have a reduction in proteinuria and an increase of serum albumin. There were no significant beneficial effects on the level of glomerular filtration. CONCLUSIONS: In this small, single-center study, a therapeutic response to MMF was obtained only in one third of patients. This response rate, though it is low, encourages us to use MMF in some patients who are resistant to conventional therapy for SRNS.
Authors: Marco Orsini; Flavio R Sztajnbok; Acary Bulle Oliveira; Marco Antonio Araújo Leite; Salem Peter Júnior; Marcos R G de Freitas; Osvaldo J M Nascimento; Júlio Guilherme Silva; Marzia Puccioni Sholer; Fernando Silva Guimarães; Alessandra Cardoso Pereira; Sara Lúcia Silveira de Menezes; Antonio Marcos da Silva Catharino; Fabrício Bino Journal: Neurol Int Date: 2011-09-20
Authors: Agnes Trautmann; Sven Schnaidt; Beata S Lipska-Ziętkiewicz; Monica Bodria; Fatih Ozaltin; Francesco Emma; Ali Anarat; Anette Melk; Marta Azocar; Jun Oh; Bassam Saeed; Alaleh Gheisari; Salim Caliskan; Jutta Gellermann; Lina Maria Serna Higuita; Augustina Jankauskiene; Dorota Drozdz; Sevgi Mir; Ayse Balat; Maria Szczepanska; Dusan Paripovic; Alexandra Zurowska; Radovan Bogdanovic; Alev Yilmaz; Bruno Ranchin; Esra Baskin; Ozlem Erdogan; Giuseppe Remuzzi; Agnieszka Firszt-Adamczyk; Elzbieta Kuzma-Mroczkowska; Mieczyslaw Litwin; Luisa Murer; Marcin Tkaczyk; Helena Jardim; Anna Wasilewska; Nikoleta Printza; Kibriya Fidan; Eva Simkova; Halina Borzecka; Hagen Staude; Katharina Hees; Franz Schaefer Journal: J Am Soc Nephrol Date: 2017-05-31 Impact factor: 10.121