Objectives Eosinophilic granulomatosis with polyangiitis (EGPA) is frequently associated with refractory chronic rhinosinusitis with nasal polyps (CRSwNP), despite current treatments. Dupilumab demonstrated efficacy in the treatment of severe and uncontrolled CRSwNP, with improvements in patient-reported outcome measures and in objective measurements. This study aims to evaluate efficacy and safety of dupilumab in refractory CRSwNP in EGPA patients. Methods A prospective observational study was conducted on EGPA patients treated with dupilumab between 2021 and 2023. Patients in a phase of prolonged remission of vasculitis manifestations but still experiencing active CRSwNP were included. Clinical, biological, and rhinologic evaluations were performed, alongside with patient-reported outcomes measures (PROMs) and nasal cytology. Complete response was defined by Birmingham Vasculitis Activity Score (BVAS)=0 and prednisone dose ≤ 4 mg/day, while partial response by BVAS = 0 and prednisone dose >4 mg/day. Results Nine EGPA patients were included. After 3 months, 55.6% achieved complete response, increasing to 83.3% at 12 months. Nasal symptoms and patient-reported outcomes improved significantly, with sustained efficacy over 12 months. An improvement in quality of life was also observed, with a significant reduction of the AAV-PRO score. Nasal cytology revealed reductions in eosinophils and neutrophils counts. Adverse events occurred in 44.4%, including hypereosinophilia in 2 cases, which led to dupilumab discontinuation. Conclusions Dupilumab is an effective treatment option for severe and refractory ENT manifestations in EGPA, as it improves symptoms, reduces inflammation, and leads to better a quality of life. However, careful patient selection and monitoring are necessary to minimize adverse events and optimize outcomes.

中文翻译：

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度普利尤单抗治疗嗜酸性肉芽肿性多血管炎患者的难治性慢性鼻-鼻窦炎


目的 嗜酸性肉芽肿性多血管炎 （EGPA） 经常与难治性慢性鼻-鼻窦炎伴鼻息肉 （CRSwNP） 相关，尽管目前进行了治疗。Dupilumab 在治疗严重和不受控制的 CRSwNP 方面显示出疗效，患者报告的结果测量和客观测量有所改善。本研究旨在评估 dupilumab 在 EGPA 患者难治性 CRSwNP 中的疗效和安全性。方法 对 2021 年至 2023 年接受 dupilumab 治疗的 EGPA 患者进行了一项前瞻性观察研究。纳入处于血管炎表现长期缓解阶段但仍经历活动性 CRSwNP 的患者。进行临床、生物学和鼻学评估，以及患者报告结果测量 （PROM） 和鼻细胞学。完全缓解定义为伯明翰血管炎活动评分 （BVAS）=0 且泼尼松剂量≤ 4 mg/天，而 BVAS = 0 且泼尼松剂量 >4 mg/天的部分缓解。结果 共纳入 9 例 EGPA 患者。3 个月后，55.6% 的患者达到完全缓解，12 个月时增加到 83.3%。鼻部症状和患者报告的结局显著改善，疗效持续超过 12 个月。还观察到生活质量的改善，AAV-PRO 评分显著降低。鼻细胞学显示嗜酸性粒细胞和中性粒细胞计数减少。不良事件发生率为 44.4%，其中 2 例嗜酸性粒细胞增多，导致 dupilumab 停药。结论 Dupilumab 是 EGPA 中严重难治性耳鼻喉科表现的有效治疗选择，因为它可以改善症状、减少炎症并提高生活质量。 然而，仔细选择和监测患者对于最大限度地减少不良事件和优化结果是必要的。