Early experience of avacopan for ANCA-associated vasculitis in an Australian paediatric population: a case series.

Avacopan, a complement inhibitor targeting C5a receptor, is a novel and effective agent in the treatment of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) in adults. However, paediatric data of efficacy and safety are scarce. This report outlines our nationwide experience with avacopan for AAV in 8 adolescents. Avacopan was used as an adjunct following induction with glucocorticoids, rituximab, and/or cyclophosphamide at a median of 2 months postdiagnosis. There was good clinical improvement in 5 of the patients with an ability to wean steroid therapy; however, for 3, the disease progressed to end-organ damage despite avacopan introduction. There were no significant adverse events or serious infections reported with follow-up of a median of 6 months of avacopan use. The efficacy for most and short-term safety in all our cohort of avacopan is reassuring in the infancy of paediatric use and supports the need for further research in this orphan disease.