Open AccessLong-term use and remission of granulomatosis with polyangiitis with the oral C5a receptor inhibitor avacopan
Author(s) -
Daniel B. Ennis,
Rae S. M. Yeung,
Christian Pagnoux
Publication year - 2020
Publication title -
bmj case reports
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.231
H-Index - 26
ISSN - 1757-790X
DOI - 10.1136/bcr-2020-236236
Subject(s) - medicine , granulomatosis with polyangiitis , discontinuation , vasculitis , microscopic polyangiitis , anti neutrophil cytoplasmic antibody , adverse effect , refractory (planetary science) , gastroenterology , dermatology , surgery , disease , physics , astrobiology
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.