A phase 3 trial tested whether mycophenolate mofetil (MMF) could replace prednisone for children ages 1 to 10 with a first episode of steroid-sensitive nephrotic syndrome. The study involved 272 children across 37 hospitals in Germany. Half received MMF for 12 weeks, and the other half received prednisone for 12 weeks. Researchers followed them for 24 months.
The main finding: MMF was non-inferior to prednisone in preventing relapses. About 79% of children in the MMF group had a treated relapse, compared to 75% in the prednisone group. The difference was small enough to conclude MMF works just as well.
Importantly, MMF caused fewer steroid-related side effects. Fewer children on MMF developed high blood pressure (59% vs 87%), had lower BMI scores, and had fewer psychological issues. However, infections were more common with MMF (70% vs 56%). Gastrointestinal problems were similar between groups.
This study suggests MMF could be a good alternative to steroids for initial treatment, reducing common steroid side effects. But the higher infection rate with MMF needs careful monitoring. Parents should discuss options with their child's doctor, as this is one study and individual factors matter.