Many of the disease modifying therapies prescribed for adults with MS are also prescribed for pediatric MS. These include conventional first line therapies like interferon beta 1A (Avonex®, Betaseron®) and Glatirimer acetate (Copaxone®). Safety and efficacy of these self-injected disease modifying MS drugs have been demonstrated in small retrospective studies, case studies and unblinded controlled trials (Banwell et al., 2006
, Tenenbaum et al., 2013
, Kornek et al., 2003
). Lack of tolerability or continued progression of disease despite these therapies may necessitate the use of other newer therapies.
Pediatric MS is considered highly active, characterized by more frequent relapses, rapid lesion accrual early in the disease course and more cognitive and physical disability than in adult-onset MS (Hacohen et al., 2020
). In a cohort study comparing initial treatment with newer DMTs versus injectables in children with MS/CIS, newer DMTs provided better disease control, though long-term safety data are needed (Krysko et al., 2020
). Due to the highly active nature of pediatric MS, and earlier age of conversion to secondary progressive MS, treatment with a high-efficacy DMT has been recommended (Hacohen et al., 2020
In 2018 the U.S. Food and Drug Administration approved the use of the oral MS therapy fingolimod (Gilenya®, Novartis AG) for the treatment of children and adolescents 10 years of age or older with relapsing MS.
Other oral therapies for MS, including dimethyl fumarate (Tecfidera®) and teriflunomide (Aubagio®), are currently under investigation in clinical trials for the treatment of pediatric MS.
An observational study of natalizumab (Tysabri®) showed that the safety and efficacy in children was similar to that seen in the adult MS population (Ghezzi et al., 2015
In addition to the FDA-approved therapies used in pediatric MS, another treatment that is not FDA-approved for MS, known as rituximab (Rituxan®), has been studied in small trials of pediatric patients, demonstrating both safety and efficacy. Rituximab is widely used in other pediatric autoimmune disorders and has a favorable safety profile (Dale et al., 2014
Ultimately, starting or switching a disease modifying therapy in children and adolescents requires an in-depth discussion between the provider, child and family. It should include the goals and expectations of the child and family, how the drug is expected to control MS and the side effects, risks, alternatives and any monitoring that must be performed before and during therapy. In this way, providers, patients and families can participate in a shared decision-making process to determine the therapy that best meets the individual needs of each patient.
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 as a supplement
in the journal, Neurology
A publication from the MS International Federation (MSIF) summarizes the key points from each of these articles.
In any patient diagnosed with pediatric MS, poor academic performance, difficulty with peer relations, low self-esteem, difficulty with accepting the diagnosis and depression are all important considerations. While a detailed psychosocial evaluation by a trained professional should always be pursued, pediatric MS support groups
can provide additional crucial information, resources and networks for both patients and their families who are faced with this complex and life-long disease.
Reviewed by Carla Francisco, MD and Aaron Abrams, MD, September, 2020