Division of Rheumatology, St Paul's Hospital, University of British Columbia, Vancouver, British Columbia, Canada.
Department of Paediatrics, Immunology, and Institute of Medical Science, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
BMJ Case Rep. 2020 Oct 29;13(10):e236236. doi: 10.1136/bcr-2020-236236.
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.
肉芽肿性多血管炎（GPA）是一种罕见的抗中性粒细胞胞浆抗体相关性血管炎。在过去的二十年中，已经有了几项治疗进展，但复发率仍然很高，难治性病例也不少见。在这里，我们报告了一例 9 岁女性 GPA 患者，其疾病严重且多次复发，常规治疗反应不佳。基于 II 期研究显示其具有作为类固醇节约辅助药物的潜力，开始使用新型 C5a 受体抑制剂阿伐考帕。患者成功地减少了糖皮质激素剂量并减少了免疫抑制治疗，而没有再次发作。她已经使用阿伐考帕 35 个月，没有因不良反应而停药，疾病处于持续缓解状态。
