Service de Médecine Interne, Centre National de Référence Vascularites Nécrosantes et Sclérodermie Systémique, Hôpital Cochin, AP-HP, Université Paris Descartes, INSERM U 1060, Paris, France.
J Autoimmun. 2013 Jun;43:60-9. doi: 10.1016/j.jaut.2013.03.003. Epub 2013 Apr 13.
The purpose of this study was to assess the outcomes of 118 patients with eosinophilic granulomatosis with polyangiitis (EGPA) enrolled in 2 prospective, randomized, open-label clinical trials (1994-2005), with or without Five-Factor Score (FFS)-defined poor-prognosis factors, focusing on survival, disease-free survival, relapses, clinical and laboratory findings, therapeutic responses, and factors predictive of relapse. Forty-four patients with FFS ≥ 1 were assigned to receive 6 or 12 cyclophosphamide pulses plus corticosteroids and the seventy-four with FFS = 0 received corticosteroids alone, with immunosuppressant adjunction when corticosteroids failed. Patients were followed (2005-2011) under routine clinical care in an extended study and data were recorded prospectively. Mean ± SD follow-up was 81.3 ± 39.6 months. Among the 118 patients studied, 29% achieved long-term remission and 10% died. Among the 115 patients achieving a first remission, 41% experienced ≥1 relapses, 26.1 ± 26.8 months after treatment onset, with 57% of relapses occurring when corticosteroid-tapering reached <10 mg/day. Treatment achieved new remissions in >90%, but relapses recurred in 38%. Overall survival was good, reaching 90% at 7 years, regardless of baseline severity. Age ≥65 years was the only factor associated with a higher risk of death during follow-up. The risk of relapse was higher for patients with anti-myeloperoxidase antibodies and lower for those with >3000 eosinophils/mm(3). Sequelae remained frequent, usually chronic asthma and peripheral neuropathy. In conclusion, EGPA patients' survival rate is very good when treatment is stratified according to the baseline FFS. Relapses are frequent, especially in patients with anti-myeloperoxidase antibodies and baseline eosinophilia <3000/mm(3).
本研究旨在评估纳入 2 项前瞻性、随机、开放性临床试验（1994-2005 年）的 118 例嗜酸性肉芽肿性多血管炎（EGPA）患者的结局，这些患者存在或不存在五因素评分（FFS）定义的不良预后因素，重点关注生存、无疾病生存、复发、临床和实验室发现、治疗反应以及预测复发的因素。44 例 FFS≥1 的患者接受 6 或 12 个环磷酰胺脉冲加皮质类固醇治疗，74 例 FFS=0 的患者接受单独皮质类固醇治疗，当皮质类固醇治疗失败时加用免疫抑制剂。在一项扩展研究中，患者在常规临床护理下进行随访（2005-2011 年），数据前瞻性记录。平均随访时间为 81.3±39.6 个月。在 118 例研究患者中，29%达到长期缓解，10%死亡。在 115 例达到首次缓解的患者中，41%经历≥1 次复发，治疗开始后 26.1±26.8 个月，57%的复发发生在皮质类固醇逐渐减量至<10mg/天时。治疗使超过 90%的患者获得新的缓解，但 38%的患者复发。总体生存良好，7 年时达到 90%，与基线严重程度无关。年龄≥65 岁是随访期间死亡风险增加的唯一因素。抗髓过氧化物酶抗体阳性的患者复发风险较高，而嗜酸性粒细胞计数>3000/mm3 的患者复发风险较低。后遗症仍然很常见，通常是慢性哮喘和周围神经病。总之，根据基线 FFS 分层治疗，EGPA 患者的生存率非常好。复发很常见，尤其是在抗髓过氧化物酶抗体阳性和基线嗜酸性粒细胞计数<3000/mm3 的患者中。
