Abstract
Objective Early recognition of markers of faster disability worsening in paediatric-onset multiple sclerosis (POMS) is a key requisite of personalised therapy for children with MS at the earliest possible time after symptoms onset. We sought to identify early predictors of rapid disability accrual in patients with POMS, considering the potential for commencing higher-efficacy disease-modifying therapies (DMTs).
Methods Using the global MSBase registry, we identified patients who were <18 years at the onset of MS symptoms. The clinico-demographic characteristics examined as predictors of future MS Severity Score (MSSS) included: sex, age at symptoms onset, absence of disability at the initial assessment, and maximum Expanded Disability Status Scale (EDSS) score, relapse frequency, and presence of brainstem, pyramidal, visual, or cerebellar symptoms during the first 12 months. The Bayesian log-normal mixed models were adjusted for cumulative proportion of time on higher-efficacy DMTs.
Results 672 patients (70% female) contributing 9357 visits were included. Older age at symptoms onset (exp(β)=1·10 [95% Credible Interval: 1·04,1·17]), higher EDSS score (1·22 [1·12,1·34]), and pyramidal (1·31 [1·11,1·55]), visual (1·25 [1·10,1·44]), or cerebellar (1·18 [1·01, 1·38]) symptoms during the first 12 months were associated with higher MSSS. MSSS was reduced by 4% for every 24% increase in the proportion of time on higher-efficacy DMTs (0·96 [0·93,0·99]).
Conclusions MS symptoms presented later in the childhood, higher disability, and pyramidal, visual, or cerebellar symptoms during the first year predicted more rapid accrual of disability in patients with POMS. Persistent treatment with higher-efficacy DMTs was associated with a reduced rate of disability worsening.
