Fingolimod (Gilenya®, Tascenso ODT®) is indicated for the treatment of children and adolescents 10 years of age or older with relapsing-remitting MS. Other DMTs are frequently used off-label in this population.
- A study looking at the use of teriflunomide (Aubagio ®) in treating POMS did not meet its primary endpoint of decreasing time to first confirmed clinical relapse, however, it did meet its secondary endpoint of reducing disease activity on MRI scans significantly more than placebo.
- Other oral therapies for MS, including dimethyl fumarate (Tecfidera®) continue to be studied in clinical trials for the treatment of POMS.
Prior to oral and high efficacy therapies becoming available, many of the older disease modifying therapies prescribed for adults with MS were also prescribed in children. These included interferon beta-1a (Avonex®, Betaseron®) and glatiramer acetate (Copaxone®).
- Safety and efficacy of these self-injectable 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).
- Initial treatment of POMS with newer DMTs (oral and infusible medications) has shown better disease activity control compared to 1st generation injectables in clinical trials, showcasing the effectiveness of newer therapies. However, long-term safety data for newer DMTs are still needed (Krysko et al., 2020).
With regards to infusion therapies, 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). Additional infusion trials are currently underway.
In addition to the adult FDA-approved therapies used in POMS, 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 safety profile similar to other B-cell depleting agents widely used in adults (e.g. ocrelizumab) (Dale et al., 2014).
POMS is usually highly active, characterized by more frequent relapses, rapid lesion accrual early in the disease course and more cognitive and physical disability at an earlier age than in adult-onset MS (Hacohen et al., 2020). In a cohort study comparing initial treatment with newer DMTs versus older 1st-generation injectables in POMS patients or those with CIS, newer DMTs provided better disease control and safety profiles were similar to what has been observed in adults. However, long-term safety data are still needed in children (Krysko et al., 2020). Due to the highly active nature of POMS and earlier age of conversion to secondary progressive MS, initial treatment with newer, higher-efficacy DMTs is being increasingly recommended (Hacohen et al., 2020).
Starting or switching disease modifying therapies
Ultimately, starting or switching a disease modifying therapy in children and adolescents requires an in-depth discussion between the provider, patient and family. It should include:
- Understanding the goals and expectations of the child and family
- Sharing the safety, efficacy and monitoring profile of the various treatment options
- Proactively discussing factors that could affect adherence (cost, route of administration, dosage, transportation)
By understanding what is important to the child and family and addressing concerns, fears, and potential challenges, 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 (IPMSSG) 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