Highly Effective Therapies as First-Line Treatment for Pediatric-Onset Multiple Sclerosis
- Type de publi. : Article dans une revue
- Date de publi. : 01/03/2024
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Auteurs :
Naïl BenallegueFabien RollotS WiertlewskiRomain CaseyMarc DebouverieAnne KerbratJérôme de SèzeJonathan CironAurelie RuetPierre LabaugeElisabeth MaillartHélène ZéphirCaroline PapeixGilles DeferChristine Lebrun-FrenayThibault MoreauEric BergerBruno StankoffPierre ClavelouOlivier HeinzlefJean PelletierEric ThouvenotAbdullatif Al KhedrBertrand BourreOlivier CasezPhilippe CabreAbir WahabLaurent MagySandra VukusicDavid-Axel Laplaud
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Organismes :
Centre Hospitalier Universitaire d'Angers
Centre Hospitalier Universitaire de Nantes = Nantes University Hospital
Centre de Recherche en Transplantation et Immunologie - Center for Research in Transplantation and Translational Immunology
CIC Plurithématique de Nantes
Université Claude Bernard Lyon 1
Hôpital neurologique et neurochirurgical Pierre Wertheimer [CHU - HCL]
Centre de recherche en neurosciences de Lyon - Lyon Neuroscience Research Center
Observatoire Français de la Sclérose En Plaques [Lyon]
Fondation Eugène Devic EDMUS
Centre Hospitalier Universitaire de Nantes = Nantes University Hospital
CIC Plurithématique de Nantes
Centre de Recherche en Transplantation et Immunologie - Center for Research in Transplantation and Translational Immunology
Université Claude Bernard Lyon 1
Hôpital neurologique et neurochirurgical Pierre Wertheimer [CHU - HCL]
Centre de recherche en neurosciences de Lyon - Lyon Neuroscience Research Center
Observatoire Français de la Sclérose En Plaques [Lyon]
Fondation Eugène Devic EDMUS
Service de neurologie [CHRU Nancy]
INterdisciplinarité en Santé Publique Interventions et Instruments de mesure complexes
Service de Neurologie [Rennes] = Neurology [Rennes]
Neuroimagerie: méthodes et applications
Hôpitaux Universitaires de Strasbourg
CIC Strasbourg
Centre Hospitalier Universitaire de Toulouse
Institut Toulousain des Maladies Infectieuses et Inflammatoires
Centre Hospitalier Universitaire de Bordeaux
Neurocentre Magendie : Physiopathologie de la Plasticité Neuronale
Centre Hospitalier Régional Universitaire [Montpellier]
CHU Pitié-Salpêtrière [AP-HP]
Hôpital Roger Salengro [Lille]
Lille Neurosciences & Cognition - U 1172
Service de neurologie [Hôpital Fondation Adolphe de Rothschild]
CHU Caen Normandie – Centre Hospitalier Universitaire de Caen Normandie
Centre Hospitalier Universitaire de Nice
Unité de Recherche Clinique Côte d’Azur
Centre Hospitalier Universitaire de Dijon - Hôpital François Mitterrand
Service de Neurologie [CHRU Besançon]
CHU Saint-Antoine [AP-HP]
CHU Clermont-Ferrand
Centre hospitalier intercommunal de Poissy/Saint-Germain-en-Laye - CHIPS [Poissy]
Hôpital de la Timone [CHU - APHM]
Centre de résonance magnétique biologique et médicale
Service de Neurologie [CHU Nimes]
Institut de Génomique Fonctionnelle
CHU Amiens-Picardie
CHU Rouen
CHU de Grenoble-Alpes - Centre Hospitalier Universitaire CHU Grenoble
Centre Hospitalier Universitaire de Martinique [Fort-de-France, Martinique]
Hôpital Henri Mondor
CHU Limoges
Université Claude Bernard Lyon 1
Hôpital neurologique et neurochirurgical Pierre Wertheimer [CHU - HCL]
Centre de recherche en neurosciences de Lyon - Lyon Neuroscience Research Center
Observatoire Français de la Sclérose En Plaques [Lyon]
Fondation Eugène Devic EDMUS
Centre Hospitalier Universitaire de Nantes = Nantes University Hospital
Centre de Recherche en Transplantation et Immunologie - Center for Research in Transplantation and Translational Immunology
CIC Plurithématique de Nantes
- Publié dans JAMA neurology le 24/10/2020
Résumé : Importance: 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.
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jamaneurology_benallegue_2024_oi_230102_1707244155.42328.pdf
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