Study: Disease-modifying therapies in managing disability worsening in paediatric-onset multiple sclerosis: a longitudinal analysis of global and national registries.
A recent study, the largest of its kind in the world, demonstrated that treating children with multiple sclerosis (MS) early and with the same high-efficacy medicines as adults improves outcomes. The results were published in Lancet Child and Adolescent Health.
There are only a few medicines licensed for children with multiple sclerosis, and only one is designated high effectiveness, which is highly successful.
However, A Royal Melbourne Hospital (RMH) observational research found that pediatric patients should be treated with the same high-efficacy medicines as adults as early as feasible to avoid the start of substantial handicap.
“We found that patients who were treated with high-efficacy disease-modifying therapies during the initial phases of their disease benefited the most compared to patients who were not treated,” said Dr. Sifat Sharmin, a Research Fellow at the Royal Melbourne Hospital’s Neuroimmunology Center and the University of Melbourne’s Department of Medicine.
“Based on our findings we recommend that patients with pediatric-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 examined global data from over 5,000 people diagnosed with MS as children over the previous 30 years, including data from MSBase, a huge international registry spanning 41 countries, and a national registry in Italy, where the condition is very prevalent.
It linked the strength of treatment to the severity of the disease later in life and found that
people who had the most effective treatments early in their diagnosis were less likely to have their handicap deteriorate. These disease-modifying medicines include extremely effective antibodies that alter how an individual’s immune system functions.
The study also confirmed that any treatment—including low-efficacy treatments—was superior to no treatment.
Dr. Sharmin, who led the study, stated that because pediatric-onset MS is a rare disease (about 4-8% of MS patients are diagnosed before the age of 18), it has not received adequate attention.
“This is the largest study of its kind for pediatric MS,” she went on to say.
“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 disorder in which the immune system targets the brain and spinal cord. There is currently no cure for this illness.
More information: Disease-modifying therapies in managing disability worsening in paediatric-onset multiple sclerosis: a longitudinal analysis of global and national registries, The Lancet Child & Adolescent Health (2024). www.thelancet.com/journals/lan … (24)00047-6/fulltext
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