Ushio Yusuke, Shimada Hiromi, Wakiya Risa, Nakashima Shusaku, Miyagi Taichi, Sugihara Koichi, Mino Rina, Mizusaki Mao, Chujo Kanako, Manabe Naoto, Kadowaki Norimitsu, Dobashi Hiroaki
Division of Hematology, Faculty of Medicine, Rheumatology and Respiratory Medicine, Department of Internal Medicine, Kagawa University, 1750-1 Ikenobe, Miki-cho, Kita-gun, Kagawa, 761-0793, Japan.
BMC Rheumatol. 2025 Aug 11;9(1):99. doi: 10.1186/s41927-025-00555-2.
Avacopan, a selective oral C5a receptor antagonist, was approved for the treatment of microscopic polyangiitis (MPA) and granulomatosis with polyangiitis (GPA) in 2021. However, there are still limited reports on its efficacy and safety in real-world settings, specifically regarding its impact on the Vasculitis Damage Index (VDI), and its effects on serum biomarkers are poorly understood. This study aimed to evaluate the efficacy and safety of avacopan in remission induction therapy for MPA/GPA in a real-world setting, as well as its effect on serum C5a levels.
This retrospective study investigated patients with MPA/GPA who received remission induction therapy with a 6-month follow-up at our institution, comparing those who received avacopan with those who did not. Efficacy and safety were evaluated by comparing the remission rate, changes in Birmingham Vasculitis Activity Score (BVAS) and VDI score after 6 months, daily glucocorticoid (GC) dose, and incidence of adverse events (AEs). Changes in serum C5a levels, measured using ELISA, were compared between both groups at baseline and 3 months.
A total of 66 patients with MPA/GPA were included, with 14 and 52 patients in the avacopan and non-avacopan groups, respectively. The remission rate and decrease in BVAS was comparable between both groups. However, those who received avacopan had a significantly smaller increase in VDI score, significantly lower daily GC dose at 1, 3, and 6 months, and significantly lower incidence of GC-related AEs within 6 months. Serum C5a levels did not significantly change in the avacopan group but significantly decreased in the non-avacopan group. Remission was achieved in the avacopan group regardless of whether serum C5a decreased or increased.
Treatment with avacopan appears to effectively suppress the increase in VDI score, enable reduced GC dosage, and lower the incidence of GC-related AEs during remission induction therapy for MPA/GPA in a real-world setting. Furthermore, avacopan may suppress disease activity regardless of serum C5a levels.
Not applicable.
阿伐库潘是一种选择性口服C5a受体拮抗剂，于2021年被批准用于治疗显微镜下多血管炎（MPA）和肉芽肿性多血管炎（GPA）。然而，关于其在实际临床环境中的疗效和安全性的报道仍然有限，特别是关于其对血管炎损伤指数（VDI）的影响，以及其对血清生物标志物的影响尚不清楚。本研究旨在评估阿伐库潘在实际临床环境中对MPA/GPA缓解诱导治疗的疗效和安全性，以及其对血清C5a水平的影响。
本回顾性研究调查了在我院接受缓解诱导治疗并随访6个月的MPA/GPA患者，比较接受阿伐库潘治疗的患者和未接受阿伐库潘治疗的患者。通过比较缓解率、6个月后伯明翰血管炎活动评分（BVAS）和VDI评分的变化、每日糖皮质激素（GC）剂量以及不良事件（AE）的发生率来评估疗效和安全性。使用酶联免疫吸附测定法（ELISA）测量的血清C5a水平在基线和3个月时在两组之间进行比较。
共纳入66例MPA/GPA患者，阿伐库潘组和非阿伐库潘组分别有14例和52例患者。两组之间的缓解率和BVAS降低情况相当。然而，接受阿伐库潘治疗的患者VDI评分增加明显较小，在1、3和6个月时每日GC剂量明显较低，并且在6个月内GC相关AE的发生率明显较低。阿伐库潘组血清C5a水平无明显变化，而非阿伐库潘组血清C5a水平明显降低。无论血清C5a降低还是升高，阿伐库潘组均实现了缓解。
在实际临床环境中，阿伐库潘治疗似乎能有效抑制MPA/GPA缓解诱导治疗期间VDI评分的增加，减少GC用量，并降低GC相关AE的发生率。此外，无论血清C5a水平如何，阿伐库潘可能都能抑制疾病活动。
不适用。
