Objective: We sought to identify the early predictors of disability progression in a multi-national cohort of patients with paediatric-onset multiple sclerosis (POMS), considering the potential for commencing high-efficacy disease-modifying therapies (DMTs). Background:Early recognition of markers of faster disability worsening in POMS is important for treatment decisions at the earliest possible time after symptoms onset. Design/Methods: We identified patients who were <18 years at the onset of MS symptoms from the global MSBase registry. Patients with first neurological assessment within 12 months from symptoms onset were included. The variables examined as predictors of future MS Severity Score (MSSS; disability ranked by disease duration) included: age at symptoms onset, complete recovery from first relapse, EDSS score, relapse phenotypes and frequency during the first 12 months. The analyses were adjusted for cumulative proportion of time on high-efficacy DMTs. A Bayesian log-normal generalized linear mixed model was used to analyse the longitudinal data. Results: 672 patients (70% women) contributing 9357 visits were included. The median (quartiles) age at symptoms onset was 16 (15–17) years. Older age at symptoms onset (exp(β)=1.09 [95% Credible Interval: 1.03,1.16]), higher EDSS score (1.25 [1.13,1.36]), and pyramidal (1.34 [1.13,1.58]), visual (1.28 [1.10,1.48]), and cerebellum (1.17 [1.00,1.39]) symptoms during the first 12 months were associated with higher MSSS. Interestingly, MSSS was reduced by 4% for each unit increase in the proportion of time on high-efficacy DMTs (0.96 [0.93,0.99]). Conclusions: MS symptoms presented later in the childhood, higher disability, and pyramidal, visual, or cerebellum symptoms during the first year predicted significant worsening in disability in patients with paediatric MS. Persistent treatment with high-efficacy DMTs was associated with reduced rate of disability worsening.
Sharmin, S., Malpas, C., Roos, I., Diouf, I., Alroughani, R., Ozakbas, S., et al. (2022). Early Predictors of Disability in Paediatric Multiple Sclerosis: Evidence from a Multi -National Registry. NEUROLOGY, 98(18s), P17-4.001-P17-4.001 [10.1212/WNL.98.18_supplement.2668].
Early Predictors of Disability in Paediatric Multiple Sclerosis: Evidence from a Multi -National Registry
Lugaresi, AWriting – Review & Editing
;
2022
Abstract
Objective: We sought to identify the early predictors of disability progression in a multi-national cohort of patients with paediatric-onset multiple sclerosis (POMS), considering the potential for commencing high-efficacy disease-modifying therapies (DMTs). Background:Early recognition of markers of faster disability worsening in POMS is important for treatment decisions at the earliest possible time after symptoms onset. Design/Methods: We identified patients who were <18 years at the onset of MS symptoms from the global MSBase registry. Patients with first neurological assessment within 12 months from symptoms onset were included. The variables examined as predictors of future MS Severity Score (MSSS; disability ranked by disease duration) included: age at symptoms onset, complete recovery from first relapse, EDSS score, relapse phenotypes and frequency during the first 12 months. The analyses were adjusted for cumulative proportion of time on high-efficacy DMTs. A Bayesian log-normal generalized linear mixed model was used to analyse the longitudinal data. Results: 672 patients (70% women) contributing 9357 visits were included. The median (quartiles) age at symptoms onset was 16 (15–17) years. Older age at symptoms onset (exp(β)=1.09 [95% Credible Interval: 1.03,1.16]), higher EDSS score (1.25 [1.13,1.36]), and pyramidal (1.34 [1.13,1.58]), visual (1.28 [1.10,1.48]), and cerebellum (1.17 [1.00,1.39]) symptoms during the first 12 months were associated with higher MSSS. Interestingly, MSSS was reduced by 4% for each unit increase in the proportion of time on high-efficacy DMTs (0.96 [0.93,0.99]). Conclusions: MS symptoms presented later in the childhood, higher disability, and pyramidal, visual, or cerebellum symptoms during the first year predicted significant worsening in disability in patients with paediatric MS. Persistent treatment with high-efficacy DMTs was associated with reduced rate of disability worsening.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
