Dr Julia Donald1, Dr SWASTI CHATURVEDI1
1Sydney Children's Hospital, Sydney, Australia
Biography:
Bio to come
Background:
Complement activation plays a key role in the pathogenesis of IgA nephropathy (IgAN) and complement inhibition has been shown to reduce proteinuria in adults. We report the first use of complement blockade for treatment-resistant IgAN in a 10-year-old boy.
Case Report:
A 10-year-old boy presented with recurrent episodes of macrohaematuria and proteinuria in 2022. He had normal renal function but persistently elevated urine albumin:creatinine ratio (ACR), between 130-170mg/mmol. He underwent renal biopsy, showing IgAN with an Oxford score of M1E0S1T0C1. He was treated with three months of 1mg/kg prednisolone and lisinopril, with no change to his proteinuria. Mycophenolate (MMF) and pulsed methylprednisolone were also trialled with no response. He had a partial response to tacrolimus, with urine ACR decreasing to 80mg/mmol. Repeat renal biopsy in 2024 showed persistent active disease with an Oxford score of M1E1S1T0C1. He was commenced on oral budesonide and tacrolimus was ceased. His urine ACR increased to 400mg/mmol with this, so he was re-started on tacrolimus and prednisolone at 2mg/kg, which he received for 1 month. His urine ACR decreased to 220-260mg/mmol. Compassionate access to Ravulizumab (a complement C5 inhibitor) was granted in January 2025, and he received two doses, two weeks apart in February 2025. By the following month, his urine ACR had decreased to 9-11mg/mmol, and he has had a sustained response in his proteinuria to date with a combination of Ravulizumab, tacrolimus and ACE inhibition.
Conclusions:
While complement blockade has been shown to effectively reduce proteinuria in adults with IgAN, we found no reports in the literature concerning its use in paediatric IgAN. We report our experience of Ravulizumab use for paediatric IgAN with a promising result.