@article {Camerajnnp-2021-327206, author = {Valentina Camera and Silvia Messina and Kariem Tarek Elhadd and Julia Sanpera-Iglesias and Romina Mariano and Yael Hacohen and Ruth Dobson and Stefano Meletti and Evangeline Wassmer and Ming J Lim and Saif Huda and Cheryl Hemingway and Maria Isabel Leite and Sithara Ramdas and Jacqueline Palace}, title = {Early predictors of disability of paediatric-onset AQP4-IgG-seropositive neuromyelitis optica spectrum disorders}, elocation-id = {jnnp-2021-327206}, year = {2021}, doi = {10.1136/jnnp-2021-327206}, publisher = {BMJ Publishing Group Ltd}, abstract = {Objective To 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).Methods In 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.Results We 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{\textpm}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{\textendash}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).Conclusions Age 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.Data are available upon reasonable request. All data relevant to the study are included in the article or uploaded as supplementary information.}, issn = {0022-3050}, URL = {https://jnnp.bmj.com/content/early/2021/09/27/jnnp-2021-327206}, eprint = {https://jnnp.bmj.com/content/early/2021/09/27/jnnp-2021-327206.full.pdf}, journal = {Journal of Neurology, Neurosurgery \& Psychiatry} }