Marchese Alessandra, Frizzelli Annalisa, Accogli Rocco, Aluia Agnese, Tessitore Carolina, Bondarenko Olha, Caramori Gaetano, Chetta Alfredo, Aiello Marina
Pneumology, Department of Medicine and Surgery, Parma, Italy.
Curr Med Res Opin. 2025 May;41(5):761-766. doi: 10.1080/03007995.2025.2517693. Epub 2025 Jun 11.
Eosinophilic granulomatosis with polyangiitis (EGPA) is a small-vessel vasculitis primarily affecting individuals with asthma and hypereosinophilia. Among the available treatment options for EGPA, biological therapies such as Mepolizumab have emerged for controlling eosinophilic inflammation and reducing corticosteroid dependence. Mepolizumab is a humanized monoclonal antibody that inhibits the interleukin-5 binding to its alpha-subunit receptor, which is predominantly expressed in human eosinophils. While Mepolizumab effectively reduces exacerbations and corticosteroid use, a significant proportion of patients continue to experience disease activity and relapses. Moreover, the implications of treatment discontinuation remain poorly understood.
We present a clinical case of a 38 year-old patient diagnosed with anti-neutrophil cytoplasmic antibody (ANCA)-negative EGPA in 2015 based on clinical and laboratory criteria. The patient underwent a treatment regimen that included Methotrexate, Mepolizumab, and oral corticosteroids, achieving significant improvement in respiratory symptoms. However, approximately seven months after discontinuing a five-year treatment with Mepolizumab due to personal choice, the patient experienced sudden, progressively worsening and permanent blindness in the right eye.
The permanent blindness observed in our patient may be associated with Mepolizumab withdrawal. Currently, there is limited clinical evidence regarding the effects of biologic therapy discontinuation, even in patients with stable diseases. To our knowledge, this is the first reported case of EGPA-associated blindness following Mepolizumab withdrawal. Our case underscores the need for long-term monitoring and careful risk assessment before considering treatment discontinuation, even in the absence of active symptoms.
嗜酸性肉芽肿性多血管炎（EGPA）是一种主要影响哮喘和嗜酸性粒细胞增多患者的小血管血管炎。在EGPA的现有治疗选择中，诸如美泊利单抗等生物疗法已出现，用于控制嗜酸性粒细胞炎症并减少对皮质类固醇的依赖。美泊利单抗是一种人源化单克隆抗体，可抑制白细胞介素-5与其α亚基受体结合，该受体主要在人类嗜酸性粒细胞中表达。虽然美泊利单抗有效减少发作次数和皮质类固醇的使用，但仍有很大一部分患者继续经历疾病活动和复发。此外，治疗中断的影响仍知之甚少。
我们介绍了一例临床病例，一名38岁患者在2015年根据临床和实验室标准被诊断为抗中性粒细胞胞浆抗体（ANCA）阴性的EGPA。该患者接受了包括甲氨蝶呤、美泊利单抗和口服皮质类固醇的治疗方案，呼吸症状有显著改善。然而，由于个人选择，在停用美泊利单抗五年治疗约七个月后，该患者右眼突然出现逐渐加重的永久性失明。
我们患者中观察到的永久性失明可能与停用美泊利单抗有关。目前，关于生物治疗中断的影响，即使在病情稳定的患者中，临床证据也有限。据我们所知，这是第一例报告的停用美泊利单抗后出现EGPA相关失明的病例。我们的病例强调，即使在没有活动症状的情况下，在考虑停止治疗之前也需要进行长期监测和仔细的风险评估。
