National Referral Center for Rare Systemic Autoimmune Diseases, Université Paris Descartes, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris (APHP), 27, rue du faubourg Saint-Jacques, Paris, Cedex 14 75679, France; Division of Rheumatology, Department of Internal Medicine Specialties, Geneva University Hospitals, Switzerland.
National Referral Center for Rare Systemic Autoimmune Diseases, Université Paris Descartes, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris (APHP), 27, rue du faubourg Saint-Jacques, Paris, Cedex 14 75679, France; Present address: Vasculitis Clinic, Mount Sinai Hospital, University of Toronto, Toronto, Canada.
Semin Arthritis Rheum. 2021 Apr;51(2):339-346. doi: 10.1016/j.semarthrit.2021.02.002. Epub 2021 Feb 10.
To describe the characteristics and long-term outcomes of patients with granulomatosis with polyangiitis (GPA) from the French Vasculitis Study Group database.
Patients' clinical and laboratory characteristics, Birmingham Vasculitis Activity Score (BVAS)-assessed disease activity, malignancies, opportunistic infections, and vital status were collected at diagnosis and each visit. Estimated probabilities and predictors of overall (OS) and relapse-free survival (RFS) were analyzed by Cox regression.
We enrolled 795 newly diagnosed patients, followed for a median of 3.5 years. Initial clinical manifestations involved ear, nose & throat (ENT; 80%), lungs (68%) and kidneys (56%). Among the 728 available ELISA results, 75.0% were PR3-ANCA-positive, 16.5% MPO-ANCA-positive and 62 (8.5%) ANCA-negative. Relapses occurred in 394 (50%) patients, involving ≥1 organ(s) affected at onset in 179 (46%), mainly ENT, lungs and kidneys, with mean BVAS 10.2 points below that at diagnosis (p<0.001). Five- and 10-year RFS rates were 37% and 17%, respectively. PR3-ANCA-positivity independently predicted relapse (p = 0.05) and prolonged survival (p = 0.038). OS-but not RFS-improved significantly over time (p<0.001); 10-year OS reached 88.2% (95% CI 83.9 to 92.7) for the 660 patients diagnosed after 2000. Infections were the main causes of death. Malignancy or opportunistic infection each occurred in ≤5% of the patients.
Survival has improved dramatically over the last decades but the high relapse rate remains a major concern for GPA patients, once again stressing the need for therapeutic strategy optimization to lower it. PR3-ANCA-positivity was associated with increased probability of relapse and survival.
描述法国血管炎研究组数据库中肉芽肿性多血管炎（GPA）患者的特征和长期预后。
收集患者的临床和实验室特征、Birmingham 血管炎活动评分（BVAS）评估的疾病活动度、恶性肿瘤、机会性感染和生存状况，分别在诊断时和每次就诊时进行评估。通过 Cox 回归分析总生存（OS）和无复发生存（RFS）的估计概率和预测因素。
我们纳入了 795 例新诊断的 GPA 患者，中位随访时间为 3.5 年。最初的临床表现涉及耳部、鼻部和喉部（ENT；80%）、肺部（68%）和肾脏（56%）。在 728 份可获得的 ELISA 结果中，75.0%为 PR3-ANCA 阳性，16.5%为 MPO-ANCA 阳性，62 份（8.5%）为 ANCA 阴性。394 例（50%）患者发生了复发，179 例（46%）复发涉及≥1个初发受累器官，主要为 ENT、肺部和肾脏，平均 BVAS 较初诊时降低 10.2 分（p<0.001）。5 年和 10 年的 RFS 率分别为 37%和 17%。PR3-ANCA 阳性独立预测复发（p=0.05）和延长生存（p=0.038）。OS 但不是 RFS 随时间显著改善（p<0.001）；660 例 2000 年后诊断的患者中，10 年 OS 达到 88.2%（95%CI 83.9 至 92.7）。感染是死亡的主要原因。恶性肿瘤或机会性感染各发生在≤5%的患者中。
过去几十年，生存状况显著改善，但高复发率仍是 GPA 患者的主要关注点，再次强调需要优化治疗策略以降低复发率。PR3-ANCA 阳性与更高的复发概率和生存相关。
