First treatment for pediatric multiple sclerosis
Multiple Sclerosis (MS) is a common disease in young adults but rare in children. More than a dozen treatments have been approved by the
US Food and Drug Administration for treating MS in adults, and several of these treatments are prescribed in children. However, high-quality targeted evidence for children has been lacking. A recent phase III double-blind randomised controlled trial of fingolimod (PARADIGMS) versus interferon beta-1a was the first to show that fingolimod significantly reduced the relapse rate by 82% and the appearance of new or enlarging lesions on T2-weighted MRI by 53%. These benefits came at the cost of more serious adverse events in children with MS.
It is well known that similar to adults, loss of brain tissue (or brain atrophy) occurs in children with MS.
However, it was not known if brain atrophy could be slowed in children with MS, until now.
As a follow-up study to PARADIGMS, investigators Arnold et al have reported the brain MRI results of this trial: fingolimod reduced the accrual of lesions on T2-weighted, gadolinium-enhancing and new hypointense lesions on T1-weighted MRI. Importantly, this paper shows that fingolimod reduced the rate of brain atrophy in children with MS up to 40% compared with the interferon beta-1a treatment (absolute difference of 0.32% per year). Therefore, similar to adults, children with MS benefit from fingolimod treatment due to a direct anti-inflammatory effect, with a possible secondary effect on brain atrophy.