Recommendations for the treatment of myasthenia gravis (MG) have been difficult to develop because of limited evidence from large randomized controlled trials. New drugs and treatment approaches have recently been shown to be effective in phase 3 studies in seropositive generalized (g) MG. One such drug is efgartigimod, a human-Fc-fragment of IgG1, with a high affinity for the endosomal FcRn. We conducted a multicenter study to evaluate the real-world clinical and safety effects of efgartigimod in 22 gMG patients. We evaluated the strategies for the timing of re-treatment with it. The participants received a total of 59 efgartigimod -treatment cycles. The median number of cycles was 2 (range 1–6). Twenty patients (86.3%) improved by at least 2 MG-ADL points after the first treatment cycle. The median MG-ADL score at baseline was 6.5 (range: 3–17) and 2.5 (range: 0–9) post-treatment (p < 0.001). A consistent improvement of at least 2 points in the MG-ADL score after each cycle occurs in 18 patients. The effect duration of the treatment was usually between 4 and 12 weeks. Two major clinical patterns of treatment response were found. Treatment with efgartigimod was also associated with significant reductions of prednisone doses Overall, the treatment was safe and associated with only minor adverse events. The single fatality was apparently due tosevere respiratory failure. We found that efgartigimod is clinically effective, may be used as a steroid sparing agent and is generally safe for gMG patients. We recommend a personalized preventive treatment approach until clinical stabilization, followed by discontinuation and periodic evaluations.

由于大型随机对照试验的证据有限，很难制定重症肌无力 (MG) 的治疗建议。最近，新药物和治疗方法在血清阳性全身性 (g) 重症肌无力的 3 期研究中被证明是有效的。其中一种药物是 efgartigimod，一种 IgG1 的人 Fc 片段，与内体 FcRn 具有高亲和力。我们进行了一项多中心研究，以评估 efgartigimod 在 22 名 gMG 患者中的真实临床和安全性效果。我们评估了再治疗时机的策略。参与者总共接受了 59 个 efgartigimod 治疗周期。中位周期数为 2（范围 1-6）。第一个治疗周期后，20 名患者 (86.3%) 的 MG-ADL 改善至少 2 分。基线时的中位 MG-ADL 评分为 6.5（范围：3-17）和治疗后 2.5（范围：0-9）（p  < 0.001）。 18 名患者在每个周期后 MG-ADL 评分持续改善至少 2 分。治疗的效果持续时间通常为4至12周。发现了治疗反应的两种主要临床模式。 efgartigimod 治疗还与泼尼松剂量的显着减少相关。总体而言，该治疗是安全的，并且仅与轻微的不良事件相关。单例死亡显然是由于严重呼吸衰竭造成的。我们发现 efgartigimod 在临床上有效，可用作类固醇节约剂，并且对于 gMG 患者通常是安全的。我们建议采用个性化的预防性治疗方法，直到临床稳定，然后停药并定期评估。