Abstract
Background: Pediatric patients with steroid-resistant nephrotic syndrome (SRNS) and focal segmental glomerulosclerosis (FSGS) may relapse and current second line agents include mycophenolate mofetil. However, there is no current information about the use of the sodium salt of mycophenolic acid (SMPA) in this population. Objectives: We conducted a prospective study on the efficacy and pharmacokinetics of SMPA in children with FSGS. Patients and Methods: Patients without NPHS2 pathogenic variants received SMPA at dosages between 460 to 720 mg/m2/d for 12 months after previous treatments failure. Clinical and biochemical assessments were performed. Blood samples were obtained after the first dose and at steady state (3 months after the onset of treatment) and total and free mycophenolic acid (MPA) was quantitated using HPLC-UV. Results: Two patients showed partial remission after the 12-month period of SMPA treatment with a notable decrease in proteinuria and an increase in serum albumin levels. Maximum MPA concentrations after the first dose and at steady state were 11.6 µg/mL and 10.5 µg/mL, respectively, without drug accumulation. Maximum MPA free levels after the first dose and at steady state were 192.9 and 120.6 ng/mL, respectively. MPA levels became undetectable after 4 hours of the administration in all cases. Conclusions: SMPA is a promising agent for pediatric patients with SRNS and FSGS but SMPA schedule of treatment should be revised with shorter intervals of administration and higher doses than those used in the present study in order to attain higher systemic exposures and accumulation of the immunosuppressant drug. Further efficacy and pharmacokinetic studies should be performed to confirm these findings.
Highlights
Pediatric patients with steroid-resistant nephrotic syndrome (SRNS) and focal segmental glomerulosclerosis (FSGS) may relapse and current second line agents includeArticle history: Received: 10 March 2017 Accepted: 9 September 2017 Published online: 2 October 2017 mycophenolate mofetil
Despite mycophenolate mofetil has been proposed as a new treatment, side-effects may lead to treatment withdrawal and data on clinical response is scarce
The present study shows that salt of mycophenolic acid (SMPA) could be a second line option for children with FSGS resistant to steroids
Summary
Pediatric patients with steroid-resistant nephrotic syndrome (SRNS) and focal segmental glomerulosclerosis (FSGS) may relapse and current second line agents includeArticle history: Received: 10 March 2017 Accepted: 9 September 2017 Published online: 2 October 2017 mycophenolate mofetil. Pediatric patients with steroid-resistant nephrotic syndrome (SRNS) and focal segmental glomerulosclerosis (FSGS) may relapse and current second line agents include. Pharmacological treatment of children with FSGS is still a challenge to achieve proteinuria control and preserve kidney function [4]. Other factors related to sodium salt of mycophenolic acid (SMPA) response include hypoalbuminemia and high inter-individual variability in the pharmacokinetics of the drug [19,20]. In this sense, underexposure to the drug may lead to the lack of efficacy but overexposure to the incidence of adverse events that lead to treatment discontinuation. Scarce data has been reported on the pharmacokinetics of MPA in children and even less information is available in pediatric patients with FGSC [23]
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