Journal Article

Early predictors of disability of paediatric-onset AQP4-IgG-seropositive neuromyelitis optica spectrum disorders

Lim, Ming J, Camera, Valentina, Elhadd, Kariem Tarek, Dobson, Ruth, Meletti, Stefano, Hemingway, Cheryl, Hacohen, Yael, Ramdas, Sithara, Mariano, Romina, Leite, Maria Isabel, Wassmer, Evangeline, Huda, Saif, Sanpera-Iglesias, Julia, Palace, Jacqueline, Messina, Silvia

2022

Overview

ObjectiveTo describe onset clinical features predicting time to first relapse and time to long-term visual, motor and cognitive disabilities in paediatric-onset aquaporin-4 antibody (AQP4-IgG) neuromyelitis optica spectrum disorders (NMOSDs).MethodsIn this retrospective UK multicentre cohort study, we recorded clinical data of paediatric-onset AQP4-IgG NMOSD. Univariate and exploratory multivariable Cox proportional hazard models were used to identify long-term predictors of permanent visual disability, Expanded Disability Status Scale (EDSS) score of 4 and cognitive impairment.ResultsWe included 49 paediatric-onset AQP4-IgG patients (38.8% white, 34.7% black, 20.4% Asians and 6.1% mixed), mean onset age of 12±4.1 years, and 87.7% were female. Multifocal onset presentation occurred in 26.5% of patients, and optic nerve (47%), area postrema/brainstem (48.9%) and encephalon (28.6%) were the most involved areas. Overall, 52.3% of children had their first relapse within 1 year from disease onset. Children with onset age <12 years were more likely to have an earlier first relapse (p=0.030), despite showing no difference in time to immunosuppression compared with those aged 12–18 years at onset. At the cohort median disease duration of 79 months, 34.3% had developed permanent visual disability, 20.7% EDSS score 4 and 25.8% cognitive impairment. Visual disability was associated with white race (p=0.032) and optic neuritis presentations (p=0.002). Cognitive impairment was predicted by cerebral syndrome presentations (p=0.048), particularly if resistant to steroids (p=0.034).ConclusionsAge at onset, race, onset symptoms and resistance to acute therapy at onset attack predict first relapse and long-term disabilities. The recognition of these predictors may help to power future paediatric clinical trials and to direct early therapeutic decisions in AQP4-IgG NMOSD.

• Publisher: BMJ Publishing Group Ltd,BMJ Publishing Group LTD
• Subject: Adolescent / Adult / Age / Age of Onset / Antibodies - blood / Aquaporin 4 / Aquaporins / Asian People - statistics & numerical data / Black People - statistics & numerical data / Brain - pathology / Child / Cognition & reasoning / Cognitive ability / Cohort Studies / Demography / Disability Evaluation / Disease / Disease Progression / Female / Humans / Immunoglobulin G - therapeutic use / Learning disabilities / Male / Middle Aged / Neuro-inflammation / Neuromyelitis Optica - diagnosis / Neuropsychology / Optic Nerve - pathology / paediatric neurology / Pediatrics / Persons with Disabilities - statistics & numerical data / Proportional Hazards Models / Retrospective Studies / Statistical analysis / United Kingdom / Variables / White People - statistics & numerical data / Young Adult