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Background and objectives <p>Acute exacerbations/worsening of generalized myasthenia gravis (gMG) require urgent intervention to prevent progression to life-threatening myasthenic crisis. This multicenter study evaluate the efficacy and safety of efgartigimod in gMG patients experiencing acute exacerbations/worsening to validate its potential as a rapid-acting therapeutic option.</p> Methods <p>In this multicenter retrospective study (Jan 2024–Feb 2025), 61 gMG patients with acute exacerbations/worsening received weekly efgartigimod infusions for 4&#xa0;weeks. Efficacy was assessed at baseline (W0) and weekly throughout the treatment cycle (W1–W7) using Myasthenia Gravis Foundation of America Post-Intervention Status (MGFA-PIS), Quantitative Myasthenia Gravis (QMG), and Myasthenia Gravis Activities of Daily Living (MG-ADL) scores, along with prednisone dosage reductions. Safety assessments were concurrently performed.</p> Results <p>Median age was 56&#xa0;years (range: 39–68&#xa0;years). Pulmonary infection (16/61, 26.2%) was the predominant trigger of myasthenic crisis (MC) red flgs. Median duration from symptom deterioration to efgartigimod initiation was 18&#xa0;days. Clinical meaningful improvement occurred in 81.9% at W1 (post-single dose), 98.1% at W4, and 91.9% at W7. MG-ADL and QMG scores decreased substantially from baseline (W4: 80.86% and 55.81% reduction; W7: 70.78% and 47.76% reduction), Patients with MC red flags achieved comparable outcomes. Greater MG-ADL imrovements were observed in those with thymoma history, severe MGFA classification, prior MC, or MC red flags. No treatment-related allergic reactions, infections, or serious adverse events were observed.</p> Conclusion <p>Efgartigimod demonstrates rapid, significant efficacy in gMG acute exacerbations/worsening, supporting its role as a valuable therapeutic option for this high-risk population.</p>

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Rapid efficacy of efgartigimod for generalized myasthenia gravis patients in acute exacerbations/worsening: multicenter real-world retrospective study

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年2月16日

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  • Jing Lin,
  • Ting Chang,
  • Lianchen Xiao,
  • Jinquan Hu,
  • Li Di,
  • Chunsheng Yang,
  • Yan Wu,
  • Yan Jiang,
  • Juanjuan Zhang,
  • Bitao Bu,
  • Zhijun Li

摘要

Background and objectives

Acute exacerbations/worsening of generalized myasthenia gravis (gMG) require urgent intervention to prevent progression to life-threatening myasthenic crisis. This multicenter study evaluate the efficacy and safety of efgartigimod in gMG patients experiencing acute exacerbations/worsening to validate its potential as a rapid-acting therapeutic option.

Methods

In this multicenter retrospective study (Jan 2024–Feb 2025), 61 gMG patients with acute exacerbations/worsening received weekly efgartigimod infusions for 4 weeks. Efficacy was assessed at baseline (W0) and weekly throughout the treatment cycle (W1–W7) using Myasthenia Gravis Foundation of America Post-Intervention Status (MGFA-PIS), Quantitative Myasthenia Gravis (QMG), and Myasthenia Gravis Activities of Daily Living (MG-ADL) scores, along with prednisone dosage reductions. Safety assessments were concurrently performed.

Results

Median age was 56 years (range: 39–68 years). Pulmonary infection (16/61, 26.2%) was the predominant trigger of myasthenic crisis (MC) red flgs. Median duration from symptom deterioration to efgartigimod initiation was 18 days. Clinical meaningful improvement occurred in 81.9% at W1 (post-single dose), 98.1% at W4, and 91.9% at W7. MG-ADL and QMG scores decreased substantially from baseline (W4: 80.86% and 55.81% reduction; W7: 70.78% and 47.76% reduction), Patients with MC red flags achieved comparable outcomes. Greater MG-ADL imrovements were observed in those with thymoma history, severe MGFA classification, prior MC, or MC red flags. No treatment-related allergic reactions, infections, or serious adverse events were observed.

Conclusion

Efgartigimod demonstrates rapid, significant efficacy in gMG acute exacerbations/worsening, supporting its role as a valuable therapeutic option for this high-risk population.