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Abstract Details

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
Autoimmune Neurology
S6 - Autoimmune Neurology: NMOSD and MOGAD (2:36 PM-2:48 PM)
009

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).

The global NMO SPOTLIGHT Registry (NCT05966467) collects data on real-world clinical safety and effectiveness of ALXN-C5ITs in adults with AQP4-Ab+ NMOSD.

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).
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.

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.
Authors/Disclosures
Ahmed Z. Obeidat, MD, PhD (Medical College of Wisconsin)
PRESENTER 		Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Bristol Myers Squibb. Dr. Obeidat has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Genentech. Dr. Obeidat has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for EMD Serono. Dr. Obeidat has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi/Genzyme . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Horizon pharmaceuticals . Dr. Obeidat has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sandoz. Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for TG Therapeutics. Dr. Obeidat has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Alexion. Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen. Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Bristol Myers Squibb. Dr. Obeidat has received personal compensation in the range of $100,000-$499,999 for serving on a Speakers Bureau for EMD Serono. Dr. Obeidat has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Banner life sciences . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Sanofi/genzyme. Dr. Obeidat has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Horizon therapeutics . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Amgen. Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for TG Therapeutics . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for AstraZeneca. The institution of Dr. Obeidat has received research support from NIH. The institution of Dr. Obeidat has received research support from NMSS and PCORI. Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Expert opinion and key opinion leader with MJH life sciences . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Faculty Speaker with CMSC. Dr. Obeidat has a non-compensated relationship as a Board member with CMSC that is relevant to AAN interests or activities. Dr. Obeidat has a non-compensated relationship as a Editorial Board Member with IJMSC that is relevant to AAN interests or activities. Dr. Obeidat has a non-compensated relationship as a Reviewer Editor with Frontiers Neurology that is relevant to AAN interests or activities. Dr. Obeidat has a non-compensated relationship as a Board of Trustee Member with National MS Society that is relevant to AAN interests or activities.
Elias S. Sotirchos, MD (Johns Hopkins University) Dr. Sotirchos has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech. Dr. Sotirchos has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Sotirchos has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen. Dr. Sotirchos has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for TG Therapeutics. The institution of Dr. Sotirchos has received research support from National Institutes of Health. The institution of Dr. Sotirchos has received research support from National Multiple Sclerosis Society. The institution of Dr. Sotirchos has received research support from Sumaira Foundation. The institution of Dr. Sotirchos has received research support from Genentech. The institution of Dr. Sotirchos has received research support from UCB. The institution of Dr. Sotirchos has received research support from Astoria Biologica. The institution of Dr. Sotirchos has received research support from Ad Scientiam. The institution of Dr. Sotirchos has received research support from Alexion. The institution of Dr. Sotirchos has received research support from Corevitas. Dr. Sotirchos has received personal compensation in the range of $500-$4,999 for serving as a Ad Hoc Reviewer with National Institutes of Health.
Ho Jin Kim, MD (National Cancer Center) Dr. Kim has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Altos Biologics, AstraZeneca, Biogen, Daewoong Pharmaceutical, Kaigene, Kolon Life Science, MDimune, Roche, and Sanofi. Dr. Kim has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion, AstraZeneca, Eisai, GC Pharma, Merck, Mitsubishi Tanabe Pharma, and Roche. Dr. Kim has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Multiple Sclerosis Journal, Journal of Clinical Neurology.
Jin Nakahara, MD, PhD, FAAN (Keio University School of Medicine) Prof. Nakahara has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Prof. Nakahara has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Mitsubishi-Tanabe. Prof. Nakahara has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Prof. Nakahara has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Chugai. Prof. Nakahara has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Novartis. Prof. Nakahara has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biogen. Prof. Nakahara has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Prof. Nakahara has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Chugai. Prof. Nakahara has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Mitsubishi-Tanabe. Prof. Nakahara has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Takeda. Prof. Nakahara has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for UCB. Prof. Nakahara has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Amgen. Prof. Nakahara has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Eisai. Prof. Nakahara has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for The Japanese Society for Internal Medicine. The institution of Prof. Nakahara has received research support from Chugai. The institution of Prof. Nakahara has received research support from Japan Society for the Promotion of Science.
Veronica A. Tkachuk Veronica A. Tkachuk has nothing to disclose.
Lindsey Przybyl Lindsey Przybyl has received personal compensation for serving as an employee of Alexion, AstraZeneca Rare Disease. Lindsey Przybyl has stock in AstraZeneca.
Sami Fam Sami Fam has received personal compensation for serving as an employee of Alexion Pharmaceuticals. Sami Fam has or had stock in Astra Zeneca.