• Daniel Ennis, Rae Sm Yeung, and Christian Pagnoux.
• Division of Rheumatology, St Paul's Hospital, University of British Columbia, Vancouver, British Columbia, Canada.
• BMJ Case Rep. 2020 Oct 29; 13 (10).

AbstractGranulomatosis with polyangiitis (GPA) is a rare antineutrophil cytoplasm antibody-associated vasculitis. Several therapeutic advances have occurred over the past two decades, but relapse rate remains high and refractory cases are not uncommon. Here, we present the case of a female patient diagnosed with GPA at the age of 9 years with a severe, multirelapsing disease course which failed to adequately respond to conventional therapies. Avacopan, a novel C5a receptor inhibitor, was started based on phase II studies that showed promise as a steroid-sparing adjunct. The patient was able to successfully reduce her glucocorticoid dose and reduce her immunosuppressive treatments without another flare. She has been on avacopan for 35 months, had no adverse events that required its discontinuation, and her disease is in sustained remission.© BMJ Publishing Group Limited 2020. No commercial re-use. See rights and permissions. Published by BMJ.

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