Efficacy of Budesonide Delayed-Release Capsules in the Treatment of IgAN and IgA Vasculitis Nephritis in Pediatric Patients

Background: Nefecon, a targeted-released budesonide for the gut-associated lymphoid tissue at Peyer’s patches (the principal site of Gd-IgA1 production), has been approved for the treatment of adult IgA nephropathy (IgAN) worldwide. However, critical knowledge gaps persist regarding its therapeutic potential in pediatric populations with IgAN or IgA vasculitis nephritis (IgAVN). Methods: In this real-world study, six IgAN or IgAVN children treated with nefecon were included between January 2025 and May 2025. All patients were stable on maximum tolerated doses of RAS inhibitors, with an eGFR >60 mL/min/1.73 m2 and moderate hematuria and/or proteinuria. Results: A total of 6 pediatric patients (4 males, 2 females, median age 10 yr) were analyzed, including 3 IgAN and 3 IgAVN cases. Treatment with Nefecon induced rapid proteinuria reduction, achieving median UPCR decreases of 34.6% (range: 15.2-72.2) at 4 weeks (365mg/g vs 272 mg/g) and 47.4% (range: 22.2-65.8) at 12 weeks (365mg/g vs 138 mg/g). A marked reduction was also observed in both the urine albumin/creatinine ratio and the urine red blood cell count. Renal function remained stable throughout observation. Aside from 2 patients who presented with a distinctive "moon face" appearance, no treatment discontinuation or serious adverse event occurred. Conclusion: This first real-world evidence in pediatric IgAN/IgAVN indicates that Nefecon achieved significant reductions in both proteinuria and hematuria within 12 weeks. However, long-term efficacy and safety data warrant further investigation.