Application of TRF-budesonide (Nefecon) in the treatment of pediatric patient with IgA nephropathy: A case report

 

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Application of TRF-budesonide (Nefecon) in the treatment of pediatric patient with IgA nephropathy: A case report

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Jingyuan
Xie
Xinyi Zhu zhuxxyy12@163.com Shanghai Jiao Tong University School of Medicine Department of Nephrology, Ruijin Hospital Shanghai China -
Yiming Tong goodtyme@163.com Shanghai Jiao Tong University School of Medicine Department of Nephrology, Ruijin Hospital Shanghai China -
Yan Ouyang doring323@163.com Shanghai Jiao Tong University School of Medicine Department of Nephrology, Ruijin Hospital Shanghai China -
Jing Xu xjjesse@126.com Shanghai Jiao Tong University School of Medicine Department of Nephrology, Ruijin Hospital Shanghai China -
Xiaofan Hu huxiaofan1210@163.com Shanghai Jiao Tong University School of Medicine Department of Nephrology, Ruijin Hospital Shanghai China -
Xiaoxia Pan pxx10768@rjh.com.cn Shanghai Jiao Tong University School of Medicine Department of Nephrology, Ruijin Hospital Shanghai China -
Lei Yin scmckidney2022@163.com Shanghai Jiao Tong University School of Medicine Department of Nephrology, Shanghai Children's Medical Center Shanghai China -
Jingyuan Xie nephroxie@163.com Shanghai Jiao Tong University School of Medicine Department of Nephrology, Ruijin Hospital Shanghai China *
 
 
 
 
 
 
 

Immunoglobulin A nephropathy (IgAN) is one of the most common primary glomerular diseases in children and adolescents, with 20% of affected children progressing to end-stage kidney disease (ESKD) within 20 years of diagnosis. While glucocorticoids are used in adult IgAN to protect renal function, their systemic toxicity is a concern, and there is no consensus on their use in pediatric IgAN due to insufficient evidence and population differences. The pathogenesis of IgAN is not fully understood, but growing evidence highlights the role of the gastrointestinal mucosal immune system. Targeted-release formulation budesonide (TRF-budesonide), such as Nefecon, has been approved for adult IgAN ( NEFIGAN and NefIgArd trials showing efficacy in reducing proteinuria and preserving eGFR with fewer side effects). However, its safety and efficacy in pediatric patients remain unvalidated, as existing pediatric cases used TRF-budesonide approved for inflammatory bowel disease (IBD), not Nefecon.

A 14-year-old Chinese male with macroscopic hematuria (preceded by vomiting/diarrhea) was diagnosed with primary IgAN via renal biopsy.(Figure1). He had no extrarenal symptoms, family history of kidney disease, or infections (HIV, HBV, TB); autoantibody tests were negative. Initial treatment included 2.5mg/day Benazepril (a renin–angiotensin system inhibitor) for 1 month. Due to persistent hematuria and active renal lesions, with parental consent (informed of off-label use), 16mg/day TRF-budesonide (Nefecon) was added for 9 months. Post-treatment, he was followed up for 6 months with only Benazepril. Key outcomes (24-hour proteinuria, serum creatinine, hematuria, adverse events) were monitored throughout treatment and follow-up.

Figure1 Endocapillary hypercellularity (black arrows) and cellular crescent (white arrows) under light microscopy (A, B). Immunofluorescence demonstrated IgA (+~++) and C3 (+~++) mesangial deposition (C, D). Electron-dense materials (blue arrows) and foot process fusion (red arrows) under electron microscope (E, F). EM, electron microscope.

Figure1 Endocapillary hypercellularity (black arrows) and cellular crescent (white arrows) under light microscopy (A, B). Immunofluorescence demonstrated IgA (+~++) and C3 (+~++) mesangial deposition (C, D). Electron-dense materials (blue arrows) and foot process fusion (red arrows) under electron microscope (E, F). EM, electron microscope.


Benazepril phase: 24-hour proteinuria decreased from 2.9g to 0.56g, serum creatinine from 129μmol/L to 82μmol/L, but hematuria persisted (50 RBCs/HPF).

TRF-budesonide phase: After 4 months, microscopic hematuria resolved completely (no recurrence thereafter). 24-hour proteinuria dropped to <0.15g and remained stable; serum creatinine stayed <90μmol/L. Only facial acne was reported as an adverse event; blood glucose/lipids were normal.

Follow-up phase: Hematuria was undetectable, 24-hour proteinuria remained <0.35g (Figure2), and renal function stayed stable with Benazepril alone.

Figure2 Change in 24h proteinuria, serum creatinine and hematuria from baseline over the Nefecon treatment phase and follow-up phase. Scr, serum creatinine. RBC, red blood cells. HPF, high power field.

Figure2 Change in 24h proteinuria, serum creatinine and hematuria from baseline over the Nefecon treatment phase and follow-up phase. Scr, serum creatinine. RBC, red blood cells. HPF, high power field.

This is the first reported case of pediatric IgAN treated with Nefecon (TRF-budesonide). The 9-month Nefecon therapy effectively reduced proteinuria/hematuria and preserved renal function, with minimal side effects (only facial acne). A 6-month follow-up confirmed sustained benefits with Benazepril alone. These findings suggest Nefecon may be a safe and effective option for pediatric IgAN, but further large-scale studies are needed to confirm long-term safety, optimal dosing, and efficacy across diverse pediatric populations.

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