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Introduction <p>Ravulizumab, a monoclonal antibody targeting C5, was recently approved for the treatment of anti-AChR positive generalized myasthenia gravis (gMG) patients. The objective of this study is to present the Italian multicenter real-world experience evaluating the safety and efficacy of ravulizumab in gMG within the context of the Expanded Early Access Program (EAP).</p> Methods <p>We conducted a retrospective study in 7 gMG referral centres in Italy. Demographic and clinical characteristics were recorded at baseline and during follow-up through clinical scale changes including Myasthenia Gravis-Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis Composite (MGC). Frequency of minimal symptom expression (MSE) and changes in concomitant medications were also evaluated.</p> Results <p>Twenty-four gMG patients (10/24 females) aged between 24 and 82&#xa0;years (Median 60.5, IQR 52.5–67.5), were included. Fifteen patients had undergone thymectomy, and 14 had a thymoma. Median follow-up duration was 26&#xa0;weeks (range 10–74, IQR 26–42). MG-ADL and QMG scores showed a significant decrease with respect to baseline (<i>p</i> &lt; 0.001). MSE was achieved by 37.5% patients at the last available follow-up. Tapering of prednisone daily dosage was possible in 76% of patients. Thymoma was significantly associated with QMG score reduction and the frequency of QMG responders at week 2 (<i>p</i> = 0.03).</p> <p>Three patients discontinued treatment. One patient experienced a myasthenic exacerbation and needed rescue therapy. Infectious adverse events were reported in 5/24 patients, and a Stevens-Johnson syndrome in one patient.</p> Conclusions <p>Real-world data confirm the effectiveness, safety, and prednisone-sparing effect of ravulizumab in patients with gMG, especially in those with thymoma.</p>

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Ravulizumab for generalized Myasthenia Gravis: a multicenter real-life experience

  • 期刊论文

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  • 发表日期：

    2025年5月14日

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  • Elena Rossini,
  • Vincenzo Di Stefano,
  • Raffaele Iorio,
  • Francesco Habetswallner,
  • Michelangelo Maestri,
  • Claudia Vinciguerra,
  • Elena Maria Pennisi,
  • Giuseppe Di Martino,
  • Nicasio Rini,
  • Silvia Falso,
  • Sofia Marini,
  • Dario Ricciardi,
  • Melania Guida,
  • Stefania Morino,
  • Matteo Garibaldi,
  • Luca Leonardi,
  • Demetrio Marando,
  • Laura Tufano,
  • Giovanni Antonini,
  • Laura Fionda

摘要

Introduction

Ravulizumab, a monoclonal antibody targeting C5, was recently approved for the treatment of anti-AChR positive generalized myasthenia gravis (gMG) patients. The objective of this study is to present the Italian multicenter real-world experience evaluating the safety and efficacy of ravulizumab in gMG within the context of the Expanded Early Access Program (EAP).

Methods

We conducted a retrospective study in 7 gMG referral centres in Italy. Demographic and clinical characteristics were recorded at baseline and during follow-up through clinical scale changes including Myasthenia Gravis-Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis Composite (MGC). Frequency of minimal symptom expression (MSE) and changes in concomitant medications were also evaluated.

Results

Twenty-four gMG patients (10/24 females) aged between 24 and 82 years (Median 60.5, IQR 52.5–67.5), were included. Fifteen patients had undergone thymectomy, and 14 had a thymoma. Median follow-up duration was 26 weeks (range 10–74, IQR 26–42). MG-ADL and QMG scores showed a significant decrease with respect to baseline (p < 0.001). MSE was achieved by 37.5% patients at the last available follow-up. Tapering of prednisone daily dosage was possible in 76% of patients. Thymoma was significantly associated with QMG score reduction and the frequency of QMG responders at week 2 (p = 0.03).

Three patients discontinued treatment. One patient experienced a myasthenic exacerbation and needed rescue therapy. Infectious adverse events were reported in 5/24 patients, and a Stevens-Johnson syndrome in one patient.

Conclusions

Real-world data confirm the effectiveness, safety, and prednisone-sparing effect of ravulizumab in patients with gMG, especially in those with thymoma.