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Real-World experience with efgartigimod in patients with myasthenia gravis

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Abstract

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.

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Funding

No funding was received for conducting this study.

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Authors and Affiliations

  1. Faculty of Medicine & Health Sciences, Tel Aviv University, Tel Aviv-Yafo, Israel

    Lior Fuchs, Ifat Vigiser, Amir Dori & Arnon Karni

  2. Department of Neurology, Rambam Medical Center, Haifa, Israel

    Shahar Shelly

  3. Rappaport Faculty of Medicine, Technion-Israel Institute of Technology, Haifa, Israel

    Shahar Shelly

  4. Neuroimmunology and MS Unit, Neurology Institute, Tel Aviv Sourasky Medical Center, Tel Aviv, Israel

    Ifat Vigiser, Hadar Kolb, Keren Regev & Arnon Karni

  5. Department of Neurology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel

    Yoel Schwartzmann & Adi Vaknin-Dembinsky

  6. Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel

    Adi Vaknin-Dembinsky

  7. Department of Neurology, Sheba Medical Center, Ramat-Gan, Israel

    Amir Dori

  8. Sagol School of Neuroscience, Tel Aviv University, Tel Aviv, Israel

    Arnon Karni

  9. The Neurology Institute, Tel Aviv Sourasky Medical Center, 6 Weizmann Street, 6423906, Tel Aviv, Israel

    Arnon Karni

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  1. Lior Fuchs
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  2. Shahar Shelly
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  3. Ifat Vigiser
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Correspondence to Arnon Karni.

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Fuchs, L., Shelly, S., Vigiser, I. et al. Real-World experience with efgartigimod in patients with myasthenia gravis. J Neurol (2024). https://doi.org/10.1007/s00415-024-12293-5

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  • DOI: https://doi.org/10.1007/s00415-024-12293-5

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