Tagami Genri, Yamaguchi Makoto, Sugiyama Hirokazu, Kinashi Hiroshi, Imai Kentaro, Kamiya Keisuke, Katsuno Takayuki, Imaizumi Takahiro, Banno Shogo, Ito Yasuhiko, Ishimoto Takuji
Department of Nephrology and Rheumatology, Aichi Medical University, 1-1 Karimata, Yazako, Nagakute, Aichi, 480-1195, Japan.
Department of Nephrology and Rheumatology, Aichi Medical University Medical Center, Okazaki, Aichi, Japan.
BMC Rheumatol. 2025 Jan 23;9(1):8. doi: 10.1186/s41927-025-00456-4.
Avacopan, an oral C5a receptor antagonist, demonstrated efficacy as an alternative to glucocorticoid therapy in patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) in the phase 3 ADVOCATE trial. However, limited real-world data exist on the outcomes and experiences associated with avacopan use for AAV in Japan.
We performed a single-centre retrospective analysis and evaluated 21 patients with newly diagnosed or relapsed AAV who received avacopan. The co-primary outcomes were clinical remission at 6 and 12 months.
Among the 21 patients, 20 (95.2%) achieved clinical remission at 6 months, and 19 (90.4%) sustained remission at 12 months. The median time from initiation of immunosuppressive therapy to the start of avacopan was 12 days (interquartile range, 5-26). Adverse events were reported in 10 patients (47.6%), with elevated liver enzyme levels observed in eight patients (38.1%) as the most frequent complication. Avacopan was discontinued in nine patients (42.9%). Despite early discontinuation, these patients achieved comparable rates of clinical remission at 6 months, sustained remission at 12 months, and experienced a reduction in glucocorticoid doses relative to those who continued avacopan.
A high incidence of adverse events, particularly liver enzyme elevation, and frequent early discontinuations of avacopan were observed. Nevertheless, favourable clinical outcomes and reduced glucocorticoid doses were achieved regardless of avacopan discontinuation. Further studies are warranted to validate the optimal use of avacopan in clinical practice.
在3期ADVOCATE试验中，口服C5a受体拮抗剂阿伐库潘在抗中性粒细胞胞浆抗体（ANCA）相关血管炎（AAV）患者中显示出可替代糖皮质激素治疗的疗效。然而，在日本，关于阿伐库潘用于AAV的结局和经验的真实世界数据有限。
我们进行了一项单中心回顾性分析，评估了21例接受阿伐库潘治疗的新诊断或复发AAV患者。共同主要结局是6个月和12个月时的临床缓解。
21例患者中，20例（95.2%）在6个月时实现临床缓解，19例（90.4%）在12个月时维持缓解。从免疫抑制治疗开始至开始使用阿伐库潘的中位时间为12天（四分位间距，5 - 26）。10例患者（47.6%）报告了不良事件，其中8例患者（38.1%）出现肝酶水平升高，为最常见的并发症。9例患者（42.9%）停用了阿伐库潘。尽管早期停药，但这些患者在6个月时的临床缓解率、12个月时的维持缓解率与继续使用阿伐库潘的患者相当，且糖皮质激素剂量有所减少。
观察到不良事件发生率高，尤其是肝酶升高，且阿伐库潘频繁早期停药。然而，无论阿伐库潘是否停药，均取得了良好的临床结局并减少了糖皮质激素剂量。有必要进一步研究以验证阿伐库潘在临床实践中的最佳使用方法。
