In 2018 the U.S. Food and Drug Administration approved the expansion of the use of the oral MS therapy Gilenya® (fingolimod, Novartis AG) to include the treatment of children and adolescents 10 years of age or older with relapsing MS.
In addition, many of the disease modifying therapies that are FDA approved for adults are also prescribed off-label for pediatric MS.
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.