| Title: |
Disease-modifying therapies in managing disability worsening in paediatric-onset multiple sclerosis: a longitudinal analysis of global and national registries. |
| Authors: |
Sharmin, Sifat; Roos, Izanne; Malpas, Charles B; Iaffaldano, Pietro; Simone, Marta; Filippi, Massimo; Kubala Havrdova, Eva; Ozakbas, Serkan; Brescia Morra, Vincenzo; Alroughani, Raed; Zaffaroni, Mauro; Patti, Francesco; Eichau, Sara; Salemi, Giuseppe; Di Sapio, Alessia; Inglese, Matilde; Portaccio, Emilio; Trojano, Maria; Amato, Maria Pia; Kalincik, Tomas; Writing Group; Italian Multiple Sclerosis and Related Disorders Register and MSBase Study Group; Van Pesch, Vincent |
| Contributors: |
UCL - SSS/IONS/CEMO - Pôle Cellulaire et moléculaire; UCL - (SLuc) Service de neurologie |
| Source: |
The Lancet. Child & adolescent health, Vol. 8, no.5, p. 348-357 (2024) |
| Publisher Information: |
Elsevier Ltd. |
| Publication Year: |
2024 |
| Collection: |
DIAL@USL-B (Université Saint-Louis, Bruxelles) |
| Subject Terms: |
Adult; Child; Male; Humans; Female; Adolescent; Multiple Sclerosis; Relapsing-Remitting; Chronic Progressive; Fingolimod Hydrochloride; Registries |
| Description: |
BACKGROUND: High-efficacy disease-modifying therapies have been proven to slow disability accrual in adults with relapsing-remitting multiple sclerosis. However, their impact on disability worsening in paediatric-onset multiple sclerosis, particularly during the early phases, is not well understood. We evaluated how high-efficacy therapies influence transitions across five disability states, ranging from minimal disability to gait impairment and secondary progressive multiple sclerosis, in people with paediatric-onset multiple sclerosis. METHODS: Longitudinal data were obtained from the international MSBase registry, containing data from people with multiple sclerosis from 151 centres across 41 countries, and the Italian Multiple Sclerosis and Related Disorders Register, containing data from people with multiple sclerosis from 178 Italian multiple sclerosis centres. People younger than 18 years at the onset of multiple sclerosis symptoms were included, provided they had a confirmed diagnosis of relapsing-remitting multiple sclerosis and at least four Expanded Disability Status Scale (EDSS) scores recorded within 12-month intervals. The primary outcome was the time to change in disability state: minimal disability (EDSS scores 0, 1·0, and 1·5), mild disability (EDSS scores 2·0 and 2·5), moderate disability (EDSS scores 3·0 and 3·5), gait impairment (EDSS scores ≥4·0), and clinician diagnosed secondary progressive multiple sclerosis. A multi-state model was constructed to simulate the natural course of multiple sclerosis, modelling the probabilities of both disability worsening and improvement simultaneously. The impact of high-efficacy disease-modifying therapies (alemtuzumab, cladribine, daclizumab, fingolimod, mitoxantrone, natalizumab, ocrelizumab, rituximab, or autologous haematopoietic stem cell transplantation) and low-efficacy disease-modifying therapies (dimethyl fumarate, glatiramer acetate, interferon beta, or teriflunomide), compared with no treatment, on the course of disability was assessed. Apart ... |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| Relation: |
boreal:302354; http://hdl.handle.net/2078.1/302354; info:pmid/38547883 |
| DOI: |
10.1016/S2352-4642(24)00047-6 |
| Availability: |
http://hdl.handle.net/2078.1/302354; https://doi.org/10.1016/S2352-4642(24)00047-6 |
| Rights: |
info:eu-repo/semantics/openAccess |
| Accession Number: |
edsbas.5635FF35 |
| Database: |
BASE |