There are no US Food and Drug Administration (FDA) approved disease modifying therapies for children or adolescents with MS (pediatric MS). However, many of the disease modifying therapies prescribed for adults with MS are also prescribed for pediatric MS.
Results from a clinical trial of fingolimod (Gilenya®) in children with MS 10 years of age and older showed a reduction in the number of relapses (new episodes of symptoms) when compared to a group of children taking interferon beta-1a (Avonex®). The results also indicate less new MRI activity in the Gilenya treated group. Gilenya is not yet FDA approved for pediatric MS but was granted “Breakthrough Therapy designation” by the US Food and Drug Administration (FDA), which will expedite the review process.
Other oral therapies for MS, including dimethyl fumarate (Tecfidera®) and teriflunomide (Aubagio®), are currently under study in clinical trials for the treatment of pediatric MS.
An observational study of natalizumab showed that the safety and efficacy in children were similar to that in the adult MS population.
Smaller retrospective studies, case studies and unblinded controlled trials have demonstrated safety and efficacy of the self-injected MS disease modifying therapies.
In addition to the FDA approved therapies used for pediatrics, another treatment, that is not FDA approved for MS, known as rituximab (Rituxan®), has been studied in small trials of pediatric patients and showed that it was safe and effective.
Ultimately, starting or switching a disease modifying therapy in children and adolescents requires that the provider, child and family have an in-depth discussion. This discussion should include the goals and expectations of the child and family, how the drug is expected to control the MS, the side effects, the risks and any monitoring (blood tests, MRI and other tests) after the therapy is started. In this way, providers, patients and families can participate in a shared decision-making process to determine the therapy that best meets individual needs.
The International Pediatric MS Study Group has written a series of articles, highlighting the advances, unanswered questions and challenges in diagnosing and treating MS in children. These articles have been published in a supplement
to the journal Neurology. A publication from the Multiple Sclerosis International Federation (MSIF) summarizes the key points from each of these articles.