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Introduction <p>Generalized myasthenia gravis (gMG) is a rare chronic autoimmune disorder of the neuromuscular junction caused by pathogenic autoantibodies directed against a postsynaptic target. The therapeutic landscape of gMG has recently expanded with the introduction of FcRn inhibitors. This study aimed to assess the real-world effectiveness and safety of efgartigimod (EFG) in AChR-positive gMG patients who failed or were intolerant to intravenous immunoglobulin (IVIg).</p> Methods <p>EFG was administered as four consecutive weekly intravenous infusions at 10&#xa0;mg/kg. Treatment efficacy was evaluated using the Myasthenia Gravis Activity of Daily Living (MG-ADL) and Myasthenia Gravis quantitative (QMG) scales at baseline and after 4 weeks. Incidence of adverse events and prednisone use were collected at each time point.</p> Results <p>Thirteen patients (6 women and 7 men, mean age 52.9 years) received EFG following IVIg therapy. After one treatment cycle, both MG-ADL and QMG scores showed significant clinical improvement. MG-ADL responder rate (MG-ADL reduction &gt; 2) was 84.6% while 69.2% on QMG (QMG reduction &gt; 3). Minimal symptom expression was reached in one patient, accompanied by a mean reduction in daily prednisone dose of 6.9&#xa0;mg.</p> Conclusion <p>In this real-world cohort, efgartigimod demonstrated a rapid and meaningful clinical benefit with a favorable tolerability profile in patients with AChR-positive gMG after IVIg failure or intolerance. These findings support the potential role of EFG as an effective therapeutic option in this difficult-to-treat population.</p>

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Efgartigimod in Patients with Generalized Myasthenia Gravis Refractory or Intolerant to IVIg

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年4月1日

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  • Flora D’Amico,
  • Sofia Campo,
  • Nicasio Rini,
  • Claudia Vinciguerra,
  • Liliana Bevilacqua,
  • Carmen Erra,
  • Paolo Barone,
  • Francesco Tuccillo,
  • Francesco Habetswallner,
  • Filippo Brighina,
  • Vincenzo Di Stefano

摘要

Introduction

Generalized myasthenia gravis (gMG) is a rare chronic autoimmune disorder of the neuromuscular junction caused by pathogenic autoantibodies directed against a postsynaptic target. The therapeutic landscape of gMG has recently expanded with the introduction of FcRn inhibitors. This study aimed to assess the real-world effectiveness and safety of efgartigimod (EFG) in AChR-positive gMG patients who failed or were intolerant to intravenous immunoglobulin (IVIg).

Methods

EFG was administered as four consecutive weekly intravenous infusions at 10 mg/kg. Treatment efficacy was evaluated using the Myasthenia Gravis Activity of Daily Living (MG-ADL) and Myasthenia Gravis quantitative (QMG) scales at baseline and after 4 weeks. Incidence of adverse events and prednisone use were collected at each time point.

Results

Thirteen patients (6 women and 7 men, mean age 52.9 years) received EFG following IVIg therapy. After one treatment cycle, both MG-ADL and QMG scores showed significant clinical improvement. MG-ADL responder rate (MG-ADL reduction > 2) was 84.6% while 69.2% on QMG (QMG reduction > 3). Minimal symptom expression was reached in one patient, accompanied by a mean reduction in daily prednisone dose of 6.9 mg.

Conclusion

In this real-world cohort, efgartigimod demonstrated a rapid and meaningful clinical benefit with a favorable tolerability profile in patients with AChR-positive gMG after IVIg failure or intolerance. These findings support the potential role of EFG as an effective therapeutic option in this difficult-to-treat population.