NMO SPOTLIGHT Registry: Real-World Clinical Outcomes With Eculizumab and Ravulizumab in Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD)
Ahmed Obeidat1, Elias Sotirchos2, Veronica Tkachuk3, Ho Jin Kim4, Lindsey Przybyl5, Sami Fam5
1The Medical College of Wisconsin, 2Johns Hopkins University School of Medicine, 3Instituto de Investigaciones Metabolicas, 4National Cancer Center, 5Alexion, AstraZeneca Rare Disease
Objective:
To describe the initial characteristics and clinical outcomes of Registry participants with AQP4-Ab+ NMOSD treated with eculizumab or ravulizumab in real-world clinical practice.
Background:
The Alexion complement component 5 inhibitor therapies eculizumab and ravulizumab (ALXN-C5ITs) are approved in multiple countries to treat adults with AQP4-Ab+ NMOSD. The Alexion-sponsored global NMO SPOTLIGHT Registry (NCT05966467) collects ALXN-C5IT real-world safety and clinical effectiveness data in adults aged ≥18yrs with AQP4-Ab+ NMOSD.
Design/Methods:
Adults with AQP4-Ab+ NMOSD receiving ALXN-C5ITs were enrolled in the Registry starting August 2023. Demographics, number and types of relapses from 1yr prior to and during ALXN-C5IT therapy, meningococcal infections, and vaccination history were collected. An NMOSD relapse was defined as new onset/worsening of neurologic symptoms for >24hrs requiring acute standard-of-care treatment preceded by ≥30d of clinical stability.
Results:
As of 21Jun2024, 30 adults were enrolled in the Registry and available for analysis (female, 86.7%; White, 50.0%; Black, 46.7%; median [interquartile range (IQR)] age at initial diagnosis, 47 [35-57] yrs; ALXN-C5IT monotherapy, 83.3%). Median (IQR) duration of exposure to eculizumab (n=30) and ravulizumab (n=2) was 40.2 (24.0-49.7) mo and 1.0 (0.7-1.3) mo, respectively. In the 1yr prior to ALXN-C5IT, 10 (33.3%) patients had 15 relapses (annualized relapse rate [ARR]: 0.50 [95% CI: 0.28-0.82]); 2/10 (20.0%) patients had >1 relapse. While on ALXN-C5IT, 2 (6.7%) patients had relapses (ARR: 0.02 [95% CI: 0.00-0.08]); no patients had >1 relapse. Most relapses were transverse myelitis. In the 1yr prior to ALXN-C5IT through Registry enrollment, 29 (96.7%) patients received ≥1 meningococcal vaccination. No vaccinations were reported within 4 weeks before any relapse; no meningococcal infections were reported.
Conclusions:
Consistent with clinical trial and real-world data previously reported on eculizumab and/or ravulizumab among patients with AQP4-Ab+ NMOSD, initial results demonstrate the strong clinical benefit of ALXN-C5ITs in relapse prevention. No new safety signals were detected; no meningococcal infections were reported.
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