Ravulizumab in neuromyelitis optica spectrum disorder: first clinical experience in Chile
7 affiliations
- 1Neuroimmunology Unit, Department of Neurology, Clínica Alemana de Santiago
- 2Hospital Dra. Eloísa Díaz Inzunza, La Florida
- 3Clínica RedSalud
- 4Hospital Clínico Universidad de Chile
- 5Clínica Universidad de los Andes
- 6Clínica Las Condes
- 7Clínica Dávila
Abstract
Background: Neuromyelitis optica spectrum disorder (NMOSD) is a neuroimmunological astrocytopathy mediated by anti-aquaporin-4 (AQP4-IgG) antibodies that trigger complement-dependent cytotoxicity and activation of the terminal complement cascade in the central nervous system. Ravulizumab, a long-acting terminal complement C5 inhibitor, received regulatory approval in Chile in January 2025; the seven patients reported here represent the entire nationally treated cohort to date.
Objectives: To describe the first clinical experience with ravulizumab in NMOSD at three Chilean centers (Clínica Alemana de Santiago, a private center, Clínica Dávila, a private center, and Hospital Clínico Universidad de Chile, a public university hospital).
Methods: Retrospective, multicenter cohort study of patients with NMOSD fulfilling the 2015 International Panel for NMO Diagnosis (IPND) consensus criteria who were receiving ravulizumab. Demographic and clinical characteristics, AQP4-IgG status, baseline and follow-up Expanded Disability Status Scale (EDSS) scores, treatment indication, relapses and adverse events were analyzed through medical chart review.
Results: Seven patients (100% female) were included, with a mean age at disease onset of 51.0 years (range 38–69). AQP4-IgG was positive in all patients (100%), confirmed by cell-based assay. The most common presentation over the disease course was longitudinally extensive transverse myelitis (LETM; 7/7); four patients (57.1%) also developed optic neuritis. Median baseline EDSS was 6.0. Five patients had previously failed rituximab, one discontinued rituximab because of active cancer, and one was treatment naïve. Over a median follow-up of 15 months (range 4–24) on ravulizumab, no clinical relapses were observed (0/7 patients), compared with approximately 4.5 relapses expected across the cohort’s person-time had the pre-treatment relapse rate continued unchanged (observed-vs-expected analysis; see Results). EDSS declined from a baseline median of 6.0 to 2.5 (median within-patient change 1.5 points, IQR 1.0–4.0); because baseline EDSS in several patients was recorded during or shortly after an acute attack, this decrease likely reflects post-attack recovery in addition to any disability-modifying effect, and three patients remained at EDSS ≥5. Headache was the only adverse event (7/7), mild to moderate and self-limited.
Conclusions: In this real-world Chilean cohort, ravulizumab was associated with complete relapse suppression over a median follow-up of 15 months and was well tolerated. These preliminary, uncontrolled observations are consistent with the relapse-prevention efficacy reported in pivotal trials and with regional consensus guidance on its use after rituximab failure, and they underscore the need to expand equitable access to complement-targeted therapies within the Chilean healthcare system.
Related
- AQP4 antibody-induced astrocyte injury reshapes CNS immune architecture in a combined EAE–AQP4-IgG model of neuromyelitis optica spectrum disorder
- Soluble TIM-3 and galectin-9 in cerebrospinal fluid are associated with disability at 6 months in aquaporin-4 IgG-positive neuromyelitis optica spectrum disorder
- Characteristics of serum Metrnl levels in patients with neuromyelitis optica spectrum disorder and their associations with disease activity and inflammatory biomarkers
- Higher observed 12-month relapse-free survival with satralizumab than with rituximab in aquaporin-4 immunoglobulin G-positive neuromyelitis optica spectrum disorder: a multicenter retrospective cohort study
- Why repair fails in AQP4-IgG-positive neuromyelitis optica spectrum disease: an astrocyte-centered framework for remyelination and neuroaxonal preservation
BibTeX
@article{santibanez2026ravulizumab,
title = {{Ravulizumab in neuromyelitis optica spectrum disorder: first clinical experience in Chile}},
author = {Tamara Santibáñez and José Miguel Valdés and César Romero and Manuel Fruns and Lorna Galleguillos},
journal = {Frontiers in Immunology},
year = {2026},
month = oct,
doi = {10.3389/fimmu.2026.1932448},
url = {https://www.frontiersin.org/articles/10.3389/fimmu.2026.1932448/full},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Ravulizumab in neuromyelitis optica spectrum disorder: first clinical experience in Chile
AU - Tamara Santibáñez
AU - José Miguel Valdés
AU - César Romero
AU - Manuel Fruns
AU - Lorna Galleguillos
PY - 2026
DA - 2026/10/06
JO - Frontiers in Immunology
DO - 10.3389/fimmu.2026.1932448
UR - https://www.frontiersin.org/articles/10.3389/fimmu.2026.1932448/full
AB - Background: Neuromyelitis optica spectrum disorder (NMOSD) is a neuroimmunological astrocytopathy mediated by anti-aquaporin-4 (AQP4-IgG) antibodies that trigger complement-dependent cytotoxicity and activation of the terminal complement cascade in the central nervous system. Ravulizumab, a long-acting terminal complement C5 inhibitor, received regulatory approval in Chile in January 2025; the seven patients reported here represent the entire nationally treated cohort to date. Objectives: To describe the first clinical experience with ravulizumab in NMOSD at three Chilean centers (Clínica Alemana de Santiago, a private center, Clínica Dávila, a private center, and Hospital Clínico Universidad de Chile, a public university hospital). Methods: Retrospective, multicenter cohort study of patients with NMOSD fulfilling the 2015 International Panel for NMO Diagnosis (IPND) consensus criteria who were receiving ravulizumab. Demographic and clinical characteristics, AQP4-IgG status, baseline and follow-up Expanded Disability Status Scale (EDSS) scores, treatment indication, relapses and adverse events were analyzed through medical chart review. Results: Seven patients (100% female) were included, with a mean age at disease onset of 51.0 years (range 38–69). AQP4-IgG was positive in all patients (100%), confirmed by cell-based assay. The most common presentation over the disease course was longitudinally extensive transverse myelitis (LETM; 7/7); four patients (57.1%) also developed optic neuritis. Median baseline EDSS was 6.0. Five patients had previously failed rituximab, one discontinued rituximab because of active cancer, and one was treatment naïve. Over a median follow-up of 15 months (range 4–24) on ravulizumab, no clinical relapses were observed (0/7 patients), compared with approximately 4.5 relapses expected across the cohort’s person-time had the pre-treatment relapse rate continued unchanged (observed-vs-expected analysis; see Results). EDSS declined from a baseline median of 6.0 to 2.5 (median within-patient change 1.5 points, IQR 1.0–4.0); because baseline EDSS in several patients was recorded during or shortly after an acute attack, this decrease likely reflects post-attack recovery in addition to any disability-modifying effect, and three patients remained at EDSS ≥5. Headache was the only adverse event (7/7), mild to moderate and self-limited. Conclusions: In this real-world Chilean cohort, ravulizumab was associated with complete relapse suppression over a median follow-up of 15 months and was well tolerated. These preliminary, uncontrolled observations are consistent with the relapse-prevention efficacy reported in pivotal trials and with regional consensus guidance on its use after rituximab failure, and they underscore the need to expand equitable access to complement-targeted therapies within the Chilean healthcare system.
SN - 1664-3224
ER -