ESPN 54th Annual Meeting

ESPN 2022


 
Successful treatment of IgA Nephropathy (IgAN) with targeted-release budesonide in a 13-year-old boy
Śladowska-Kozłowska Joanna 1 Galata Barbara 1 Tönshoff Burkhard 1

1- Children’s Hospital, University of Heidelberg, Germany
 
Introduction:

The NEFIGAN and the NefIgArd trials demonstrated that the oral, targeted-release-formulation (TRF) of the glucocorticoid budesonide, delivering the drug to the distal ileum, suppressed the dysfunctional mucosal immune system and, in conjunction with optimal renin-angiotensin-aldosterone system blockade (RAASB), safely reduced proteinuria and improved renal function in IgA-Nephropathy (IgAN) in adults.  Also, the safety profile of TRF-budesonide is proposed to be superior to systemic glucocorticoid therapy given its extensive first pass metabolism with <10% entering the systemic circulation. To our knowledge, only one case report detailing the first successful trial of this therapy in a paediatric patient was published. Here, we want to share our experience with TRF-budesonide therapy.

Material and methods:

A 13-year-old boy who underwent a routine laboratory blood examination for a scheduled jaw cysts extraction was unexpectedly diagnosed with IgAN. At the time of the diagnosis his serum creatinine was 2.4 mg/dl, and urinary protein-to-creatinine ratio (uPCR) was >700 g/mol. He had hypertension and left ventricular hypertrophy (LVH); the family history was positive for celiac disease of the father. A kidney biopsy demonstrated features of IgA mesangioproliferative glomerulonephritis with focal segmental sclerosis and crescents (14%). Celiac disease was excluded. He received pulse methylprednisolone therapy on 3 consecutive days, followed by oral prednisone over 2 months (for 1 month 75 mg  daily, then 50 mg every other day) as well as maximally tolerated RAASB. Because of persisting proteinuria in the nephrotic range therapy with mycophenolate mofetil (MMF) was initiated. After 3 months only small reductions of proteinuria and serum creatinine were achieved. A second course of systemic glucocorticoids was not considered, because he had suffered from the steroid-associated side effect of depressive mood during the first course of steroids. We therefore initiated treatment with 15 mg TRF-budesonide daily continuing MMF and RAASB.

Results:

 The blood pressure normalised reaching values <50thpercentile,  the LVH decreased. 3 and 6 months after initiation of TRF-budesonide the patient responded well with a 86% decrease of uPCR down to 100 g/mol and a 34% decrease of serum creatinine down to 1.6 mg/dL. Budesonide was well tolerated, and no side effects were observed.

Conclusions:

This case report of successful and well tolerated treatment of IgAN with TRF-budesonide in a paediatric patient underlines the potential of this drug for this disease, for which no approved therapy is currently available. Controlled trails on TRF-budesonide in children with IgAN are required.