Article ID: oa.2022.0203
In Japan, mycophenolate mofetil (MMF) is used off-label for the treatment of nephrotic syndrome; however, there are no clear guidelines regarding the duration of its use in for this condition. In this retrospective study, we evaluated the efficacy and safety of the long-term use of MMF after an initial period of treatment with rituximab (RTX) treatment for refractory nephrotic syndrome (RNS). The study participants were 13 patients with childhood-onset RNS who used MMF as a single therapeutic agent for more than two years after initially using RTX. The median duration of MMF use was 4 years and 2 months. Eleven patients remained “relapse-free” during the observation period, while the remaining two relapsed after cessation of MMF treatment. All 12 patients who underwent renal biopsy diagnosed with minimal change nephrotic syndrome (MCNS). No serious complications necessitating the discontinuation of MMF, such as neutropenia and prolonged hypo-IgG were observed. This study has shown that administration of MMF is a safe and efficacious therapeutic option following treatment with RTX for long-term remission in most patients with RNS. This could have a considerable impact on the quality of life for children with RNS as they advance through their formative years of the education and employment.
