National Referral Center for Rare Systemic Autoimmune Diseases, Université de Paris, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris (APHP), Paris, France.
Division of Rheumatology, Department of Internal Medicine Specialties, Geneva University Hospitals, Switzerland.
Rheumatology (Oxford). 2022 May 30;61(6):2464-2471. doi: 10.1093/rheumatology/keab719.
To describe the main features at diagnosis and evolution over time of patients with localized granulomatosis with polyangiitis (L-GPA) compared with those of systemic GPA (S-GPA).
EULAR definitions of L-GPA, i.e. upper and/or lower respiratory tract involvement, and S-GPA were applied to patients from the French Vasculitis Study Group Registry. L-GPA and S-GPA patients' characteristics at diagnosis and long-term outcomes were analysed and compared.
Among the 795 Registry patients, 79 (10%) had L-GPA. Their main clinical manifestations were rhinitis, lung nodules, sinusitis and otitis. L-GPA vs S-GPA patients at diagnosis, respectively, were younger, more frequently had saddle nose deformity or subglottic stenosis and were less often PR3-ANCA-positive. L-GPA vs S-GPA induction therapy less frequently included CYC but more often a combination of MTX and glucocorticoids; 64% of MTX-treated patients experienced disease progression within 18 months post-diagnosis. L- and S-GPA patients' estimated relapse-free-survival probabilities, relapse rates and refractory disease rates at each time point were comparable, but L-GPA patients had more frequent ENT and lung relapses, and higher overall survival rates (P<0.02). Over a median follow-up of 3.5 years, 18 (22.8%) L-GPA progressed to S-GPA, either as a relapse after a period in remission or more frequently in the context of refractory disease. L-GPA patients experienced more ENT-related damage.
The relapse risks of L-GPA and S-GPA were similar, but relapse patterns differed and L-GPA overall survival rate was higher. About one-quarter of L-GPA patients developed S-GPA over time, but without end-stage organ involvement.
描述局限性肉芽肿伴多血管炎（L-GPA）患者与系统性 GPA（S-GPA）患者在诊断时的主要特征和随时间的演变。
采用 EULAR 对 L-GPA（即上呼吸道和/或下呼吸道受累）和 S-GPA 的定义，对法国血管炎研究组登记处的患者进行应用。分析和比较 L-GPA 和 S-GPA 患者的诊断时特征和长期结局。
在登记处的 795 例患者中，有 79 例（10%）为 L-GPA。其主要临床表现为鼻炎、肺结节、鼻窦炎和中耳炎。与 S-GPA 患者相比，L-GPA 患者在诊断时更年轻，更常出现鞍鼻畸形或声门下狭窄，抗蛋白酶 3 抗体（PR3-ANCA）阳性率较低。与 S-GPA 相比，L-GPA 患者诱导治疗较少使用环磷酰胺（CYC），但更常使用甲氨蝶呤（MTX）和糖皮质激素联合治疗；64%的 MTX 治疗患者在诊断后 18 个月内出现疾病进展。在每个时间点，L-GPA 和 S-GPA 患者的无复发生存率、复发率和难治性疾病率估计值均相似，但 L-GPA 患者更常出现 ENT 和肺部复发，总生存率更高（P<0.02）。在中位随访 3.5 年期间，18 例（22.8%）L-GPA 进展为 S-GPA，要么是在缓解期后复发，要么更常发生在难治性疾病的情况下。L-GPA 患者出现更多的 ENT 相关损害。
L-GPA 和 S-GPA 的复发风险相似，但复发模式不同，L-GPA 的总生存率更高。随着时间的推移，约四分之一的 L-GPA 患者发展为 S-GPA，但没有终末期器官受累。
