4.4 Article

A pediatric case of IgA nephropathy benefitting from targeted release formulation-budesonide

Journal

PEDIATRIC NEPHROLOGY
Volume 38, Issue 11, Pages 3849-3852

Publisher

SPRINGER
DOI: 10.1007/s00467-023-05968-0

Keywords

IgA nephropathy; TRF-budesonide; NEFIGAN; NEFIGARD; Children

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This case demonstrates that TRF-budesonide could be considered an effective second-line treatment in pediatric IgAN, especially when a long course of steroids is necessary to control active inflammation. However, pediatric clinical trials are urgently needed to identify the correct dosage and tolerability of TRF-budesonide.
BackgroundThe best treatment for IgAN is still debated. The trials NEFIGAN and NEFIGARD have demonstrated that TRF-budesonide (Nefecon) efficiently and safely reduced proteinuria in adults, leading to FDA approval of Nefecon for adult IgAN. In pediatric IgAN, an etiological treatment does not yet exist, and the main therapies remain RAAS inhibitors and oral steroids. To our knowledge, this is one of the few pediatric reports of TRF-budesonide therapy.Case report-diagnosis/treatmentA 13-year-old boy underwent a kidney biopsy for recurrent macrohematuria and proteinuria, resulting in an IgAN diagnosis (MEST-C score M1-E1-S0-T0-C1). At admission, serum creatinine and UPCR were slightly increased. Three methylprednisolone pulses were performed, followed by prednisone and RAAS inhibitors therapy. However, after 10 months, macrohematuria became constant, and UPCR increased. A new kidney biopsy was performed, showing an increase in sclerotic lesions. Prednisone was discontinued, and a trial with IBD TRF-budesonide 9 mg/day started. One month later, macrohematuria episodes disappeared and UPCR decreased, with a stable kidney function. After 5 months, due to a reduction in morning cortisol levels and difficulty in drug provisioning, we started to wean TRF-budesonide by 3 mg every 3 months, with complete withdrawal after 1 year. During this period, episodes of macrohematuria dramatically decreased, and UPCR and kidney function were maintained stable.ConclusionOur case demonstrates that TRF-budesonide could be considered an effective second-line treatment in pediatric IgAN, particularly when a long course of steroids is necessary to control active inflammation. However, pediatric clinical trials to identify the correct dosage and tolerability of TRF-budesonide are urgently needed.

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