Efgartigimod as Rescue Medication in a Patient with Therapy-Refractory Myasthenic Crisis.

IF 0.9 Q4 CLINICAL NEUROLOGY Case Reports in Neurological Medicine Pub Date : 2024-06-14 eCollection Date: 2024-01-01 DOI:10.1155/2024/9455237
Omar Alhaj Omar, Norma J Diel, Stefan T Gerner, Anna Mück, Hagen B Huttner, Heidrun H Krämer-Best
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Abstract

Myasthenic crises (MC) are potentially life-threatening acute exacerbations of myasthenia gravis (MG) characterized by profound muscle weakness, bulbar symptoms, and potential for respiratory failure. Intravenous immunoglobulins (IVIG) and plasma exchange (PLEX) are conventional treatments for myasthenic exacerbations. Recently, new therapeutic options for generalized acetylcholine-receptor antibody positive (AchR+) MG were approved as an add-on therapy. They mainly consist of complement C5 inhibitors such as eculizumab and ravulizumab and neonatal Fc receptor antagonists such as efgartigimod with the approval of more options pending, e.g., zilucoplan and rozanolixizumab. More therapeutic options are in the pipeline. Although the data show a quick and reliable treatment response, these medications have not been studied for the therapy of myasthenic crisis. We present the case of a 57-year-old male with his first episode of generalized myasthenia gravis (MG) and positive acetylcholine-receptor antibodies (AchR+) who was transferred to our neurological intensive care unit with worsening generalized weakness, dysphagia, and respiratory distress. The crisis was triggered by pneumonia due to dysphagia. He was diagnosed with myasthenic crisis and treated with intravenous pyridostigmine, plasmapheresis (PLEX), and continued prednisone. Initial improvement was followed by deterioration, requiring readmission and additional PLEX. After a further decline, efgartigimod was administered, leading to significant improvement within 48 hours, as evidenced by reduced MG-ADL and QMG scores. The patient continued to improve and was stable enough for transfer to a rehabilitation facility. This case illustrates the potential of efgartigimod as a novel treatment for refractory myasthenic crises.

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将依夫加替莫德作为治疗难治性肌无力危象患者的救治药物
肌无力危象(MC)是一种可能危及生命的重症肌无力症(MG)急性加重期,其特点是肌肉极度无力、出现球部症状并可能导致呼吸衰竭。静脉注射免疫球蛋白(IVIG)和血浆置换(PLEX)是治疗肌无力加重的传统方法。最近,针对全身乙酰胆碱受体抗体阳性(AchR+)的 MG 的新疗法被批准作为附加疗法。它们主要包括补体 C5 抑制剂(如 eculizumab 和 ravulizumab)和新生儿 Fc 受体拮抗剂(如 efgartigimod),还有更多治疗方案(如 zilucoplan 和 rozanolixizumab)有待批准。更多的治疗方案正在审批中。尽管数据显示治疗反应迅速可靠,但这些药物尚未用于肌无力危象的治疗研究。我们报告了一例 57 岁男性患者的病例,他是第一次全身性肌无力(MG)发作,乙酰胆碱受体抗体(AchR+)阳性,因全身乏力、吞咽困难和呼吸窘迫恶化而转入我们的神经重症监护病房。由于吞咽困难,肺炎引发了危机。他被诊断为肌无力危象,接受了静脉注射吡啶斯的明、血浆置换术(PLEX)和持续泼尼松治疗。最初病情有所好转,但随后又出现恶化,需要再次入院并追加 PLEX。病情进一步恶化后,患者接受了依加替莫德治疗,48 小时内病情明显好转,MG-ADL 和 QMG 评分均有所下降。患者病情持续好转,稳定到足以转入康复机构。本病例说明了依加替莫德作为一种新型疗法治疗难治性肌无力危象的潜力。
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审稿时长
11 weeks
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