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 (IDIM), 6Alexion, AstraZeneca Rare Disease
Objective:
Describe the characteristics and clinical outcomes of patients with AQP4-Ab+ NMOSD treated with Alexion complement component 5 inhibitor therapies (ALXN-C5ITs, eculizumab and ravulizumab).
Background:
The global NMO SPOTLIGHT Registry (NCT05966467) collects data on real-world clinical safety and effectiveness of ALXN-C5ITs in adults with AQP4-Ab+ NMOSD.
Design/Methods:
Adults with AQP4-Ab+ NMOSD receiving ALXN-C5ITs were enrolled. Demographics, number and characteristics of relapses 1yr before and after initiating ALXN-C5IT, meningococcal infections, and vaccination data were collected. NMOSD relapses were defined as new onset/worsening of neurologic symptoms for >24hrs requiring acute standard-of-care treatment preceded by ≥30d of clinical stability. Data were summarized for all patients, including those who switched from rituximab (RTX).
Results:
As of 14Oct2024, 56 patients fulfilled inclusion criteria for analysis (female, 89.3%; White, 35.7%; Black, 33.9%; median age at diagnosis, 46.5yrs [interquartile range (IQR), 33.5-56.5]). Median (IQR) duration of exposure to eculizumab (n=52) and ravulizumab (n=12) was 42.5 (22.2-54.3) mo and 5.3 (4.0-14.1) mo, respectively. In the 1yr before 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 relapsed (ARR: 0.02 [95% CI: 0.00-0.05]); none 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), 6 (40.0%) had 7 relapses in the 1yr before ALXN-C5IT initiation (ARR: 0.47 [95% CI: 0.19-0.96]); none relapsed while on ALXN-C5IT. In the 1yr before ALXN-C5IT through Registry enrollment, 52 (92.9%) patients received ≥1 meningococcal vaccination. No meningococcal or other vaccination in the 4wks prior to relapse was reported; no meningococcal infections were reported.
Conclusions:
Consistent with clinical trial and real-world data previously reported on ALXN-C5ITs in AQP4-Ab+ NMOSD, these results demonstrate the strong clinical benefit of eculizumab and ravulizumab in relapse prevention. No new safety signals were detected.
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