Draibe Juliana, Espigol-Frigolé Georgina, Cid Maria Cinta, Prados M C, Guillén E, Villacorta J, Vega C, Martins J, daSilva I, Martin-Gomez Mª Adoración, Huerta A, Martinez-Valenzuela L, Morales Enrique
Department of Nephrology, Bellvitge University Hospital, Bellvitge Biomedical Research Institute (IDIBELL), Hospitalet de Llobregat, Barcelona, Spain.
Department of Autoimmune Diseases, Hospital Clinic, University of Barcelona, Institut d'Investigacions Biomèdiques August Pi I Sunyer (IDIBAPS), Barcelona, Spain.
Rheumatology (Oxford). 2025 Apr 1;64(4):2019-2026. doi: 10.1093/rheumatology/keae534.
ANCA-associated vasculitis (AAV) is a group of chronic diseases with relapses that associate organic damage because of the disease and its treatment. Avacopan is a new treatment indicated for AAV. We present the first experiences with avacopan in Spain as part of an Early Access program.
Patients with AAV who started avacopan between June 2022 and September 2023 were included. For comparison, a historical cohort of patients diagnosed with AAV around the same time and treated without avacopan was also included.
Twenty-nine patients treated with avacopan were analysed. Twelve patients (41.4%) were male, and median age was 56 years. Most patients were ANCA MPO positive (21/29, 72.4%). The most frequently affected organ was the kidney (23/29, 79.31%), with a mean estimated glomerular filtration rate (eGFR) of 23.2 ml/min. Mean follow-up was 456.8 (±181.7) days with a remission rate of 86.2%. eGFR increased from 23.2 (11.2) to 38.38 (18.55) ml/min after 12 months of diagnosis. Two patients had adverse events related to avacopan (severe neutropenia and a gastrointestinal affectation), 13 infections were reported and one death. Patients treated with avacopan received a significantly lower cumulative dose of prednisone at 6 and 12 months (P-values of 0.02 and <0.01, respectively) compared with historical controls. The evolution of GFR at 1 year of follow-up and the incidence of relapse were similar in both groups.
The combination of avacopan with standard immunosuppressive therapy presents a good safety profile and provides added value by contributing to the control of AAV activity, increase GFR and removal of steroids.
抗中性粒细胞胞浆抗体相关性血管炎（AAV）是一组伴有复发的慢性疾病，因其疾病本身及其治疗会导致器官损害。阿伐可泮是一种用于治疗AAV的新疗法。作为早期准入计划的一部分，我们介绍了在西班牙使用阿伐可泮的首批经验。
纳入2022年6月至2023年9月开始使用阿伐可泮的AAV患者。为作比较，还纳入了一组在同一时间左右被诊断为AAV且未接受阿伐可泮治疗的历史队列患者。
对29例接受阿伐可泮治疗的患者进行了分析。12例患者（41.4%）为男性，中位年龄为56岁。大多数患者抗中性粒细胞胞浆抗体髓过氧化物酶（ANCA MPO）呈阳性（21/29，72.4%）。最常受累的器官是肾脏（23/29，79.31%），平均估计肾小球滤过率（eGFR）为23.2 ml/分钟。平均随访时间为456.8（±181.7）天，缓解率为86.2%。诊断12个月后，eGFR从23.2（11.2）升至38.38（18.55）ml/分钟。两名患者出现与阿伐可泮相关的不良事件（严重中性粒细胞减少和胃肠道感染），报告了13例感染，1例死亡。与历史对照相比，接受阿伐可泮治疗的患者在6个月和12个月时接受的泼尼松累积剂量显著更低（P值分别为0.02和<0.01）。两组在随访1年时的肾小球滤过率变化和复发率相似。
阿伐可泮与标准免疫抑制疗法联合使用具有良好的安全性，通过有助于控制AAV活动、提高肾小球滤过率和减少类固醇使用提供了附加价值。
