1. Prognostic indicators in pediatric clinically isolated syndrome
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Pietro Iaffaldano, Damiano Paolicelli, Eleonora Cocco, Gianfranco Costantino, Maria Pia Amato, Patrizia Sola, Ricardo Fernandez Bolanos, Marta Simone, Tim Spelman, Giuseppe Lucisano, Francesco Patti, Carlo Pozzilli, G. B. Zimatore, Giancarlo Comi, Helmut Butzkueven, Carlo Avolio, Pierre Duquette, Jose Antonio Cabrera-Gomez, Seyed Aidin Sajedi, Roberto Bergamaschi, Enrico Millefiorini, Thor Petersen, G. Coniglio, Alessandra Lugaresi, Carla Tortorella, Guillermo Izquierdo, Eugenio Pucci, Angelo Ghezzi, Giuseppe Salemi, Cavit Boz, Vahid Shaygannejad, Davide Maimone, Maria Trojano, Vincenzo Brescia Morra, Lucia Margari, Giacomo Lus, and Raed Alroughani
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0301 basic medicine ,medicine.medical_specialty ,education.field_of_study ,Clinically isolated syndrome ,Expanded Disability Status Scale ,business.industry ,Proportional hazards model ,Hazard ratio ,Population ,Retrospective cohort study ,Surgery ,03 medical and health sciences ,030104 developmental biology ,0302 clinical medicine ,Neurology ,Internal medicine ,Medicine ,Neurology (clinical) ,Age of onset ,Risk factor ,business ,education ,030217 neurology & neurosurgery - Abstract
Objective To assess prognostic factors for a second clinical attack and a first disability-worsening event in pediatric clinically isolated syndrome (pCIS) suggestive of multiple sclerosis (MS) patients. Methods A cohort of 770 pCIS patients was followed up for at least 10 years. Cox proportional hazard models and Recursive Partitioning and Amalgamation (RECPAM) tree-regression were used to analyze data. Results In pCIS, female sex and a multifocal onset were risk factors for a second clinical attack (hazard ratio [HR], 95% confidence interval [CI] = 1.28, 1.06–1.55; 1.42, 1.10–1.84, respectively), whereas disease-modifying drug (DMD) exposure reduced this risk (HR, 95% CI = 0.75, 0.60–0.95). After pediatric onset MS (POMS) diagnosis, age at onset younger than 15 years and DMD exposure decreased the risk of a first Expanded Disability Status Scale (EDSS)-worsening event (HR, 95% CI = 0.59, 0.42–0.83; 0.75, 0.71–0.80, respectively), whereas the occurrence of relapse increased this risk (HR, 95% CI = 5.08, 3.46–7.46). An exploratory RECPAM analysis highlighted a significantly higher incidence of a first EDSS-worsening event in patients with multifocal or isolated spinal cord or optic neuritis involvement at onset in comparison to those with an isolated supratentorial or brainstem syndrome. A Cox regression model including RECPAM classes confirmed DMD exposure as the most protective factor against EDSS-worsening events and relapses as the most important risk factor for attaining EDSS worsening. Interpretation This work represents a step forward in identifying predictors of unfavorable course in pCIS and POMS and supports a protective effect of early DMD treatment in preventing MS development and disability accumulation in this population. Ann Neurol 2017;81:729–739
- Published
- 2017
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