New generation treatments safe to use in children with MS

• While there is not much information about the effects of MS treatments in children, children with MS are often treated with adult MS medications.
• A new study aimed to capture the use of disease modifying therapies in children, in particular the new generation oral and infusion therapies.
• In a large study of children with MS conducted at a network of US clinics, 78% of children with MS were given disease modifying therapies and over 40% were given new generation medications.
• The safety and side effects seen in the children with MS were similar to that seen in the adult MS population.

MS most commonly strikes during young adulthood, but up to 5% of diagnoses occur in children, including some as young as two years of age. As the numbers affected are much smaller and the challenges greater, clinical trials in children with MS have been harder to conduct. Some trials have only recently begun, with many yet to report their findings, meaning there is a lack of information about the safety and side effects of MS medications in children. Despite this, in the clinic, children with MS have often been treated “off-label” with adult MS medications – making it even more urgent that the effects of MS medications in children are better defined.

What are children with MS in the US treated with?

A new study just published by US researchers has looked at the use of medications in a large group of children with MS. The study included 1,019 children with either MS or clinically isolated syndrome (CIS, a precursor that may develop into MS) from a network of clinics around the US. While older therapies such as interferon beta (Betaseron, Rebif) and glatiramer acetate (Copaxone) are usually the first choice for children with MS, the researchers sought to understand the use and effects of the newer generation MS therapies in children including dimethyl fumarate (Tecfidera), natalizumab (Tysabri), rituximab (Rituxan), fingolimod (Gilenya), daclizumab (Zinbryta) and teriflunomide (Aubagio).

In this study, most of the children with MS (78%) and a smaller proportion (11%) of the children with CIS were given disease modifying therapy. Over 40% had been given one of the newer generation therapies, with this being the first option tried in 20% of cases. In those who were given an injectable therapy first, close to one in three changed to a newer medication before the age of 18.

Children under ten are excluded from clinical trials and the number of children under 12 that were included in this observational study is low. As such, the researchers also looked at the children who were under 12 as a separate group. They found the number receiving treatment was still high, although somewhat lower than the group overall. Daclizumab and teriflunomide were not given to children under the age of 12.

Do children experience different side effects?

The children were then followed up to determine the effects of these treatments in a real world setting. Over time, the use of the newer oral and infusion therapies has increased and it has also become more common to use these medications as the first choice in children who need them. No new side effects were seen in the children (compared those that adult’s have experienced with the same medications) and the rates of known side effects were similar to those seen in adults. None of the children in the study developed progressive multifocal leukoencephalopathy (PML), a brain infection which can be a rare side-effect of some MS medications. It was slightly more common for children with MS to stop using the newer generation therapies than seen in the adult MS population, but the reasons for this were unclear.

The only formal, gold-standard randomised controlled clinical trial of disease modifying therapy in children with MS to be completed so far has shown that fingolimod performs better than interferon beta 1a (injected into the muscle) in terms of suppressing relapses and lesions. Other trials looking at teriflunomide and dimethyl fumarate in children with MS are ongoing and we await these results with interest. In the meantime, this real world study has provided reassurance that the newer generation MS medications are safe to use in children.