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度普利尤单抗用于治疗嗜酸性肉芽肿性多血管炎相关的难治性慢性鼻-鼻窦炎。

Dupilumab for refractory chronic rhinosinusitis in eosinophilic granulomatosis with polyangiitis.

作者信息

Padoan Roberto, Davanzo Federica, Iorio Luca, Saccardo Tommaso, Roccuzzo Giuseppe, Zampollo Sonny, Nicolai Piero, Doria Andrea, Ottaviano Giancarlo

机构信息

Rheumatology Unit, Department of Medicine, University of Padua, Padua, Italy.

Otolaryngology Section, Department of Neurosciences, University of Padua, Padua, Italy.

出版信息

Rheumatology (Oxford). 2025 May 1;64(5):2892-2901. doi: 10.1093/rheumatology/keae554.

Abstract

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 (PROMs) 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 PROMs and nasal cytology. Complete response was defined by 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 in the AAV-PRO score. Nasal cytology revealed reductions in eosinophils and neutrophils counts. Adverse events occurred in 44.4%, including hypereosinophilia in two cases, which led to dupilumab discontinuation.

CONCLUSION

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.

摘要

目的

尽管有当前的治疗方法,但嗜酸性肉芽肿性多血管炎(EGPA)常与伴有鼻息肉的难治性慢性鼻-鼻窦炎(CRSwNP)相关。度普利尤单抗在治疗重度且未得到控制的CRSwNP方面显示出疗效,患者报告的结局指标(PROMs)和客观测量指标均有改善。本研究旨在评估度普利尤单抗在EGPA患者难治性CRSwNP中的疗效和安全性。

方法

对2021年至2023年期间接受度普利尤单抗治疗的EGPA患者进行了一项前瞻性观察性研究。纳入处于血管炎表现长期缓解期但仍有活动性CRSwNP的患者。进行了临床、生物学和鼻科学评估,同时进行了PROMs和鼻细胞学检查。完全缓解定义为BVAS = 0且泼尼松剂量≤4mg/天,部分缓解定义为BVAS = 0且泼尼松剂量>4mg/天。

结果

纳入了9例EGPA患者。3个月后,55.6%的患者达到完全缓解,12个月时增至83.3%。鼻部症状和患者报告的结局有显著改善,在12个月内疗效持续。还观察到生活质量有所改善,AAV-PRO评分显著降低。鼻细胞学检查显示嗜酸性粒细胞和中性粒细胞计数减少。44.4%的患者发生了不良事件,包括2例出现嗜酸性粒细胞增多症,导致停用度普利尤单抗。

结论

度普利尤单抗是EGPA中重度难治性耳鼻喉表现的有效治疗选择,因为它能改善症状、减轻炎症并带来更好的生活质量。然而,需要仔细选择患者并进行监测,以尽量减少不良事件并优化治疗结果。

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