Granulomatosis 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.
- therapeutic indications
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Contributors DE and CP contributed equally to the drafting and revising of the manuscript. RSMY contributed to the critical review and finalising of the manuscript.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests CP reports receiving fees for serving on advisory boards from Chemocentryx, GlaxoSmithKline, Sanofi and Hoffman-LaRoche; he also reports lecture fees and research grant support from Hoffman-La Roche and GlaxoSmithKline. RSMY is the site primary investigator for a Roche funded clinical trial.
Patient consent for publication Obtained.
Provenance and peer review Not commissioned; externally peer reviewed.
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