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dc.rights.licenseopenen_US
dc.contributor.authorBENALLEGUE, Nail
dc.contributor.authorROLLOT, Fabien
dc.contributor.authorWIERTLEWSKI, Sandrine
dc.contributor.authorCASEY, Romain
dc.contributor.authorDEBOUVERIE, Marc
dc.contributor.authorKERBRAT, Anne
dc.contributor.authorDE SEZE, Jérôme
dc.contributor.authorCIRON, Jonathan
hal.structure.identifierNeurocentre Magendie : Physiopathologie de la Plasticité Neuronale [U1215 Inserm - UB]
dc.contributor.authorRUET, Aurelie
dc.contributor.authorLABAUGE, Pierre
dc.contributor.authorMAILLART, Elisabeth
dc.contributor.authorZEPHIR, Helene
dc.contributor.authorPAPEIX, Caroline
dc.contributor.authorDEFER, Gilles
dc.contributor.authorLEBRUN-FRENAY, Christine
dc.contributor.authorMOREAU, Thibault
dc.contributor.authorBERGER, Eric
dc.contributor.authorSTANKOFF, Bruno
dc.contributor.authorCLAVELOU, Pierre
dc.contributor.authorHEINZLEF, Olivier
dc.contributor.authorPELLETIER, Jean
dc.contributor.authorTHOUVENOT, Eric
dc.contributor.authorAL KHEDR, Abdullatif
dc.contributor.authorBOURRE, Bertrand
dc.contributor.authorCASEZ, Olivier
dc.contributor.authorCABRE, Philippe
dc.contributor.authorWAHAB, Abir
dc.contributor.authorMAGY, Laurent
dc.contributor.authorVUKUSIC, Sandra
dc.contributor.authorLAPLAUD, David-Axel
dc.date.accessioned2024-04-23T14:11:06Z
dc.date.available2024-04-23T14:11:06Z
dc.date.issued2024-03-01
dc.identifier.issn2168-6149en_US
dc.identifier.urihttps://oskar-bordeaux.fr/handle/20.500.12278/199289
dc.description.abstractEnImportance: Moderately effective therapies (METs) have been the main treatment in pediatric-onset multiple sclerosis (POMS) for years. Despite the expanding use of highly effective therapies (HETs), treatment strategies for POMS still lack consensus.Objective: To assess the real-world association of HET as an index treatment compared with MET with disease activity.Design, setting, and participants: This was a retrospective cohort study conducted from January 1, 2010, to December 8, 2022, until the last recorded visit. The median follow-up was 5.8 years. A total of 36 French MS centers participated in the Observatoire Français de la Sclérose en Plaques (OFSEP) cohort. Of the total participants in OFSEP, only treatment-naive children with relapsing-remitting POMS who received a first HET or MET before adulthood and at least 1 follow-up clinical visit were included in the study. All eligible participants were included in the study, and none declined to participate.Exposure: HET or MET at treatment initiation.Main outcomes and measures: The primary outcome was the time to first relapse after treatment. Secondary outcomes were annualized relapse rate (ARR), magnetic resonance imaging (MRI) activity, time to Expanded Disability Status Scale (EDSS) progression, tertiary education attainment, and treatment safety/tolerability. An adapted statistical method was used to model the logarithm of event rate by penalized splines of time, allowing adjustment for effects of covariates that is sensitive to nonlinearity and interactions.Results: Of the 3841 children (5.2% of 74 367 total participants in OFSEP), 530 patients (mean [SD] age, 16.0 [1.8] years; 364 female [68.7%]) were included in the study. In study patients, both treatment strategies were associated with a reduced risk of first relapse within the first 2 years. HET dampened disease activity with a 54% reduction in first relapse risk (adjusted hazard ratio [HR], 0.46; 95% CI, 0.31-0.67; P < .001) sustained over 5 years, confirmed on MRI activity (adjusted odds ratio [OR], 0.34; 95% CI, 0.18-0.66; P = .001), and with a better tolerability pattern than MET. The risk of discontinuation at 2 years was 6 times higher with MET (HR, 5.97; 95% CI, 2.92-12.20). The primary reasons for treatment discontinuation were lack of efficacy and intolerance. Index treatment was not associated with EDSS progression or tertiary education attainment (adjusted OR, 0.51; 95% CI, 0.24-1.10; P = .09).Conclusions and relevance: Results of this cohort study suggest that compared with MET, initial HET in POMS was associated with a reduction in the risk of first relapse with an optimal outcome within the first 2 years and was associated with a lower rate of treatment switching and a better midterm tolerance in children. These findings suggest prioritizing initial HET in POMS, although long-term safety studies are needed.
dc.description.sponsorshipObservatoire Français de la Sclérose en Plaquesen_US
dc.language.isoENen_US
dc.rightsAttribution 3.0 United States*
dc.rights.urihttp://creativecommons.org/licenses/by/3.0/us/*
dc.subject.enAdolescent
dc.subject.enAdult
dc.subject.enChild
dc.subject.enCohort Studies
dc.subject.enFemale
dc.subject.enHumans
dc.subject.enMultiple Sclerosis* / drug therapy
dc.subject.enMultiple Sclerosis* / therapy
dc.subject.enMultiple Sclerosis
dc.subject.enRelapsing-Remitting* / drug therapy
dc.subject.enNeoplasm Recurrence
dc.subject.enLocal
dc.subject.enRecurrence
dc.subject.enRetrospective Studies
dc.title.enHighly Effective Therapies as First-Line Treatment for Pediatric-Onset Multiple Sclerosis
dc.title.alternativeJAMA Neurolen_US
dc.typeArticle de revueen_US
dc.identifier.doi10.1001/jamaneurol.2023.5566en_US
dc.subject.halSciences du Vivant [q-bio]/Médecine humaine et pathologieen_US
dc.identifier.pubmed38345791en_US
bordeaux.journalJAMA neurologyen_US
bordeaux.page273-282en_US
bordeaux.volume81en_US
bordeaux.hal.laboratoriesNeurocentre Magendie - U1215en_US
bordeaux.issue3en_US
bordeaux.institutionUniversité de Bordeauxen_US
bordeaux.institutionINSERMen_US
bordeaux.teamRelations glie-neuroneen_US
bordeaux.peerReviewedouien_US
bordeaux.inpressnonen_US
bordeaux.import.sourcehal
hal.identifierhal-04458180
hal.version1
hal.popularnonen_US
hal.audienceInternationaleen_US
hal.exportfalse
workflow.import.sourcehal
dc.rights.ccCC BYen_US
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