Uchi Takafumi, Konno Shingo, Kihara Hideo, Sugimoto Hideki
Neurology, Toho University Ohashi Medical Center, Tokyo, JPN.
Cureus. 2024 Nov 20;16(11):e74117. doi: 10.7759/cureus.74117. eCollection 2024 Nov.
This case study describes the successful use of ravulizumab in treating a 71-year-old woman with myasthenia gravis experiencing a myasthenic crisis. The patient initially presented with hypernasality and dysphagia; her medical history included untreated, complicated type 1 diabetes. The patient received several treatments approved in Japan for general myasthenia gravis, including immunoadsorption plasmapheresis, tacrolimus, intravenous immunoglobulin, and intravenous methylprednisolone. Despite these treatments, the patient's condition fluctuated, and she ultimately experienced a myasthenic crisis, which required ventilator management. The introduction of ravulizumab, a complement inhibitor targeting the complement protein C5, marked a significant turning point in the patient's treatment. Ravulizumab improved the patient's respiratory function, allowing ventilator weaning and discharge from the hospital. To the best of our knowledge, this case is the first report of successful weaning from mechanical ventilation after treatment with ravulizumab in a patient with a myasthenic crisis. This finding suggests the efficacy of ravulizumab in the management of refractory myasthenia gravis and highlights the potential of novel therapeutic approaches and combination strategies in improving the condition. Future clinical trials are needed to assess the efficacy and safety of ravulizumab in larger, more diverse populations of patients with myasthenia gravis.
本病例研究描述了ravulizumab在治疗一名患有重症肌无力危象的71岁女性患者中的成功应用。患者最初表现为鼻音过重和吞咽困难;其病史包括未经治疗的复杂性1型糖尿病。该患者接受了日本批准的几种用于治疗一般重症肌无力的疗法,包括免疫吸附血浆置换、他克莫司、静脉注射免疫球蛋白和静脉注射甲泼尼龙。尽管进行了这些治疗,患者的病情仍有波动,最终出现了重症肌无力危象,需要呼吸机管理。引入ravulizumab,一种靶向补体蛋白C5的补体抑制剂,标志着患者治疗过程中的一个重要转折点。Ravulizumab改善了患者的呼吸功能,使其能够脱机并出院。据我们所知,本病例是关于ravulizumab治疗重症肌无力危象患者后成功脱机的首例报告。这一发现表明ravulizumab在治疗难治性重症肌无力方面的疗效,并突出了新型治疗方法和联合策略在改善病情方面的潜力。未来需要进行临床试验,以评估ravulizumab在更大、更多样化的重症肌无力患者群体中的疗效和安全性。