Real-world Clinical Outcomes with Eculizumab and Ravulizumab in Anti–Aquaporin-4 Antibody–Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD): Results From the Global NMO SPOTLIGHT Registry
Elias Sotirchos1, Ahmed Obeidat2, Ho Jin Kim3, Jin Nakahara4, Veronica Tkachuk5, Lindsey Przybyl6, Sami Fam6
1Johns Hopkins University School of Medicine, 2Medical College of Wisconsin, 3National Cancer Center, 4Keio University School of Medicine, 5Instituto de Investigaciones Metabolicas, 6Alexion, AstraZeneca Rare Disease
Objective:
Describe global NMO SPOTLIGHT Registry (NCT05966467) patients with AQP4-Ab+ NMOSD treated with Alexion complement component 5 inhibitor therapies (ALXN-C5ITs) in real-world clinical practice.
Background:
ALXN-C5ITs eculizumab and ravulizumab are approved in multiple countries for adults with AQP4-Ab+ NMOSD. The Registry collects ALXN-C5IT real-world safety and clinical effectiveness data in patients aged ≥18y.
Design/Methods:
Adults with AQP4-Ab+ NMOSD receiving ALXN-C5ITs were enrolled in the Registry starting August 2023. Data on demographics, relapses, infections, and vaccinations were collected. NMOSD relapse was defined as new onset/worsening neurologic symptoms for >24h requiring acute standard-of-care treatment preceded by ≥30d of clinical stability. Data were summarized for all patients, including patients who switched from rituximab (RTX).
Results:
As of 14Oct2024, 56 adults were enrolled in the Registry (median age at diagnosis, 46.5y). Median (IQR) duration of eculizumab (n=52) and ravulizumab (n=12) exposure was 42.5 (22.2-54.3) mo and 5.3 (4.0-14.1) mo, respectively. In 1y prior to ALXN-C5IT, 21 (37.5%) patients had 28 relapses (annualized relapse rate [ARR]: 0.50 [95% CI: 0.33-0.72]); 4/21 (19.0%) patients had >1 relapse. While on ALXN-C5IT, 3 (5.4%) patients had relapses (ARR: 0.02 [95% CI: 0.00-0.05]); no patients had >1 relapse. Among patients who switched from RTX to ALXN-C5IT (n=15; median time from last RTX discontinuation date to ALXN-C5IT initiation=141.5d, meningococcal vaccination=112d), 6 (40.0%) had 7 relapses in the 1y prior to ALXN-C5IT initiation (ARR: 0.47 [95% CI: 0.19-0.96]); none experienced a relapse while on ALXN-C5IT. In the 1y prior to ALXN-C5IT through Registry enrollment, 52 (92.9%) patients received ≥1 meningococcal vaccination. No receipt of a meningococcal or other vaccination in the 4w prior to relapse was reported; no meningococcal infections were reported.
Conclusions:
Consistent with previous clinical trial and real-world data, these results demonstrate the strong clinical benefit of eculizumab and ravulizumab in relapse prevention. No new safety signals were detected; no meningococcal infections were reported.
Generative AI Usage
No, did not use generative AI in the drafting or editing in this abstract.
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