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Abstract Details

A Global, Long-Term, Prospective, Observational Registry of Patients With Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) Treated With Complement Component 5 Inhibitor Therapies (C5ITs) Eculizumab or Ravulizumab
Autoimmune Neurology
P2 - Poster Session 2 (2:45 PM-3:45 PM)
015
To assess long-term effectiveness and safety of C5ITs eculizumab and ravulizumab in AQP4-Ab+ NMOSD.
Eculizumab and ravulizumab have been approved/submitted for approval in several regions for AQP4-Ab+ NMOSD. Both target the same complement component 5 epitope; however, ravulizumab has a longer elimination half-life, allowing for extended dosing intervals. Real-world effectiveness and safety data on patients treated with C5ITs for AQP4-Ab+ NMOSD are needed to inform clinical practice.

This global, long-term, prospective, multicenter, observational registry will enroll adults with AQP4-Ab+ NMOSD treated with C5ITs who received ≥1 dose of eculizumab or ravulizumab ≤4 or ≤12 weeks prior to enrollment, respectively. Inclusion criteria include available historical C5IT dosing data since initiation and relapse data from ≤1 year before C5IT initiation through enrollment. Retrospective outcomes include disease onset and diagnosis. Relapse data, treatment history, vaccinations, and available Expanded Disability Status Scale (EDSS) scores will be collected from 1 year prior to C5IT initiation through enrollment. The primary outcome is annualized relapse rate; effectiveness outcomes include C5IT effects on disability (EDSS score), ambulation (Hauser Ambulation Index), and vision (Best Corrected Visual Acuity). Collected patient-reported outcomes (PROs) include pain, bladder and bowel dysfunction, and quality of life. Utilization of eculizumab and ravulizumab, other therapies for management of AQP4-Ab+ NMOSD, vaccination and antibiotic use related to risk of meningococcal infection, and hospitalizations will be assessed. Safety outcomes include serious adverse events, meningococcal infections, and pregnancy, breastfeeding, and neonatal outcomes. Data will be collected prospectively for ≤5 years; clinical data will be collected at least annually and PRO data every 6 months. Approximately 130–200 patients will be enrolled in ≤10 countries. Descriptive statistics will be used.

N/A

This registry will collect data to characterize long-term effectiveness and safety of eculizumab and ravulizumab in patients with AQP4-Ab+ NMOSD and the real-world impact of C5ITs in this population.

Authors/Disclosures
Ukwen Akpoji, PharmD (Alexion, AstraZeneca Rare Disease)
PRESENTER
Dr. Akpoji has received personal compensation for serving as an employee of Alexion, AstraZeneca Rare Disease. Dr. Akpoji has received personal compensation for serving as an employee of Pfizer, Inc. Dr. Akpoji has stock in Alexion, AstraZeneca Rare Disease.
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.
Lindsey Przybyl Lindsey Przybyl has received personal compensation for serving as an employee of Alexion, AstraZeneca Rare Disease. Lindsey Przybyl has stock in AstraZeneca.
Tania Azad Tania Azad has nothing to disclose.
Jeannette Stankowski, PhD (Alexion) Dr. Stankowski has nothing to disclose.
Kristin Moy No disclosure on file