A ground-breaking study – the largest of its kind globally – has found children with multiple sclerosis (MS) have better outcomes if treated early and with the same high-efficacy therapies as adults.
There are a limited number of therapies approved for children with MS, with only one considered to be of high-efficacy – meaning highly effective.
However, a Royal Melbourne Hospital (RMH) observational study has determined that paediatric patients should be treated with the same high-efficacy treatments offered to adults as early in their diagnosis as possible to avoid the onset of significant disability.
“We found that patients who were treated with high-efficacy disease-modifying therapies during the initial phases of their disease benefitted the most compared to patients who were not treated,” Dr Sifat Sharmin, a Research Fellow at the Royal Melbourne Hospital’s Neuroimmunology Centre, and the University of Melbourne’s Department of Medicine, said.
“Based on our findings we recommend that patients with paediatric-onset multiple sclerosis should be treated early in the disease course, when the disability is still minimal, to preserve neurological capacity before it’s damaged.”
The observational study analysed global data of more than 5000 people diagnosed with MS during childhood over the last 30 years – including from MSBase, a large international registry encompassing 41 countries, and a national registry in Italy, where the disease is highly prevalent.
It compared the strength of treatment with the severity of the disease later in life, and concluded patients treated with the most effective treatments early on in their diagnosis were less likely to experience disability worsening. These disease-modifying therapies include highly effective antibodies that change the way in which an individual’s immune system behaves.
The findings were published in the prestigious journal the Lancet Child and Adolescent Health this week.
The research also confirmed that any treatment – including low-efficacy treatments – was better than no treatment
Dr Sharmin, who led the study, said because paediatric-onset MS was a rare disease – about four to eight per cent of MS patients are diagnosed before age 18 – it wasn’t as well investigated.
“This is the largest study of its kind for paediatric MS,” she said.
“We hope this may have some policy implications so children with MS can access the most effective therapies as early as possible.”
MS is a chronic condition that occurs when the immune system attacks the brain and spinal cord. There is currently no cure for the condition.
View the paper in full once published via www.thelancet.com/journals/lanchi/article/PIIS2352-4642(24)00047-6/fulltext
For interview and patient case study requests, please contact:
Alanah Frost, Media and Content Advisor, the Royal Melbourne Hospital, 0472 767 760
Journal
The Lancet Child & Adolescent Health
Method of Research
Observational study
Subject of Research
People
Article Title
Disease-modifying therapies in managing disability worsening in paediatric-onset multiple sclerosis: a longitudinal analysis of global and national registries
Article Publication Date
26-Mar-2024
COI Statement
SS has received research support from the MSBase Foundation and MS Australia. IR served on scientific advisory boards and received conference travel support or speaker honoraria from Roche, Novartis, Merck, and Biogen. CBM has received conference travel support from Merck, Novartis, and Biogen, and research support from the National Health and Medical Research Council Australia, MS Australia, The University of Melbourne, The Royal Melbourne Hospital Neuroscience Foundation, and Dementia Australia. PI has served on scientific advisory boards for Biogen Idec, Bayer, Bristol Myers Squibb (BMS), Teva, Roche, Merck Serono, Novartis, and Genzyme, and has received funding for travel or speaker honoraria from Sanofi Aventis, Genzyme, Roche, Biogen Idec, Teva, Merck Serono, Alexion, BMS, and Novartis. MF received compensation for consulting services or speaking activities from Almiral, Alexion, Bayer, Biogen, Celgene, Eli Lilly, Genzyme, Merck-Serono, Novartis, Roche, Sanofi, Takeda, and Teva Pharmaceutical Industries, and receives research support from Biogen Idec, Merck-Serono, Novartis, Roche, Teva Pharmaceutical Industries, Italian Ministry of Health, Fondazione Italiana Sclerosi Multipla, and ARiSLA (Fondazione Italiana di Ricerca per la SLA). EKH received honoraria or research support from Biogen, Merck Serono, Novartis, Roche, and Teva; has been member of advisory boards for Actelion, Biogen, Celgene, Merck Serono, Novartis, and Sanofi Genzyme; and has been supported by the Czech Ministry of Education (project Cooperatio LF1) and the National Institute for Neurological Research of the Czech Republic (programme EXCELES, project number LX22NPO5107) funded by the Next Generation EU. VBM received compensation for public speaking or consultancy from Merck, Novartis, Biogen, Genzyme, Teva, and Alrmirall. RA received honoraria as a speaker and for serving on scientific advisory boards from Bayer, Biogen, GSK, Merck, Novartis, Roche, and Sanofi-Genzyme. MZ received travel support and fees for lecturing or participating in advisory boards from Almirall, Biogen, Merck, Novartis, and Sanofi-Genzyme. FP received personal compensation for serving on advisory boards by Almirall, Alexion, Biogen, BMS, Janssen, Merck, Novartis, and Roche, and research grants from Alexion, Almirall, Biogen, BMS, Merck, Novartis, Roche, Fondazione Italiana Sclerosi Multipla, Reload Association (Onlus), Italian Health Minister, and University of Catania. SE received speaker honoraria and consultant fees from Biogen Idec, Novartis, Merck, Bayer, Sanofi Genzyme, Roche, and Teva. GS received grants and honoraria from Roche, Sanofi, Merck, Biogen, and Novartis. ADS received speaker honoraria and consultant fees from Biogen, Novartis, Merck, Roche, Alexion, and Sanofi. EP received compensation for travel grants, participation in advisory boards, or speaking activities from Biogen, Merck Serono, Sanofi, Teva, and Novartis, and serves on the editorial boards of Frontiers in Neurology and Brain Sciences. MT has served on scientific advisory boards for Biogen, Novartis, Roche, Merck, and Genzyme; received speaker honoraria from Biogen, Roche, Sanofi, Merck, Genzyme, and Novartis; and received research grants for her institution from Biogen, Merck, and Novartis. MPA served on scientific advisory boards for and has received speaker honoraria and research support from Biogen Idec, Merck Serono, Bayer Schering Pharma, Novartis, Roche, BMS Celgene, and Sanofi Aventis, and is Editor of Multiple Sclerosis Journal. TK served on scientific advisory boards for MS International Federation and WHO, BMS, Roche, Janssen, Sanofi Genzyme, Novartis, Merck, and Biogen, and the steering committee for Brain Atrophy Initiative by Sanofi Genzyme; received conference travel support or speaker honoraria from WebMD Global, Eisai, Novartis, Biogen, Roche, Sanofi-Genzyme, Teva, BioCSL, and Merck; and received research or educational event support from Biogen, Novartis, Genzyme, Roche, Celgene, and Merck. All other authors declare no competing interests.