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Objective <p>To evaluate the real-world effectiveness and safety of eculizumab in patients with AQP4-IgG–positive neuromyelitis optica spectrum disorder (NMOSD) and to identify predictors of disability outcomes.</p> Methods <p>This multinational, retrospective cohort study analyzed data from 46 patients across 26 centers. The outcomes included the annualized relapse rate (ARR), relapse-free status, change in expanded disability status scale (EDSS) scores, and adverse events. To identify predictors of EDSS improvement or worsening, patients were stratified into subgroups (improved vs. stable/worsened) at each follow-up time point and compared based on demographic, clinical, and radiological variables.</p> Results <p>This retrospective cohort study included 46 patients with AQP4-IgG-positive NMOSD from 26 centers, followed for a mean of 27.3&#xa0;months. The mean ARR significantly decreased from 1.1 in the 2&#xa0;years pre-treatment to 0.1 during eculizumab therapy. The relapse-free rate increased from 6.5% pre-treatment to 80.4%. Mean EDSS scores improved from 4.2 at baseline to 3.6 at 24&#xa0;months. The presence of area postrema syndrome was associated with a favorable prognosis, while the presence of spinal attacks was associated with a poor prognosis at 12&#xa0;months. Adverse events occurred in 7 patients (18.9%), leading to permanent discontinuation in only two.</p> Conclusion <p>Eculizumab demonstrated robust real-world effectiveness in reducing relapse rates and stabilizing disability, with an acceptable safety profile. Clinical outcomes may be influenced by attack phenotype, underscoring the importance of early intervention.</p>

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Real-world effectiveness and safety of eculizumab in AQP4-IgG-positive neuromyelitis optica spectrum disorder

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年1月8日

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  • Emine Rabia Koc,
  • Mehmet Fatih Yetkin,
  • Furkan Saridas,
  • Omer Faruk Turan,
  • Serhan Sevim,
  • Murat Terzi,
  • Sedat Sen,
  • Melih Tutuncu,
  • Ugur Uygunoglu,
  • Murat Kurtuncu,
  • Tuncay Gunduz,
  • Nermin Tepe,
  • Sibel Canbaz-Kabay,
  • Husnu Efendi,
  • Sena Destan Bunul,
  • Damla Cetinkaya-Tezer,
  • İpek Gungor-Dogan,
  • Serkan Demir,
  • Burcu Altunrende,
  • Nilufer Kale-Icen,
  • Sami Omerhoca,
  • Ozden Kamisli,
  • Caner Fevzi Demir,
  • Basak Karakurum-Yuksel,
  • Cihat Uzunkopru,
  • Yesim Beckmann,
  • Vedat Cilingir,
  • Kadriye Agan,
  • Haluk Gumus,
  • Merve Ercan,
  • Belgin Kocer,
  • Sanja Gluscevic,
  • Edgar Carnero-Contentti,
  • Adriana Casallas-Vanegas,
  • Valentina Camera,
  • Massimiliano Calabrese,
  • Guven Ozkaya,
  • Gulsen Akman-Demir,
  • Cavit Boz,
  • Ayse Altıntas,
  • Aksel Siva

摘要

Objective

To evaluate the real-world effectiveness and safety of eculizumab in patients with AQP4-IgG–positive neuromyelitis optica spectrum disorder (NMOSD) and to identify predictors of disability outcomes.

Methods

This multinational, retrospective cohort study analyzed data from 46 patients across 26 centers. The outcomes included the annualized relapse rate (ARR), relapse-free status, change in expanded disability status scale (EDSS) scores, and adverse events. To identify predictors of EDSS improvement or worsening, patients were stratified into subgroups (improved vs. stable/worsened) at each follow-up time point and compared based on demographic, clinical, and radiological variables.

Results

This retrospective cohort study included 46 patients with AQP4-IgG-positive NMOSD from 26 centers, followed for a mean of 27.3 months. The mean ARR significantly decreased from 1.1 in the 2 years pre-treatment to 0.1 during eculizumab therapy. The relapse-free rate increased from 6.5% pre-treatment to 80.4%. Mean EDSS scores improved from 4.2 at baseline to 3.6 at 24 months. The presence of area postrema syndrome was associated with a favorable prognosis, while the presence of spinal attacks was associated with a poor prognosis at 12 months. Adverse events occurred in 7 patients (18.9%), leading to permanent discontinuation in only two.

Conclusion

Eculizumab demonstrated robust real-world effectiveness in reducing relapse rates and stabilizing disability, with an acceptable safety profile. Clinical outcomes may be influenced by attack phenotype, underscoring the importance of early intervention.