The safety and efficacy of mycophenolate mofetil in children and adolescents with steroid-dependent nephrotic syndrome: a single-centre study

BackgroundSteroid-dependent nephrotic syndrome (SDNS) patients experience frequent relapse or adverse effects on long-term treatment with steroids or cyclophosphamide. This study assessed the efficacy and side effect profile of mycophenolate mofetil (MMF) therapy in children with nephrotic syndrome...

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Published in:Clinical kidney journal 2020-04, Vol.13 (2), p.179-183
Main Authors: Karunamoorthy, Saravanakumar, Thanigachalam, Dineshkumar, Jeyachandran, Dhanapriya, Ramanathan, Sakthirajan, Natarajan, Gopalakrishnan, Thoppalan, Balasubramaniam
Format: Article
Language:English
Subjects:
Care and treatment
Children
Diseases
Mycophenolate mofetil
Nephrotic syndrome
Original
Patient outcomes
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Summary:BackgroundSteroid-dependent nephrotic syndrome (SDNS) patients experience frequent relapse or adverse effects on long-term treatment with steroids or cyclophosphamide. This study assessed the efficacy and side effect profile of mycophenolate mofetil (MMF) therapy in children with nephrotic syndrome in our population.MethodsA retrospective study was performed on children with SDNS who were on MMF therapy for a minimum period of 1 year, and were on regular follow-up in the Department of Nephrology at the Institute of Child Health and hospital for children attached to Madras Medical College.ResultsThe study included 87 patients, with a male:female ratio of 2:1. The median age at diagnosis of nephrotic syndrome was 3 years [95% confidence interval (CI): 1–8 years], which was found to be a statistically significant risk factor for MMF failure. The median duration of follow-up after initiation of MMF therapy was 3 years and 3 months (95% CI: 1 year and 3 months to 6 years and 6 months). At initial evaluation, 31 (36%) patients presented with SDNS while the remaining had frequently relapsing nephrotic syndrome progressing to SDNS. Intravenous cyclophosphamide was used as first-line therapy in 82 patients, of whom 24 patients had persistent proteinuria while the remaining 58 had attained remission for a median duration of 6 months. The median duration of treatment with MMF was 2 years and 6 months (95% CI: 1 year and 3 months to 4 years and 6 months). MMF was used at a mean dose of 28.5 mg/kg. Seventy-two (83%) patients were MMF-sensitive, and these patients had a reduction in mean prednisolone dose from 1.28 to 0.35 mg/kg (P 
ISSN:2048-8505
2048-8513
DOI:10.1093/ckj/sfz061